Status:
TERMINATED
A Study of IMR-687 in Subjects With Beta Thalassemia
Lead Sponsor:
Cardurion Pharmaceuticals, Inc.
Collaborating Sponsors:
Imara, Inc.
Conditions:
β Thalassemia
Eligibility:
All Genders
18-65 years
Phase:
PHASE2
Brief Summary
A Study to Evaluate the Safety and Tolerability of IMR-687 in Subjects with Beta Thalassemia
Detailed Description
A phase 2, randomized, double-blind, placebo-controlled study to evaluate the safety, tolerability, PK, and PD of IMR-687 (phosphodiesterase (PDE) 9 inhibitor) administered once daily (qd) orally for ...
Eligibility Criteria
Inclusion
- 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.
Exclusion
- 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
Key Trial Info
Start Date :
October 16 2020
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
May 4 2022
Estimated Enrollment :
122 Patients enrolled
Trial Details
Trial ID
NCT04411082
Start Date
October 16 2020
End Date
May 4 2022
Last Update
May 15 2025
Active Locations (36)
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1
Herlev Hospital
Herlev, Capital Region, Denmark, 2730
2
Institut Universitaire du Cancer de Toulouse Oncopole
Toulouse, Haute-Garonn, France, 31059
3
Hôpital Edouard Herriot
Lyon, Rhone, France, 69437
4
Hôpital Necker-Enfants Malades
Paris, France, 75015