Health TrialsbyHealthPanel

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

ConditionFragile X Syndrome
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexMale
Age13-45
SponsorMirum Pharmaceuticals, Inc.

About this trial

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

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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 .

Disqualifiers

None

Trial design

Design model

Parallel

Treatments tested in this trial

  • Low dose of MRM-3379

    Drug

    Oral capsule

  • Middle Dose of MRM-3379

    Drug

    Oral capsule

  • High dose of MRM-3379

    Drug

    Oral capsule

  • Placebo

    Drug

    Capsules matched to study drug without the active pharmaceutical ingredient

  • Low dose of MRM-3379 Open-Label

    Drug

    Oral capsule

Treatment groups

60 Participants
are divided into 5 treatment groups
Group A: Arm 1: Low Dose of MRM-3379Experimental treatment 1 intervention
Group B: Arm 2: Middle dose of MRM-3379Experimental treatment 1 intervention
Group C: Arm 3: High dose of MRM-3379Experimental treatment 1 intervention
Group D: Arm 4 :PlaceboPlacebo comparator 1 intervention
Group E: Arm 5: Low dose of MRM-3379 Open-LabelExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Incidence and severity of treatment-emergent adverse events and Withdrawal due to adverse events and withdrawal due to adverse events

Time frame
Baseline to Week 12

Secondary outcomes

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Mirum Pharmaceuticals, Inc.

Lead sponsor

Source ClinicalTrials.gov
Apply now