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

Last updated: April 11, 2025
Sponsor: Regeneron Pharmaceuticals
Overall Status: Active - Recruiting

Phase

2

Condition

Thalassemia

Treatment

Placebo

REGN7999

Clinical Study ID

NCT06421636
R7999-BThal-2350
2023-508604-37-00
  • Ages 18-60
  • All Genders

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 Criteria

Inclusion

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

Exclusion

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 asdescribed in the protocol

  4. For Part B only: If on ICT, any change in Iron chelation therapy (ICT) dose inapproximately 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 inthe 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

Total Participants: 95
Treatment Group(s): 2
Primary Treatment: Placebo
Phase: 2
Study Start date:
September 30, 2024
Estimated Completion Date:
January 24, 2028

Connect with a study center

  • K Eristavi National Center of Experimental and Clinical Surgery

    Tbilisi, 0159
    Georgia

    Active - Recruiting

  • Medinvest Institute of Hematology and Transfusiology Limited

    Tbilisi, 0160
    Georgia

    Active - Recruiting

  • All India Institute of Medical Sciences, New Delhi

    New Delhi, Delhi 110029
    India

    Active - Recruiting

  • Hemato-Oncology Clinic (HOC)

    Ahmedabad, Gujarat 380009
    India

    Active - Recruiting

  • Hemato-Oncology Clinic Ahmedabad PVT. LTD.

    Ahmedabad, Gujarat 380009
    India

    Active - Recruiting

  • Nirmal Hospital Pvt. Ltd - Surat

    Surat, Gujarat 395002
    India

    Active - Recruiting

  • Amrita Institute of Medical Sciences and Research Centre (AIMS)

    Kochi, Kerala 682041
    India

    Active - Recruiting

  • K J Somaiya Super Specialty Hospital & Research Centre

    Mumbai, Maharashtra 400022
    India

    Active - Recruiting

  • JK Lon Hospital

    Jaipur, Rajasthan 302004
    India

    Active - Recruiting

  • Indraprastha Apollo Hospitals

    Delhi, 110048
    India

    Active - Recruiting

  • Hospital Tunku Ampuan Besar Tuanku Aishah Rohani (HPKK UKM)

    Cheras, Kuala Lumpur 56000
    Malaysia

    Active - Recruiting

  • Hospital Queen Elizabeth

    Kota Kinabalu, Sabah 88586
    Malaysia

    Active - Recruiting

  • Sarawak General Hospital

    Kuching, Sarawak 93586
    Malaysia

    Active - Recruiting

  • Hospital Ampang

    Ampang, Selangor 68000
    Malaysia

    Active - Recruiting

  • Srinagarind Hospital

    Maung, Khon Kaen 40000
    Thailand

    Active - Recruiting

  • Songklanagarind Hospital, Prince of Songkla University

    Hat Yai, Songkhla 90110
    Thailand

    Active - Recruiting

  • Chulalongkorn University

    Bangkok, 10330
    Thailand

    Active - Recruiting

  • Chiang Mai University

    Chiang Mai, 50200
    Thailand

    Active - Recruiting

  • Imperial College Healthcare NHS Trust

    London, W12 0HS
    United Kingdom

    Active - Recruiting

  • University College London Hospitals NHS Trust

    London, NW1 2PG
    United Kingdom

    Active - Recruiting

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