A Study to Determine Eligibility for CAH-301 (A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH) [NCT04783181])

• Sponsor: Adrenas Therapeutics Inc (Industry)
• Overall Status: Recruiting
• CT.gov ID: NCT05101902
• Collaborator: (none)
• Enrollment: 300
• Location: 1
• Anticipated Duration (Months): 23
• Patients Per Site Per Month: 13

Study Details

Study Description

Brief Summary

This pre-screening study is designed to determine potential eligibility of adults with classic CAH due to 21-hydroxylase deficiency (21-OHD) for participation in the CAH-301 [NCT04783181] gene therapy trial with BBP-631.

Condition or Disease	Intervention/Treatment	Phase
Congenital Adrenal Hyperplasia	Other: Pre-Screening

Detailed Description

Adult participants diagnosed with classic CAH due to 21-OHD and who are taking glucocorticoids as treatment for CAH will undergo assessments to determine their potential eligibility for participation in the adult CAH-301 treatment trial with BBP-631.

Study Design

• Study Type: Observational
• Anticipated Enrollment: 300 participants
• Observational Model: Cohort
• Time Perspective: Prospective
• Official Title: Pre-screening Study to Identify Adult Participants With Classic Congenital Adrenal Hyperplasia Due to 21-Hydroxylase Deficiency Who May Be Eligible for Treatment in the CAH-301 Trial With BBP-631, an Adeno-associated Virus (AAV) Serotype 5-Based Recombinant Vector Encoding the Human CYP21A2 Gene
• Actual Study Start Date: Jul 1, 2021
• Anticipated Primary Completion Date: Jun 1, 2023
• Anticipated Study Completion Date: Jun 1, 2023

Arms and Interventions

Arm

Intervention/Treatment

Overall Study Participants will undergo antibody and genetic screen, initial pre-screening, eligibility monitoring and eligibility confirmation for potential transition to the treatment trial center once a treatment assignment in the study CAH-301 becomes available. Participation in Study CAH-300 is permitted to continue until treatment assignment is available in the treatment trial or unless, during eligibility monitoring or eligibility

Other: Pre-Screening; No intervention given

Outcome Measures

Primary Outcome Measures

1. Number of Participants Potentially Eligible for CAH-301 Study [Up to 3 years]

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years and Older
• Sexes Eligible for Study: All

Inclusion Criteria:

• Adult male and non-pregnant females with classic CAH (simple virilizing or salt-wasting) due to 21-OHD

• Screening/baseline 17-OHP levels > 5-10 × ULN

• Is on a daily regimen of glucocorticoid

• Naïve to prior gene therapy or AAV-mediated therapy

Exclusion Criteria:

• Positive for anti-AAV5 antibodies

• History of adrenalectomy and has no significant liver disease

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Science37	Los Angeles	California	United States	90094

Sponsors and Collaborators

• Adrenas Therapeutics Inc

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Adrenas Therapeutics Inc
• ClinicalTrials.gov Identifier: NCT05101902
• Other Study ID Numbers: CAH-300
• First Posted: Nov 1, 2021
• Last Update Posted: Mar 2, 2022
• Last Verified: Mar 1, 2022

• Individual Participant Data (IPD) Sharing Statement: Yes
• Plan to Share IPD: Yes
• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No
• Keywords provided by Adrenas Therapeutics Inc: CAH
• Additional relevant MeSH terms: Adrenal Hyperplasia, Congenital; Adrenogenital Syndrome; Adrenocortical Hyperfunction; Hyperplasia

Study Results

No Results Posted as of Mar 2, 2022