NCT06421636Active, not recruitingPhase 2
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
A PHASE 2, TWO-PART, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, MULTICENTER STUDY TO EVALUATE THE EFFICACY, SAFETY, AND TOLERABILITY OF SUBCUTANEOUSLY ADMINISTERED REGN7999 (AN INHIBITOR OF TMPRSS6) IN PARTICIPANTS WITH IRON OVERLOAD DUE TO NON-TRANSFUSION DEPENDENT β-THALASSEMIA
Source: ClinicalTrials.gov · Last updated 1 Jul 2026
Brief summary (registry, in English)
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)
- Status (registry)
- Active, not recruiting
- Phase (registry)
- Phase 2
- Study type
- Interventional
- Enrollment
- 19 (actual)
- First submitted
- 14 May 2024
- Start date
- 30 Sept 2024
- Primary completion date
- 3 Dec 2026
- Lead sponsor
- Regeneron Pharmaceuticals
- Diseases
- Thalassaemia
- NCT number
- NCT06421636
- EU CT number
- 2023-508604-37-00
What you commit to
- Study type: Interventional · Phase 2
- Participants enrolled: 19
- From 30 Sept 2024 to the primary completion date, 3 Dec 2026
- 29 sites in 9 countries
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- For Part A only: Any Iron Chelation Therapy (ICT) use in approximately 12 weeks prior to screening as described in the protocol
- For Part B only: If on ICT, any change in 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
Other illness
- Diagnosis of Chronic kidney disease (CKD) stage 4 or higher
- Note: Other protocol-defined Inclusion/ Exclusion Criteria apply
Lab values
- Hemoglobin ≤ 8 g/dL at screening
Other
- Any RBC transfusion within 12 weeks of visit 3
- Absolute contraindication to MRI
- Diagnosis of cirrhosis of the liver
Who the study is looking for
- 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
Age in the registry: 18 years to 65 years · Sex: All · Does not accept healthy volunteers
Sites (29)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Imperial College Healthcare NHS Trust Coordinator of this site? Confirm its status | London | United Kingdom | Unknown status |
| University College London Hospitals NHS Trust Coordinator of this site? Confirm its status | London | United Kingdom | Unknown status |
| Medinvest Institute of Hematology and Transfusiology Limited Coordinator of this site? Confirm its status | Tbilisi | Georgia | Unknown status |
| K. Eristavi National Center of Experimental and Clinical Surgery Coordinator of this site? Confirm its status | Tbilisi | Georgia | Unknown status |
| Laiko General Hospital of Athens Coordinator of this site? Confirm its status | Athens | Greece | Completed |
| Nosokomeio Paidon I Agia Sofia Coordinator of this site? Confirm its status | Athens | Greece | Completed |
| University General Hospital of Ioannina Coordinator of this site? Confirm its status | Ioannina | Greece | Completed |
| Hemato Oncology Clinic Ahmedabad Pvt. Ltd. Coordinator of this site? Confirm its status | Ahmedabad | India | Unknown status |
| JK Lon Hospital Coordinator of this site? Confirm its status | Jaipur | India | Unknown status |
| Amrita Institute of Medical Sciences and Research Centre (AIMS) Coordinator of this site? Confirm its status | Kochi | India | Unknown status |
| K J Somaiya Super Specialty Hospital & Research Centre Coordinator of this site? Confirm its status | Mumbai | India | Unknown status |
| All India Institute of Medical Sciences, New Delhi Coordinator of this site? Confirm its status | New Delhi | India | Unknown status |
| Indraprastha Apollo Hospitals Coordinator of this site? Confirm its status | New Delhi | India | Unknown status |
| Nirmal Hospital Pvt. Ltd - Surat Coordinator of this site? Confirm its status | Surat | India | Unknown status |
| University Hospital Of Ferrara Coordinator of this site? Confirm its status | Ferrara | Italy | Completed |
| Ente Ospedaliero Ospedali Galliera Di Genova Coordinator of this site? Confirm its status | Genoa | Italy | Completed |
| Azienda Ospedaliera Ospedali Riuniti Villa Sofia - Cervello Coordinator of this site? Confirm its status | Palermo | Italy | Completed |
| Ospedale Pediatrico Bambino Gesù Coordinator of this site? Confirm its status | Rome | Italy | Completed |
| Hospital Ampang Coordinator of this site? Confirm its status | Ampang | Malaysia | Unknown status |
| Hospital Queen Elizabeth Coordinator of this site? Confirm its status | Kota Kinabalu | Malaysia | Unknown status |
| Hospital Tunku Ampuan Besar Tuanku Aishah Rohani (HPKK UKM) Coordinator of this site? Confirm its status | Kuala Lumpur | Malaysia | Unknown status |
| Sarawak General Hospital Coordinator of this site? Confirm its status | Kuching | Malaysia | Unknown status |
| Phramongkutklao Hospital Coordinator of this site? Confirm its status | Bangkok | Thailand | Unknown status |
| Chulalongkorn University Coordinator of this site? Confirm its status | Bangkok | Thailand | Unknown status |
| Chiang Mai University Coordinator of this site? Confirm its status | Chiang Mai | Thailand | Unknown status |
| Songklanagarind Hospital Prince of Songkla University Coordinator of this site? Confirm its status | Hat Yai | Thailand | Unknown status |
| Srinagarind Hospital Coordinator of this site? Confirm its status | Khon Kaen | Thailand | Unknown status |
| Weill Cornell Medicine Coordinator of this site? Confirm its status | New York | United States | Unknown status |
| Hacettepe University Coordinator of this site? Confirm its status | Ankara | Turkey (Türkiye) | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.