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
NCT ID: NCT06421636
Last Updated: 2025-11-12
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE2
95 participants
INTERVENTIONAL
2024-09-30
2028-01-24
Brief Summary
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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
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Conditions
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Keywords
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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Part A High Dose
Randomized 2:2:1
REGN7999
Administered subcutaneous (SC)
Part A Low Dose
Randomized 2:2:1
REGN7999
Administered subcutaneous (SC)
Part A Placebo
Randomized 2:2:1
Placebo
Administered SC
Part B High Dose
Randomized 2:2:1
REGN7999
Administered subcutaneous (SC)
Part B Low Dose
Randomized 2:2:1
REGN7999
Administered subcutaneous (SC)
Part B Placebo
Randomized 2:2:1
Placebo
Administered SC
Interventions
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REGN7999
Administered subcutaneous (SC)
Placebo
Administered SC
Eligibility Criteria
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Inclusion Criteria
2. IOL, defined as LIC ≥ 5 mg Fe/g DW as measured by R2\* MRI at screening
3. Serum ferritin ≥ 300 ng/mL as described in the protocol
Exclusion Criteria
2. Any RBC transfusion within 12 weeks of visit 3
3. For Part A only: Any Iron Chelation Therapy (ICT) use in approximately 12 weeks prior to screening as described in the protocol
4. For Part B only: If on ICT, any change in ICT dose in approximately 12 weeks prior to screening as described in the protocol
5. Any use of luspatercept or mitapivat in 6 months prior to screening as described in the protocol
6. Absolute contraindication to MRI
7. Diagnosis of cirrhosis of the liver
8. Diagnosis of Chronic kidney disease (CKD) stage 4 or higher
18 Years
65 Years
ALL
No
Sponsors
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Regeneron Pharmaceuticals
INDUSTRY
Responsible Party
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Principal Investigators
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Clinical Trial Management
Role: STUDY_DIRECTOR
Regeneron Pharmaceuticals
Locations
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Weill Cornell Medicine
New York, New York, United States
K Eristavi National Center of Experimental and Clinical Surgery
Tbilisi, , Georgia
Medinvest Institute of Hematology and Transfusiology Limited
Tbilisi, , Georgia
Children's Hospital Agia Sophia
Athens, Attica, Greece
Laiko General Hospital of Athens
Athens, Attica, Greece
University Hospital of Ioannina
Ioannina, , Greece
Hemato-Oncology Clinic Ahmedabad PVT. LTD.
Ahmedabad, Gujarat, India
Nirmal Hospital Pvt. Ltd - Surat
Surat, Gujarat, India
Amrita Institute of Medical Sciences and Research Centre (AIMS)
Kochi, Kerala, India
K J Somaiya Super Specialty Hospital & Research Centre
Mumbai, Maharashtra, India
All India Institute of Medical Sciences, New Delhi
New Delhi, National Capital Territory of Delhi, India
JK Lon Hospital
Jaipur, Rajasthan, India
Indraprastha Apollo Hospitals
Delhi, , India
Day Hospital Thalassemia and Haemoglobinopathies (DHTE); A.O.U S.Anna
Ferrara, , Italy
Hospital Queen Elizabeth
Kota Kinabalu, Sabah, Malaysia
Sarawak General Hospital
Kuching, Sarawak, Malaysia
Hospital Ampang
Ampang, Selangor, Malaysia
Hospital Tunku Ampuan Besar Tuanku Aishah Rohani (HPKK UKM)
Kuala Lumpur, , Malaysia
Songklanagarind Hospital, Prince of Songkla University
Hat Yai, Changwat Songkhla, Thailand
Chulalongkorn University
Bangkok, , Thailand
Phramongkutklao Hospital
Bangkok, , Thailand
Chiang Mai University
Chiang Mai, , Thailand
Srinagarind Hospital
Khon Kaen, , Thailand
University College London Hospitals NHS Trust
London, , United Kingdom
Imperial College Healthcare NHS Trust
London, , United Kingdom
Countries
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Central Contacts
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Other Identifiers
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2023-508604-37-00
Identifier Type: CTIS
Identifier Source: secondary_id
R7999-BThal-2350
Identifier Type: -
Identifier Source: org_study_id