Adrenal InsufficiencyPhase 2NCT01859312

Comparison of Cortisol Pump With Standard Treatment for Congenital Adrenal Hyperplasia

A Phase 2 study testing Hydrocortisone (Solucortef) and Insulin pump (Medtronic) in people with Adrenal Insufficiency and Excess Androgen, run by National Institutes of Health Clinical Center (CC) at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

Background: Congenital adrenal hyperplasia (CAH) is a genetic disorder of the adrenal gland. The adrenal gland is located in the abdomen and produces small amounts of hormones such as cortisol, aldosterone, and androgen. Read more about this study

Who is it for?: Ages 18 to 99 years
Conditions the study is for: Adrenal Insufficiency, Excess Androgen, Congenital Adrenal Hyperplasia (CAH)
What is being tested?: Hydrocortisone (Solucortef) and Insulin pump (Medtronic)
Type of treatment: Injection or infusion, Medical device
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 8 participants
Where: 1 siteUnited States
Study dates: 6 May 2013to 2 Dec 2016
Phase: Phase 2
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 18 to 99 years
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

Patients with known classic CAH due to 21-hydroxylase deficiency as evidenced by hormonal and genetic testing
Male or female patients 18 years or older
Females must have a negative pregnancy test initially and at all visits. Sexually active females must be using a medically acceptable method of contraception.
Patients with elevated adrenal androgens (defined as 17-OHP >1200 ng/dL and androstenedione >210 ng/dL)
One or more co-morbidities:<TAB>
Obesity [body mass index (BMI) greater than 30.0 kg/m(2)]

Show all 10Show fewer

Fatty liver disease; assessed by AST/ALT liver enzyme ratio (AST to ALT ratio <1 (11)) liver ultrasound or MRI imaging (Steatosis score as previously described)
Low insulin sensitivity; assessed by the Homeostasis Model Assessment Insulin Resistance (HOMA-IR) method [HOMA-IR = insulin (micro U/ml) times glucose (mmol/L)/ 22.5]. Elevated HOMA-IR index is defined as >2.6 in adults17.
Osteopenia [bone mineral density by DEXA (at the spine, hip, or forearm) with T-score of -1 to -2.5) or osteoporosis (bone mineral density by DEXA (at the spine, hip, or forearm) with T-score of <-2.5] defined according to World Health Organization (WHO).
Glucocorticoid-related gastrointestinal side effects (nausea, vomiting, dyspepsia, anorexia, gastritis, peptic ulcer disease and gastric bleeding)

Who cannot join

Co-morbid conditions requiring daily administration of medications that induce hepatic enzymes or interfere with the metabolism of glucocorticoids
Females who are pregnant or lactating
Patients on inhaled or oral steroids given for reasons other than treatment of CAH
Women who have taken estrogen-containing oral contraceptive pills within 6 weeks of recruitment
Patients who required stress dose glucocorticoids for an illness within 4 weeks of recruitment
Patients who changed their glucocorticoid agent within 3 months of recruitment
Patients who underwent bilateral adrenalectomy
Co-morbid conditions that could interfere with the ability to comply to the protocol

Trial locations and contacts

1 site in 1 country.

United States1 site

National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, 20892

Other trials in United States

About this study

Official title: A Pilot Study Assessing the Use of Continuous Subcutaneous Hydrocortisone Infusion in the Treatment of Congenital Adrenal Hyperplasia

Background:

Congenital adrenal hyperplasia (CAH) is a genetic disorder of the adrenal gland. The adrenal gland is located in the abdomen and produces small amounts of hormones such as cortisol, aldosterone, and androgen. These hormones help control blood pressure, protect the body, and maintain good health, especially during development. People with CAH do not make enough cortisol and aldosterone, and make too much androgen. This can lead to serious medical problems. The standard treatment is to take pills that mimic the effects of cortisol and aldosterone. However, treatment with pills can have long-term side effects because of the higher doses needed, and may not work well for some people.
A possible new treatment for CAH is to use a pump to deliver cortisol under the skin. Similar pumps are often used to give insulin to people with diabetes. Researchers think that a cortisol pump might be able to help the body use the cortisol more effectively than taking pills. They want to compare the results of a cortisol pump and standard pill treatments for CAH.

Objectives:

To compare the effectiveness of a cortisol pump with standard cortisol pill therapy for CAH.

Eligibility:

Men and women at least 18 years of age who have CAH (see more details in Eligibility section below).

Design:

This study will involve four inpatient hospital stays at the National Institutes of Health in Bethesda, MD over 6 months (spaced 2 months apart). The first and last stays will last about 5 days. The second and third stays will last about 3 days.
Participants will be screened with a physical exam and medical history. Blood and urine samples will be collected.
At the first study visit, participants will provide regular blood and urine samples. They will also have imaging studies. These studies will look at the bones, fat, and muscles in the abdomen and pelvis.
Participants will receive a cortisol pump during the first visit. They will be shown how to use the pump. They will also learn what to do, if they need to take extra "stress dose" cortisol pills.
At the second and third visits, the cortisol dose given with the pump will be adjusted as needed. Blood and urine samples will also be collected. No imaging studies are scheduled for these visits.
The last study visit will have the same tests as the first visit. Participants will be offered the chance to continue with the pump treatment for 1 more year, or go back to their standard pill treatment.

Study type: Interventional non-randomized trial

Official title: A Pilot Study Assessing the use of Continuous Subcutaneous Hydrocortisone Infusion In the Treatment of Congenital Adrenal Hyperplasia

Estimated enrollment: 8

Study Start Date: May 2013

Estimated Study Completion Date: December 2016

Sponsoring Institute: National Institute of Child Health and Human Development

<TAB>ELIGIBILITY

Inclusion criteria

Men and women 18 years of age or older with classic congenital adrenal hyperplasia (21-Hydroxylase deficiency)
High adrenal androgens in the blood, and
One or more of the following conditions: obesity, fatty liver, risk for diabetes, low bone mass, inability to tolerate cortisol pills

Exclusion criteria

Pregnancy
Breast feeding
Use of inhaled or oral steroids for diseases other than CAH
Use of estrogen-containing birth control pills
Use of medicines that cross-react with hydrocortisone
Use of stress dose steroids for illness during the last 30 days prior to joining the study

Read the detailed description

Congenital adrenal hyperplasia (CAH) is a common genetic endocrine disorder, with 21-hydroxylase enzyme deficiency accounting for 95% of the cases. 21-hydroxylase deficiency presents with a spectrum of clinical manifestations ranging from salt-wasting and virilization of female neonates (classic CAH) to symptomatic (precocious puberty, short stature, acne) or asymptomatic hyperandrogenemia (non-classic CAH). Classic CAH is characterized by impaired cortisol and mineralocorticoid biosynthesis, which triggers adrenocorticotropic hormone (ACTH) hyper-secretion and accumulation of adrenal androgens. Glucocorticoid treatment of patients with classic CAH focuses on cortisol replacement and prevention of the ACTH-driven androgen excess. Current conventional glucocorticoid treatment regimens (short or long-acting agents dosed once, twice or thrice daily) have failed to simulate physiological cortisol secretion and suppress adrenal androgen overproduction, without supraphysiologic replacement. Short-term overtreatment with glucocorticoids can lead to iatrogenic Cushing syndrome and long-term use has been associated with the development of obesity, visceral adiposity, insulin resistance and osteoporosis. Isolated case reports have provided evidence that continuous subcutaneous hydrocortisone infusion (CSHI) can mimic physiologic cortisol release and lead to improved CAH control at doses similar to or lower than the traditional treatment. This pilot study aims to test the hypothesis that difficult-to-treat adult patients with classic CAH will have better adrenal androgen control and improved CAH and glucocorticoid-related comorbidities, when they receive near-physiologic cortisol replacement therapy via CSHI compared to conventional glucocorticoid treatment. In addition, this study will provide information on the safety and tolerability of CSHI, and will generate data that will be used in the design of future pediatric studies.

Conditions: Adrenal Insufficiency, Excess Androgen, Congenital Adrenal Hyperplasia (CAH)
Being tested: Hydrocortisone (Solucortef), Insulin pump (Medtronic)
Keywords: Glucocorticoids, Adrenal Insufficiency, Congenital Adrenal Hyperplasia (CAH), Adrenal
Medical subject headings: Adrenal Insufficiency, Hyperandrogenism, Adrenal Hyperplasia, Congenital

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Hydrocortisone (Solucortef)Drug
Also called CSHI
Continuous subcutaneous hydrocortisone infusion (CSHI) via Medtronic insulin pump (MMT-722NA). Total daily hydrocortisone dose was calculated based on the patient's estimated cortisol clearance. Rates were established to achieve peak and trough concentrations within the normal circadian cortisol range.
Insulin pump (Medtronic)Device
Also called CSHI
Continuous subcutaneous hydrocortisone infusion (CSHI) via Medtronic insulin pump (MMT-722NA)

Study groups in NCT01859312
Study group	Role	What they receive
Continuous Subcutaneous Hydrocortisone Infusion	Experimental	Enrolled participants with congenital adrenal hyperplasia (CAH) received continuous subcutaneous hydrocortisone (Solucortef) infusion (CSHI) via insulin pump (Medtronic) (MMT-722Na) to achieve near-physiologic cortisol replacement therapy. Participants were their own controls; participant's baseline outcomes/lab values while on conventional glucocorticoid therapy were compared to outcomes/lab values after 6 months of treatment using CSHI via insulin pump. Receives: Drug: Hydrocortisone (Solucortef); Device: Insulin pump (Medtronic)

What is being measured

The primary outcome is the main question the study is designed to answer.

Number of Patients With 17-OHP Levels Equal or Below 1,200 ng/dL at 0700
17-OHP (17-hydroxyprogesterone) is a laboratory blood test to test for congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency
Measured: At baseline
Number of Patients With 17-OH Progesterone Levels Equal or Below 1,200 ng/dL at 0700
17-OHP (17-hydroxyprogesterone) is a laboratory blood test to test for congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency
Measured: At 6 months

Secondary outcomes (42)

Participants Mean Level of 17-OHP at 0700
17-OHP (17-hydroxyprogesterone) is a laboratory blood test to test for congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency
Measured: At baseline
Participants Mean Level of 17-OHP at 0700
17-OHP (17-hydroxyprogesterone) is a laboratory blood test to test for congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency
Measured: At 6 months
Participants Mean Level of 17-OHP Area Under the Curve (AUC) - 24 Hours
17-OHP (17-hydroxyprogesterone) is a laboratory blood test to test for congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency. AUC was calculated using the linear-up log-down trapezoidal rule for the entire 24 hours and samples were obtained every 2 hours beginning at 2300, 0100, 0300, 0500, 0700, 0900, 1100, 1300, 1500, 1700, 1900, 2100, 2300. Actual collection times were used to define peak plasma concentration (Cmax) and time to peak plasma concentration (Tmax).
Measured: At baseline
Participants Mean Level of 17-OHP Area Under the Curve (AUC) - 24 Hours
17-OHP (17-hydroxyprogesterone) is a laboratory blood test to test for congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency. AUC was calculated using the linear-up log-down trapezoidal rule for the entire 24 hours and samples were obtained every 2 hours beginning at 2300, 0100, 0300, 0500, 0700, 0900, 1100, 1300, 1500, 1700, 1900, 2100, 2300. Actual collection times were used to define peak plasma concentration (Cmax) and time to peak plasma concentration (Tmax).
Measured: At 6 months
Participants Mean Level of 17-OHP Area Under the Curve (AUC) - Daytime (0700-1500)
17-OHP (17-hydroxyprogesterone) is a laboratory blood test to test for congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency. AUC was calculated using the linear-up log-down trapezoidal rule for the 8 hour daytime level, samples were obtained every 2 hours beginning at 0700, 0900, 1100, 1300, 1500. Actual collection times were used to define peak plasma concentration (Cmax) and time to peak plasma concentration (Tmax).
Measured: At baseline
Participants Mean Level of 17-OHP Area Under the Curve (AUC) - Daytime (0700-1500)
17-OHP (17-hydroxyprogesterone) is a laboratory blood test to test for congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency. AUC was calculated using the linear-up log-down trapezoidal rule for the 8 hour daytime level, samples were obtained every 2 hours beginning at 0700, 0900, 1100, 1300, 1500. Actual collection times were used to define peak plasma concentration (Cmax) and time to peak plasma concentration (Tmax).
Measured: At 6 months
Participants Mean Level of 17-OHP Area Under the Curve (AUC) - Midday (1500 - 2300)
17-OHP (17-hydroxyprogesterone) is a laboratory blood test to test for congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficienc. AUC was calculated using the linear-up log-down trapezoidal rule for the 8 hour midday level, samples were obtained every 2 hours beginning at 1500, 1700, 1900, 2100, 2300. Actual collection times were used to define peak plasma concentration (Cmax) and time to peak plasma concentration (Tmax).
Measured: At baseline
Participants Mean Level of 17-OHP Area Under the Curve (AUC) - Midday (1500 - 2300)
17-OHP (17-hydroxyprogesterone) is a laboratory blood test to test for congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency. AUC was calculated using the linear-up log-down trapezoidal rule for the 8 hour midday level, samples were obtained every 2 hours beginning at 1500, 1700, 1900, 2100, 2300. Actual collection times were used to define peak plasma concentration (Cmax) and time to peak plasma concentration (Tmax).
Measured: At 6 months
Participants Mean Level of 17-OHP Area Under the Curve (AUC) - Nighttime (2300 - 0700)
17-OHP (17-hydroxyprogesterone) is a laboratory blood test to test for congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency. AUC was calculated using the linear-up log-down trapezoidal rule for the 8 hour nighttime level, samples were obtained every 2 hours beginning at 2300, 0100, 0300, 0500, 0700. Actual collection times were used to define peak plasma concentration (Cmax) and time to peak plasma concentration (Tmax).
Measured: At baseline
Participants Mean Level of 17-OHP Area Under the Curve (AUC) - Nighttime (2300 - 0700)
17-OHP (17-hydroxyprogesterone) is a laboratory blood test to test for congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency. AUC was calculated using the linear-up log-down trapezoidal rule for the 8 hour nighttime level, samples were obtained every 2 hours beginning at 2300, 0100, 0300, 0500, 0700. Actual collection times were used to define peak plasma concentration (Cmax) and time to peak plasma concentration (Tmax).
Measured: At 6 months
Participants Mean Level of Androstenedione at 0700
Participants Mean Level of Androstenedione at 0700.
Measured: At baseline
Participants Mean Level of Androstenedione at 0700
Participants Mean Level of Androstenedione at 0700.
Measured: At 6 months
Participants Mean Level of Androstenedione Area Under the Curve (AUC) - 24 Hours
Participants Mean Level of Androstenedione Area Under the Curve (AUC) - 24 hours. AUC was calculated using the linear-up log-down trapezoidal rule for the entire 24 hours and samples were obtained every 2 hours beginning at 2300, 0100, 0300, 0500, 0700, 0900, 1100, 1300, 1500, 1700, 1900, 2100, 2300. Actual collection times were used to define peak plasma concentration (Cmax) and time to peak plasma concentration (Tmax).
Measured: At baseline
Participants Mean Level of Androstenedione Area Under the Curve (AUC) - 24 Hours
Participants Mean Level of Androstenedione area under the curve (AUC) - 24 hours. AUC was calculated using the linear-up log-down trapezoidal rule for the entire 24 hours and samples were obtained every 2 hours beginning at 2300, 0100, 0300, 0500, 0700, 0900, 1100, 1300, 1500, 1700, 1900, 2100, 2300. Actual collection times were used to define peak plasma concentration (Cmax) and time to peak plasma concentration (Tmax).
Measured: At 6 months
Participants Mean Level of Androstenedione Area Under the Curve (AUC) - Daytime (0700-1500)
Participants Mean Level of Androstenedione Area Under the Curve (AUC) - Daytime (0700-1500). AUC was calculated using the linear-up log-down trapezoidal rule for the 8 hour daytime level, samples were obtained every 2 hours beginning at 0700, 0900, 1100, 1300, 1500. Actual collection times were used to define peak plasma concentration (Cmax) and time to peak plasma concentration (Tmax).
Measured: At baseline
Participants Mean Level of Androstenedione Area Under the Curve (AUC) - Daytime (0700-1500)
Participants Mean Level of Androstenedione Area Under the Curve (AUC) - Daytime (0700-1500). AUC was calculated using the linear-up log-down trapezoidal rule for the 8 hour daytime level, samples were obtained every 2 hours beginning at 0700, 0900, 1100, 1300, 1500. Actual collection times were used to define peak plasma concentration (Cmax) and time to peak plasma concentration (Tmax).
Measured: At 6 months
Participants Mean Level of Androstenedione Area Under the Curve (AUC) - Midday (1500 - 2300)
Participants Mean Level of Androstenedione Area Under the Curve (AUC) - Midday (1500 - 2300). AUC was calculated using the linear-up log-down trapezoidal rule for the 8 hour midday level, samples were obtained every 2 hours beginning at 1500, 1700, 1900, 2100, 2300. Actual collection times were used to define peak plasma concentration (Cmax) and time to peak plasma concentration (Tmax).
Measured: At baseline
Participants Mean Level of Androstenedione Area Under the Curve (AUC) - Midday (1500 - 2300)
Participants Mean Level of Androstenedione Area Under the Curve (AUC) - Midday (1500 - 2300). AUC was calculated using the linear-up log-down trapezoidal rule for the 8 hour midday level, samples were obtained every 2 hours beginning at 1500, 1700, 1900, 2100, 2300. Actual collection times were used to define peak plasma concentration (Cmax) and time to peak plasma concentration (Tmax).
Measured: At 6 months
Participants Mean Level of Androstenedione Area Under the Curve (AUC) - Nighttime (2300 - 0700)
Participants Mean Level of Androstenedione Area Under the Curve (AUC) - Nighttime (2300 - 0700). AUC was calculated using the linear-up log-down trapezoidal rule for the 8 hour nighttime level, samples were obtained every 2 hours beginning at 2300, 0100, 0300, 0500, 0700. Actual collection times were used to define peak plasma concentration (Cmax) and time to peak plasma concentration (Tmax).
Measured: At baseline
Participants Mean Level of Androstenedione Area Under the Curve (AUC) - Nighttime (2300 - 0700)
Participants Mean Level of Androstenedione Area Under the Curve (AUC) - Nighttime (2300 - 0700). AUC was calculated using the linear-up log-down trapezoidal rule for the 8 hour nighttime level, samples were obtained every 2 hours beginning at 2300, 0100, 0300, 0500, 0700. Actual collection times were used to define peak plasma concentration (Cmax) and time to peak plasma concentration (Tmax).
Measured: At 6 months
Participants Mean Level of ACTH at 0700
Participants Mean Level of ACTH at 0700.
Measured: At baseline
Participants Mean Level of ACTH at 0700
Participants Mean Level of ACTH at 0700.
Measured: At 6 months
Participants Mean Level of ACTH Area Under the Curve (AUC) - 24 Hours
Participants Mean Level of ACTH Area Under the Curve (AUC) - 24 Hours. AUC was calculated using the linear-up log-down trapezoidal rule for the entire 24 hours and samples were obtained every 2 hours beginning at 2300, 0100, 0300, 0500, 0700, 0900, 1100, 1300, 1500, 1700, 1900, 2100, 2300.Actual collection times were used to define peak plasma concentration (Cmax) and time to peak plasma concentration (Tmax).
Measured: At baseline
Participants Mean Level of ACTH Area Under the Curve (AUC) - 24 Hours
Participants Mean Level of ACTH Area Under the Curve (AUC) - 24 Hours. AUC was calculated using the linear-up log-down trapezoidal rule for the entire 24 hours and samples were obtained every 2 hours beginning at 2300, 0100, 0300, 0500, 0700, 0900, 1100, 1300, 1500, 1700, 1900, 2100, 2300. Actual collection times were used to define peak plasma concentration (Cmax) and time to peak plasma concentration (Tmax).
Measured: At 6 months
Participants Mean Level of ACTH Area Under the Curve (AUC) - Daytime (0700-1500)
Participants Mean Level of ACTH Area Under the Curve (AUC) - Daytime (0700-1500). AUC was calculated using the linear-up log-down trapezoidal rule for the 8 hour daytime level, samples were obtained every 2 hours beginning at 0700, 0900, 1100, 1300, 1500. Actual collection times were used to define peak plasma concentration (Cmax) and time to peak plasma concentration (Tmax).
Measured: At baseline
Participants Mean Level of ACTH Area Under the Curve (AUC) - Daytime (0700-1500)
Participants Mean Level of ACTH Area Under the Curve (AUC) - Daytime (0700-1500). AUC was calculated using the linear-up log-down trapezoidal rule for the 8 hour daytime level, samples were obtained every 2 hours beginning at 0700, 0900, 1100, 1300, 1500. Actual collection times were used to define peak plasma concentration (Cmax) and time to peak plasma concentration (Tmax).
Measured: At 6 months
Participants Mean Level of ACTH Area Under the Curve (AUC) - Midday (1500 - 2300)
Participants Mean Level of ACTH Area Under the Curve (AUC) - Midday (1500 - 2300). AUC was calculated using the linear-up log-down trapezoidal rule for the 8 hour midday level, samples were obtained every 2 hours beginning at 1500, 1700, 1900, 2100, 2300. Actual collection times were used to define peak plasma concentration (Cmax) and time to peak plasma concentration (Tmax).
Measured: At baseline
Participants Mean Level of ACTH Area Under the Curve (AUC) - Midday (1500 - 2300)
Participants Mean Level of ACTH Area Under the Curve (AUC) - Midday (1500 - 2300). AUC was calculated using the linear-up log-down trapezoidal rule for the 8 hour midday level, samples were obtained every 2 hours beginning at 1500, 1700, 1900, 2100, 2300. Actual collection times were used to define peak plasma concentration (Cmax) and time to peak plasma concentration (Tmax).
Measured: At 6 months
Participants Mean Level of ACTH Area Under the Curve (AUC) - Nighttime (2300 - 0700)
Participants Mean Level of ACTH Area Under the Curve (AUC) - Nighttime (2300 - 0700). AUC was calculated using the linear-up log-down trapezoidal rule for the 8 hour nighttime level, samples were obtained every 2 hours beginning at 2300, 0100, 0300, 0500, 0700. Actual collection times were used to define peak plasma concentration (Cmax) and time to peak plasma concentration (Tmax).
Measured: At baseline
Participants Mean Level of ACTH Area Under the Curve (AUC) - Nighttime (2300 - 0700)
Participants Mean Level of ACTH Area Under the Curve (AUC) - Nighttime (2300 - 0700). AUC was calculated using the linear-up log-down trapezoidal rule for the 8 hour nighttime level, samples were obtained every 2 hours beginning at 2300, 0100, 0300, 0500, 0700. Actual collection times were used to define peak plasma concentration (Cmax) and time to peak plasma concentration (Tmax).
Measured: At 6 months
Participants Mean Progesterone Levels at 0700
Participants Mean Level of Progesterone at 0700
Measured: At baseline
Participants Mean Progesterone Level at 0700
Participants Mean Level of Progesterone at 0700
Measured: At 6 months
Participants Mean Level of Progesterone Area Under the Curve (AUC) - 24 Hours
Participants Mean Level of Progesterone area under the curve (AUC) - 24 hours. AUC was calculated using the linear-up log-down trapezoidal rule for the entire 24 hours and samples were obtained every 2 hours beginning at 2300, 0100, 0300, 0500, 0700, 0900, 1100, 1300, 1500, 1700, 1900, 2100, 2300. Actual collection times were used to define peak plasma concentration (Cmax) and time to peak plasma concentration (Tmax).
Measured: At baseline
Participants Mean Level of Progesterone Area Under the Curve (AUC) - 24 Hours
Participants Mean Level of Progesterone area under the curve (AUC) - 24 hours. AUC was calculated using the linear-up log-down trapezoidal rule for the entire 24 hours and samples were obtained every 2 hours beginning at 2300, 0100, 0300, 0500, 0700, 0900, 1100, 1300, 1500, 1700, 1900, 2100, 2300. Actual collection times were used to define peak plasma concentration (Cmax) and time to peak plasma concentration (Tmax).
Measured: At 6 months
Participants Mean Level of Progesterone Area Under the Curve (AUC) - Daytime (0700-1500)
Participants Mean Level of Progesterone area under the curve (AUC) - Daytime (0700-1500). AUC was calculated using the linear-up log-down trapezoidal rule for the 8 hour daytime level, samples were obtained every 2 hours beginning at 0700, 0900, 1100, 1300, 1500. Actual collection times were used to define peak plasma concentration (Cmax) and time to peak plasma concentration (Tmax).
Measured: At baseline
Participants Mean Level of Progesterone Area Under the Curve (AUC) - Daytime (0700-1500)
Participants Mean Level of Progesterone area under the curve (AUC) - Daytime (0700-1500). AUC was calculated using the linear-up log-down trapezoidal rule for the 8 hour daytime level, samples were obtained every 2 hours beginning at 0700, 0900, 1100, 1300, 1500. Actual collection times were used to define peak plasma concentration (Cmax) and time to peak plasma concentration (Tmax).
Measured: At 6 months
Participants Mean Level of Progesterone Area Under the Curve (AUC) - Midday (1500 - 2300)
Participants Mean Level of Progesterone area under the curve (AUC) - Midday (1500 - 2300). AUC was calculated using the linear-up log-down trapezoidal rule for the 8 hour midday level, samples were obtained every 2 hours beginning at 1500, 1700, 1900, 2100, 2300. Actual collection times were used to define peak plasma concentration (Cmax) and time to peak plasma concentration (Tmax).
Measured: At baseline
Participants Mean Level of Progesterone Area Under the Curve (AUC) - Midday (1500 - 2300)
Participants Mean Level of Progesterone area under the curve (AUC) - Midday (1500 - 2300). AUC was calculated using the linear-up log-down trapezoidal rule for the 8 hour midday level, samples were obtained every 2 hours beginning at 1500, 1700, 1900, 2100, 2300. Actual collection times were used to define peak plasma concentration (Cmax) and time to peak plasma concentration (Tmax).
Measured: At 6 months
Participants Mean Level of Progesterone Area Under the Curve (AUC) - Nighttime (2300 - 0700)
Participants Mean Level of Progesterone area under the curve (AUC) - Nighttime (2300 - 0700). AUC was calculated using the linear-up log-down trapezoidal rule for the 8 hour nighttime level, samples were obtained every 2 hours beginning at 2300, 0100, 0300, 0500, 0700. Actual collection times were used to define peak plasma concentration (Cmax) and time to peak plasma concentration (Tmax).
Measured: At baseline
Participants Mean Level of Progesterone Area Under the Curve (AUC) - Nighttime (2300 - 0700)
Participants Mean Level of Progesterone area under the curve (AUC) - Nighttime (2300 - 0700). AUC was calculated using the linear-up log-down trapezoidal rule for the 8 hour nighttime level, samples were obtained every 2 hours beginning at 2300, 0100, 0300, 0500, 0700. Actual collection times were used to define peak plasma concentration (Cmax) and time to peak plasma concentration (Tmax).
Measured: At 6 months
Participant Lean Body Mass
lean body mass measured by DEXA, Hologic Discovery-A
Measured: At baseline
Participant Lean Body Mass
lean body mass measured by DEXA, Hologic Discovery-A
Measured: At 6 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

6 May 20132 Dec 2016

First posted21 May 2013 (estimated)
Study start6 May 2013
Primary completion2 Dec 2016
Study completion2 Dec 2016

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (5)Papers the sponsor has linked to this study.
Other trials for Adrenal InsufficiencyStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 2
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 8 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01859312
Sponsor's protocol ID: 130121
Other IDs: 13-CH-0121
Status last verified: 2 Oct 2017
First submitted: 17 May 2013
Last updated: 22 Dec 2017
Results posted: Yes, on ClinicalTrials.gov
FDA-regulated drug: Yes
FDA-regulated device: No

Publications

Debono M, Ghobadi C, Rostami-Hodjegan A, Huatan H, Campbell MJ, Newell-Price J, Darzy K, Merke DP, Arlt W, Ross RJ. Modified-release hydrocortisone to provide circadian cortisol profiles. J Clin Endocrinol Metab. 2009 May;94(5):1548-54. doi: 10.1210/jc.2008-2380. Epub 2009 Feb 17.
PubMed 19223520
Finkielstain GP, Kim MS, Sinaii N, Nishitani M, Van Ryzin C, Hill SC, Reynolds JC, Hanna RM, Merke DP. Clinical characteristics of a cohort of 244 patients with congenital adrenal hyperplasia. J Clin Endocrinol Metab. 2012 Dec;97(12):4429-38. doi: 10.1210/jc.2012-2102. Epub 2012 Sep 18.
PubMed 22990093
Arlt W, Willis DS, Wild SH, Krone N, Doherty EJ, Hahner S, Han TS, Carroll PV, Conway GS, Rees DA, Stimson RH, Walker BR, Connell JM, Ross RJ; United Kingdom Congenital Adrenal Hyperplasia Adult Study Executive (CaHASE). Health status of adults with congenital adrenal hyperplasia: a cohort study of 203 patients. J Clin Endocrinol Metab. 2010 Nov;95(11):5110-21. doi: 10.1210/jc.2010-0917. Epub 2010 Aug 18.
PubMed 20719839
Nella AA, Mallappa A, Perritt AF, Gounden V, Kumar P, Sinaii N, Daley LA, Ling A, Liu CY, Soldin SJ, Merke DP. A Phase 2 Study of Continuous Subcutaneous Hydrocortisone Infusion in Adults With Congenital Adrenal Hyperplasia. J Clin Endocrinol Metab. 2016 Dec;101(12):4690-4698. doi: 10.1210/jc.2016-1916. Epub 2016 Sep 28.
PubMed 27680873
Mallappa A, Nella AA, Sinaii N, Rao H, Gounden V, Perritt AF, Kumar P, Ling A, Liu CY, Soldin SJ, Merke DP. Long-term use of continuous subcutaneous hydrocortisone infusion therapy in patients with congenital adrenal hyperplasia. Clin Endocrinol (Oxf). 2018 Oct;89(4):399-407. doi: 10.1111/cen.13813. Epub 2018 Aug 8.
PubMed 30003563

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What is the study trying to find out?
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See locations

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All Adrenal Insufficiency trials

Study at a glance

From the registry record, last updated by the sponsor on 22 Dec 2017. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Pilot Study Assessing the Use of Continuous Subcutaneous Hydrocortisone Infusion in the Treatment of Congenital Adrenal Hyperplasia
ClinicalTrials.gov ID: NCT01859312
Acronym / sponsor ID: 130121
Conditions: Adrenal Insufficiency, Excess Androgen, Congenital Adrenal Hyperplasia (CAH)
Keywords: Glucocorticoids, Adrenal Insufficiency, Congenital Adrenal Hyperplasia (CAH), Adrenal
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 8 participants (actual)
Lead sponsor: National Institutes of Health Clinical Center (CC)US National Institutes of Health
Collaborators: Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Study start: 6 May 2013
Primary completion: 2 Dec 2016 (actual)
Study completion: 2 Dec 2016 (actual)
First posted: 21 May 2013 (estimated)
Results: Posted 22 Nov 2017

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Background:

Congenital adrenal hyperplasia (CAH) is a genetic disorder of the adrenal gland. The adrenal gland is located in the abdomen and produces small amounts of hormones such as cortisol, aldosterone, and androgen. These hormones help control blood pressure, protect the body, and maintain good health, especially during development. People with CAH do not make enough cortisol and aldosterone, and make too much androgen. This can lead to serious medical problems. The standard treatment is to take pills that mimic the effects of cortisol and aldosterone. However, treatment with pills can have long-term side effects because of the higher doses needed, and may not work well for some people.
A possible new treatment for CAH is to use a pump to deliver cortisol under the skin. Similar pumps are often used to give insulin to people with diabetes. Researchers think that a cortisol pump might be able to help the body use the cortisol more effectively than taking pills. They want to compare the results of a cortisol pump and standard pill treatments for CAH.

Objectives:

To compare the effectiveness of a cortisol pump with standard cortisol pill therapy for CAH.

Eligibility:

Men and women at least 18 years of age who have CAH (see more details in Eligibility section below).

Design:

This study will involve four inpatient hospital stays at the National Institutes of Health in Bethesda, MD over 6 months (spaced 2 months apart). The first and last stays will last about 5 days. The second and third stays will last about 3 days.
Participants will be screened with a physical exam and medical history. Blood and urine samples will be collected.
At the first study visit, participants will provide regular blood and urine samples. They will also have imaging studies. These studies will look at the bones, fat, and muscles in the abdomen and pelvis.
Participants will receive a cortisol pump during the first visit. They will be shown how to use the pump. They will also learn what to do, if they need to take extra "stress dose" cortisol pills.
At the second and third visits, the cortisol dose given with the pump will be adjusted as needed. Blood and urine samples will also be collected. No imaging studies are scheduled for these visits.
The last study visit will have the same tests as the first visit. Participants will be offered the chance to continue with the pump treatment for 1 more year, or go back to their standard pill treatment.

Study type: Interventional non-randomized trial

Official title: A Pilot Study Assessing the use of Continuous Subcutaneous Hydrocortisone Infusion In the Treatment of Congenital Adrenal Hyperplasia

Estimated enrollment: 8

Study Start Date: May 2013

Estimated Study Completion Date: December 2016

Sponsoring Institute: National Institute of Child Health and Human Development

<TAB>ELIGIBILITY

Inclusion criteria

Men and women 18 years of age or older with classic congenital adrenal hyperplasia (21-Hydroxylase deficiency)
High adrenal androgens in the blood, and
One or more of the following conditions: obesity, fatty liver, risk for diabetes, low bone mass, inability to tolerate cortisol pills

Exclusion criteria

Pregnancy
Breast feeding
Use of inhaled or oral steroids for diseases other than CAH
Use of estrogen-containing birth control pills
Use of medicines that cross-react with hydrocortisone
Use of stress dose steroids for illness during the last 30 days prior to joining the study

Detailed description

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
Continuous Subcutaneous Hydrocortisone Infusion	Experimental	Drug: Hydrocortisone (Solucortef), Device: Insulin pump (Medtronic)Enrolled participants with congenital adrenal hyperplasia (CAH) received continuous subcutaneous hydrocortisone (Solucortef) infusion (CSHI) via insulin pump (Medtronic) (MMT-722Na) to achieve near-physiologic cortisol replacement therapy. Participants were their own controls; participant's baseline outcomes/lab values while on conventional glucocorticoid therapy were compared to outcomes/lab values after 6 months of treatment using CSHI via insulin pump.

Who may qualify

Ages: 18 to 99 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (10 listed)

Patients with known classic CAH due to 21-hydroxylase deficiency as evidenced by hormonal and genetic testing
Male or female patients 18 years or older
Females must have a negative pregnancy test initially and at all visits. Sexually active females must be using a medically acceptable method of contraception.
Patients with elevated adrenal androgens (defined as 17-OHP >1200 ng/dL and androstenedione >210 ng/dL)
One or more co-morbidities:<TAB>

Key exclusion criteria (8 listed)

Co-morbid conditions requiring daily administration of medications that induce hepatic enzymes or interfere with the metabolism of glucocorticoids
Females who are pregnant or lactating
Patients on inhaled or oral steroids given for reasons other than treatment of CAH
Women who have taken estrogen-containing oral contraceptive pills within 6 weeks of recruitment
Patients who required stress dose glucocorticoids for an illness within 4 weeks of recruitment

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
Hydrocortisone (Solucortef)Also: CSHIContinuous subcutaneous hydrocortisone infusion (CSHI) via Medtronic insulin pump (MMT-722NA). Total daily hydrocortisone dose was calculated based on the patient's estimated cortisol clearance. Rates were established to achieve peak and trough concentrations within the normal circadian cortisol range.	Drug	Continuous Subcutaneous Hydrocortisone Infusion
Insulin pump (Medtronic)Also: CSHIContinuous subcutaneous hydrocortisone infusion (CSHI) via Medtronic insulin pump (MMT-722NA)	Device	Continuous Subcutaneous Hydrocortisone Infusion

Primary outcome

Number of Patients With 17-OHP Levels Equal or Below 1,200 ng/dL at 0700
Time frame: At baseline
17-OHP (17-hydroxyprogesterone) is a laboratory blood test to test for congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency
Number of Patients With 17-OH Progesterone Levels Equal or Below 1,200 ng/dL at 0700
Time frame: At 6 months
17-OHP (17-hydroxyprogesterone) is a laboratory blood test to test for congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency

42 secondary outcomes

Participants Mean Level of 17-OHP at 0700
Time frame: At baseline
Participants Mean Level of 17-OHP at 0700
Time frame: At 6 months
Participants Mean Level of 17-OHP Area Under the Curve (AUC) - 24 Hours
Time frame: At baseline
Participants Mean Level of 17-OHP Area Under the Curve (AUC) - 24 Hours
Time frame: At 6 months
Participants Mean Level of 17-OHP Area Under the Curve (AUC) - Daytime (0700-1500)
Time frame: At baseline
Participants Mean Level of 17-OHP Area Under the Curve (AUC) - Daytime (0700-1500)
Time frame: At 6 months
Participants Mean Level of 17-OHP Area Under the Curve (AUC) - Midday (1500 - 2300)
Time frame: At baseline
Participants Mean Level of 17-OHP Area Under the Curve (AUC) - Midday (1500 - 2300)
Time frame: At 6 months
Participants Mean Level of 17-OHP Area Under the Curve (AUC) - Nighttime (2300 - 0700)
Time frame: At baseline
Participants Mean Level of 17-OHP Area Under the Curve (AUC) - Nighttime (2300 - 0700)
Time frame: At 6 months
Participants Mean Level of Androstenedione at 0700
Time frame: At baseline
Participants Mean Level of Androstenedione at 0700
Time frame: At 6 months
Participants Mean Level of Androstenedione Area Under the Curve (AUC) - 24 Hours
Time frame: At baseline
Participants Mean Level of Androstenedione Area Under the Curve (AUC) - 24 Hours
Time frame: At 6 months
Participants Mean Level of Androstenedione Area Under the Curve (AUC) - Daytime (0700-1500)
Time frame: At baseline
Participants Mean Level of Androstenedione Area Under the Curve (AUC) - Daytime (0700-1500)
Time frame: At 6 months
Participants Mean Level of Androstenedione Area Under the Curve (AUC) - Midday (1500 - 2300)
Time frame: At baseline
Participants Mean Level of Androstenedione Area Under the Curve (AUC) - Midday (1500 - 2300)
Time frame: At 6 months
Participants Mean Level of Androstenedione Area Under the Curve (AUC) - Nighttime (2300 - 0700)
Time frame: At baseline
Participants Mean Level of Androstenedione Area Under the Curve (AUC) - Nighttime (2300 - 0700)
Time frame: At 6 months
Participants Mean Level of ACTH at 0700
Time frame: At baseline
Participants Mean Level of ACTH at 0700
Time frame: At 6 months
Participants Mean Level of ACTH Area Under the Curve (AUC) - 24 Hours
Time frame: At baseline
Participants Mean Level of ACTH Area Under the Curve (AUC) - 24 Hours
Time frame: At 6 months
Participants Mean Level of ACTH Area Under the Curve (AUC) - Daytime (0700-1500)
Time frame: At baseline
Participants Mean Level of ACTH Area Under the Curve (AUC) - Daytime (0700-1500)
Time frame: At 6 months
Participants Mean Level of ACTH Area Under the Curve (AUC) - Midday (1500 - 2300)
Time frame: At baseline
Participants Mean Level of ACTH Area Under the Curve (AUC) - Midday (1500 - 2300)
Time frame: At 6 months
Participants Mean Level of ACTH Area Under the Curve (AUC) - Nighttime (2300 - 0700)
Time frame: At baseline
Participants Mean Level of ACTH Area Under the Curve (AUC) - Nighttime (2300 - 0700)
Time frame: At 6 months
Participants Mean Progesterone Levels at 0700
Time frame: At baseline
Participants Mean Progesterone Level at 0700
Time frame: At 6 months
Participants Mean Level of Progesterone Area Under the Curve (AUC) - 24 Hours
Time frame: At baseline
Participants Mean Level of Progesterone Area Under the Curve (AUC) - 24 Hours
Time frame: At 6 months
Participants Mean Level of Progesterone Area Under the Curve (AUC) - Daytime (0700-1500)
Time frame: At baseline
Participants Mean Level of Progesterone Area Under the Curve (AUC) - Daytime (0700-1500)
Time frame: At 6 months
Participants Mean Level of Progesterone Area Under the Curve (AUC) - Midday (1500 - 2300)
Time frame: At baseline
Participants Mean Level of Progesterone Area Under the Curve (AUC) - Midday (1500 - 2300)
Time frame: At 6 months
Participants Mean Level of Progesterone Area Under the Curve (AUC) - Nighttime (2300 - 0700)
Time frame: At baseline
Participants Mean Level of Progesterone Area Under the Curve (AUC) - Nighttime (2300 - 0700)
Time frame: At 6 months

Results and safety

Results posted to the registry on 22 Nov 2017, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Continuous Sub-Q Hydrocortisone Infusion	8	8

Primary outcome: Number of Patients With 17-OHP Levels Equal or Below 1,200 ng/dL at 0700

Time frame: At baseline. Count of participants. Unit: Participants

Measure	Continuous Subcutaneous Hydrocortisone Infusion n = 8
Value	0

Primary outcome: Number of Patients With 17-OH Progesterone Levels Equal or Below 1,200 ng/dL at 0700

Time frame: At 6 months. Count of participants. Unit: Participants

Measure	Continuous Subcutaneous Hydrocortisone Infusion n = 8
Value	3

42 secondary outcomes are reported in the full results.

Adverse events

Time frame: At 6 months. Subjects that select the option of continuing CSHI therapy for 1 additional year will be monitored for 1 year, 6 months.. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Continuous Subcutaneous Hydrocortisone Infusion	0 of 8 (0%)	8 of 8 (100%)	0 of 8

Limitations and caveats (from the sponsor): All patients completed the study; according to medication and supply accountability, one patient was found to be partially non-compliant, the pump was disconnected approximately 30% of the time. When this patient was excluded, analysis were the same.

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 22 Dec 2017.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Background: PubMed 19223520Debono M, Ghobadi C, Rostami-Hodjegan A, Huatan H, Campbell MJ, Newell-Price J, Darzy K, Merke DP, Arlt W, Ross RJ. Modified-release hydrocortisone to provide circadian cortisol profiles. J Clin Endocrinol Metab. 2009 May;94(5):1548-54. doi: 10.1210/jc.2008-2380. Epub 2009 Feb 17.
Background: PubMed 22990093Finkielstain GP, Kim MS, Sinaii N, Nishitani M, Van Ryzin C, Hill SC, Reynolds JC, Hanna RM, Merke DP. Clinical characteristics of a cohort of 244 patients with congenital adrenal hyperplasia. J Clin Endocrinol Metab. 2012 Dec;97(12):4429-38. doi: 10.1210/jc.2012-2102. Epub 2012 Sep 18.
Background: PubMed 20719839Arlt W, Willis DS, Wild SH, Krone N, Doherty EJ, Hahner S, Han TS, Carroll PV, Conway GS, Rees DA, Stimson RH, Walker BR, Connell JM, Ross RJ; United Kingdom Congenital Adrenal Hyperplasia Adult Study Executive (CaHASE). Health status of adults with congenital adrenal hyperplasia: a cohort study of 203 patients. J Clin Endocrinol Metab. 2010 Nov;95(11):5110-21. doi: 10.1210/jc.2010-0917. Epub 2010 Aug 18.
Results publication: PubMed 27680873Nella AA, Mallappa A, Perritt AF, Gounden V, Kumar P, Sinaii N, Daley LA, Ling A, Liu CY, Soldin SJ, Merke DP. A Phase 2 Study of Continuous Subcutaneous Hydrocortisone Infusion in Adults With Congenital Adrenal Hyperplasia. J Clin Endocrinol Metab. 2016 Dec;101(12):4690-4698. doi: 10.1210/jc.2016-1916. Epub 2016 Sep 28.
Derived from this study: PubMed 30003563Mallappa A, Nella AA, Sinaii N, Rao H, Gounden V, Perritt AF, Kumar P, Ling A, Liu CY, Soldin SJ, Merke DP. Long-term use of continuous subcutaneous hydrocortisone infusion therapy in patients with congenital adrenal hyperplasia. Clin Endocrinol (Oxf). 2018 Oct;89(4):399-407. doi: 10.1111/cen.13813. Epub 2018 Aug 8.
NIH Clinical Center Detailed Web PageLink provided by the sponsor.

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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Deborah P Merke, M.D. principal investigator

National Institutes of Health Clinical Center (CC)

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 10 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 22 Dec 2017. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 22 Nov 2017). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of Patients With 17-OHP Levels Equal or Below 1,200 ng/dL at 0700 (time frame: At baseline). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01859312, which also lists 5 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Dec 2017.