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

ConditionVanishing White Matter Disease
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age6+
SponsorCalico Life Sciences LLC

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Males and females >= 6 months of age at the time of Screening.

A clinical diagnosis by a physician experienced in the assessment of VWM disease; and

A molecular diagnosis of VWM disease, and

A magnetic resonance imaging (MRI) presentation consistent with VWM disease.

Disqualifiers

Pediatric participants >= 6 months and < 6 years of age must not be on any form of respiratory support at the time of Screening.

Changes in medication use for the management of VWM disease symptoms within the 4 weeks preceding Screening.

Seizure disorder not considered adequately controlled by the investigator within the 6 months preceding Screening.

Participant who, in the opinion of the investigator, is incapable of completing study-required visits and procedures to assess primary and secondary endpoints.

Trial design

Design model

Parallel

Treatments tested in this trial

  • Fosigotifator

    Drug

    Oral Use

Treatment groups

50 Participants
are divided into 5 treatment groups
Group A: Fosigotifator - Cohort 1Experimental treatment 1 intervention
Group B: Fosigotifator - Cohort 1bExperimental treatment 1 intervention
Group C: Fosigotifator - Cohort 2Experimental treatment 1 intervention
Group D: Fosigotifator - Cohort 3Experimental treatment 1 intervention
Group E: Fosigotifator - Cohort 4Experimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Incidence of Treatment-Emergent Adverse Events

Number of participants with treatment-related adverse events as assessed by CTCAE v4.03

Time frame
Baseline up to Approximately Day 28
2

Number of Participants with Change in Vital Signs

Number of Participants with Change in Vital Signs will be assessed.

Time frame
Baseline up to Approximately Day 28
3

Number of Participants with Change in ECG

Number of Participants with Change in ECG will be assessed.

Time frame
Baseline up to Approximately Day 28
4

Number of Participants with Change in Clinical Laboratory Tests

Number of participants with change in clinical laboratory tests will be assessed.

Time frame
Baseline up to Approximately Day 28

Secondary outcomes

1

Incidence of Treatment-Emergent Adverse Events

Number of patients with treatment-related adverse events as assessed by CTCAE v4.03

Time frame
Baseline up to Approximately Week 197
2

Number of Participants with Change in Vital Signs

Number of Participants with Change in Vital Signs will be assessed.

Time frame
Baseline up to approximately Week 197
3

Number of Participants with Change in ECG

Number of Participants with Change in ECG will be assessed.

Time frame
Baseline up to approximately Week 197
4

Number of Participants with Change in Clinical Laboratory Tests

Number of participants with change in clinical laboratory tests will be assessed.

Time frame
Baseline up to approximately Week 197

Other outcomes

Sponsors and contacts

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

Calico Life Sciences LLC

Lead sponsor

Source ClinicalTrials.gov
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