Phase 1b Study to Investigate ABBV-CLS-7262's Safety, Tolerability & Pharmacokinetics in Vanishing White Matter Patients

Study Overview
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Brief Summary
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ABBV-CLS-7262 is an investigational drug being researched for the treatment of Vanishing White Matter disease. This is a 96-week, open-label, single arm study. Subjects will attend regular visits during the course of the study and complete medical assessments, blood tests, checking for side effects, and completing questionnaires.

Official Title
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A Phase 1b Open-label Study to Evaluate the Safety, Tolerability, and Pharmacokinetics Following ABBV-CLS-7262 Administration in Adult Subjects With Vanishing White Matter Disease

Conditions
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Vanishing White Matter Disease

Intervention / Treatment
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* Drug: ABBV-CLS-7262


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Males and females ≥18 y of age at the time of Screening. Have VWM disease defined as: A clinical diagnosis by a physician; and A molecular diagnosis of VWM disease confirmed through mutation(s) in eIF2B, and A magnetic resonance imaging (MRI) presentation consistent with VWM disease. Have a designated caregiver who is able to complete the respective caregiver-centered assessments. Signed and dated informed consent provided by the subject, or from a legally authorized representative (LAR) if subject is incapable to consent themselves. All male subjects who are sexually active and not surgically sterilized must agree to use an acceptable contraceptive method. Additionally, male subjects must agree to not donate sperm during the study until 30 days after the final dose of study drug. All female subjects who are sexually active and of childbearing potential must agree to use a highly effective contraceptive method. Additionally, female subjects must agree to not donate eggs during the study and for 30 days after the final dose of study drug. Exclusion Criteria: 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. Subject who, in the opinion of the investigator, is incapable of completing study-required visits and procedures. Pregnant or breastfeeding. Treatment with any other investigational treatment within 30 days or 5 half-lives (whichever is longer) prior to Baseline.

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: ABBV-CLS-7262<br> | Drug: ABBV-CLS-7262<br>* ABBV-CLS-7262 is an eIF2B activator<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability] | Number of patients with treatment-related adverse events as assessed by CTCAE v4.03 | Baseline up to Approximately Day 49 | 
| Plasma Concentration | Maximum Plasma Concentration [Cmax] | Baseline up to approximately Day 28 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability] | Number of patients with treatment-related adverse events as assessed by CTCAE v4.03 | Baseline up to Approximately Week 96 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Neurodegenerative Diseases, Nervous System Diseases, Central Nervous System Brain Diseases, Hereditary Central Nervous System, Leukoencephalopathy with Vanishing White Matter