MRM-3379 for Fragile X syndrome

Inclusion criteria

• {"criterion_text":"- Willing and able to provide signed informed consent/assent. Where local regulations permit inclusion of participants deemed unable to provide informed consent, a legally authorized representative must provide informed consent on the participant’s behalf, and the participant must provide assent if applicable.\n- Male, 13–45 years of age (inclusive).\n- Weight ≥30 kg and BMI ≥18 and <36 kg/m2 (inclusive)\n- Diagnosis of FXS with a molecular genetic ≥200 CGG repetitions.\n- Have a consistent caregiver(s) who is willing and able to be present regularly and reliably with the participant.\n- Able to swallow tablets or capsules"}

Exclusion criteria

• {"criterion_text":"- History of or current medical condition other than FXS and related issues that would place the participant at higher risk from study participation."}

Primary endpoints

• {"endpoint_text":"- To evaluate the safety and tolerability of MRM-3379 in male participants with FXS on the bases of the following endpoints: -Incidence and severity of TEAEs; - Withdrawal due to AEs.","definition_or_measurement_approach":"Incidence and severity of TEAEs; Withdrawal due to AEs."}

Secondary endpoints

• {"endpoint_text":"- To evaluate the preliminary efficacy of MRM-3379 in male participants with FXS on the basis of the following endpoints: - Key secondary endpoint: Change from baseline on the NIH-TCB Crystallized Cognition Composite (CCC): Composed from the Picture Vocabulary Test (PVT) and Oral Reading Recognition Test (ORRT) change sensitive scores; - Other secondary endpoint: Clinician Global Impression-Change (CGI-C) score","definition_or_measurement_approach":"Change from baseline on the NIH-TCB Crystallized Cognition Composite (CCC) composed from PVT and ORRT change-sensitive scores; Clinician Global Impression-Change (CGI-C) score."}
• {"endpoint_text":"- To describe the PK properties of MRM-3379 on the basis of the following endpoint: Steady-state MRM-3379 and metabolite plasma concentrations","definition_or_measurement_approach":"Steady-state plasma concentrations of MRM-3379 and metabolites."}

Methods

• Site-based recruitment at listed hospitals in Spain (Hospital Sant Joan De Deu Barcelona; Parc Tauli Hospital Universitari; Hospital Universitario Regional De Malaga).
• Recruitment materials and patient referral provided by Subject Well Inc. ("Recruitment materials and patient referral").
• Travel arrangements and travel reimbursement support provided by Meeting Protocol Worldwide LP (duty described as "Travel arrangements and travel reimbursement").
• Scheduling and conducting Spanish-speaking interviews for exit interviews by QualWorld (duty described as "QualWorld will be scheduling and conducting Spanish-speaking interviews").
• Exit interviews with caregivers conducted/analysed by Lumanity (duty described as "Lumanity will be conducting/analysing exit interviews with caregivers of ENV-202 trial participants").

Spain

Earliest CTIS Part Ii Submission Date

16-03-2026

Latest Decision Or Authorization Date

27-04-2026

Processing Time Days

42

Number Of Sites

3

Number Of Participants

15

Primary sponsor

Full Name

Mirum Pharmaceuticals Inc.

Organisation Type

Pharmaceutical company

Country Of Registered Address

United States

Contract research organisations

Name

Parexel International (IRL) Limited

Responsibilities

Multiple study management responsibilities (sponsorDuties codes: 1,10,11,12,2,5,6)

Name

Icon Clinical Research Limited

Responsibilities

Clinical research services (sponsorDuties code: 4)

Third parties

• {"country":"United States","full_name":"Medidata Solutions Inc.","duties_or_roles":"Electronic Clinical Outcome Assessments","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United States","full_name":"Cogstate Inc.","duties_or_roles":"Rater training and administration of Vineland-3","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Icon Laboratory Services Inc.","duties_or_roles":"Laboratory services (sponsorDuties code: 4)","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"United States","full_name":"Primevigilance USA Inc.","duties_or_roles":"Pharmacovigilance / Safety reporting (sponsorDuties code: 8)","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Lumanity Patient Centered Outcomes LLC","duties_or_roles":"Conducting/analysing exit interviews with caregivers of ENV-202 trial participants","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"United States","full_name":"Meeting Protocol Worldwide LP","duties_or_roles":"Travel arrangements and travel reimbursement","organisation_type":"Pharmaceutical company"}
• {"country":"France","full_name":"Qualworld","duties_or_roles":"Scheduling and conducting Spanish-speaking interviews","organisation_type":"Pharmaceutical company"}
• {"country":"Ireland","full_name":"Icon Clinical Research Limited","duties_or_roles":"Clinical research services (sponsorDuties code: 4)","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Subject Well Inc.","duties_or_roles":"Recruitment materials and patient referral","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"Germany","full_name":"Centogene GmbH","duties_or_roles":"Laboratory services (sponsorDuties code: 4)","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"Ireland","full_name":"Parexel International (IRL) Limited","duties_or_roles":"Multiple study management responsibilities (sponsorDuties codes: 1,10,11,12,2,5,6)","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Edetek Inc.","duties_or_roles":"Data management / other (sponsorDuties code: 10)","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Suvoda LLC","duties_or_roles":"Quality assurance (sponsorDuties code: 3)","organisation_type":"Non-Pharmaceutical company"}
• {"country":"France","full_name":"Biotrial","duties_or_roles":"EEG and ECG Central Reader","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Alturas Analytics Inc.","duties_or_roles":"Bioanalytics","organisation_type":"Pharmaceutical company"}