A Study of IMR-687 in Subjects With Beta Thalassemia

NCT04411082 | Terminated Phase 2 Results DMC FDA Drug

• 2 other identifiers: IMR-BTL-2012019-002989-12
• Study Type: interventional
• Target: 122
• Locations: 12 countries
• Sites: 32

Brief Summary

A Study to Evaluate the Safety and Tolerability of IMR-687 in Subjects with Beta Thalassemia

Conditions

β Thalassemia

Keywords

Transfusion; TDT; NTDT

Outcome Measures

Primary Outcomes (1)

• IMR-687 Safety and Tolerability

  Incidence and severity of Adverse Events Incidence and severity of Serious Adverse Events

  Baseline to Week 40

Secondary Outcomes (8)

• TDT Patients: Reduction in Red Blood Cell (RBC) Transfusion Burden With ≥33% Hematological Improvement From Week 12 to Week 24

  Baseline to Week 24

• NTDT Patients: Proportion of Subjects With an Increase From Baseline of Hb at Week 12 to Week 24 in the Absence of Transfusions.

  Baseline to Week 24

• NTDT Patients: Proportion of Subjects With an Increase From Baseline of ≥3% in Mean HbF Values at Week 12 to Week 24 in Absence of Transfusions

  Baseline to Week 24

• TDT Patients: Reduction in Red Blood Cell (RBC) Transfusion Burden With ≥33% Hematological Improvement From Week 24 to Week 36

  Baseline to Week 36

• TDT Patients: Reduction in Red Blood Cell (RBC) Transfusion Burden With ≥50 % Hematological Improvement From Week 12 to Week 24

  Baseline to Week 24

Study Arms (3)

Lower Dose IMR-687

EXPERIMENTAL

Oral administration of once daily IMR-687

Drug: IMR-687

Higher dose IMR-687

EXPERIMENTAL

Oral administration of once daily IMR-687

Drug: IMR-687

Placebo

PLACEBO COMPARATOR

Oral administration of once daily placebo

Drug: Placebo

Interventions

IMR-687 DRUG

Oral administration of once daily IMR-687

Higher dose IMR-687; Lower Dose IMR-687

Placebo DRUG

Oral administration of once daily Placebo

Placebo

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Documented diagnosis of β-thalassemia or HbE/ β-thalassemia in their medical history. Concomitant alpha gene deletion, duplication, or triplication is allowed.
• Documentation of the dates of transfusion events and the number of all pRBC units per event within the 12 weeks prior to the Baseline (Day 1) visit. .
• Must be willing and able to complete all study assessments and procedures, and to communicate effectively with the investigator and site staff.
• TDT Subjects: subjects must be regularly transfused, defined as \>3 to 10 pRBC units in the12 weeks prior to Baseline (Day 1) visit and no transfusion-free period for \>35 days during that period.
• NTDT subjects: Subjects must be transfusion independent, defined as 0 to ≤3 units of pRBCs received during the 12-week period prior to the Baseline (Day 1) visit, must not be on a regular transfusion program, must be RBC transfusion-free for at least ≥ 4 weeks prior to randomization, and must not be scheduled to start a regular
• hematopoietic stem cell transplantation within 9 months.
• NTDT subjects: Subjects must have Hb ≤10.0 g/dL at Screening; the screening Hb sample must be collected 7 to 28 days prior to randomization. Hb values within 21 days post-transfusion will be excluded.
• ECOG performance score of 0 to 1
• Female subjects must not be pregnant, or breastfeeding and be highly unlikely to become pregnant. Male subjects must be unlikely to impregnate a partner.

You may not qualify if:

• Diagnosis of α-thalassemia (e.g., hemoglobin H \[HbH\]) or hemoglobin S (HbS)/ β thalassemia.
• Body mass index (BMI) \<17.0 kg/m2 or a total body weight \<45 kg; or BMI \>35 kg/m2
• Subjects with known active hepatitis A, hepatitis B, or hepatitis C, with active or acute event of malaria, or who are known to be positive for human immunodeficiency virus (HIV).
• Stroke requiring medical intervention ≤24 weeks prior to randomization.
• Platelet count \>1000 × 109/L.
• Participated in another clinical study of an investigational agent (or device) within 30 days or 5-half-lives of date of informed consent, whichever is longer, or is currently participating in another study.
• For Subjects on iron chelation therapy (ICT) at the time of ICF signing, initiation of ICT less than 24 weeks before the predicted randomization date.
• Prior exposure to sotatercept or luspatercept, IMR-687, or gene therapy within 6 months prior to randomization (Day 1).
• Subjects who have major organ damage

Sponsors & Collaborators

• Cardurion Pharmaceuticals, Inc.: lead
• Imara, Inc.: collaborator

Study Sites (36)

Herlev Hospital

Herlev, Capital Region, 2730, Denmark

Location

Institut Universitaire du Cancer de Toulouse Oncopole

Toulouse, Haute-Garonn, 31059, France

Location

Hôpital Edouard Herriot

Lyon, Rhone, 69437, France

Location

Hôpital Necker-Enfants Malades

Paris, 75015, France

Location

M. Zodelava Hematology Centre

Tbilisi, Borjomi, 0112, Georgia

Location

National Center of Surgery

Tbilisi, 0159, Georgia

Location

Medinvest - Institute of Hematology and Transfusiology

Tbilisi, 0186, Georgia

Location

Aghia Sofia General Children's Hospital

Athens, Attica, 11527, Greece

Location

Laiko General Hospital of Athens

Athens, Attica, 11527, Greece

Location

Ippokrateio General Hospital of Thessaloniki

Thessaloniki, Central Macedonia, 54642, Greece

Location

University General Hospital of Patras

Patra, Peloponnese, 26504, Greece

Location

Rambam Health Care Campus

Haifa, Haifa District, 3109601, Israel

Location

Hadassah University Hospital Ein Kerem

Jerusalem, Jerusalem, 9112001, Israel

Location

The Galilee Medical Center

Nahariya, Northern District, 2210001, Israel

Location

Emek Medical Center

Afula, 18101, Israel

Location

Azienda Ospedaliera Giuseppe Brotzu

Orbassano, Turin, 10043, Italy

Location

Azienda Ospedaliera Universitaria - Università degli Studi della Campania Luigi Vanvitelli

Orbassano, Turin, 10043, Italy

Location

Chronic Care Center

Hazmiyeh, 213, Lebanon

Location

Hospital Sultanah Aminah Johor Bharu

Johor Bahru, Johor, 80100, Malaysia

Location

Hospital Sultanah Bahiyah

Alor Star, Kedah, 05460, Malaysia

Location

Hospital Raja Permaisuri Bainun

Ipoh, Perak, 30450, Malaysia

Location

Hospital Pulau Pinang

George Town, Pulau Pinang, 10450, Malaysia

Location

Hospital Queen Elizabeth - Kota Kinabalu

Kota Kinabalu, Sabah, 88586, Malaysia

Location

Hospital Umum Sarawak

Kuching, Sarawak, 93586, Malaysia

Location

Hôpital d'Enfants Rabat

Rabat, 10100, Morocco

Location

Amsterdam Universitair Medische Centra - Academisch Medisch Centrum

Amsterdam, North Holland, 1105 AZ, Netherlands

Location

Centre Hôpital Universitaire Farhat Hached

Sousse, 4000, Tunisia

Location

Centre National de Greffe de la Moelle Osseuse

Tunis, 1006, Tunisia

Location

Hospital Aziza Othmana

Tunis, 1008, Tunisia

Location

Akdeniz Üniversitesi

Mersin, Mersin, 33110, Turkey (Türkiye)

Location

Mersin Üniversitesi Tıp Fakültesi

Mersin, Mersin, 33110, Turkey (Türkiye)

Location

Hacettepe Üniversitesi

Ankara, 06230, Turkey (Türkiye)

Location

Ege Universitesi Tip Fakultesi

Izmir, 35100, Turkey (Türkiye)

Location

Whittington Health NHS Trust

London, England, N19 5NF, United Kingdom

Location

University College London Hospitals NHS Foundation Trust

London, England, NW1 2PG, United Kingdom

Location

Manchester University NHS Foundation Trust

Manchester, England, M13 9WL, United Kingdom

Location

Related Publications (1)

• Foong WC, Loh CK, Ho JJ, Lau DS. Foetal haemoglobin inducers for reducing blood transfusion in non-transfusion-dependent beta-thalassaemias. Cochrane Database Syst Rev. 2023 Jan 13;1(1):CD013767. doi: 10.1002/14651858.CD013767.pub2.

  PMID: 36637054 DERIVED

Results Point of Contact

• Title: Rahul Ballal
• Organization: Imara, Inc.

rballal@imaratx.com

617-206-2020

Study Officials

• Steve Luperchio

  Cardurion Pharmaceuticals

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Masking Details: Double-Blind
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 15, 2020
• First Posted: June 2, 2020
• Study Start: October 16, 2020
• Primary Completion: March 11, 2022
• Study Completion: May 4, 2022
• Last Updated: May 15, 2025
• Results First Posted: June 30, 2022

Record last verified: 2025-05

Locations

FR (3); IL (4); NL (1); LE (1); GR (4); GE (3); MY (6); TU (3); GB (3); MO (1); DK (1); IT (2)