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

NCT06421636 | Active Not Recruiting Phase 2 FDA Drug

• 2 other identifiers: R7999-BThal-23502023-508604-37-00
• Study Type: interventional
• Target: 19
• Locations: 9 countries
• Sites: 26

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)

Conditions

Non-Transfusion Dependent Beta-Thalassemia (NTDT)

Keywords

Beta-Thalassemia; Resultant Iron Overload (IOL)

Outcome Measures

Primary Outcomes (3)

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

  At week 24

• Incidence of Treatment-Emergent Adverse Events (TEAEs)

  Up to week 72

• Severity of TEAEs

  Up to week 72

Secondary Outcomes (13)

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

  Through week 52

• Change from baseline in hemoglobin

  To week 24

• Change from baseline in LIC by R2* MRI

  At week 52

• Percent change from baseline in LIC by R2* MRI

  To week 24 and week 52

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

  Through week 24

Study Arms (6)

Part A High Dose

EXPERIMENTAL

Randomized 2:2:1

Drug: REGN7999

Part A Low Dose

EXPERIMENTAL

Randomized 2:2:1

Drug: REGN7999

Part A Placebo

PLACEBO COMPARATOR

Randomized 2:2:1

Drug: Placebo

Part B High Dose

EXPERIMENTAL

Randomized 2:2:1

Drug: REGN7999

Part B Low Dose

EXPERIMENTAL

Randomized 2:2:1

Drug: REGN7999

Part B Placebo

PLACEBO COMPARATOR

Randomized 2:2:1

Drug: Placebo

Interventions

REGN7999 DRUG

Administered subcutaneous (SC)

Part A High Dose; Part A Low Dose; Part B High Dose; Part B Low Dose

Placebo DRUG

Administered SC

Part A Placebo; Part B Placebo

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Regeneron Pharmaceuticals: lead

Study Sites (26)

Weill Cornell Medicine

New York, New York, 10065, United States

Location

K Eristavi National Center of Experimental and Clinical Surgery

Tbilisi, 0159, Georgia

Location

Medinvest Institute of Hematology and Transfusiology Limited

Tbilisi, 0160, Georgia

Location

Children's Hospital Agia Sophia

Athens, Attica, 11521, Greece

Location

Laiko General Hospital of Athens

Athens, Attica, 11527, Greece

Location

University Hospital of Ioannina

Ioannina, 45110, Greece

Location

Hemato-Oncology Clinic Ahmedabad PVT. LTD.

Ahmedabad, Gujarat, 380009, India

Location

Nirmal Hospital Pvt. Ltd - Surat

Surat, Gujarat, 395002, India

Location

Amrita Institute of Medical Sciences and Research Centre (AIMS)

Kochi, Kerala, 682041, India

Location

K J Somaiya Super Specialty Hospital & Research Centre

Mumbai, Maharashtra, 400022, India

Location

All India Institute of Medical Sciences, New Delhi

New Delhi, National Capital Territory of Delhi, 110029, India

Location

Indraprastha Apollo Hospitals

New Delhi, National Capital Territory of Delhi, 110048, India

Location

JK Lon Hospital

Jaipur, Rajasthan, 302004, India

Location

Day Hospital Thalassemia and Haemoglobinopathies (DHTE); A.O.U S.Anna

Ferrara, 44124, Italy

Location

Hospital Queen Elizabeth

Kota Kinabalu, Sabah, 88586, Malaysia

Location

Sarawak General Hospital

Kuching, Sarawak, 93586, Malaysia

Location

Hospital Ampang

Ampang, Selangor, 68000, Malaysia

Location

Hospital Tunku Ampuan Besar Tuanku Aishah Rohani (HPKK UKM)

Kuala Lumpur, 56000, Malaysia

Location

Songklanagarind Hospital, Prince of Songkla University

Hat Yai, Changwat Songkhla, 90110, Thailand

Location

Chulalongkorn University

Bangkok, 10330, Thailand

Location

Phramongkutklao Hospital

Bangkok, 10400, Thailand

Location

Chiang Mai University

Chiang Mai, 50200, Thailand

Location

Srinagarind Hospital

Khon Kaen, 40000, Thailand

Location

Hacettepe University

Ankara, 06100, Turkey (Türkiye)

Location

University College London Hospitals NHS Trust

London, NW1 2PG, United Kingdom

Location

Imperial College Healthcare NHS Trust

London, W12 0HS, United Kingdom

Location

MeSH Terms

Conditions

beta-Thalassemia

Condition Hierarchy (Ancestors)

Thalassemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Clinical Trial Management

  Regeneron Pharmaceuticals

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 14, 2024
• First Posted: May 20, 2024
• Study Start: September 30, 2024
• Primary Completion (Estimated): December 3, 2026
• Study Completion (Estimated): December 3, 2026
• Last Updated: July 1, 2026

Record last verified: 2026-06

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR, ANALYTIC CODE
• Time Frame: When Regeneron has: * received marketing authorization from major health authorities (e.g., FDA, European Medicines Agency (EMA), Pharmaceuticals and Medical Devices Agency (PMDA), etc.) for the product and indication or has globally discontinued development of the product for all indications on or after April 2020 and has no plans for future development * made the study results publicly available (e.g., scientific publication, scientific conference, clinical trial registry) * the legal authority to share the data, and * ensured the ability to protect participant privacy
• Access Criteria: Qualified researchers can submit a proposal for access to individual patient or aggregate level data from a Regeneron-sponsored clinical trial through Vivli. Regeneron's Independent Research Request Evaluation Criteria can be found at: https://www.regeneron.com/sites/default/files/Regeneron-External-Data-Sharing-Policy-and-Independent-Research-Request-Evaluation-Criteria.pdf

Locations

GE (2); MY (4); GB (2); TH (5); GR (3); US (1); IN (7); TU (1); IT (1)