REGN7999 for Non-transfusion dependent beta-thalassemia (NTDT) | Iron overload due to beta-thalassemia

Inclusion criteria

• {"criterion_text":"- Clinical diagnosis of NTDT as described in the protocol .\n- IOL, defined as LIC ≥ 5 mg Fe/g DW as measured by R2* MRI at screening.\n- Serum ferritin ≥ 300 ng/mL as described in the protocol.\n- Other protocol defined inclusion criteria apply."}

Exclusion criteria

• {"criterion_text":"- Hemoglobin ≤ 8 g/dL at screening.\n- Any RBC transfusion within 12 weeks of visit 3.\n- For Part A only: Any Iron Chelation Therapy (ICT) use in approximately 12 weeks prior to screening as described in the protocol.\n- For Part B only: If on ICT, any change in ICT dose in approximately 12 weeks prior to screening as described in the protocol.\n- Any use of luspatercept or mitapivat in 6 months prior to screening as described in the protocol.\n- Absolute contraindication to MRI.\n- Diagnosis of cirrhosis of the liver.\n- Diagnosis of Chronic kidney disease (CKD) stage 4 or higher.\n- Other protocol defined exclusion criteria apply."}

Primary endpoints

• {"endpoint_text":"- Change from baseline in Liver iron concentration (LIC) by R2* magnetic resonance imaging (MRI).","definition_or_measurement_approach":"LIC measured by R2* MRI; change from baseline."}
• {"endpoint_text":"- Incidence of Treatment-emergent adverse events (TEAEs).","definition_or_measurement_approach":"Incidence of TEAEs as reported during the on-study period (adverse event reporting)."}
• {"endpoint_text":"- Severity of Treatment-emergent adverse events (TEAEs).","definition_or_measurement_approach":"Severity grading of TEAEs (as recorded per protocol-specified AE severity criteria)."}

Secondary endpoints

• {"endpoint_text":"- Achievement of ≥20% reduction in LIC by R2* MRI.","definition_or_measurement_approach":"LIC measured by R2* MRI; achievement of ≥20% reduction from baseline."}
• {"endpoint_text":"- Change from baseline in hemoglobin.","definition_or_measurement_approach":"Change in hemoglobin concentration from baseline (laboratory measurement)."}
• {"endpoint_text":"- Change from baseline in LIC by R2* MRI.","definition_or_measurement_approach":"LIC measured by R2* MRI; change from baseline."}
• {"endpoint_text":"- Percent change from baseline in LIC by R2* MRI.","definition_or_measurement_approach":"Percent change in LIC measured by R2* MRI from baseline."}
• {"endpoint_text":"- Achievement of ≥20% reduction from baseline LIC by R2*.","definition_or_measurement_approach":"LIC measured by R2* MRI; achievement of ≥20% reduction from baseline."}
• {"endpoint_text":"- Change in hemoglobin over time.","definition_or_measurement_approach":"Serial hemoglobin measurements over study visits."}
• {"endpoint_text":"- Achievement of ≥1.5 g/dL increase in hemoglobin for two consecutive assessments in the absence of red blood cell (RBC) transfusions.","definition_or_measurement_approach":"Hemoglobin increase ≥1.5 g/dL for two consecutive assessments without intervening RBC transfusion (laboratory measurement and transfusion records)."}
• {"endpoint_text":"- Number of RBC transfusions required.","definition_or_measurement_approach":"Count of RBC transfusions recorded during study period."}
• {"endpoint_text":"- Achievement of transfusion independence.","definition_or_measurement_approach":"Assessment of transfusion independence per protocol-defined criteria (based on transfusion records)."}
• {"endpoint_text":"- Change in RBC counts over time.","definition_or_measurement_approach":"Serial RBC count measurements over study visits."}
• {"endpoint_text":"- Concentrations of REGN7999 in serum over time.","definition_or_measurement_approach":"Pharmacokinetic serum concentrations of REGN7999 measured at scheduled timepoints."}
• {"endpoint_text":"- Incidence of anti-drug antibody (ADA) to REGN7999 over time.","definition_or_measurement_approach":"Incidence of ADA measured by immunogenicity assays at scheduled timepoints."}
• {"endpoint_text":"- Magnitude of ADA to REGN7999 over time.","definition_or_measurement_approach":"Titer/level of ADA measured over time using immunogenicity assays."}

Methods

• Study website / Website transcript (document titles: K2_R7999-BTHAL-2350_Recruit Material_Basic Website; Recruitment material-Website Transcript; Recruitment material-Website Privacy Policy; Recruitment material-Website Terms of Use).
• Banner advertisements (document titles: K2_R7999-BTHAL-2350_Recruit Material_Banner Pt Layout; Recruitment material-Banner Ads-Patient).
• Study brochure / patient brochure (document titles: K2_R7999-BTHAL-2350_Recruit Material_Study Brochure; Recruitment material-Study Brochure_FP).
• Recruitment arrangements and ICF process (document titles: K1_R7999-BTHAL-2350_Recruit-ICF proces_FP / K1_R7999-BTHAL-2350_Recruit-ICF process_FP).

Greece

Earliest CTIS Part Ii Submission Date

18-12-2024

Latest Decision Or Authorization Date

21-10-2025

Processing Time Days

307

Number Of Sites

3

Number Of Participants

5

Italy

Earliest CTIS Part Ii Submission Date

17-03-2025

Latest Decision Or Authorization Date

17-12-2025

Processing Time Days

275

Number Of Sites

4

Number Of Participants

5

Primary sponsor

Full Name

Regeneron Pharmaceuticals Inc.

Organisation Type

Pharmaceutical company

Country Of Registered Address

United States

Contract research organisations

Name

Icon Clinical Research Limited

Responsibilities

CRO (and separate Central Lab role listed)

Name

Parexel International (IRL) Limited

Responsibilities

CRO

Name

PRA Hellas CRO A.E.

Responsibilities

CRO-related duties (codes provided: 1,5)

Third parties

• {"country":"United States","full_name":"Iqvia Biotech LLC","duties_or_roles":"code:6","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Perceptive Informatics Inc.","duties_or_roles":"Imaging Service Provider","organisation_type":"Pharmaceutical company"}
• {"country":"Ireland","full_name":"Icon Clinical Research Limited","duties_or_roles":"CRO","organisation_type":"Pharmaceutical company"}
• {"country":"Ireland","full_name":"Icon Clinical Research Limited","duties_or_roles":"Central Lab","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Fisher Clinical Services Inc.","duties_or_roles":"Clinical Supply","organisation_type":"Pharmaceutical company"}
• {"country":"Ireland","full_name":"Parexel International (IRL) Limited","duties_or_roles":"CRO","organisation_type":"Pharmaceutical company"}
• {"country":"Greece","full_name":"PRA Hellas CRO A.E.","duties_or_roles":"codes:1,5","organisation_type":"Pharmaceutical company"}