An Open-Label Exploratory Study of Fosigotifator in Participants With Vanishing White Matter Disease

Conditions

• Vanishing White Matter Disease

Eligibility Criteria

Sex

ALL

Age

6 Months - N/A

Healthy Volunteers

Not accepted

Interventions

• Fosigotifator — DRUG
  Oral Use

Study Details

Fosigotifator is an investigational drug being researched for the treatment of Vanishing White Matter disease in adult, pediatric and infant participants. This is a 201-week, open-label, multiple cohort study enrolling adults, pediatric and infant participants with Vanishing White Matter disease. Participants will attend regular visits during the course of the study and complete medical assessments, blood tests, questionnaires, and be evaluated for side effects.

Key Dates

Start date

Mar 13, 2023

Status verified

May 2026

Primary completion

Nov 30, 2027

Completion

Nov 30, 2029

Study Design

Enrollment

50 participants (estimated)

Allocation

NON_RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Arms

• Experimental: Fosigotifator - Cohort 1
  Cohort 1: VWM adults \>= 18 years.
• Experimental: Fosigotifator - Cohort 1b
  Cohort 1b: VWM adults \>= 18 years.
• Experimental: Fosigotifator - Cohort 2
  Cohort 2: VWM children\>= 12 y and \<18 years.
• Experimental: Fosigotifator - Cohort 3
  Cohort 3: VWM children \>= 6 y and \<12 years.
• Experimental: Fosigotifator - Cohort 4
  Cohort 4: VWM children \>= 6 months and \<6 years.

Primary Outcome Measure

Incidence of Treatment-Emergent Adverse Events [ Time Frame: Baseline up to Approximately Day 28 ]

Central Contacts

• Call Center - English
  1-833-250-9660
  [email protected]

Locations (3)

Find similar trials in Boston, MA

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