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
Status: Recruiting
Location: See all (22) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2
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
Participation Requirements
Sex: All
Minimum Age: 18
Maximum Age: 65
Healthy Volunteers: f
View:
• 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
Locations
United States
New York
Weill Cornell Medicine
RECRUITING
New York
Other Locations
Georgia
K Eristavi National Center of Experimental and Clinical Surgery
RECRUITING
Tbilisi
Medinvest Institute of Hematology and Transfusiology Limited
RECRUITING
Tbilisi
India
Hemato-Oncology Clinic Ahmedabad PVT. LTD.
RECRUITING
Ahmedabad
Indraprastha Apollo Hospitals
RECRUITING
Delhi
JK Lon Hospital
RECRUITING
Jaipur
Amrita Institute of Medical Sciences and Research Centre (AIMS)
RECRUITING
Kochi
K J Somaiya Super Specialty Hospital & Research Centre
RECRUITING
Mumbai
All India Institute of Medical Sciences, New Delhi
RECRUITING
New Delhi
Nirmal Hospital Pvt. Ltd - Surat
RECRUITING
Surat
Italy
Day Hospital Thalassemia and Haemoglobinopathies (DHTE); A.O.U S.Anna
RECRUITING
Ferrara
Malaysia
Hospital Ampang
RECRUITING
Ampang
Hospital Queen Elizabeth
RECRUITING
Kota Kinabalu
Hospital Tunku Ampuan Besar Tuanku Aishah Rohani (HPKK UKM)
RECRUITING
Kuala Lumpur
Sarawak General Hospital
RECRUITING
Kuching
Thailand
Chulalongkorn University
RECRUITING
Bangkok
Phramongkutklao Hospital
RECRUITING
Bangkok
Chiang Mai University
RECRUITING
Chiang Mai
Songklanagarind Hospital, Prince of Songkla University