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NCT Number: NCT06421636

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

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)

Active, Not Recruiting

This study is active but is not currently recruiting participants.

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Key information

Conditions

Non-Transfusion Dependent Beta-Thalassemia (NTDT) Anemia Anemia, Hemolytic Anemia, Hemolytic, Congenital Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinopathies Thalassemia beta-Thalassemia

Age range

18 year–65 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

K Eristavi National Center of Experimental and Clinical Surgery, Tbilisi, Georgia

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Key Inclusion Criteria:

  • 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

Key Exclusion Criteria:

  • Hemoglobin ≤ 8 g/dL at screening
  • Any RBC transfusion within 12 weeks of visit 3
  • 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
  • Absolute contraindication to MRI
  • Diagnosis of cirrhosis of the liver
  • Diagnosis of Chronic kidney disease (CKD) stage 4 or higher

Note: Other protocol-defined Inclusion/ Exclusion Criteria apply

Treatment and study plan

REGN7999

Drug

Administered subcutaneous (SC)

Placebo

Drug

Administered SC

Primary outcomes

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

    Time frame: At week 24

  2. Incidence of Treatment-Emergent Adverse Events (TEAEs)

    Time frame: Up to week 72

  3. Severity of TEAEs

    Time frame: Up to week 72

Secondary outcomes

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

    Time frame: Through week 52

  2. Change from baseline in hemoglobin

    Time frame: To week 24

  3. Change from baseline in LIC by R2* MRI

    Time frame: At week 52

  4. Percent change from baseline in LIC by R2* MRI

    Time frame: To week 24 and week 52

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

    Time frame: Through week 24

  6. Change in hemoglobin over time

    Time frame: Up to week 56

  7. 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

  8. Number of RBC transfusions required

    Time frame: Baseline to week 72

  9. Achievement of transfusion independence

    Time frame: Baseline to week 72

  10. Change in RBC counts over time

    Time frame: Baseline to week 56

  11. Concentrations of REGN7999 in serum over time

    Time frame: Up to week 56

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

    Time frame: Up to week 56

  13. Magnitude of ADA to REGN7999 over time

    Time frame: Up to week 56

Sponsors and collaborators

Lead sponsor

Regeneron Pharmaceuticals

Industry

Registry information

Official study title

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

Acronym: FERVENT-1

Important dates

Study start
2024
Primary completion
2026
Study completion
2026
First posted
May 20, 2024
Registry last updated
Jul 1, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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