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Abiraterone Acetate for Congenital Adrenal Hyperplasia

Phase 1
Waitlist Available
Led By Perrin C White, MD
Research Sponsored by University of Texas Southwestern Medical Center
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Requirement for standard of care fludrocortisone (any dose) and ≥10 mg/m2/day of hydrocortisone for at least 1 month prior to the study consent
Confirmed classic 21-hydroxylase deficiency evident by genotype groups A, A1 or B or clinical course (e.g., adrenal crisis with documented hyperkalemia and hyponatremia, at diagnosis or during a later evaluation; ambiguous genitalia in females). Documentation of one or both parents' genotypes may be required to confirm the subject's genotype
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 7 days
Awards & highlights

Study Summary

This trial is testing a new drug, abiraterone acetate, to see if it can be used to help treat children with a congenital adrenal hyperplasia.

Who is the study for?
This trial is for pre-pubescent children with classic 21-hydroxylase deficiency, which causes congenital adrenal hyperplasia. Eligible participants are girls aged 2-8 and boys aged 2-9 who have not yet reached advanced stages of puberty and require specific hormone treatments. Children with significant heart, liver, kidney issues, or other serious health conditions are excluded.Check my eligibility
What is being tested?
The study tests the effectiveness of abiraterone acetate in reducing the need for high doses of hydrocortisone in children with CAH. It aims to find the smallest dose that normalizes hormone levels over a week while maintaining standard therapy with hydrocortisone and fludrocortisone.See study design
What are the potential side effects?
Potential side effects include liver problems since abiraterone can affect liver enzymes; it may also cause hormonal imbalances or reactions related to its active ingredients. The exact side effects will be monitored closely due to the young age of participants.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have been taking fludrocortisone and at least 10 mg/m2/day of hydrocortisone for over a month.
Select...
I have a confirmed diagnosis of classic 21-hydroxylase deficiency.
Select...
My child is between 2-8 years old for girls, or 2-9 for boys, and meets the weight and skeletal age requirements.
Select...
Both my parents (or guardians) have signed the consent form, and I have given my assent if I'm 10 or older.
Select...
My androstenedione levels are high even after taking hydrocortisone.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~7 days
This trial's timeline: 3 weeks for screening, Varies for treatment, and 7 days for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Normalization of serum androstenedione level
Secondary outcome measures
17-hydroxyprogesterone levels
Dihydrotestosterone levels
Other outcome measures
Area under the plasma concentration versus time curve (AUC) of abiraterone
Number of adverse events
Peak Plasma Concentration (Cmax)

Side effects data

From 2021 Phase 3 trial • 32 Patients • NCT01517802
10%
Pulmonary Embolism
6%
Diarrhoea
6%
Fall
6%
Skin Laceration
3%
Acute Kidney Injury
3%
Urinary Retention
3%
Cardiac Failure Congestive
3%
Myocardial Infarction
3%
Vomiting
3%
Aortic Thrombosis
3%
Hypertension
3%
Dyspnoea
3%
Nausea
3%
Oesophagitis
3%
Musculoskeletal Pain
3%
Fatigue
3%
Weight Decreased
3%
Lower Respiratory Tract Infection
3%
Urinary Tract Infection
3%
Dehydration
3%
Cerebrovascular Accident
3%
Encephalopathy
3%
Spinal Cord Compression
3%
Syncope
3%
Cardiac Failure
3%
Upper Limb Fracture
3%
Aortic Valve Replacement
100%
80%
60%
40%
20%
0%
Study treatment Arm
Abiraterone Acetate + Prednisone/Prednisolone

Trial Design

3Treatment groups
Experimental Treatment
Group I: Abiraterone acetate 4 mg/kg/dExperimental Treatment1 Intervention
If the 2 mg/kg/d dosing does not result in androstenedione level normalization, abiraterone acetate will be administered orally at a daily dose of 4 mg/kg for 7 days in addition to the standard of care treatment of hydrocortisone and fludrocortisone.
Group II: Abiraterone acetate 2 mg/kg/dExperimental Treatment1 Intervention
If the 1 mg/kg/d dosing does not result in androstenedione level normalization, abiraterone acetate will be administered orally at a daily dose of 2 mg/kg for 7 days in addition to the standard of care treatment of hydrocortisone and fludrocortisone.
Group III: Abiraterone acetate 1 mg/kg/dExperimental Treatment1 Intervention
Abiraterone acetate will be administered orally at a daily dose of 1 mg/kg for 7 days in addition to the standard of care treatment of hydrocortisone and fludrocortisone.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Abiraterone acetate
2014
Completed Phase 3
~3440

Find a Location

Who is running the clinical trial?

University of MichiganOTHER
1,782 Previous Clinical Trials
6,364,671 Total Patients Enrolled
Children's Hospital Los AngelesOTHER
230 Previous Clinical Trials
5,076,546 Total Patients Enrolled
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)NIH
1,952 Previous Clinical Trials
2,660,818 Total Patients Enrolled

Media Library

Abiraterone acetate Clinical Trial Eligibility Overview. Trial Name: NCT02574910 — Phase 1
Congenital Adrenal Hyperplasia Research Study Groups: Abiraterone acetate 4 mg/kg/d, Abiraterone acetate 1 mg/kg/d, Abiraterone acetate 2 mg/kg/d
Congenital Adrenal Hyperplasia Clinical Trial 2023: Abiraterone acetate Highlights & Side Effects. Trial Name: NCT02574910 — Phase 1
Abiraterone acetate 2023 Treatment Timeline for Medical Study. Trial Name: NCT02574910 — Phase 1

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Could I be a valid candidate for this research endeavor?

"Those who wish to be accepted into this research must have been diagnosed with adrenal hyperplasia, congenital and should lie between the ages of 2 and 9. A total of 36 participants are expected to join the study."

Answered by AI

Does this research encompass seniors over the age of seventy?

"This medical research study is only open to minors aged between 2 and 9 years old. There are 32 trial sites for younger patients and 200 for elderly individuals over 65."

Answered by AI

How many participants are eligible for enrollment in this scientific experiment?

"At present, this clinical trial is not actively enrolling; it was first published on 8/1/2017 and modified for the last time on 2/22/2022. For those seeking alternative studies, 137 trials are recruiting patients with adrenal hyperplasia congenital and 98 trials require Abiraterone acetate participants."

Answered by AI

Has Abiraterone acetate been granted sanction by the FDA?

"Abiraterone acetate has only been tested in a limited capacity and therefore received a score of 1 on our safety scale due to it being an early-stage trial."

Answered by AI

Are there any opportunities to participate in this research initiative currently?

"At the present time, this clinical trial is not enrolling any new participants. Initially posted on August 1st 2017 and last updated February 22nd 2022, individuals in search of other studies may have luck with 137 trials actively recruiting for adrenal hyperplasia congenital or 98 studies searching for Abiraterone acetate subjects."

Answered by AI

Is Abiraterone acetate being tested in any additional experiments?

"Abiraterone acetate was first researched in 2005 at Rosemere Cancer Centre of Royal Preston Hospital. Over the course of 100 trials, 98 are still active with some based out of Dallas, Texas."

Answered by AI

How prevalent is this clinical trial in the US health care system?

"This clinical trial is offering enrolment across seven sites, including Children's Medical Center in Dallas, Children's Hospital of Los Angeles in LA and University of Michigan Ann Arbor. Additionally there are four other recruiting locations."

Answered by AI
~4 spots leftby Jan 2025