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| ID | Type | Description | Link |
|---|---|---|---|
| 2023-508604-37-00 | EU Trial (CTIS) Number |
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This study is researching an experimental drug called REGN7999 (called "study drug"). The study is focused on patients with non-transfusion dependent beta-thalassemia. The aim of the study is to see how safe and effective the study drug is.
The study is looking at several other research questions, including:
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Part A High Dose | Experimental | Randomized 2:2:1 |
|
| Part A Low Dose | Experimental | Randomized 2:2:1 |
|
| Part A Placebo | Placebo Comparator | Randomized 2:2:1 |
|
| Part B High Dose | Experimental | Randomized 2:2:1 |
|
| Part B Low Dose | Experimental | Randomized 2:2:1 |
|
| Part B Placebo | Placebo Comparator | Randomized 2:2:1 |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| REGN7999 | Drug | Administered subcutaneous (SC) |
|
| Measure | Description | Time Frame |
|---|---|---|
| Change from baseline in Liver Iron Concentration (LIC) by R2* Magnetic Resonance Imaging (MRI) | At week 24 | |
| Incidence of Treatment-Emergent Adverse Events (TEAEs) | Up to week 72 | |
| Severity of TEAEs | Up to week 72 |
| Measure | Description | Time Frame |
|---|---|---|
| Achievement of ≥20% reduction from baseline in LIC by R2* MRI | Through week 52 | |
| Change from baseline in hemoglobin | To week 24 | |
| Change from baseline in LIC by R2* MRI |
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Key Inclusion Criteria:
Key Exclusion Criteria:
Note: Other protocol-defined Inclusion/ Exclusion Criteria apply
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| Name | Affiliation | Role |
|---|---|---|
| Clinical Trial Management | Regeneron Pharmaceuticals | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Weill Cornell Medicine | New York | New York | 10065 | United States | ||
| K Eristavi National Center of Experimental and Clinical Surgery |
All Individual Patient Data (IPD) that underlie publicly available results will be considered for sharing.
When Regeneron has:
Qualified researchers can submit a proposal for access to individual patient or aggregate level data from a Regeneron-sponsored clinical trial through Vivli. Regeneron's Independent Research Request Evaluation Criteria can be found at: https://www.regeneron.com/sites/default/files/Regeneron-External-Data-Sharing-Policy-and-Independent-Research-Request-Evaluation-Criteria.pdf
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| Placebo | Drug | Administered SC |
|
| At week 52 |
| Percent change from baseline in LIC by R2* MRI | To week 24 and week 52 |
| Achievement of ≥20% reduction from baseline in LIC by R2* MRI | Through week 24 |
| Change in hemoglobin over time | Up to week 56 |
| Achievement of ≥1.5 g/dL increase in hemoglobin for two consecutive assessments in the absence of Red Blood Cell (RBC) transfusions | Baseline to week 56 |
| Number of RBC transfusions required | Baseline to week 72 |
| Achievement of transfusion independence | Baseline to week 72 |
| Change in RBC counts over time | Baseline to week 56 |
| Concentrations of REGN7999 in serum over time | Up to week 56 |
| Incidence of Anti-Drug Antibody (ADA) to REGN7999 over time | Up to week 56 |
| Magnitude of ADA to REGN7999 over time | Up to week 56 |
| Tbilisi |
| 0159 |
| Georgia |
| Medinvest Institute of Hematology and Transfusiology Limited | Tbilisi | 0160 | Georgia |
| Children's Hospital Agia Sophia | Athens | Attica | 11521 | Greece |
| Laiko General Hospital of Athens | Athens | Attica | 11527 | Greece |
| University Hospital of Ioannina | Ioannina | 45110 | Greece |
| Hemato-Oncology Clinic Ahmedabad PVT. LTD. | Ahmedabad | Gujarat | 380009 | India |
| Nirmal Hospital Pvt. Ltd - Surat | Surat | Gujarat | 395002 | India |
| Amrita Institute of Medical Sciences and Research Centre (AIMS) | Kochi | Kerala | 682041 | India |
| K J Somaiya Super Specialty Hospital & Research Centre | Mumbai | Maharashtra | 400022 | India |
| All India Institute of Medical Sciences, New Delhi | New Delhi | National Capital Territory of Delhi | 110029 | India |
| Indraprastha Apollo Hospitals | New Delhi | National Capital Territory of Delhi | 110048 | India |
| JK Lon Hospital | Jaipur | Rajasthan | 302004 | India |
| Day Hospital Thalassemia and Haemoglobinopathies (DHTE); A.O.U S.Anna | Ferrara | 44124 | Italy |
| Hospital Queen Elizabeth | Kota Kinabalu | Sabah | 88586 | Malaysia |
| Sarawak General Hospital | Kuching | Sarawak | 93586 | Malaysia |
| Hospital Ampang | Ampang | Selangor | 68000 | Malaysia |
| Hospital Tunku Ampuan Besar Tuanku Aishah Rohani (HPKK UKM) | Kuala Lumpur | 56000 | Malaysia |
| Songklanagarind Hospital, Prince of Songkla University | Hat Yai | Changwat Songkhla | 90110 | Thailand |
| Chulalongkorn University | Bangkok | 10330 | Thailand |
| Phramongkutklao Hospital | Bangkok | 10400 | Thailand |
| Chiang Mai University | Chiang Mai | 50200 | Thailand |
| Srinagarind Hospital | Khon Kaen | 40000 | Thailand |
| Hacettepe University | Ankara | 06100 | Turkey (Türkiye) |
| University College London Hospitals NHS Trust | London | NW1 2PG | United Kingdom |
| Imperial College Healthcare NHS Trust | London | W12 0HS | United Kingdom |
| ID | Term |
|---|---|
| D017086 | beta-Thalassemia |
| ID | Term |
|---|---|
| D013789 | Thalassemia |
| D000745 | Anemia, Hemolytic, Congenital |
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D006453 | Hemoglobinopathies |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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