ß Thalassemia Clinical Trial
Official title:
A Phase 2 Study to Evaluate the Safety and Tolerability of IMR-687 in Subjects With Beta Thalassemia
| Verified date | June 2022 |
| Source | Imara, Inc. |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | |
| Study type | Interventional |
A Study to Evaluate the Safety and Tolerability of IMR-687 in Subjects with Beta Thalassemia
| Status | Terminated |
| Enrollment | 122 |
| Est. completion date | May 4, 2022 |
| Est. primary completion date | March 11, 2022 |
| Accepts healthy volunteers | No |
| Gender | All |
| Age group | 18 Years to 65 Years |
| Eligibility | Inclusion Criteria: 1. Documented diagnosis of ß-thalassemia or HbE/ ß-thalassemia in their medical history. Concomitant alpha gene deletion, duplication, or triplication is allowed. 2. 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. . 3. Must be willing and able to complete all study assessments and procedures, and to communicate effectively with the investigator and site staff. 4. 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. 5. 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 6. hematopoietic stem cell transplantation within 9 months. 7. 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. 8. ECOG performance score of 0 to 1 9. 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 Criteria: 1. Diagnosis of a-thalassemia (e.g., hemoglobin H [HbH]) or hemoglobin S (HbS)/ ß thalassemia. 2. Body mass index (BMI) <17.0 kg/m2 or a total body weight <45 kg; or BMI >35 kg/m2 3. 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). 4. Stroke requiring medical intervention =24 weeks prior to randomization. 5. Platelet count >1000 × 109/L. 6. 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. 7. 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. 8. Prior exposure to sotatercept or luspatercept, IMR-687, or gene therapy within 6 months prior to randomization (Day 1). 9. Subjects who have major organ damage |
| Country | Name | City | State |
|---|---|---|---|
| Denmark | Herlev Hospital | Herlev | Hovedstaden |
| France | Hôpital Edouard Herriot | Lyon Cedex 03 | Rhone |
| France | Hôpital Necker-Enfants Malades | Paris | |
| France | Institut Universitaire du Cancer de Toulouse Oncopole | Toulouse cedex 9 | Haute-Garonn |
| Georgia | M. Zodelava Hematology Centre | Tbilisi | Borjomi |
| Georgia | Medinvest - Institute of Hematology and Transfusiology | Tbilisi | |
| Georgia | National Center of Surgery | Tbilisi | |
| Greece | Aghia Sofia General Children's Hospital | Athens | Attica |
| Greece | Laiko General Hospital of Athens | Athens | Attica |
| Greece | University General Hospital of Patras | Patra | Peloponnese |
| Greece | Ippokrateio General Hospital of Thessaloniki | Thessaloníki | Central Macedonia |
| Israel | Emek Medical Center | Afula | |
| Israel | Rambam Health Care Campus | Haifa | Haifa District |
| Israel | Hadassah University Hospital Ein Kerem | Jerusalem | Jerusalem District |
| Israel | The Galilee Medical Center | Nahariya | Northern District |
| Italy | Azienda Ospedaliera Giuseppe Brotzu | Orbassano | Turin |
| Italy | Azienda Ospedaliera Universitaria - Università degli Studi della Campania Luigi Vanvitelli | Orbassano | Turin |
| Lebanon | Chronic Care Center | Hazmiyeh | |
| Malaysia | Hospital Sultanah Bahiyah | Alor Setar | Kedah |
| Malaysia | Hospital Pulau Pinang | George Town | Penang |
| Malaysia | Hospital Raja Permaisuri Bainun | Ipoh | Perak |
| Malaysia | Hospital Sultanah Aminah Johor Bharu | Johor Bahru | Johor |
| Malaysia | Hospital Queen Elizabeth - Kota Kinabalu | Kota Kinabalu | Sabah |
| Malaysia | Hospital Umum Sarawak | Kuching | Sarawak |
| Morocco | Hôpital d'Enfants Rabat | Rabat | |
| Netherlands | Amsterdam Universitair Medische Centra - Academisch Medisch Centrum | Amsterdam | North Holland |
| Tunisia | Centre Hôpital Universitaire Farhat Hached | Sousse | |
| Tunisia | Centre National de Greffe de la Moelle Osseuse | Tunis | |
| Tunisia | Hospital Aziza Othmana | Tunis | |
| Turkey | Hacettepe Üniversitesi | Ankara | |
| Turkey | Ege Universitesi Tip Fakultesi | Izmir | |
| Turkey | Akdeniz Üniversitesi | Mersin | Icel |
| Turkey | Mersin Üniversitesi Tip Fakültesi | Mersin | Icel |
| United Kingdom | University College London Hospitals NHS Foundation Trust | London | England |
| United Kingdom | Whittington Health NHS Trust | London | England |
| United Kingdom | Manchester University NHS Foundation Trust | Manchester | England |
| Lead Sponsor | Collaborator |
|---|---|
| Imara, Inc. |
Denmark, France, Georgia, Greece, Israel, Italy, Lebanon, Malaysia, Morocco, Netherlands, Tunisia, Turkey, United Kingdom,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | IMR-687 Safety and Tolerability | Incidence and severity of Adverse Events Incidence and severity of Serious Adverse Events | Baseline to Week 40 | |
| Secondary | TDT Patients: Reduction in Red Blood Cell (RBC) Transfusion Burden With =33% Hematological Improvement From Week 12 to Week 24 | Proportion of patients with =33% hematological improvement (as measured by reduced transfusion burden) from Week 12 to Week 24 compared to the 12 weeks prior to Baseline (Day 1) | Baseline to Week 24 | |
| Secondary | NTDT Patients: Proportion of Subjects With an Increase From Baseline of Hb at Week 12 to Week 24 in the Absence of Transfusions. | Proportion of subjects with an increase from baseline of =1.0 g/dL in mean Hb values at Week 12 to Week 24 in the absence of transfusions. | Baseline to Week 24 | |
| Secondary | NTDT Patients: Proportion of Subjects With an Increase From Baseline of =3% in Mean HbF Values at Week 12 to Week 24 in Absence of Transfusions | Proportion of subjects with an increase from baseline of =3% in mean HbF values at Week 12 to Week 24 in absence of transfusions | Baseline to Week 24 | |
| Secondary | TDT Patients: Reduction in Red Blood Cell (RBC) Transfusion Burden With =33% Hematological Improvement From Week 24 to Week 36 | Proportion of patients with =33% hematological improvement from Week 24 to Week 36 compared to the 12 weeks prior to Baseline (Day 1) | Baseline to Week 36 | |
| Secondary | TDT Patients: Reduction in Red Blood Cell (RBC) Transfusion Burden With =50 % Hematological Improvement From Week 12 to Week 24 | Proportion of patients with =50% hematological improvement from Week 12 to Week 24 compared to the 12 weeks prior to Baseline (Day 1) | Baseline to Week 24 | |
| Secondary | TDT Patients: Reduction in Red Blood Cell (RBC) Transfusion Burden With =50% Hematological Improvement From Week 24 to Week 36 | Proportion of patients with =50% hematological improvement from Week 24 to Week 36 compared to the 12 weeks prior to Baseline (Day 1) | Baseline to Week 36 | |
| Secondary | NTDT Patients: Proportion of Subjects With an Increase From Baseline of Hb at Week 24 to Week 36 in the Absence of Transfusions | Proportion of subjects with an increase from baseline of =1.0 g/dL in mean Hb values at Week 24 to Week 36 in the absence of transfusions. | Baseline to Week 36 | |
| Secondary | NTDT: Proportion of Subjects With an Increase From Baseline of =3% in Mean HbF Values at Week 24 to Week 36 in Absence of Transfusions | Proportion of subjects with an increase from baseline of =3% in mean HbF values at Week 24 to Week 36 in absence of transfusions | Baseline to Week 36 |