MIBAVADEMAB for Monogenic obesity|Congenital leptin deficiency

Inclusion criteria

• {"criterion_text":"- Has documented medical history of biallelic loss of function variants of the LEP gene prior to the screening visit."}
• {"criterion_text":"- Has class ≥2 obesity at screening and at baseline, as defined in the protocol."}
• {"criterion_text":"- NOTE: Other Protocol-defined inclusion criteria apply."}

Exclusion criteria

• {"criterion_text":"- Unwilling or unable to provide, or have the treating physician provide, documented historical weight for at least 6 months prior to screening."}
• {"criterion_text":"- History of bariatric surgery within approximately the past 12 months of study screening."}
• {"criterion_text":"- History of weight loss of ≥5% of body weight in approximately the past 3 months of study screening."}
• {"criterion_text":"- History of genetic causes of obesity other than/in addition to biallelic loss of function variants of the LEP gene."}
• {"criterion_text":"- History of schizophrenia, bipolar disorder, or other mental illness that, in the opinion of the investigator, might pose a safety risk to participation in a clinical study of an investigational drug."}
• {"criterion_text":"- Any suicidal ideation of type 4 or 5 on the Columbia Suicide Severity Rating Scale (C-SSRS) during screening or baseline, or any lifetime history of a suicide attempt, or any lifetime history of suicidal behavior, including ideation."}
• {"criterion_text":"- Treatment with medications for weight loss or medications with known side effects of weight loss within the past approximately 3 months of study screening."}
• {"criterion_text":"- Participants currently being treated with metreleptin, or a history of prior treatment with metreleptin as defined in the protocol."}
• {"criterion_text":"- NOTE: Other Protocol-defined exclusion criteria apply."}

Primary endpoints

• {"endpoint_text":"- Percent change in Body Mass Index (BMI) from baseline to week 24.","definition_or_measurement_approach":"Percent change in BMI measured from baseline to week 24."}

Secondary endpoints

• {"endpoint_text":"- Percent change in BMI from baseline to week 52.","definition_or_measurement_approach":"Percent change in BMI measured from baseline to week 52."}
• {"endpoint_text":"- Absolute change in BMI from baseline, through week 52.","definition_or_measurement_approach":"Absolute change in BMI measured from baseline through week 52."}
• {"endpoint_text":"- Percent change in body weight from baseline, through week 52.","definition_or_measurement_approach":"Percent change in body weight measured from baseline through week 52."}
• {"endpoint_text":"- Absolute change in body weight from baseline, through week 52.","definition_or_measurement_approach":"Absolute change in body weight measured from baseline through week 52."}
• {"endpoint_text":"- Change in waist circumference from baseline, through week 52 in participants aged ≥18 years.","definition_or_measurement_approach":"Change in waist circumference measured from baseline through week 52 (adults ≥18 years)."}
• {"endpoint_text":"- Change in absolute total fat mass by whole body DXA from baseline, through week 52.","definition_or_measurement_approach":"Change in absolute total fat mass measured by whole body DXA from baseline through week 52."}
• {"endpoint_text":"- Change in percent total fat mass by whole body DXA from baseline, through week 52.","definition_or_measurement_approach":"Change in percent total fat mass measured by whole body DXA from baseline through week 52."}
• {"endpoint_text":"- Change in absolute regional fat mass by whole body DXA from baseline, through week 52.","definition_or_measurement_approach":"Change in absolute regional fat mass measured by whole body DXA from baseline through week 52."}
• {"endpoint_text":"- Change in percent regional fat mass by whole body DXA from baseline, through week 52.","definition_or_measurement_approach":"Change in percent regional fat mass measured by whole body DXA from baseline through week 52."}
• {"endpoint_text":"- Change in scores in the symptoms of hyperphagia questionnaire from baseline, through week 52.","definition_or_measurement_approach":"Change in questionnaire scores (symptoms of hyperphagia) from baseline through week 52."}
• {"endpoint_text":"- Change in scores in the impacts of hyperphagia questionnaire from baseline, through week 52.","definition_or_measurement_approach":"Change in questionnaire scores (impacts of hyperphagia) from baseline through week 52."}
• {"endpoint_text":"- Change in scores in the hunger questionnaire from baseline, through week 52.","definition_or_measurement_approach":"Change in questionnaire scores (hunger questionnaire) from baseline through week 52."}
• {"endpoint_text":"- Number of treatment-emergent adverse events (TEAEs) through week 65.","definition_or_measurement_approach":"Count of TEAEs collected through week 65."}
• {"endpoint_text":"- Severity of TEAEs through week 65.","definition_or_measurement_approach":"Assessment of severity of TEAEs collected through week 65."}
• {"endpoint_text":"- Concentrations of total mibavademab in serum through week 65.","definition_or_measurement_approach":"Serum concentration measurements of total mibavademab through week 65 (PK sampling)."}
• {"endpoint_text":"- Incidence of anti-drug antibody (ADA) to mibavademab through week 65.","definition_or_measurement_approach":"Incidence of ADA to mibavademab measured through week 65."}
• {"endpoint_text":"- Titer of ADA to mibavademab through week 65.","definition_or_measurement_approach":"ADA titer measurements to mibavademab through week 65."}

Germany

Earliest CTIS Part Ii Submission Date

22-10-2025

Latest Decision Or Authorization Date

06-11-2025

Processing Time Days

15

Number Of Sites

1

Number Of Participants

2

Primary sponsor

Full Name

Regeneron Pharmaceuticals Inc.

Organisation Type

Pharmaceutical company

Country Of Registered Address

United States

Contract research organisations

Name

Syneos Health Inc.

Responsibilities

CRO and Trial Management

Third parties

• {"country":"United States","full_name":"Clario Medical Imaging Inc.","duties_or_roles":"Imaging vendor","organisation_type":"Pharmaceutical company"}
• {"country":"Slovakia","full_name":"SanaClis s.r.o.","duties_or_roles":"Drug logistics","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Syneos Health Inc.","duties_or_roles":"CRO and Trial Management","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Perceptive Informatics Inc.","duties_or_roles":"IVRS (Interactive Voice Response System)","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Iqvia Inc.","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Labcorp Central Laboratory Services LP","duties_or_roles":"Central Lab Testing and Sample Logistics","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Datacubed Health Inc.","duties_or_roles":"eCOA","organisation_type":"Pharmaceutical company"}