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A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH)

Primary Purpose

Congenital Adrenal Hyperplasia

Status
Recruiting
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
AAV BBP-631
Sponsored by
Adrenas Therapeutics Inc
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Congenital Adrenal Hyperplasia focused on measuring CAH, Gene therapy, AAV, AAV5

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Key Inclusion Criteria

  1. Adult male and non-pregnant females with classic CAH (simple virilizing or salt-wasting) due to 21-OHD
  2. Screening/baseline 17-OHP levels > 5-10 × ULN and < 40 × ULN (upper limit of normal)
  3. Stable oral hydrocortisone (HC) regimen as the only glucocorticoid (GC) maintenance therapy
  4. Naïve to prior gene therapy or AAV-mediated therapy

Key Exclusion Criteria

  1. Positive for anti-AAV5 (Adeno-Associated Virus Type 5) antibodies
  2. History of adrenalectomy and has no significant liver disease

Sites / Locations

  • UCSF Benioff Children's Hospital, OaklandRecruiting
  • Ann and Robert H. Lurie Children's Hospital of ChicagoRecruiting
  • National Institutes of Health Clinical CenterRecruiting
  • University of MinnesotaRecruiting
  • Lucas Research, Inc.Recruiting
  • Children's Hospital of Philadelphia

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Experimental

Experimental

Arm Label

Dose Level 1

Dose Level 2

Dose Level 3

Arm Description

BBP-631 lowest dose, administered once, intravenously (IV)

BBP-631 middle dose, administered once, IV

BBP-631, highest dose, administered once, IV

Outcomes

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 endogenous cortisol levels
Change from Baseline in androstenedione (A4) levels

Full Information

First Posted
March 1, 2021
Last Updated
May 16, 2023
Sponsor
Adrenas Therapeutics Inc
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1. Study Identification

Unique Protocol Identification Number
NCT04783181
Brief Title
A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH)
Official Title
A Phase 1/2, First-in-Human, Open-Label, Dose-Escalation Study of the Safety and Efficacy of Gene Therapy for Congenital Adrenal Hyperplasia Through Administration of an Adeno-Associated Virus (AAV) Serotype 5-Based Recombinant Vector Encoding the Human CYP21A2 Gene
Study Type
Interventional

2. Study Status

Record Verification Date
May 2023
Overall Recruitment Status
Recruiting
Study Start Date
July 1, 2021 (Actual)
Primary Completion Date
June 2024 (Anticipated)
Study Completion Date
November 2028 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Adrenas Therapeutics Inc

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
This study is designed to evaluate the safety, tolerability, and efficacy of AAV5 based BBP-631 in adult participants diagnosed with classic congenital adrenal hyperplasia.
Detailed Description
Participants will receive a single dose of AAV5 based intravenous (IV) BBP-631 and will be followed for safety and efficacy for at least 5 years after the date of treatment with BBP-631.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Congenital Adrenal Hyperplasia
Keywords
CAH, Gene therapy, AAV, AAV5

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Sequential Assignment
Model Description
Participants will be assigned sequentially to one of 3 dose levels depending on the date of determination of eligibility.
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
25 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Dose Level 1
Arm Type
Experimental
Arm Description
BBP-631 lowest dose, administered once, intravenously (IV)
Arm Title
Dose Level 2
Arm Type
Experimental
Arm Description
BBP-631 middle dose, administered once, IV
Arm Title
Dose Level 3
Arm Type
Experimental
Arm Description
BBP-631, highest dose, administered once, IV
Intervention Type
Biological
Intervention Name(s)
AAV BBP-631
Intervention Description
intravenous
Primary Outcome Measure Information:
Title
Number of participants with Treatment-emergent Adverse Events that Led to Study Discontinuation
Time Frame
up to 3 years
Title
To select the optimum dose or dose range of BBP 631 for future studies
Time Frame
up to 3 years
Secondary Outcome Measure Information:
Title
Change from Baseline in 17-OHP (hydroxyprogesterone) levels
Time Frame
Baseline, Week 52
Title
Change from Baseline in endogenous cortisol levels
Time Frame
Baseline, Week 52
Title
Change from Baseline in androstenedione (A4) levels
Time Frame
Baseline, Week 52

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Key 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 and < 40 × ULN (upper limit of normal) Stable oral hydrocortisone (HC) regimen as the only glucocorticoid (GC) maintenance therapy Naïve to prior gene therapy or AAV-mediated therapy Key Exclusion Criteria Positive for anti-AAV5 (Adeno-Associated Virus Type 5) antibodies History of adrenalectomy and/or significant liver disease
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Patient Advocacy
Phone
650-391-9740
Email
ClinicalTrials@AdrenasTx.com
Facility Information:
Facility Name
UCSF Benioff Children's Hospital, Oakland
City
Oakland
State/Province
California
ZIP/Postal Code
94609
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Alyssa Kohler
Phone
510-428-3885
Ext
7442
Email
Alyssa.Kohler@ucsf.edu
First Name & Middle Initial & Last Name & Degree
Paul Harmatz, MD
First Name & Middle Initial & Last Name & Degree
Tariq E Ahmad, MD, FAAP
Facility Name
Ann and Robert H. Lurie Children's Hospital of Chicago
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60611
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Sarayu Ratnam
Phone
312-227-6617
Email
sratnam@luriechildrens.org
Phone
312-227-6090
First Name & Middle Initial & Last Name & Degree
Courtney Finlayson, MD
Facility Name
National Institutes of Health Clinical Center
City
Bethesda
State/Province
Maryland
ZIP/Postal Code
20892-1932
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Office of Patient Recruitment
Phone
800-411-1222
Email
prpl@cc.nih.gov
First Name & Middle Initial & Last Name & Degree
Elizabeth Joyal (Study Coordinator), CRNP
Phone
301-496-8542
Email
ejoyal@nih.gov
First Name & Middle Initial & Last Name & Degree
Deborah Merke, M.D., M.S.
Facility Name
University of Minnesota
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55455
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Coordinator Office
Phone
612-624-8672
Email
saraf010@umn.edu
First Name & Middle Initial & Last Name & Degree
Coordinator Office
Phone
612-626-7024
First Name & Middle Initial & Last Name & Degree
Kyriakie Sarafoglou, M.D.
Facility Name
Lucas Research, Inc.
City
Morehead City
State/Province
North Carolina
ZIP/Postal Code
28557
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Crystal Gann, CCRC
Phone
252-222-5700
Ext
135
Email
crystal.gann@lucasresearch.org
First Name & Middle Initial & Last Name & Degree
K. Jean Lucas, MD,FACP,FACE
Facility Name
Children's Hospital of Philadelphia
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Sarah McCague
Phone
267-273-8077
Email
mccagues@chop.edu
First Name & Middle Initial & Last Name & Degree
Maria Vogiatzi, MD

12. IPD Sharing Statement

Plan to Share IPD
No
Links:
URL
https://adrenastx.com
Description
Adrenas Therapeutics Website
URL
https://cahgenetherapy.com
Description
CAH Gene Therapy Website

Learn more about this trial

A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH)

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