What side effects are associated with this type of intervention?

Traditional treatments for Congenital Adrenal Hyperplasia (CAH) typically involve glucocorticoids and mineralocorticoids, which can cause side effects such as weight gain, hypertension, osteoporosis, and growth suppression in children. Gene therapy, like the AAV5-based BBP-631 being studied, aims to correct the genetic defect causing CAH. Potential side effects of gene therapy may include immune reactions to the viral vector, liver enzyme elevations, and insertional mutagenesis, although these are still under investigation and long-term data is limited.

What prior FDA approvals exist?

Currently, there are no FDA-approved gene therapies specifically for the treatment of Congenital Adrenal Hyperplasia (CAH) similar to the BBP-631 trial using the AAV5 vector. Traditional treatments for CAH typically involve glucocorticoid and mineralocorticoid replacement therapies to manage hormone levels. The BBP-631 trial represents an investigational approach aiming to correct the genetic defect in CAH through gene therapy, which is still under evaluation for safety and efficacy.

What other types of research exist?

Promising alternatives to BBP-631 for treating Congenital Adrenal Hyperplasia (CAH) include other gene therapies using different viral vectors or delivery methods, CRISPR-based gene editing techniques, and novel pharmacological treatments such as modified steroidogenesis inhibitors or enzyme replacement therapies. Additionally, advancements in personalized medicine and targeted therapies that address specific genetic mutations in CAH patients are also being explored.

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Gene Therapy

Gene Therapy for Congenital Adrenal Hyperplasia

Phase 1 & 2

Waitlist Available

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

Must not have

History of adrenalectomy and/or significant liver disease

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

No Placebo-Only Group

Summary

This trial tests a new gene therapy called BBP-631, delivered by a virus through an IV, in adults with classic congenital adrenal hyperplasia. The goal is to see if it is safe and effective over several years. BBP-631 is a gene therapy delivered by a virus, similar to other gene therapies that have shown promise in treating adrenal disorders.

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.

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.

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

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I have never received gene therapy or AAV-mediated therapy.

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I am on a stable dose of oral hydrocortisone as my only steroid treatment.

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I am an adult with classic CAH due to 21-OHD and not pregnant if female.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have had surgery to remove my adrenal gland or have significant liver disease.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

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)

0 / 4Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Congenital Adrenal Hyperplasia (CAH) is commonly treated with glucocorticoids and mineralocorticoids to replace deficient hormones and suppress excess androgen production. Glucocorticoids, such as hydrocortisone, work by providing the cortisol that the adrenal glands cannot produce, thereby reducing the overproduction of androgens. Mineralocorticoids, like fludrocortisone, help maintain salt balance and blood pressure. Gene therapy, such as the AAV5-based BBP-631, aims to correct the underlying genetic defect by delivering a functional copy of the defective gene to the patient's cells. This approach has the potential to provide a long-term solution by addressing the root cause of CAH, reducing the need for lifelong hormone replacement therapy and improving quality of life for patients.