21-hydroxylase DeficiencyPhase 1NCT01495910
A Study Examining Doses of Abiraterone Acetate in Adult Women With 21-Hydroxylase Deficiency
A Phase 1 study testing Abiraterone acetate in people with 21-hydroxylase Deficiency, run by Johnson & Johnson Pharmaceutical Research & Development, L.L.C. at 3 sites in United States.
Trial summary
The purpose of this study is to determine the minimum dose of abiraterone acetate needed to decrease serum androstenedione to age-appropriate levels in premenopausal women on steroid replacement for classic 21-hydroxylase deficiency. Read more about this study
- Who is it for?
- Ages 18 years and older, women only
- Conditions the study is for
- 21-hydroxylase Deficiency
- What is being tested?
- Abiraterone acetate
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 3 sitesUnited States
- Study dates
- Dec 2011to Feb 2013
- Phase
- Phase 1
- 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 18 years and older
- Women only
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Premenopausal women >=18 years of age.
- Must be receiving a hormonal contraceptive agent containing both estrogen and progesterone.
- Confirmed 21-hydroxylase deficiency by CYP21A2 genotype associated with classic congenital adrenal hyperplasia.
- Demonstrates a >=1.5 X ULN of morning serum androstenedione concentration while taking study-defined doses of hydrocortisone and fludrocortisone.
- No coexisting medical conditions in the opinion of the investigator that would preclude participation in the study.
Who cannot join
- Current or history of active or chronic hepatitis, including symptomatic viral hepatitis A, B, or C.
- Any active infection.
- Evidence of active malignancy.
- Serious or uncontrolled co-existent non-malignant disease.
- Receiving systemic glucocorticoids for any reason other than for the treatment of 21-hydroxylase deficiency.
- Any disorders that require treatment with anticonvulsants.
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- Patients of child-bearing potential who are not willing to use a method of birth control during the study and for 3 months after the end-of-study.
- Women who are pregnant or breast-feeding.
- Genotypes associated with non-classic congenital adrenal hyperplasia.
Trial locations and contacts
3 sites in 1 country.
United States3 sites
Study site
Ann Arbor, Michigan
Study site
Chicago, Illinois
Study site
Dallas, Texas
About this study
Official title: An Open-Label, Multiple-Dose, Dose-Finding Study of Abiraterone Acetate in Adult Women With 21-Hydroxylase Deficiency
The purpose of this study is to determine the minimum dose of abiraterone acetate needed to decrease serum androstenedione to age-appropriate levels in premenopausal women on steroid replacement for classic 21-hydroxylase deficiency.
Read the detailed description
This is a non-randomized (patients will not be assigned by chance to study treatments), open-label (patients will know the identity of study treatments), multiple-dose, intra-patient sequential dose-escalation study with a planned enrollment of approximately 10 patients. This study will consist of a screening period and a treatment period. Due to the intra-patient dose escalation, there will be multiple treatment periods consisting of 8 days each. A rest period of at least 7 days will separate each treatment period. Eligible patients will take study-defined replacement doses of hydrocortisone and fludrocortisone. Abiraterone acetate oral suspension will be administered in daily escalating doses from 100 mg to 500 mg. Patients will proceed to the next higher dose level when the majority of the treated patients have a reduction in the androstenedione level. Serial pharmacokinetic (study of what the body does to a drug) and pharmacodynamic (study of the effects of a drug on the body) samples will be collected at each treatment period as detailed in the protocol. All patients who receive at least 1 dose of abiraterone acetate will be analyzed for safety.
- Conditions
- 21-hydroxylase Deficiency
- Being tested
- Abiraterone acetate
- Keywords
- 21-hydroxylase deficiency, Androstenedione, Abiraterone acetate, Pharmacokinetics, Pharmacodynamics, Dose-finding
- Medical subject headings
- Congenital adrenal hyperplasia due to 21 hydroxylase deficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Abiraterone acetateDrug
Abiraterone acetate oral suspension administered daily from study Day 1 to study Day 6 of each treatment period: the first dose level is 100 mg with escalating doses of 250 mg and 500 mg in subsequent treatment periods.
| Study group | Role | What they receive |
|---|---|---|
| Abiraterone acetate | Experimental | Abiraterone acetate oral suspension administered daily from study Day 1 to study Day 6 of each treatment period: the first dose level is 100 mg with escalating doses of 250 mg and 500 mg in subsequent treatment periods. Receives: Drug: Abiraterone acetate |
What is being measured
The primary outcome is the main question the study is designed to answer.
The minimum dose of abiraterone acetate required to decrease serum androstenedione to the age-appropriate range for adult women with 21-hydroxylase deficiency
Normalization or reduction of age-appropriate androstenedione levels will be determined by the mean of the androstenedione values measured on Study Days 6 and 7 of the treatment period.
Measured: Up to Day 7 of each treatment period.
Secondary outcomes (12)
Mean serum concentrations of androstenedione
Measured: Up to Day 8 of each treatment period.
Mean serum concentrations of 17-hydroxyprogesterone
Measured: Up to Day 8 of each treatment period.
Mean plasma concentrations of renin activity
Measured: Up to Day 8 of each treatment period.
Mean serum concentrations of testosterone
Measured: Up to Day 8 of each treatment period.
Urine concentrations of androsterone
Measured: Up to Day 8 of each treatment period.
Urine concentrations of etiocholanolone
Measured: Up to Day 8 of each treatment period.
Maximum plasma concentration (Cmax) of abiraterone
Measured: Up to Day 8 of each treatment period.
Time to reach the maximum plasma concentration (tmax) of abiraterone
Measured: Up to Day 8 of each treatment period.
Area under the plasma concentration-time curve from time 0 to 24 hours postdose (AUC24) of abiraterone
Measured: Up to Day 8 of each treatment period.
Area under the plasma concentration-time curve from time 0 to time of the last quantifiable concentration (AUClast) of abiraterone
Measured: Up to Day 8 of each treatment period.
Time to last quantifiable plasma concentration (Tlast) of abiraterone
Measured: Up to Day 8 of each treatment period.
Elimination half-life associated with the terminal slope of the semilogarithmic drug concentration-time curve of abiraterone
Measured: Up to Day 8 of each treatment period.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Dec 2011 (estimated)
- Study startDec 2011
- Primary completionFeb 2013
- Study completionFeb 2013
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for 21-hydroxylase DeficiencyStudies 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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- Johnson & Johnson Pharmaceutical Research & Development, L.L.C.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Johnson & Johnson Pharmaceutical Research and Development, L.L.C., Clinical Trial, Study director, Johnson & Johnson Pharmaceutical Research & Development, L.L.C.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01495910
- Sponsor's protocol ID
- CR100007
- Other IDs
- 212082HPL1002
- Status last verified
- Feb 2014
- First submitted
- 23 Nov 2011
- Last updated
- 28 Feb 2014
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 28 Feb 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label, Multiple-Dose, Dose-Finding Study of Abiraterone Acetate in Adult Women With 21-Hydroxylase Deficiency
- ClinicalTrials.gov ID
- NCT01495910
- Acronym / sponsor ID
- CR100007
- Conditions
- 21-hydroxylase Deficiency
- Keywords
- 21-hydroxylase deficiency, Androstenedione, Abiraterone acetate, Pharmacokinetics, Pharmacodynamics, Dose-finding
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 6 participants (actual)
- Lead sponsor
- Johnson & Johnson Pharmaceutical Research & Development, L.L.C.Industry (pharma, biotech, devices)
- Study start
- Dec 2011
- Primary completion
- Feb 2013 (actual)
- Study completion
- Feb 2013 (actual)
- First posted
- 20 Dec 2011 (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 determine the minimum dose of abiraterone acetate needed to decrease serum androstenedione to age-appropriate levels in premenopausal women on steroid replacement for classic 21-hydroxylase deficiency.
Detailed description
This is a non-randomized (patients will not be assigned by chance to study treatments), open-label (patients will know the identity of study treatments), multiple-dose, intra-patient sequential dose-escalation study with a planned enrollment of approximately 10 patients. This study will consist of a screening period and a treatment period. Due to the intra-patient dose escalation, there will be multiple treatment periods consisting of 8 days each. A rest period of at least 7 days will separate each treatment period. Eligible patients will take study-defined replacement doses of hydrocortisone and fludrocortisone. Abiraterone acetate oral suspension will be administered in daily escalating doses from 100 mg to 500 mg. Patients will proceed to the next higher dose level when the majority of the treated patients have a reduction in the androstenedione level. Serial pharmacokinetic (study of what the body does to a drug) and pharmacodynamic (study of the effects of a drug on the body) samples will be collected at each treatment period as detailed in the protocol. All patients who receive at least 1 dose of abiraterone acetate will be analyzed for safety.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Abiraterone acetate | Experimental | Drug: Abiraterone acetateAbiraterone acetate oral suspension administered daily from study Day 1 to study Day 6 of each treatment period: the first dose level is 100 mg with escalating doses of 250 mg and 500 mg in subsequent treatment periods. |
Who may qualify
- Ages
- 18 years and older
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Premenopausal women >=18 years of age.
- Must be receiving a hormonal contraceptive agent containing both estrogen and progesterone.
- Confirmed 21-hydroxylase deficiency by CYP21A2 genotype associated with classic congenital adrenal hyperplasia.
- Demonstrates a >=1.5 X ULN of morning serum androstenedione concentration while taking study-defined doses of hydrocortisone and fludrocortisone.
- No coexisting medical conditions in the opinion of the investigator that would preclude participation in the study.
Key exclusion criteria (9 listed)
- Current or history of active or chronic hepatitis, including symptomatic viral hepatitis A, B, or C.
- Any active infection.
- Evidence of active malignancy.
- Serious or uncontrolled co-existent non-malignant disease.
- Receiving systemic glucocorticoids for any reason other than for the treatment of 21-hydroxylase deficiency.
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 |
|---|---|---|
| Abiraterone acetateAbiraterone acetate oral suspension administered daily from study Day 1 to study Day 6 of each treatment period: the first dose level is 100 mg with escalating doses of 250 mg and 500 mg in subsequent treatment periods. | Drug | Abiraterone acetate |
Primary outcome
The minimum dose of abiraterone acetate required to decrease serum androstenedione to the age-appropriate range for adult women with 21-hydroxylase deficiency
Time frame: Up to Day 7 of each treatment period.
Normalization or reduction of age-appropriate androstenedione levels will be determined by the mean of the androstenedione values measured on Study Days 6 and 7 of the treatment period.
12 secondary outcomes
Mean serum concentrations of androstenedione
Time frame: Up to Day 8 of each treatment period.
Mean serum concentrations of 17-hydroxyprogesterone
Time frame: Up to Day 8 of each treatment period.
Mean plasma concentrations of renin activity
Time frame: Up to Day 8 of each treatment period.
Mean serum concentrations of testosterone
Time frame: Up to Day 8 of each treatment period.
Urine concentrations of androsterone
Time frame: Up to Day 8 of each treatment period.
Urine concentrations of etiocholanolone
Time frame: Up to Day 8 of each treatment period.
Maximum plasma concentration (Cmax) of abiraterone
Time frame: Up to Day 8 of each treatment period.
Time to reach the maximum plasma concentration (tmax) of abiraterone
Time frame: Up to Day 8 of each treatment period.
Area under the plasma concentration-time curve from time 0 to 24 hours postdose (AUC24) of abiraterone
Time frame: Up to Day 8 of each treatment period.
Area under the plasma concentration-time curve from time 0 to time of the last quantifiable concentration (AUClast) of abiraterone
Time frame: Up to Day 8 of each treatment period.
Time to last quantifiable plasma concentration (Tlast) of abiraterone
Time frame: Up to Day 8 of each treatment period.
Elimination half-life associated with the terminal slope of the semilogarithmic drug concentration-time curve of abiraterone
Time frame: Up to Day 8 of each treatment period.
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 28 Feb 2014.
- An Open-Label, Multiple-Dose, Dose-Finding Study of Abiraterone Acetate in Adult Women With 21-Hydroxylase DeficiencyLink 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.
Johnson & Johnson Pharmaceutical Research and Development, L.L.C., Clinical Trial study director
Johnson & Johnson Pharmaceutical Research & Development, L.L.C.
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Abiraterone acetate in people with 21-hydroxylase Deficiency, run by Johnson & Johnson Pharmaceutical Research & Development, L.L.C. at 3 sites 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 9 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 28 Feb 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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?
The minimum dose of abiraterone acetate required to decrease serum androstenedione to the age-appropriate range for adult women with 21-hydroxylase deficiency (time frame: Up to Day 7 of each treatment period.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 28 Feb 2014.