A Phase 2a Proof-of-Concept Trial of Bempikibart (ADX-914) for the Treatment of Severe Alopecia Areata (SIGNAL-AA) (SIGNAL-AA)

A Phase 2a, Randomized, Double-blind, Placebo-Controlled, Proof-of-Concept Trial With Part B and Open-label Extension of Bempikibart (ADX-914) for the Treatment of Severe Alopecia Areata (SIGNAL-AA)

Part A is a randomized, double-blind, placebo-controlled, multi-center Proof-of-Concept (POC) Trial in subjects with severe Alopecia Areata. All participants in Part A have completed participation.

Part B is a multicenter, open-label study to assess the efficacy, safety, and tolerability of bempikibart (ADX-914) in participants with severe Alopecia Areata.

Study Overview

• Status: Active, not recruiting
• Conditions: Alopecia Areata
• Intervention / Treatment: Drug: bempikibart (ADX-914)

Detailed Description

Part A is a Phase IIa, Randomized, Double-blind, Placebo-controlled, multi-center Proof-of-Concept (POC) study in adult subjects with severe Alopecia Areata (AA). Bempikibart (ADX-914) or matching placebo is administered subcutaneously in the clinic setting every 2 weeks for 24 weeks, with follow-up for 12 weeks. Subjects will be randomized 3:1 to drug vs placebo. All participants in Part A have completed participation.

Part B is a multicenter, open-label study comprised of a screening period of up to 4 weeks, a treatment period of 36 weeks, and a follow-up period of 16 weeks, for a total study duration of up to 56 weeks.

Part A Open Label Extension Cohort is a multicenter, open-label extension to assess the long-term efficacy, safety, and tolerability of bempikibart (ADX-914) in participants with severe AA who experienced hair regrowth as participants in Part A of the study. Participants enrolled in the Open Label Extension Cohort will receive bempikibart (ADX-914) for a duration of 6 months followed by a 16-week follow-up period.

Part B Open Label Extension Cohort is a multicenter, open-label extension to assess the long-term efficacy, safety, and tolerability of bempikibart (ADX-914) in participants with severe AA who experienced hair regrowth as participants in Part B of the study. Participants enrolled in the Open Label Extension Cohort will receive bempikibart (ADX-914) for a treatment period of 36 weeks, and follow-up period of 16 weeks, for a total study duration of up to 52 weeks.

• Study Type: Interventional
• Enrollment (Estimated): 75
• Phase: Phase 2

Contacts and Locations

Study Locations

• Canada
  • Alberta
    • Calgary, Alberta, Canada, T1Y 0B4
      • Clinical Trials
  • Manitoba
    • Winnipeg, Manitoba, Canada, R3M 3Z4
      • Clinical Trials
  • Ontario
    • Oakville, Ontario, Canada, L6J 7W5
      • Clinical Trials
    • Peterborough, Ontario, Canada, K9J 5K2
      • Clinical Trials
    • Waterloo, Ontario, Canada, N2J 1C4
      • Clinical Trials
• United States
  • Arizona
    • Scottsdale, Arizona, United States, 85255
      • Scottsdale, Arizona
  • Arkansas
    • Fayetteville, Arkansas, United States, 72703
      • Fayetteville, Arkansas
    • Hot Springs, Arkansas, United States, 71913
      • Hot Springs, Arkansas
  • California
    • Encinitas, California, United States, 92024
      • Encinitas, California
    • Fountain Valley, California, United States, 92708
      • Fountain Valley, California
    • Lomita, California, United States, 90717
      • Lomita, California
  • Connecticut
    • New Haven, Connecticut, United States, 06519
      • New Haven, Connecticut
  • Florida
    • Miami, Florida, United States, 33173
      • Miami, Florida
    • Tampa, Florida, United States, 33613
      • Tampa, Florida
  • Indiana
    • Clarksville, Indiana, United States, 47129
      • Clarksville, Indiana
  • Kentucky
    • Bowling Green, Kentucky, United States, 42104
      • Bowling Green, Kentucky
  • Massachusetts
    • Burlington, Massachusetts, United States, 01805
      • Burlington, Massachusetts
  • Michigan
    • Troy, Michigan, United States, 48084
      • Troy, Michigan
    • Warren, Michigan, United States, 48192
      • Warren, Michigan
  • New York
    • New York, New York, United States, 10012
      • New York, New York
    • New York, New York, United States, 10023
      • New York, New York
  • North Carolina
    • Wilmington, North Carolina, United States, 28405
      • Wilmington, North Carolina
  • Ohio
    • Bexley, Ohio, United States, 43209
      • Bexley, Ohio
    • Canton, Ohio, United States, 44718
      • Clinical Trials
    • Mason, Ohio, United States, 45040
      • Mason, Ohio
    • Mayfield Heights, Ohio, United States, 44124
      • Mayfield Heights, Ohio
  • Oregon
    • Portland, Oregon, United States, 97225
      • Portland, Oregon
  • Texas
    • Austin, Texas, United States, 78759
      • Austin, Texas
    • Frisco, Texas, United States, 75034
      • Frisco, Texas
    • Houston, Texas, United States, 77056
      • Houston, Texas
    • San Antonio, Texas, United States, 78213
      • San Antonio, Texas
  • Utah
    • Jordan, Utah, United States, 84095
      • Jordan, Utah
  • Washington
    • Spokane, Washington, United States, 99202
      • Spokane, Washington

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Key Inclusion Criteria:

1. Adult men and women, aged 18 to 75 years, inclusive, at the time of informed consent.
2. Clinical diagnosis of severe to very severe alopecia areata, defined as the presence of ≥50% total scalp hair loss at screening and baseline as measured by the SALT score.

Key Exclusion Criteria:

1. History of or diagnosis at screening of any another form of alopecia based on assessment by investigator.
2. History (lifetime) or presence of hair transplants.
3. History (lifetime) or presence of micropigmentation of the scalp (Note: Microblading of the eyebrows is permitted).
4. Use of systemic, topical, or device-based therapy for AA.
5. History of, recent, or current clinically serious viral, bacterial, fungal, or parasitic infection or mycobacterial infection or at risk of serious infection.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Experimental: bempikibart (ADX-914); 200mg dose of bempikibart (ADX-914) administered via injection under the skin	Drug: bempikibart (ADX-914); Subcutaneous administration of bempikibart (ADX-914)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Part A: Mean relative percent change in SALT score	Part A: Mean relative percent change in SALT score at 24 weeks compared with baseline	24 Weeks
Part B: Mean percent change from baseline in SALT score	Part B: Mean percent change from baseline in SALT score at Week 36	36 weeks

Secondary Outcome Measures

Outcome Measure	Time Frame
Part A: Mean relative percentage change SALT score compared with baseline at Weeks 6,12, and 18.	18 Weeks
Part A: Mean relative percentage change in SALT score at 24 weeks compared with treatment nadir.	24 Weeks
Part A: Absolute change in SALT score compared with baseline at Weeks 6, 12, 18, and 24.	24 Weeks
Part A: Proportion of participants who achieve ≥30% and ≥50% relative reduction in SALT score compared with baseline at Weeks 12, 18, and 24.	24 Weeks
Part A: Proportion of participants with absolute SALT score ≤5, ≤10, ≤20, and ≤30 at Weeks 12, 18, and 24.	24 Weeks
Part A: Overall Safety as evaluated by number of adverse events (AEs).	36 Weeks
Part B: Proportion of participants who achieve ≥50% relative reduction in SALT score compared with baseline at 36 weeks	36 Weeks
Part B: Mean percent change from baseline in SALT score at Week 52	52 Weeks
Part B: Proportion of participants with absolute SALT score ≤20 and ≤30 at Week 36.	36 Weeks

Collaborators and Investigators

• Sponsor: Q32 Bio Inc.
• Collaborators: Innovaderm Research Inc.

Study record dates

Study Major Dates

• Study Start (Actual): September 12, 2023
• Primary Completion (Estimated): June 1, 2026
• Study Completion (Estimated): September 1, 2026

Study Registration Dates

• First Submitted: August 15, 2023
• First Submitted That Met QC Criteria: August 23, 2023
• First Posted (Actual): August 30, 2023

Study Record Updates

• Last Update Posted (Actual): May 8, 2026
• Last Update Submitted That Met QC Criteria: May 5, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Keywords: Alopecia; Hair Loss; Dermatitis; Atopic; Trichoscopy; Areata, Alopecia
• Additional Relevant MeSH Terms: Pathological Conditions, Anatomical; Skin Diseases; Hypotrichosis; Hair Diseases; Pathological Conditions, Signs and Symptoms; Skin and Connective Tissue Diseases; Alopecia Areata; Alopecia; Dermatitis
• Other Study ID Numbers: ADX-914-203; SIGNAL-AA (Other Identifier: Q32 Bio)

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No