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

NCT06421636 · tracked via the Priya Life Science UK tracker

Sponsor

Regeneron Pharmaceuticals

Phase

Phase 2

Started

2024-09-30

Last updated

2026-07-01

Condition(s) studied

Non-Transfusion Dependent Beta-Thalassemia (NTDT)

Investigational drug(s) / intervention(s)

REGN7999Placebo

REGN7999: Administered subcutaneous (SC)

Placebo: Administered SC

Study 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)

Eligibility

Sex

ALL

Min age

18 Years

Max age

65 Years

Healthy volunteers

Key Inclusion Criteria: 1. Clinical diagnosis of NTDT as described in the protocol 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 Key Exclusion Criteria: 1. Hemoglobin ≤ 8 g/dL at screening 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 Note: Other protocol-defined Inclusion/ Exclusion Criteria apply

Primary outcome measure(s)

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

Trial sites (26)

Facility	City	Region
Weill Cornell Medicine	New York	New York
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
Laiko General Hospital of Athens	Athens	Attica
University Hospital of Ioannina	Ioannina	Greece
Hemato-Oncology Clinic Ahmedabad PVT. LTD.	Ahmedabad	Gujarat
Nirmal Hospital Pvt. Ltd - Surat	Surat	Gujarat
Amrita Institute of Medical Sciences and Research Centre (AIMS)	Kochi	Kerala
K J Somaiya Super Specialty Hospital & Research Centre	Mumbai	Maharashtra
All India Institute of Medical Sciences, New Delhi	New Delhi	National Capital Territory of Delhi
Indraprastha Apollo Hospitals	New Delhi	National Capital Territory of Delhi
JK Lon Hospital	Jaipur	Rajasthan
Day Hospital Thalassemia and Haemoglobinopathies (DHTE); A.O.U S.Anna	Ferrara	Italy
Hospital Queen Elizabeth	Kota Kinabalu	Sabah
Sarawak General Hospital	Kuching	Sarawak
Hospital Ampang	Ampang	Selangor
Hospital Tunku Ampuan Besar Tuanku Aishah Rohani (HPKK UKM)	Kuala Lumpur	Malaysia
Songklanagarind Hospital, Prince of Songkla University	Hat Yai	Changwat Songkhla
Chulalongkorn University	Bangkok	Thailand
Phramongkutklao Hospital	Bangkok	Thailand
Chiang Mai University	Chiang Mai	Thailand
Srinagarind Hospital	Khon Kaen	Thailand
Hacettepe University	Ankara	Turkey (Türkiye)
University College London Hospitals NHS Trust	London	United Kingdom
Imperial College Healthcare NHS Trust	London	United Kingdom

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Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06421636 on ClinicalTrials.gov ↗ ← All trials in the UK