A Study of IMR-687 in Subjects With Sickle Cell Disease

NCT04474314 | Terminated Phase 2 Results DMC FDA Drug

• 2 other identifiers: IMR-SCD-3012019-004471-39
• Study Type: interventional
• Target: 115
• Locations: 13 countries
• Sites: 49

Brief Summary

A Study to Evaluate the Safety and Efficacy of IMR-687 in Subjects with Sickle Cell Disease

Conditions

Sickle Cell Disease

Keywords

Sickle Cell Disease

Outcome Measures

Primary Outcomes (2)

• Effect on the Incidence of Vaso-occlusive Crises (VOCs)

  Annualized rate of VOCs. For each subject, the total number of VOCs on treatment were divided by the time on treatment divided by 52 weeks. The median was then summarized.

  Baseline to Week 52

• Proportion of Patients With Adverse Events and Serious Adverse Events

  Incidence of Adverse Events Incidence of Serious Adverse Events

  Baseline to Week 56

Study Arms (3)

Higher dose IMR-687

EXPERIMENTAL

Oral administration of once daily IMR-687

Drug: IMR-687

Lower 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:

• Confirmed diagnosis of SCD (HbSS, HbSB0 thalassemia, or HbSB+ thalassemia)
• Hemoglobin of \>5.5 and \<10.5 g/dL; Hb values within 21 days post-transfusion will be excluded.
• Subjects must have had at least 2 and no more than 12 documented episodes of VOCs in the past 12 months at the time of informed consent signing and at randomization (Day 1).
• Subjects receiving HU must have received it continuously for at least 6 months prior to signing informed consent, and must have been on a stable dose for at least 3 months prior to signing the informed consent, with no anticipated need for dose adjustments during the study including the screening period, in the opinion of the investigator.
• Female subjects must not be pregnant or breastfeeding and be highly unlikely to become pregnant. Male subjects must be unlikely to impregnate a partner.
• Must be willing and able to complete all study assessments and procedures, and to communicate effectively with the investigator and site staff.

You may not qualify if:

• Hospital discharge for sickle cell crisis or other vaso-occlusive event within the 4 days prior to randomization (Day 1).
• Subjects participating in a chronic/prophylactic RBC transfusion program (i.e., regularly scheduled RBC transfusions); any transfusions within 21 days of screening or baseline Hb measurements
• Subjects with HbF \>25% at screening.
• Significant kidney disease (eGFR \<45mL/min) and liver dysfunction: alanine aminotransferase or aspartate aminotransferase \>3x upper limit of normal.
• Body mass index (BMI) \<17.0 kg/m2 and a total body weight \<45 kg; or a 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 within 24 weeks prior to randomization (Day 1).
• Prior exposure to IMR-687.
• Subjects taking direct acting oral anti-coagulants (apixaban, dabigatran, rivaroxaban, edoxaban, or ticagrelor) or taking warfarin unless they stopped the treatment at least 28 days prior to randomization (Day 1).
• A history of use of crizanlizumab (Adakveo®) or voxelotor (Oxbryta®) within 6 months prior to signing the informed consent.
• Receipt of erythropoietin, luspatercept (Reblozyl®)or other hematopoietic growth factor treatment within 3 months of signing the ICF or anticipated need for such agents during the study.
• Prior gene therapy.

Sponsors & Collaborators

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

Study Sites (49)

University of Alabama at Birmingham School of Medicine - 1917 Clinic

Birmingham, Alabama, 35233, United States

Location

Arkansas Primary Care Clinic

Little Rock, Arkansas, 72204, United States

Location

University of California San Diego Moores Cancer Center

La Jolla, California, 92093, United States

Location

Center For Inherited Blood Disorders

Santa Ana, California, 92705, United States

Location

The Oncology Institute Long Beach

Whittier, California, 90603, United States

Location

University of Connecticut Health Main Building

Farmington, Connecticut, 06030, United States

Location

Children's Healthcare of Atlanta

Atlanta, Georgia, 30342, United States

Location

The University of Illinois at Chicago College of Medicine

Chicago, Illinois, 60612-4333, United States

Location

Johns Hopkins Hospital

Baltimore, Maryland, 21287, United States

Location

Children's Hospital of Michigan

Detroit, Michigan, 48201, United States

Location

Newark Beth Israel Medical Center

Newark, New Jersey, 07112, United States

Location

Weill Cornell Medicine - Center for Blood Disorders

New York, New York, 10021, United States

Location

Baylor Scott & White Medical Center-Temple

Temple, Texas, 76508, United States

Location

Virginia Commonwealth University Health - Ambulatory Care Center

Richmond, Virginia, 23219, United States

Location

Korle Bu Teaching Hospital

Accra, PO Box 77, Ghana

Location

Kintampo Health Research Centre

Kintampo, Brong-Ahafo Region, Ghana

Location

Laiko General Hospital of Athens

Athens, Attica, 11526, Greece

Location

University General Hospital of Patras

Pátrai, 26504, Greece

Location

Ippokrateio General Hospital of Thessaloniki

Thessaloniki, 54642, Greece

Location

Azienda Ospedaliero - Universitaria San Luigi Gonzaga

Turin, Orbassano, 10043, Italy

Location

Fondazione Policlinico Universitario Agostino Gemelli

Roma, Rome, 00168, Italy

Location

Ente Ospedaliero Ospedali Galliera

Genoa, 16128, Italy

Location

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

Milan, 20122, Italy

Location

Azienda Ospedaliera Ospedali Riuniti Villa Sofia Cervello

Palermo, 90146, Italy

Location

Kenya Medical Research Institute - Kisumu

Kisumu, Nyanza, 40100, Kenya

Location

Gertrude's Children's Hospital

Nairobi, 00100, Kenya

Location

The Centre for Respiratory Diseases Research - Kenya Medical Research Institute

Nairobi, 00100, Kenya

Location

Hopital Nini

Tripoli, North Governorate, Lebanon

Location

American University of Beirut Medical Center

Beirut, 01107 2020, Lebanon

Location

Chronic Care Center

Hazmiyeh, Lebanon

Location

Hôpital d'Enfants Rabat

Rabat, 10100, Morocco

Location

Hagaziekenhuis Van Den Haag - Leyweg

The Hague, South Holland, 2545 AA, Netherlands

Location

Sultan Qaboos University Hospital

Muscat, 123, Oman

Location

Centre National De Transfusion Sanguine - Du Senegal

Dakar, 5002, Senegal

Location

Hedi Chaker Hospital

Sfax, 3089, Tunisia

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

Jinja Regional Referral Hospital

Jinja, PO Box 43, Uganda

Location

Uganda Cancer Institute

Kampala, PO Box 3935, Uganda

Location

Makerere University College of Health Sciences

Kampala, PO Box 7072, Uganda

Location

Joint Clinical Research Center - Lubowa

Kampala, Wskiso District, Uganda

Location

Infectious Diseases Research Collaboration - Tororo

Tororo, 256, Uganda

Location

University Hospitals Bristol NHS Foundation Trust

Bristol, England, BS1 3NU, United Kingdom

Location

University College London Hospitals NHS

London, England, NW1 2PG, United Kingdom

Location

Guy's and Saint Thomas' NHS Foundation Trust

London, England, SE1 9RT, United Kingdom

Location

King's College Hospital NHS Foundation Trust

London, England, SE5 9RS, United Kingdom

Location

Manchester University NHS Foundation Trust

Manchester, England, M13 9WL, United Kingdom

Location

Oxford University Hospitals NHS Foundation Trust

Oxford, England, OX3 7LE, United Kingdom

Location

MeSH Terms

Conditions

Anemia, Sickle Cell

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Results Point of Contact

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

rballal@imaratx.com

617-206-2020

Study Officials

• Kenneth Attie, MD

  Imara, Inc.

  STUDY DIRECTOR

Publication Agreements

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

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Masking Details: Double-Blind
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 26, 2020
• First Posted: July 16, 2020
• Study Start: August 13, 2020
• Primary Completion: March 2, 2022
• Study Completion: May 4, 2022
• Last Updated: May 15, 2025
• Results First Posted: October 18, 2023

Record last verified: 2025-05

Locations

SE (1); GB (6); OM (1); NL (1); UG (5); US (14); TU (4); KE (3); GH (2); GR (3); LE (3); MO (1); IT (5)