Adrenal Hyperplasia, CongenitalNCT00529841
Research Study for Children With Salt Wasting Congenital Adrenal Hyperplasia
A study testing Hydrocortisone sodium acetate in people with Adrenal Hyperplasia, Congenital, run by Baylor College of Medicine at 1 site in United States.
Trial summary
The purpose of this study is to develop a more physiological approach to the management of children and adolescents with salt wasting Congenital Adrenal Hyperplasia. We will administer the glucocorticosteroid via insulin infusion pump to see whether this treatment will improve the serum hormone concentrations. Read more about this study
- Who is it for?
- Ages 3 to 18 years
- Conditions the study is for
- Adrenal Hyperplasia, Congenital
- What is being tested?
- Hydrocortisone sodium acetate
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 7 participants
- Where
- 1 siteUnited States
- Study dates
- Jan 2007to Sep 2008
- Phase
- Interventional
- Are there results?
- None posted in the registry
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 3 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Children with salt wasting CAH otherwise healthy without other chronic disease
- Age: between 3 and 18 years of age
- Body weight 23 kg (50 lbs) or above
- Hemoglobin equal to or higher than 12 g/dl before the study
- Supportive family environment
Who cannot join
- Age less than 3 or older than 18 years at the time of study
- Other chronic disease
- Hemoglobin less than 12 g/dl
- Non-supportive family
- Allergy to local anesthetics
- Criteria for study termination: If the subject's parents are unable to manage/operate the pump, the subject will be withdrawn from the study.
Trial locations and contacts
1 site in 1 country.
United States1 site
BCM, Texas Children's Hospital Clinic and General Clinical Research Center
Houston, Texas, 77030
About this study
Official title: A Novel Therapeutic Modality for Congenital Adrenal Hyperplasia
The purpose of this study is to develop a more physiological approach to the management of children and adolescents with salt wasting Congenital Adrenal Hyperplasia.
We will administer the glucocorticosteroid via insulin infusion pump to see whether this treatment will improve the serum hormone concentrations.
Read the detailed description
The adrenal gland is a small organ of the body. It produces very important chemicals called hormones. One of these hormones, cortisol (the stress hormone) helps the body fight diseases. The other hormone is the aldosterone helps to maintain the normal amount of salt and water in the body. The third type of hormones are the androgens or male hormones, which cause some of the changes during puberty, like the growth of body hair and pimples.
The salt wasting Congenital Adrenal Hyperplasia or CAH disease is a disease of the adrenal gland. Patients with this disease cannot make cortisol or the aldosterone. As a result, their body cannot fight diseases and cannot keep normal amounts of salt and water in the body. At the same time, the gland makes too much of the male hormones, which is bad for the body because too much male hormone slows down growth, increases the growth of body hair, and causes pimples and abnormal period in girls.
Patients with this disease have to take medications every day. However, the treatment does not work very well, because usually the patients do not have the right amount of hormone in their body. Usually the body gets too much hormone right after taking the pills. A couple of hours later the body has too little of the hormones, because in the meantime the body gets rid of the medication.The healthy adrenal gland makes the hormones throughout the day in different amounts. The patients with this disease take the medication only a couple of times a day. They take the Florinef tablet once a day and the Cortisol tablet two or three times a day. The treatment that we use today by mouth cannot copy the natural hormone productions of the adrenal gland. Because of this it does not make much of a difference in the patient's life.
We would like to improve the treatment and find out the effect of a new treatment. In this study we will try to imitate the body's normal hormone production and will give the medication via an insulin pump to see if this treatment method will decrease the male hormones in the blood. This study will help us to develop a new and better treatment for children and adolescents.
- Conditions
- Adrenal Hyperplasia, Congenital
- Being tested
- Hydrocortisone sodium acetate
- Keywords
- Salt Wasting Congenital Adrenal Hyperplasia, Subcutaneous Hydrocortisone
- Medical subject headings
- Adrenal Hyperplasia, Congenital
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Hydrocortisone sodium acetateDrug
Also called Solu-Cortef injection
Subcutaneous administration of medication via insulin pump
| Study group | Role | What they receive |
|---|---|---|
| 1 (Hydrocortisone sodium acetate) | Experimental | Subcutaneous administration of Hydrocortisone sodium acetate via insulin pump Receives: Drug: Hydrocortisone sodium acetate |
What is being measured
The primary outcome is the main question the study is designed to answer.
Serum 17-OHP concentration in the morning
Measured: 11 days
Secondary outcomes (3)
serum steroid hormone profiles
Measured: 11 days
serum blood glucose
Measured: study days 2,3 and 11
serum sodium
Measured: study days 2,3 and 11
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Sep 2007 (estimated)
- Study startJan 2007
- Primary completionSep 2008
- Study completionSep 2008
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (12)Papers the sponsor has linked to this study.
- Other trials for Adrenal Hyperplasia, CongenitalStudies of the same condition, open and under way.
- More trials run by Baylor College of MedicineThe sponsor's other registered studies.
- 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 N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 7 participants (actual)
Sponsor and investigators
- Lead sponsor
- Baylor College of Medicine
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Morey W Haymond, MD, Principal investigator, Baylor College of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00529841
- Sponsor's protocol ID
- H-19704
- Other IDs
- GCRC # 0962
- Status last verified
- Sep 2015
- First submitted
- 12 Sep 2007
- Last updated
- 14 Sep 2015
- Results posted
- No
- Data monitoring committee
- No
Publications
Esteban NV, Loughlin T, Yergey AL, Zawadzki JK, Booth JD, Winterer JC, Loriaux DL. Daily cortisol production rate in man determined by stable isotope dilution/mass spectrometry. J Clin Endocrinol Metab. 1991 Jan;72(1):39-45. doi: 10.1210/jcem-72-1-39.
PubMed 1986026Kerrigan JR, Veldhuis JD, Leyo SA, Iranmanesh A, Rogol AD. Estimation of daily cortisol production and clearance rates in normal pubertal males by deconvolution analysis. J Clin Endocrinol Metab. 1993 Jun;76(6):1505-10. doi: 10.1210/jcem.76.6.8501158.
PubMed 8501158Speiser PW. Toward better treatment of congenital adrenal hyperplasia. Clin Endocrinol (Oxf). 1999 Sep;51(3):273-4. doi: 10.1046/j.1365-2265.1999.00780.x. No abstract available.
PubMed 10469004Cutler GB Jr, Laue L. Congenital adrenal hyperplasia due to 21-hydroxylase deficiency. N Engl J Med. 1990 Dec 27;323(26):1806-13. doi: 10.1056/NEJM199012273232605. No abstract available.
PubMed 2247119Winterer J, Chrousos GP, Loriaux DL, Cutler GB Jr. Effect of hydrocortisone dose schedule on adrenal steroid secretion in congenital adrenal hyperplasia. J Pediatr. 1985 Jan;106(1):137-42. doi: 10.1016/s0022-3476(85)80486-8.
PubMed 3871229Wallace WH, Crowne EC, Shalet SM, Moore C, Gibson S, Littley MD, White A. Episodic ACTH and cortisol secretion in normal children. Clin Endocrinol (Oxf). 1991 Mar;34(3):215-21. doi: 10.1111/j.1365-2265.1991.tb00297.x.
PubMed 1645237Merza Z, Rostami-Hodjegan A, Memmott A, Doane A, Ibbotson V, Newell-Price J, Tucker GT, Ross RJ. Circadian hydrocortisone infusions in patients with adrenal insufficiency and congenital adrenal hyperplasia. Clin Endocrinol (Oxf). 2006 Jul;65(1):45-50. doi: 10.1111/j.1365-2265.2006.02544.x.
PubMed 16817818Lukert BP. Editorial: glucocorticoid replacement--how much is enough? J Clin Endocrinol Metab. 2006 Mar;91(3):793-4. doi: 10.1210/jc.2005-2737. No abstract available.
PubMed 16522704Claude J.Migeon. Can the Long Range Results of the Treatment of Congenital Adrenal Hyperplasia be improved? JCEM 1996 Vol 81, No 9 3187-3189
Laue L, Merke DP, Jones JV, Barnes KM, Hill S, Cutler GB Jr. A preliminary study of flutamide, testolactone, and reduced hydrocortisone dose in the treatment of congenital adrenal hyperplasia. J Clin Endocrinol Metab. 1996 Oct;81(10):3535-9. doi: 10.1210/jcem.81.10.8855797.
PubMed 8855797Mah PM, Jenkins RC, Rostami-Hodjegan A, Newell-Price J, Doane A, Ibbotson V, Tucker GT, Ross RJ. Weight-related dosing, timing and monitoring hydrocortisone replacement therapy in patients with adrenal insufficiency. Clin Endocrinol (Oxf). 2004 Sep;61(3):367-75. doi: 10.1111/j.1365-2265.2004.02106.x.
PubMed 15355454Sheila K.Gunn et al Subcutaneous Hydrocortisone Delivery mimics Physiologic Cortisol Concentrations, Poster, Endocrine Society Meeting 2000
Study at a glance
From the registry record, last updated by the sponsor on 14 Sep 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Novel Therapeutic Modality for Congenital Adrenal Hyperplasia
- ClinicalTrials.gov ID
- NCT00529841
- Acronym / sponsor ID
- H-19704
- Conditions
- Adrenal Hyperplasia, Congenital
- Keywords
- Salt Wasting Congenital Adrenal Hyperplasia, Subcutaneous Hydrocortisone
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 7 participants (actual)
- Lead sponsor
- Baylor College of MedicineUniversity, hospital or other
- Study start
- Jan 2007
- Primary completion
- Sep 2008 (actual)
- Study completion
- Sep 2008 (actual)
- First posted
- 14 Sep 2007 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to develop a more physiological approach to the management of children and adolescents with salt wasting Congenital Adrenal Hyperplasia.
We will administer the glucocorticosteroid via insulin infusion pump to see whether this treatment will improve the serum hormone concentrations.
Detailed description
The adrenal gland is a small organ of the body. It produces very important chemicals called hormones. One of these hormones, cortisol (the stress hormone) helps the body fight diseases. The other hormone is the aldosterone helps to maintain the normal amount of salt and water in the body. The third type of hormones are the androgens or male hormones, which cause some of the changes during puberty, like the growth of body hair and pimples.
The salt wasting Congenital Adrenal Hyperplasia or CAH disease is a disease of the adrenal gland. Patients with this disease cannot make cortisol or the aldosterone. As a result, their body cannot fight diseases and cannot keep normal amounts of salt and water in the body. At the same time, the gland makes too much of the male hormones, which is bad for the body because too much male hormone slows down growth, increases the growth of body hair, and causes pimples and abnormal period in girls.
Patients with this disease have to take medications every day. However, the treatment does not work very well, because usually the patients do not have the right amount of hormone in their body. Usually the body gets too much hormone right after taking the pills. A couple of hours later the body has too little of the hormones, because in the meantime the body gets rid of the medication.The healthy adrenal gland makes the hormones throughout the day in different amounts. The patients with this disease take the medication only a couple of times a day. They take the Florinef tablet once a day and the Cortisol tablet two or three times a day. The treatment that we use today by mouth cannot copy the natural hormone productions of the adrenal gland. Because of this it does not make much of a difference in the patient's life.
We would like to improve the treatment and find out the effect of a new treatment. In this study we will try to imitate the body's normal hormone production and will give the medication via an insulin pump to see if this treatment method will decrease the male hormones in the blood. This study will help us to develop a new and better treatment for children and adolescents.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 1 (Hydrocortisone sodium acetate) | Experimental | Drug: Hydrocortisone sodium acetateSubcutaneous administration of Hydrocortisone sodium acetate via insulin pump |
Who may qualify
- Ages
- 3 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 listed)
- Children with salt wasting CAH otherwise healthy without other chronic disease
- Age: between 3 and 18 years of age
- Body weight 23 kg (50 lbs) or above
- Hemoglobin equal to or higher than 12 g/dl before the study
- Supportive family environment
Key exclusion criteria (6 listed)
- Age less than 3 or older than 18 years at the time of study
- Other chronic disease
- Hemoglobin less than 12 g/dl
- Non-supportive family
- Allergy to local anesthetics
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 sodium acetateAlso: Solu-Cortef injectionSubcutaneous administration of medication via insulin pump | Drug | 1 (Hydrocortisone sodium acetate) |
Primary outcome
Serum 17-OHP concentration in the morning
Time frame: 11 days
3 secondary outcomes
serum steroid hormone profiles
Time frame: 11 days
serum blood glucose
Time frame: study days 2,3 and 11
serum sodium
Time frame: study days 2,3 and 11
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Sep 2015.
- Background: PubMed 1986026Esteban NV, Loughlin T, Yergey AL, Zawadzki JK, Booth JD, Winterer JC, Loriaux DL. Daily cortisol production rate in man determined by stable isotope dilution/mass spectrometry. J Clin Endocrinol Metab. 1991 Jan;72(1):39-45. doi: 10.1210/jcem-72-1-39.
- Background: PubMed 8501158Kerrigan JR, Veldhuis JD, Leyo SA, Iranmanesh A, Rogol AD. Estimation of daily cortisol production and clearance rates in normal pubertal males by deconvolution analysis. J Clin Endocrinol Metab. 1993 Jun;76(6):1505-10. doi: 10.1210/jcem.76.6.8501158.
- Background: PubMed 10469004Speiser PW. Toward better treatment of congenital adrenal hyperplasia. Clin Endocrinol (Oxf). 1999 Sep;51(3):273-4. doi: 10.1046/j.1365-2265.1999.00780.x. No abstract available.
- Background: PubMed 2247119Cutler GB Jr, Laue L. Congenital adrenal hyperplasia due to 21-hydroxylase deficiency. N Engl J Med. 1990 Dec 27;323(26):1806-13. doi: 10.1056/NEJM199012273232605. No abstract available.
- Background: PubMed 3871229Winterer J, Chrousos GP, Loriaux DL, Cutler GB Jr. Effect of hydrocortisone dose schedule on adrenal steroid secretion in congenital adrenal hyperplasia. J Pediatr. 1985 Jan;106(1):137-42. doi: 10.1016/s0022-3476(85)80486-8.
- Background: PubMed 1645237Wallace WH, Crowne EC, Shalet SM, Moore C, Gibson S, Littley MD, White A. Episodic ACTH and cortisol secretion in normal children. Clin Endocrinol (Oxf). 1991 Mar;34(3):215-21. doi: 10.1111/j.1365-2265.1991.tb00297.x.
- Background: PubMed 16817818Merza Z, Rostami-Hodjegan A, Memmott A, Doane A, Ibbotson V, Newell-Price J, Tucker GT, Ross RJ. Circadian hydrocortisone infusions in patients with adrenal insufficiency and congenital adrenal hyperplasia. Clin Endocrinol (Oxf). 2006 Jul;65(1):45-50. doi: 10.1111/j.1365-2265.2006.02544.x.
- Background: PubMed 16522704Lukert BP. Editorial: glucocorticoid replacement--how much is enough? J Clin Endocrinol Metab. 2006 Mar;91(3):793-4. doi: 10.1210/jc.2005-2737. No abstract available.
- BackgroundClaude J.Migeon. Can the Long Range Results of the Treatment of Congenital Adrenal Hyperplasia be improved? JCEM 1996 Vol 81, No 9 3187-3189
- Background: PubMed 8855797Laue L, Merke DP, Jones JV, Barnes KM, Hill S, Cutler GB Jr. A preliminary study of flutamide, testolactone, and reduced hydrocortisone dose in the treatment of congenital adrenal hyperplasia. J Clin Endocrinol Metab. 1996 Oct;81(10):3535-9. doi: 10.1210/jcem.81.10.8855797.
- Background: PubMed 15355454Mah PM, Jenkins RC, Rostami-Hodjegan A, Newell-Price J, Doane A, Ibbotson V, Tucker GT, Ross RJ. Weight-related dosing, timing and monitoring hydrocortisone replacement therapy in patients with adrenal insufficiency. Clin Endocrinol (Oxf). 2004 Sep;61(3):367-75. doi: 10.1111/j.1365-2265.2004.02106.x.
- BackgroundSheila K.Gunn et al Subcutaneous Hydrocortisone Delivery mimics Physiologic Cortisol Concentrations, Poster, Endocrine Society Meeting 2000
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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.
Morey W Haymond, MD principal investigator
Baylor College of Medicine
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Hydrocortisone sodium acetate in people with Adrenal Hyperplasia, Congenital, run by Baylor College of Medicine at 1 site in United States.
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 5 inclusion and 6 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 14 Sep 2015. 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.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Serum 17-OHP concentration in the morning (time frame: 11 days). 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 NCT00529841, which also lists 12 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 14 Sep 2015.