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 (FERVENT-1)

Regeneron Pharmaceuticals

Status and phase: Enrolling; Phase 2

Conditions: Non-transfusion Dependent Beta-thalassemia (NTDT)

Treatments: Drug: REGN7999; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT06421636

R7999-BThal-2350

2023-508604-37-00 (Other Identifier)

Details and patient eligibility

About

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)

Enrollment: 95 estimated patients

Sex: All

Ages: 18 to 60 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

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
2. Any RBC transfusion within 12 weeks of visit 3
3. For Part A only: Any 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 Iron chelation therapy (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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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95 participants in 6 patient groups, including a placebo group

Part A High Dose

Experimental group

Description:

Randomized 2:2:1

Treatment:

Drug: REGN7999

Part A Low Dose

Experimental group

Description:

Randomized 2:2:1

Treatment:

Drug: REGN7999

Part A Placebo

Placebo Comparator group

Description:

Randomized 2:2:1

Treatment:

Drug: Placebo

Part B High Dose

Experimental group

Description:

Randomized 2:2:1

Treatment:

Drug: REGN7999

Part B Low Dose

Experimental group

Description:

Randomized 2:2:1

Treatment:

Drug: REGN7999

Part B Placebo

Placebo Comparator group

Description:

Randomized 2:2:1

Treatment:

Drug: Placebo