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 2RecruitingNCT06421636

Regeneron Pharmaceuticals95 enrolled

Overview

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)

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

Conditions

Non-Transfusion Dependent Beta-Thalassemia (NTDT)

Interventions

Eligibility

Sex: ALLMin age: 18 YearsMax age: 65 Years

Medical Language ↔ Plain English

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

Locations (25)

Weill Cornell Medicine

New York, New York, United States

RECRUITING

K Eristavi National Center of Experimental and Clinical Surgery

Tbilisi, Georgia

RECRUITING

Medinvest Institute of Hematology and Transfusiology Limited

Tbilisi, Georgia

RECRUITING

Children's Hospital Agia Sophia

Athens, Attica, Greece

Outcomes

Primary Outcomes

Change from baseline in Liver Iron Concentration (LIC) by R2* Magnetic Resonance Imaging (MRI)

Time frame: At week 24

Incidence of Treatment-Emergent Adverse Events (TEAEs)

Time frame: Up to week 72

Severity of TEAEs

Time frame: Up to week 72

Secondary Outcomes

Achievement of ≥20% reduction from baseline in LIC by R2* MRI

Time frame: Through week 52

Change from baseline in hemoglobin

Time frame: To week 24

Change from baseline in LIC by R2* MRI

Time frame: At week 52

Percent change from baseline in LIC by R2* MRI

Time frame: To week 24 and week 52

Achievement of ≥20% reduction from baseline in LIC by R2* MRI

Time frame: Through week 24

Change in hemoglobin over time

Time frame: 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

Time frame: Baseline to week 56

Number of RBC transfusions required

Time frame: Baseline to week 72

Achievement of transfusion independence

Time frame: Baseline to week 72

Change in RBC counts over time

Time frame: Baseline to week 56

Concentrations of REGN7999 in serum over time

Time frame: Up to week 56

Incidence of Anti-Drug Antibody (ADA) to REGN7999 over time

Time frame: Up to week 56

Magnitude of ADA to REGN7999 over time

Time frame: Up to week 56

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

Overview

Conditions

Interventions

Eligibility

Locations (25)

Outcomes

Primary Outcomes

Secondary Outcomes

Central Contacts