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

Stopped early · Phase 2 · Has a placebo group

Conditions studied: Sickle Cell Disease

In brief

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

Key facts

Study ID: NCT04474314
Run by: Cardurion Pharmaceuticals, Inc.
People needed: 115
Starts: 2020-08-13
Expected to finish: 2022-05-04
Last updated by the study team: 2025-05-15

Who can join

Age: 18 and older, up to 65. Sex: any. Healthy volunteers: not accepted.

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.

Where it is running

University of Alabama at Birmingham School of Medicine - 1917 Clinic — Birmingham, Alabama, United States
Arkansas Primary Care Clinic — Little Rock, Arkansas, United States
University of California San Diego Moores Cancer Center — La Jolla, California, United States
Center For Inherited Blood Disorders — Santa Ana, California, United States
The Oncology Institute Long Beach — Whittier, California, United States
University of Connecticut Health Main Building — Farmington, Connecticut, United States
Children's Healthcare of Atlanta — Atlanta, Georgia, United States
The University of Illinois at Chicago College of Medicine — Chicago, Illinois, United States
Johns Hopkins Hospital — Baltimore, Maryland, United States
Children's Hospital of Michigan — Detroit, Michigan, United States
Newark Beth Israel Medical Center — Newark, New Jersey, United States
Weill Cornell Medicine - Center for Blood Disorders — New York, New York, United States
Baylor Scott & White Medical Center-Temple — Temple, Texas, United States
Virginia Commonwealth University Health - Ambulatory Care Center — Richmond, Virginia, United States
Korle Bu Teaching Hospital — Accra, Ghana
Kintampo Health Research Centre — Kintampo, Ghana
Laiko General Hospital of Athens — Athens, Attica, Greece
University General Hospital of Patras — Pátrai, Greece
Ippokrateio General Hospital of Thessaloniki — Thessaloniki, Greece
Azienda Ospedaliero - Universitaria San Luigi Gonzaga — Turin, Orbassano, Italy
Fondazione Policlinico Universitario Agostino Gemelli — Roma, Rome, Italy
Ente Ospedaliero Ospedali Galliera — Genoa, Italy
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico — Milan, Italy
Azienda Ospedaliera Ospedali Riuniti Villa Sofia Cervello — Palermo, Italy
Kenya Medical Research Institute - Kisumu — Kisumu, Nyanza, Kenya

Full record on ClinicalTrials.gov

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