Clinical trial NCT06421636: 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
Official title: 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
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
Conditions: Non-transfusion Dependent Beta-thalassemia (NTDT)
Keywords: Beta-thalassemia
Resultant Iron overload (IOL)
Lead sponsor: Regeneron Pharmaceuticals
Sponsor class: INDUSTRY
First posted: 2024-05-20
Enrollment: 95
Sex: ALL
Minimum age (years): 18
Maximum age (years): 60
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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)


Eligibility criteria: 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
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 as described in the protocol
4. For Part B only: If on ICT, any change in Iron chelation therapy (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

Primary outcomes:
measure: Change in Liver iron concentration (LIC) by R2* magnetic resonance imaging (MRI)
timeFrame: Baseline to week 24
measure: Incidence of Treatment-emergent adverse events (TEAEs)
timeFrame: Up to week 72
measure: Severity of Treatment-emergent adverse events (TEAEs)
timeFrame: Up to week 72
Secondary outcomes:
measure: Achievement of ≥20% reduction in LIC by R2* MRI
timeFrame: Baseline to week 52
measure: Change in hemoglobin
timeFrame: Baseline to week 24
measure: Change in LIC by R2* MRI
timeFrame: Baseline to week 52
measure: Percent change in LIC by R2* MRI
timeFrame: Baseline to week 24 and week 52
measure: Achievement of ≥20% reduction in LIC by R2* MRI
timeFrame: Baseline to week 24
measure: Change in hemoglobin over time
timeFrame: Up to week 56
measure: Achievement of ≥1.5 g/dL increase in hemoglobin for two consecutive assessments in the absence of red blood cell (RBC) transfusions
timeFrame: Baseline to week 56
measure: Number of RBC transfusions required
timeFrame: Baseline to week 72
measure: Achievement of transfusion independence
timeFrame: Baseline to week 72
measure: Change in RBC counts over time
timeFrame: Baseline to week 56
measure: Concentrations of REGN7999 in serum over time
timeFrame: Up to week 56
measure: Incidence of anti-drug antibody (ADA) to REGN7999 over time
timeFrame: Up to week 56
measure: Titer of ADA to REGN7999 over time
timeFrame: Up to week 56
Other outcomes:
Locations:
city: New York
country: United States
facility: Weill Cornell Medicine
state: New York
city: Tbilisi
country: Georgia
facility: K Eristavi National Center of Experimental and Clinical Surgery
city: Tbilisi
country: Georgia
facility: Medinvest Institute of Hematology and Transfusiology Limited
city: New Delhi
country: India
facility: All India Institute of Medical Sciences, New Delhi
state: Delhi
city: Ahmedabad
country: India
facility: Hemato-Oncology Clinic Ahmedabad PVT. LTD.
state: Gujarat
city: Surat
country: India
facility: Nirmal Hospital Pvt. Ltd - Surat
state: Gujarat
city: Kochi
country: India
facility: Amrita Institute of Medical Sciences and Research Centre (AIMS)
state: Kerala
city: Mumbai
country: India
facility: K J Somaiya Super Specialty Hospital & Research Centre
state: Maharashtra
city: Jaipur
country: India
facility: JK Lon Hospital
state: Rajasthan
city: Delhi
country: India
facility: Indraprastha Apollo Hospitals
city: Cheras
country: Malaysia
facility: Hospital Tunku Ampuan Besar Tuanku Aishah Rohani (HPKK UKM)
state: Kuala Lumpur
city: Kota Kinabalu
country: Malaysia
facility: Hospital Queen Elizabeth
state: Sabah
city: Kuching
country: Malaysia
facility: Sarawak General Hospital
state: Sarawak
city: Ampang
country: Malaysia
facility: Hospital Ampang
state: Selangor
city: Maung
country: Thailand
facility: Srinagarind Hospital
state: Khon Kaen
city: Hat Yai
country: Thailand
facility: Songklanagarind Hospital, Prince of Songkla University
state: Songkhla
city: Bangkok
country: Thailand
facility: Chulalongkorn University
city: Bangkok
country: Thailand
facility: Phramongkutklao Hospital
city: Chiang Mai
country: Thailand
facility: Chiang Mai University
city: London
country: United Kingdom
facility: University College London Hospitals NHS Trust
city: London
country: United Kingdom
facility: Imperial College Healthcare NHS Trust
Study officials:
affiliation: Regeneron Pharmaceuticals
name: Clinical Trial Management
role: STUDY_DIRECTOR
References: