Status:
TERMINATED
A Study of IMR-687 in Subjects With Sickle Cell Disease
Lead Sponsor:
Cardurion Pharmaceuticals, Inc.
Collaborating Sponsors:
Imara, Inc.
Conditions:
Sickle Cell Disease
Eligibility:
All Genders
18-65 years
Phase:
PHASE2
Brief Summary
A Study to Evaluate the Safety and Efficacy of IMR-687 in Subjects with Sickle Cell Disease
Detailed Description
A phase 2b, randomized, double-blind, placebo-controlled, multicenter study of subjects with sickle cell disease (SCD; homozygous sickle hemoglobin \[HbSS\], sickle-β0 \[HbSβ0\] thalassemia, or sickle...
Eligibility Criteria
Inclusion
- 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.
Exclusion
- 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.
Key Trial Info
Start Date :
August 13 2020
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
May 4 2022
Estimated Enrollment :
115 Patients enrolled
Trial Details
Trial ID
NCT04474314
Start Date
August 13 2020
End Date
May 4 2022
Last Update
May 15 2025
Active Locations (49)
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1
University of Alabama at Birmingham School of Medicine - 1917 Clinic
Birmingham, Alabama, United States, 35233
2
Arkansas Primary Care Clinic
Little Rock, Arkansas, United States, 72204
3
University of California San Diego Moores Cancer Center
La Jolla, California, United States, 92093
4
Center For Inherited Blood Disorders
Santa Ana, California, United States, 92705