New drug aims to tackle iron overload in thalassemia patients

NCT ID NCT06421636

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

First seen Jun 27, 2026 · Last updated Jul 01, 2026 · Updated 1 time

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.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for NON-TRANSFUSION DEPENDENT BETA-THALASSEMIA (NTDT) are added.

Our safety recommendation!

By submitting, you agree to our Terms of use

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

Show contact details

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