Study of MRM-3379 in Male Participants With Fragile X Syndrome

Mirum Pharmaceuticals

Status and phase: Enrolling; Phase 2

Conditions: Fragile X Syndrome

Treatments: Drug: Low dose of MRM-3379 Open-Label; Drug: Middle Dose of MRM-3379; Drug: Placebo; Drug: Low dose of MRM-3379; Drug: High dose of MRM-3379

Study type: Interventional

Funder types: Industry

Identifiers

NCT07209462

ENV-202

Details and patient eligibility

About

This study is a multicenter, double-blind, randomized, placebo-controlled study to assess the safety and tolerability of 3 doses of MRM-3379 in male participants with Fragile X Syndrome ages 16 to 45 (inclusive). In addition, a parallel cohort of participants ages 13 to <16 will receive open-label MRM-3379. All participants will participate for 12 weeks of treatment. The study is also intended as a proof-of-concept investigation to evaluate whether MRM-3379 can improve FXS symptoms

Enrollment: 60 estimated patients

Sex: Male

Ages: 13 to 45 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• 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.
• Male, 13-45 years of age (inclusive)
• Weight ≥30 kg and BMI between 18 and 36 kg/m2 (inclusive) at the screening visit
• Diagnosis of FXS with a molecular genetic ≥200 CGG repetitions .
• Able to perform the PVT and ORRT of the NIH-TCB
• Have a consistent caregiver(s) who is willing and able to be present regularly and reliably with the participant
• Able to swallow tablets or capsules

Exclusion criteria

History of or current medical condition other than FXS and related issues that would place the participant at higher risk from study participation

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Double Blind

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60 participants in 5 patient groups, including a placebo group

Arm 1: Low Dose of MRM-3379

Experimental group

Description:

Participants 16-45 years of age randomized to this arm will receive the low dose of MRM-3379

Treatment:

Drug: Low dose of MRM-3379

Arm 2: Middle dose of MRM-3379

Experimental group

Description:

Participants 16-45 years of age randomized to this arm will receive the middle dose of MRM-3379

Treatment:

Drug: Middle Dose of MRM-3379

Arm 3: High dose of MRM-3379

Experimental group

Description:

Participants 16-45 years of age randomized to this arm will receive the high dose of MRM-3379

Treatment:

Drug: High dose of MRM-3379

Arm 4 :Placebo

Placebo Comparator group

Description:

Participants 16-45 years of age randomized to this arm will receive Placebo

Treatment:

Drug: Placebo

Arm 5: Low dose of MRM-3379 Open-Label

Experimental group

Description:

Participants 13 to \< 16 years of age will receive the low dose of MRM-3370 Open-Label

Treatment:

Drug: Low dose of MRM-3379 Open-Label