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

Phase 2

Recruiting

• Conditions: Non-Transfusion Dependent Beta-Thalassemia (NTDT)
• Interventions: Drug: REGN7999; 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

Study Timeline

• Study First Submitted: 2024-05-14
• Study First Submitted QC: 2024-05-14
• Study First Posted: 2024-05-20
• Study Start: 2024-09-30
• Status Verified: 2025-08
• Last Update Submitted: 2025-08-27
• Last Update Posted: 2025-08-28
• Primary Completion: 2028-01-24
• Study Completion: 2028-01-24

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 95

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

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 from baseline in Liver Iron Concentration (LIC) by R2* Magnetic Resonance Imaging (MRI)	At week 24
Severity of TEAEs	Up to week 72
Incidence of Treatment-Emergent Adverse Events (TEAEs)	Up to week 72

Secondary Outcome Measures

Name	Time	Method
Change from baseline in hemoglobin	To week 24
Change from baseline in LIC by R2* MRI	At week 52
Achievement of ≥20% reduction from baseline in LIC by R2* MRI	Through week 24
Percent change from baseline in LIC by R2* MRI	To week 24 and week 52
Change in RBC counts over time	Baseline to week 56
Concentrations of REGN7999 in serum over time	Up to week 56
Number of RBC transfusions required	Baseline to week 72
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
Achievement of transfusion independence	Baseline to week 72
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

Trial Locations

Locations (22)

Day Hospital Thalassemia and Haemoglobinopathies (DHTE); A.O.U S.Anna; 🇮🇹 Ferrara, Italy

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

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

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