A Study of Obexelimab in Patients With Warm Autoimmune Hemolytic Anemia (SApHiAre)

Warm Autoimmune Hemolytic Anemia Clinical Trial

Official title:

A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study With An Open Label Safety and Dose Confirmation Run-In Period, To Evaluate the Efficacy and Safety of Obexelimab in Patients With Warm Autoimmune Hemolytic Anemia (SApHiAre)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05786573
• Other study ID #: ZB012-03-002
• Status: Recruiting
• Phase: Phase 3
• Start date: September 25, 2023
• Est. completion date: June 8, 2026

Study information

• Verified date: May 2024
• Source: Zenas BioPharma (USA), LLC
• Contact: Patient and Medical Information
• Phone: 833-269-4696
• Email: clinicaltrialsinfo[@]zenasbio.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

This study aims to examine the efficacy and safety of obexelimab in participants with Warm Autoimmune Hemolytic Anemia (wAIHA).

Description:

This study consists of a 6-month open label Safety and Dose Confirmation Run-in Period (SRP), 6-month Randomized Control Period (RCP), and an additional 1-year open-label extension (OLE) period. To enter the Screening Period (Day -28 to Day -1) in the SRP or RCP, patients must have a clinical diagnosis of primary or secondary wAIHA due to an underlying autoimmune disorder, have failed at least 1 prior wAIHA treatment regimen, and have a Hgb level of ≥ 7 to < 10 g/dL with at least one sign or symptom of anemia. For the SRP only, patients with secondary wAIHA due to underlying lymphoproliferative disease may be eligible if they are receiving stable treatment.

All patients in the SRP or RCP are allowed to continue up to 2 failed wAIHA therapies throughout the 24-week study. On Day 1 of the SRP, patients receive obexelimab administered as subcutaneous (SC) injections. On Day 1 of the RCP, patients will be randomized in a ratio of 1:1 to receive either obexelimab or placebo administered as subcutaneous (SC) injections. Patients must return to the study site for the first 5 weeks and then every 2 weeks thereafter. Patients will undergo assessments for efficacy, safety, PK, PD, and immunogenicity during the 24-week SRP or RCP.

Following the 24-week SRP or RCP, patients will have the opportunity to receive obexelimab for up to 52 weeks in the Open Label Extension (OLE) Period.

Including screening and follow-up, the maximum duration of participation in this study for an individual patient is 81 weeks (i.e., 28-day screening, 24-week SRP or RCP, 52-week OLE, and an 8-week follow-up).

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 134
• Est. completion date: June 8, 2026
• Est. primary completion date: March 14, 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

1. Males and females, = 18 years of age

2. Clinically diagnosed with wAIHA for at least 3 months and currently receiving treatment for wAIHA or have previously received treatment for wAIHA.

3. Diagnosis of primary or secondary wAIHA documented by a positive direct antiglobulin test specific for anti-IgG or anti-IgA.

4. Failed at least 1 prior wAIHA treatment regimen.

5. At least one sign or symptom of anemia as assessed by the investigator at screening.

6. Other inclusion criteria apply.

Exclusion Criteria:

1. Have cold antibody AIHA, cold agglutinin syndrome, mixed type (i.e., warm, and cold) AIHA, or paroxysmal cold hemoglobinuria.

2. Have any other associated cause of hereditary or acquired hemolytic anemia.

3. For the RCP only, patients with secondary wAIHA not due to autoimmune disorders, including LPDs.

4. Received a transfusion within 2 weeks prior to randomization.

5. Use of B cell-depleting, B cell-targeted, or other biologic immunomodulatory agents within the 6 months prior to randomization.

6. Received IV Ig or epoetin alfa within 6 weeks prior to randomization.

7. Receiving more than 2 concomitant medications for the treatment of wAIHA.

8. Other exclusion criteria apply.

Related Conditions & MeSH terms

• Anemia
• Anemia, Hemolytic
• Anemia, Hemolytic, Autoimmune
• Hemolysis
• Warm Autoimmune Hemolytic Anemia

Intervention

Drug:
• Obexelimab
Obexelimab is a monoclonal antibody that simultaneously binds CD19 and Fc?RIIb, resulting in down regulation of B cell activity.
• Obexelimab
Obexelimab is a monoclonal antibody that simultaneously binds CD19 and Fc?RIIb, resulting in down regulation of B cell activity.

Other:
• Placebo
Placebo

Locations

Country	Name	City	State
Italy	Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico	Milano
Italy	A.O.U. Maggiore della Carità	Novara	Piemonte
Italy	Azienda Ospedaliera Città della Salute e della Scienza di Torino	Torino
Italy	Azienda Sanitaria Universitaria Giuliano Isontina	Udine	Friuli-Venezia Giulia
Japan	Fukushima Medical University Hospital	Fukushima
Japan	Japanese Red Cross Society Himeji Hospital	Himeji	Hyogo
Japan	Kanazawa University Hospital	Kanazawa	Ishikawa
Japan	Hospital of the University of Occupational & Environmental Health	Kitakyushu	Hukuoka
Japan	Okayama University Hospital	Okayama
Japan	Kitasato University Hospital	Sagamihara	Kanagawa
Japan	Tohoku University Hospital	Sendai	Miyagi
Japan	Osaka University Hospital	Suita	Osaka
Poland	Copernicus PL Sp. z o.o. Wojewodzkie Centrum Onkologii	Gdansk	Pomorskie
Poland	Uniwersyteckie Centrum Kliniczne - Ul. Smoluchowskiego 17	Gdansk
Poland	SPZOZ MiSWiA z Warminsko-Mazurskim Centrum Onkologii w Olsztynie	Olsztyn	Warminsko-mazurskie
Spain	Hospital Universitario Germans Trias i Pujol	Badalona	Barcelona
Spain	Hospital Universitario de Burgos	Burgos
Spain	Hospital Universitario de Gran Canaria Doctor Negrin	Las Palmas De Gran Canaria	Las Palmas
Spain	Hospital Universitario 12 de Octubre	Madrid
Spain	Hospital Universitario de Navarra	Pamplona	Navarra
Spain	Hospital Universitario Virgen del Rocio	Sevilla
Spain	Hospital Universitario Virgen del Rocio - PPDS	Sevilla
Taiwan	Changhua Christian Hospital	Chang Hua	Changhua
Taiwan	Chi Mei Medical Center, Liouying	Tainan
Taiwan	National Cheng Kung University Hospital	Tainan
Taiwan	National Taiwan University Hospital	Taipei
Turkey	Ankara Universitesi Tip Fakultesi Hastaneleri - Cebeci Hastanesi	Ankara
Turkey	Ondokuz Mayis Universitesi Tip Fakultesi Hastanesi	Samsun
Turkey	Karadeniz Technical University Faculty of Medicine	Trabzon
United Kingdom	Kent and Canterbury Hospital	Canterbury	Kent
United Kingdom	Leicester Royal Infirmary	Leicester	Leicestershire
United Kingdom	Barts Health NHS Trust	London
United Kingdom	University College London Hospitals	London
United Kingdom	Plymouth Hospitals NHS Trust	Plymouth	Devon
United States	Mercy Health Lacks Cancer Center	Grand Rapids	Michigan
United States	East Carolina University	Greenville	North Carolina
United States	MD Anderson Cancer Center	Houston	Texas
United States	University of Iowa	Iowa City	Iowa
United States	Regents of the University of California Los Angeles	Los Angeles	California
United States	University of Southern California	Los Angeles	California
United States	Memorial Sloan Kettering	New York	New York
United States	Integris Southwest Medical Center	Oklahoma City	Oklahoma
United States	Mayo Clinic	Rochester	Minnesota
United States	University of California San Diego Moores Cancer Center	San Diego	California
United States	Georgetown University Medical Center	Washington	District of Columbia

Sponsors (1)

Lead Sponsor	Collaborator
Zenas BioPharma (USA), LLC

Countries where clinical trial is conducted

United States,  Italy,  Japan,  Poland,  Spain,  Taiwan,  Turkey,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Safety and Dose Confirmation Run-in Period (SRP)	Proportion of participants with hemoglobin (Hgb) = 10 g/dL and = 2 g/dL increase from Baseline with no use of blood transfusion or glucocorticoid (GC) rescue therapy.	24 weeks
Primary	Randomized Control Period (RCP)	Proportion of participants who achieve a durable Hgb response (defined as Hgb = 10 g/dL and = 2 g/dL increase from Baseline on at least 3 of 4 consecutive available visits), at the earliest on or after Week 12, with no use of blood transfusion or GC rescue therapy prior to attaining durable response through Week 24.	24 weeks