A Study to Determine Eligibility for CAH-301 (A Study of... | NCT05101902

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

NCT05101902

Sponsor

Adrenas Therapeutics Inc

Status

Completed

Last Update Posted

Sep 19, 2024Actual

Enrollment

65Actual

Phase

Not provided

Conditions

Congenital Adrenal Hyperplasia

Interventions

Pre-Screening

Countries

United States

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

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

Status Module

Record Verification Date

Sep 2024

Overall Recruitment Status or Expanded Access Status

Completed

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Jul 1, 2021Actual

Primary Completion Date

Aug 7, 2024Actual

Completion Date

Aug 7, 2024Actual

First Submitted Date

Oct 20, 2021

First Submission Date that Met QC Criteria

Oct 20, 2021

First Posted Date

Nov 1, 2021Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Sep 12, 2024

Last Update Posted Date

Sep 19, 2024Actual

Oversight Module

Has Data Monitoring Committee (DMC)

Yes

Is FDA Regulated Drug

Yes

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

FDAAA801 Violation

Not provided

Description Module

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.

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.

Design Module

Study Type

Observational

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Target Follow-Up Duration

Not provided

Phases

Not provided

Interventional Study Design

Allocation

Not provided

Intervention Model

Biospecimen

No data available

No data is available for this block.

Enrollment

65Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
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

Interventions

Name	Type	Description	Arm Group Labels	Other Names
Pre-Screening	Other	No intervention given	Overall Study

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Number of Participants Potentially Eligible for CAH-301 Study		Up to 5 years

Secondary Outcomes

Not provided

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

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

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

18 Years

Maximum Age

Not provided

Standard Ages

AdultOlder Adult

Study Population

Adult participants diagnosed with classic CAH due to 21-OHD

Sampling Method

Non-Probability Sample

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Not provided

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Science37		Los Angeles	California	90094	United States

References Module

Citations

Not provided

Label	URL
Description: Adrenas Therapeutics Website	View source
CAH Gene Therapy Website	View source

ID	Term
D047808	Adrenogenital Syndrome
D012734	Disorders of Sex Development
D014564	Urogenital Abnormalities
D052776	Female Urogenital Diseases