A Study of IMR-687 in Subjects With Beta Thalassemia
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The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT04411082 |
Recruitment Status :
Recruiting
First Posted : June 2, 2020
Last Update Posted : October 23, 2020
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
β Thalassemia | Drug: IMR-687 Drug: Placebo | Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 120 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Masking: | None (Open Label) |
Masking Description: | Double-Blind |
Primary Purpose: | Treatment |
Official Title: | A Phase 2 Study to Evaluate the Safety and Tolerability of IMR-687 in Subjects With Beta Thalassemia |
Actual Study Start Date : | October 16, 2020 |
Estimated Primary Completion Date : | May 2022 |
Estimated Study Completion Date : | May 2022 |

Arm | Intervention/treatment |
---|---|
Experimental: Lower Dose IMR-687
Oral administration of once daily IMR-687
|
Drug: IMR-687
Oral administration of once daily IMR-687 |
Experimental: Higher dose IMR-687
Oral administration of once daily IMR-687
|
Drug: IMR-687
Oral administration of once daily IMR-687 |
Placebo Comparator: Placebo
Oral administration of once daily placebo
|
Drug: Placebo
Oral administration of once daily Placebo |
- Proportion of patients with adverse events and serious adverse events [ Time Frame: Baseline to Week 40 ]
- Incidence of Adverse Events
- Incidence of Serious Adverse Events
- TDT Patients: Reduction in red blood cell (RBC) transfusion burden [ Time Frame: Week 12 through Week 36 ]
- Proportion of patients with ≥20% hematological improvement as compared to the 12 week prescreening timeframe
- Proportion of patients with ≥33% hematological improvement as compared to the 12 week prescreening timeframe
- TDT Patients: Mean number of transfusion events [ Time Frame: Baseline to Week 36, Weeks 24 to 36 ]a. Mean number of transfusion events
- TDT Patients: Mean change in ICT dose and Serum ferritin levels [ Time Frame: Baseline to Week 36, Weeks 24 to 36 ]a. Mean change in ICT dose and Serum ferritin levels
- TDT Patients: Proportion of patients that had a decrease in ICT and serum ferritin as compared to the 12 week prescreening timeframe [ Time Frame: Baseline to Week 36, Weeks 24 to 36 ]a. Proportion of patients that had a decrease in ICT and serum ferritin as compared to the 12 week prescreening timeframe
- NTDT Patients:Mean change in HbF [ Time Frame: Baseline to Week 24, Week 12 to 24, Week 24 to 36 ]a. Mean change in HbF
- NTDT Patients: Mean change in percent HbF [ Time Frame: Baseline to Week 24, Week 12 to 24, Week 24 to 36 ]a. Mean change in percent HbF
- NTDT Patients: Mean change in HbF over a continuous 12-week interval in the absence of a transfusion [ Time Frame: Baseline to Week 24, Week 12 to 24, Week 24 to 36 ]a. Mean change in HbF over a continuous 12-week interval in the absence of a transfusion
- NTDT Patients: Subject Response in HbF (increase of ≥3%) [ Time Frame: Baseline to Week 24, Week 12 to 24, Week 24 to 36 ]a. Subject Response in HbF (increase of ≥3%)
- NTDT Patients: Mean change in Hb [ Time Frame: Baseline to Week 12 to 24, Week 24 to 36 ]a. Mean change in Hb
- NTDT Patients: Mean change in Hb over a continuous 12-week interval in the absence of a transfusion [ Time Frame: Baseline to Week 12 to 24, Week 24 to 36 ]a. Mean change in Hb over a continuous 12-week interval in the absence of a transfusion
- TDT and NTDT Patients: PK Parameter Cmax [ Time Frame: Baseline to Week 36 ]a. Peak Plasma Concentration (Cmax)
- TDT and NTDT Patients: PK Parameter Area Under the Plasma Concentration versus Time Curve (AUC) [ Time Frame: Baseline to Week 36 ]a. Area Under the Plasma Concentration versus Time Curve (AUC)
- TDT and NTDT Patients: PK Parameter Tmax [ Time Frame: Baseline to Week 36 ]a. Time to maximum concentration (tmax)
- TDT and NTDT Patients: PK Parameter t ½ [ Time Frame: Baseline to Week 36 ]a. Apparent terminal half-life t ½ (half-life)
- TDT and NTDT Patients: PK Parameter AUC 0-24 [ Time Frame: Baseline to Week 36 ]a. Area Under the Plasma Concentration versus Time Curve (AUC) from time 0 to 24 hours
- TDT and NTDT Patients: PK Parameter AUClast [ Time Frame: Baseline to Week 36 ]a. 0 to the last measurable timepoint (AUClast)
- TDT and NTDT Patients: PK Parameter AUC 0-infinity [ Time Frame: Baseline to Week 36 ]a. Extrapolated to infinity (AUC0-∞)

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Ages Eligible for Study: | 18 Years to 65 Years (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Documented diagnosis of β-thalassemia or HbE/ β-thalassemia. Concomitant single alpha gene deletion, duplication, or triplication is allowed.
- Documentation of dates of transfusions and the number of all RBC units within the 12 weeks prior to Screening.
- Must be willing and able to complete all study assessments and procedures, and to communicate effectively with the investigator and site staff.
- ECOG performance score of 0-1
- Female subjects must not be pregnant, not be breast feeding, and be highly unlikely to become pregnant. Male subjects must be unlikely to impregnate a partner.
Exclusion Criteria:
- 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.
- Subjects on iron chelation therapy (ICT) at the time of ICF signing must have initiated the treatment with ICT at least 24 weeks before the predicted randomization date.
- Prior exposure to sotatercept, luspatercept, IMR-687, or gene therapy.

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04411082
Contact: Caitlon Jacoby | 617-206-2020 | trials@imaratx.com | |
Contact: Joelle Lufkin | 617-206-2020 | trials@imaratx.com |
United States, California | |
The Oncology Institute Long Beach | Recruiting |
Long Beach, California, United States, 90805 | |
Contact: Omkar Marathe, MD 562-232-0550 | |
France | |
Hôpital Necker-Enfants Malades | Recruiting |
Paris, France, 2401 | |
Contact: Laure Joseph +33 1 71 19 64 35 | |
Georgia | |
M. Zodelava Hematology Centre | Recruiting |
Tbilisi, Georgia, 0112 | |
Contact: Mamia Zodelava, MD +995599106010 | |
National Center of Surgery | Recruiting |
Tbilisi, Georgia, 0159 | |
Contact: Levani Makhaldiani, MD +995599773340 | |
Medinvest - Institute of Hematology and Transfusiology | Recruiting |
Tbilisi, Georgia, 0186 | |
Contact: Genadi Iosava, MD +995577411239 | |
Israel | |
Shaare Zedek Medical Center | Recruiting |
Jerusalem, Israel, 1205 | |
Contact: Hagit Miskin +972-2-5645681 | |
Hadassah University Hospital Ein Kerem | Recruiting |
Jerusalem, Israel, 9112001 | |
Contact: Neta Goldschmidt, MD +97226776744 | |
Galilee Medical Center | Recruiting |
Nahariya, Israel, 2210001 | |
Contact: Amir Kuperman, MD +972 507887457 |
Study Director: | Eleanor Lisbon, MD, MPH | Imara, Inc. |
Responsible Party: | Imara, Inc. |
ClinicalTrials.gov Identifier: | NCT04411082 |
Other Study ID Numbers: |
IMR-BTL-201 2019-002989-12 ( EudraCT Number ) |
First Posted: | June 2, 2020 Key Record Dates |
Last Update Posted: | October 23, 2020 |
Last Verified: | October 2020 |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Transfusion TDT NTDT |
Thalassemia beta-Thalassemia Anemia, Hemolytic, Congenital Anemia, Hemolytic |
Anemia Hematologic Diseases Hemoglobinopathies Genetic Diseases, Inborn |