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New drug aims to tackle iron overload in thalassemia patients

NCT ID NCT06421636

Disease control Sponsor: Regeneron Pharmaceuticals Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jul 02, 2026 · Updated 2 times

Summary

This Phase 2 trial tests an experimental drug called REGN7999 in about 95 people with non-transfusion dependent beta-thalassemia who have too much iron in their bodies. The drug is given as a shot under the skin and aims to safely reduce iron levels in the liver. Researchers will compare it to a placebo to see if it works and what side effects occur.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
REGN7999 (a drug that blocks TMPRSS6 to help control iron levels)
What this could lead to
If it works, this could offer a new way to manage iron overload in people with beta-thalassemia who don't need regular blood transfusions.
What could go wrong
This is an early Phase 2 study with only 95 participants, so results may not apply to everyone. The drug may cause side effects or fail to lower iron enough.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

19 people

The number who actually took part.

Started

Sep 2024

Expected to finish

Dec 2026

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 to 65 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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

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Conditions

The condition(s) this trial relates to.

beta thalassemia beta-thalassemia intermedia

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Non-transfusion dependent beta-thalassemia (NTDT)

Contacts and locations

Locations

  • All India Institute of Medical Sciences, New Delhi

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

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

    Kochi, Kerala, 682041, India

  • Chiang Mai University

    Chiang Mai, 50200, Thailand

  • Children's Hospital Agia Sophia

    Athens, Attica, 11521, Greece

  • Chulalongkorn University

    Bangkok, 10330, Thailand

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

    Ferrara, 44124, Italy

  • Hacettepe University

    Ankara, 06100, Turkey (Türkiye)

  • Hemato-Oncology Clinic Ahmedabad PVT. LTD.

    Ahmedabad, Gujarat, 380009, India

  • Hospital Ampang

    Ampang, Selangor, 68000, Malaysia

  • Hospital Queen Elizabeth

    Kota Kinabalu, Sabah, 88586, Malaysia

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

    Kuala Lumpur, 56000, Malaysia

  • Imperial College Healthcare NHS Trust

    London, W12 0HS, United Kingdom

  • Indraprastha Apollo Hospitals

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

  • JK Lon Hospital

    Jaipur, Rajasthan, 302004, India

  • K Eristavi National Center of Experimental and Clinical Surgery

    Tbilisi, 0159, Georgia

  • K J Somaiya Super Specialty Hospital & Research Centre

    Mumbai, Maharashtra, 400022, India

  • Laiko General Hospital of Athens

    Athens, Attica, 11527, Greece

  • Medinvest Institute of Hematology and Transfusiology Limited

    Tbilisi, 0160, Georgia

  • Nirmal Hospital Pvt. Ltd - Surat

    Surat, Gujarat, 395002, India

  • Phramongkutklao Hospital

    Bangkok, 10400, Thailand

  • Sarawak General Hospital

    Kuching, Sarawak, 93586, Malaysia

  • Songklanagarind Hospital, Prince of Songkla University

    Hat Yai, Changwat Songkhla, 90110, Thailand

  • Srinagarind Hospital

    Khon Kaen, 40000, Thailand

  • University College London Hospitals NHS Trust

    London, NW1 2PG, United Kingdom

  • University Hospital of Ioannina

    Ioannina, 45110, Greece

  • Weill Cornell Medicine

    New York, New York, 10065, United States