A Study to Test the Safety, Tolerability, and Efficacy of an Antibody, REGN7999, Injected Under the Skin for the Treatment of Iron Overload in Adult Participants With Non-Transfusion Dependent β-thalassemia, Using MRI Scans to Measure Iron Levels in the Body
- Conditions
- Non-transfusion Dependent Beta-thalassemia (NTDT)
- Interventions
- Drug: Placebo
- Registration Number
- NCT06421636
- Lead Sponsor
- Regeneron Pharmaceuticals
- Brief Summary
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:
* Whether the study drug lowers extra iron levels in the body
* What side effects may happen from taking the study drug
* How much study drug is in the blood at different times
* Whether the body makes antibodies against the study drug (which could make the drug less effective or could lead to side effects)
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 95
- Clinical diagnosis of NTDT as described in the protocol
- IOL, defined as LIC ≥ 5 mg Fe/g DW as measured by R2* MRI at screening
- Serum ferritin ≥ 300 ng/mL as described in the protocol
Key
- Hemoglobin ≤ 8 g/dL
- Any RBC transfusion within 12 weeks of visit 3
- For Part A only: Any ICT use in approximately 12 weeks prior to screening as described in the protocol
- For Part B only: If on ICT, any change in Iron chelation therapy (ICT) dose in approximately 12 weeks prior to screening as described in the protocol
- Any use of luspatercept or mitapivat in 6 months prior to screening as described in the protocol
- Absolute contraindication to MRI
- Diagnosis of cirrhosis of the liver
- Diagnosis of Chronic kidney disease (CKD) stage 4 or higher
Note: Other protocol-defined Inclusion/ Exclusion Criteria apply
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Part B High Dose REGN7999 Randomized 2:2:1 Part B Low Dose REGN7999 Randomized 2:2:1 Part A Placebo Placebo Randomized 2:2:1 Part A Low Dose REGN7999 Randomized 2:2:1 Part B Placebo Placebo Randomized 2:2:1 Part A High Dose REGN7999 Randomized 2:2:1
- Primary Outcome Measures
Name Time Method Change in Liver iron concentration (LIC) by R2* magnetic resonance imaging (MRI) Baseline to week 24 Incidence of Treatment-emergent adverse events (TEAEs) Up to week 72 Severity of Treatment-emergent adverse events (TEAEs) Up to week 72
- Secondary Outcome Measures
Name Time Method Change in RBC counts over time Baseline to week 56 Change in hemoglobin over time Up to week 56 Achievement of transfusion independence Baseline to week 72 Achievement of ≥20% reduction in LIC by R2* MRI Baseline to week 24 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 Incidence of anti-drug antibody (ADA) to REGN7999 over time Up to week 56 Change in LIC by R2* MRI Baseline to week 52 Percent change in LIC by R2* MRI Baseline to week 24 and week 52 Number of RBC transfusions required Baseline to week 72 Titer of ADA to REGN7999 over time Up to week 56 Change in hemoglobin Baseline to week 24 Concentrations of REGN7999 in serum over time Up to week 56
Trial Locations
- Locations (19)
Nirmal Hospital Pvt. Ltd - Surat
🇮🇳Surat, Gujarat, India
Amrita Institute of Medical Sciences and Research Centre (AIMS)
🇮🇳Kochi, Kerala, India
Srinagarind Hospital
🇹🇭Maung, Khon Kaen, Thailand
University College London Hospitals NHS Trust
🇬🇧London, United Kingdom
K Eristavi National Center of Experimental and Clinical Surgery
🇬🇪Tbilisi, Georgia
Medinvest Institute of Hematology and Transfusiology Limited
🇬🇪Tbilisi, Georgia
All India Institute of Medical Sciences, New Delhi
🇮🇳New Delhi, Delhi, India
Hemato-Oncology Clinic Ahmedabad PVT. LTD.
🇮🇳Ahmedabad, Gujarat, India
K J Somaiya Super Specialty Hospital & Research Centre
🇮🇳Mumbai, Maharashtra, India
Indraprastha Apollo Hospitals
🇮🇳Delhi, India
Hospital Ampang
🇲🇾Ampang, Selangor, Malaysia
Songklanagarind Hospital, Prince of Songkla University
🇹🇭Hat Yai, Songkhla, Thailand
Chulalongkorn University
🇹🇭Bangkok, Thailand
Chiang Mai University
🇹🇭Chiang Mai, Thailand
JK Lon Hospital
🇮🇳Jaipur, Rajasthan, India
Imperial College Healthcare NHS Trust
🇬🇧London, United Kingdom
Hospital Tunku Ampuan Besar Tuanku Aishah Rohani (HPKK UKM)
🇲🇾Cheras, Kuala Lumpur, Malaysia
Hospital Queen Elizabeth
🇲🇾Kota Kinabalu, Sabah, Malaysia
Sarawak General Hospital
🇲🇾Kuching, Sarawak, Malaysia