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Gene Therapy

Gene Therapy for Congenital Adrenal Hyperplasia

Phase 1 & 2
Recruiting
Research Sponsored by Adrenas Therapeutics Inc
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Naïve to prior gene therapy or AAV-mediated therapy
Stable oral hydrocortisone (HC) regimen as the only glucocorticoid (GC) maintenance therapy
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 3 years
Awards & highlights

Study Summary

This trial is testing a new gene therapy for adults with a rare genetic disorder that causes adrenal problems.

Who is the study for?
Adults with classic congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency, who haven't had gene therapy before and are on a stable dose of hydrocortisone. Pregnant women, those with previous adrenalectomy or significant liver disease, or immunity against AAV5 cannot participate.Check my eligibility
What is being tested?
The trial is testing the safety and effectiveness of a gene therapy called AAV BBP-631 in adults with CAH. It aims to see if this treatment can manage the condition better than current therapies.See study design
What are the potential side effects?
Specific side effects for AAV BBP-631 aren't listed but may include typical gene therapy-related reactions such as immune response to the virus used for delivery, mild fever, muscle aches, and potential liver issues.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I have never received gene therapy or AAV-mediated therapy.
Select...
I am on a stable dose of oral hydrocortisone as my only steroid treatment.
Select...
I am an adult with classic CAH due to 21-OHD and not pregnant if female.

Timeline

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

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Number of participants with Treatment-emergent Adverse Events that Led to Study Discontinuation
To select the optimum dose or dose range of BBP 631 for future studies
Secondary outcome measures
Change from Baseline in 17-OHP (hydroxyprogesterone) levels
Change from Baseline in androstenedione (A4) levels
Change from Baseline in endogenous cortisol levels

Trial Design

4Treatment groups
Experimental Treatment
Group I: Dose Level 4Experimental Treatment1 Intervention
BBP-631, highest dose, administered once, IV
Group II: Dose Level 3Experimental Treatment1 Intervention
BBP-631, high dose, administered once, IV
Group III: Dose Level 2Experimental Treatment1 Intervention
BBP-631 middle dose, administered once, IV
Group IV: Dose Level 1Experimental Treatment1 Intervention
BBP-631 lowest dose, administered once, intravenously (IV)

Find a Location

Who is running the clinical trial?

Adrenas Therapeutics IncLead Sponsor
1 Previous Clinical Trials
300 Total Patients Enrolled

Media Library

AAV BBP-631 (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT04783181 — Phase 1 & 2
Congenital Adrenal Hyperplasia Research Study Groups: Dose Level 4, Dose Level 1, Dose Level 2, Dose Level 3
Congenital Adrenal Hyperplasia Clinical Trial 2023: AAV BBP-631 Highlights & Side Effects. Trial Name: NCT04783181 — Phase 1 & 2
AAV BBP-631 (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04783181 — Phase 1 & 2

Frequently Asked Questions

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

Are there any still available positions for volunteers in this experiment?

"That is accurate. The clinicaltrials.gov website contains information which suggests this study is looking for enrollees. This trial was originally posted on July 1st, 2021 and updated October 27th, 2022. There are 8 locations signing up 25 patients each."

Answered by AI

How many people can join this clinical trial at the most?

"The sponsor, Adrenas Therapeutics Inc., needs to enroll 25 patients that fit the clinical trial's inclusion criteria in order to begin. The study will be conducted across multiple sites including University of Minnesota and Children's Hospital of Philadelphia."

Answered by AI

How many medical facilities are participating in this experiment?

"At the moment, this trial is taking place in 8 different locations. These include Minneapolis, Philadelphia and Bethesda among others. If you are interested in participating, try to select a location that is close to minimize travel time and inconvenience."

Answered by AI

Who else is applying?

What state do they live in?
North Carolina
Pennsylvania
How old are they?
18 - 65
What site did they apply to?
Lucas Research, Inc.
Children's Hospital of Philadelphia
What portion of applicants met pre-screening criteria?
Did not meet criteria
Met criteria
How many prior treatments have patients received?
0

How responsive is this trial?

Average response time
  • < 1 Day
Most responsive sites:
  1. Lucas Research, Inc.: < 24 hours
Typically responds via
Phone Call
Recent research and studies
clinicaltrials.govStudy
A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH)
2021. "A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04783181.
All > Cushings Syndrome
~1 spots leftby Jun 2024
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