NCT06421636Active, not recruitingPhase 2
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
Regeneron Pharmaceuticals50 sites in 9 countries19 target enrollmentStarted: September 30, 2024Last updated:
Interventions
Drugs
Trial Snapshot
- Phase
- Phase 2
- Status
- Active, not recruiting
- Sponsor
- Regeneron Pharmaceuticals
- Enrollment
- 19
- Locations
- 50
- Primary Endpoint
- Change from baseline in Liver Iron Concentration (LIC) by R2* Magnetic Resonance Imaging (MRI)
Study Overview
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)
Study Design
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel
- Primary Purpose
- Treatment
- Masking
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Eligibility Criteria
- Ages
- 18 Years to 65 Years (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •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
Exclusion Criteria
- •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
- •Note: Other protocol-defined Inclusion/ Exclusion Criteria apply
Arms & Interventions
Part A Placebo
Placebo Comparator
Randomized 2:2:1
Intervention: Placebo (Drug)
Part A High Dose
Experimental
Randomized 2:2:1
Intervention: REGN7999 (Drug)
Part A Low Dose
Experimental
Randomized 2:2:1
Intervention: REGN7999 (Drug)
Part B High Dose
Experimental
Randomized 2:2:1
Intervention: REGN7999 (Drug)
Part B Placebo
Placebo Comparator
Randomized 2:2:1
Intervention: Placebo (Drug)
Part B Low Dose
Experimental
Randomized 2:2:1
Intervention: REGN7999 (Drug)
Outcomes
Primary Outcomes
Change from baseline in Liver Iron Concentration (LIC) by R2* Magnetic Resonance Imaging (MRI)
Time Frame: At week 24
Severity of TEAEs
Time Frame: Up to week 72
Incidence of Treatment-Emergent Adverse Events (TEAEs)
Time Frame: Up to week 72
Secondary Outcomes
- Change from baseline in hemoglobin(To week 24)
- Change from baseline in LIC by R2* MRI(At week 52)
- Achievement of ≥20% reduction from baseline in LIC by R2* MRI(Through week 24)
- Percent change from baseline in LIC by R2* MRI(To week 24 and week 52)
- Change in RBC counts over time(Baseline to week 56)
- Concentrations of REGN7999 in serum over time(Up to week 56)
- Number of RBC transfusions required(Baseline to week 72)
- Change in hemoglobin over time(Up to week 56)
- Achievement of ≥1.5 g/dL increase in hemoglobin for two consecutive assessments in the absence of Red Blood Cell (RBC) transfusions(Baseline to week 56)
- Achievement of transfusion independence(Baseline to week 72)
- Incidence of Anti-Drug Antibody (ADA) to REGN7999 over time(Up to week 56)
- Magnitude of ADA to REGN7999 over time(Up to week 56)
- Change in hemoglobin over time(Up to week 56)
- Number of RBC transfusions required(Baseline to week 72)
- Achievement of transfusion independence(Baseline to week 72)
- Concentrations of REGN7999 in serum over time(Up to week 56)
- Achievement of ≥20% reduction from baseline in LIC by R2* MRI(Through week 52)
Investigators
Study Sites (50)
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