An Open-Label Exploratory Study of ABBV-CLS-7262 Subjects With Vanishing White Matter Disease

Vanishing White Matter Disease Clinical Trial

Official title:

A Phase 1b/2 Open-label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Exploratory Efficacy Following ABBV-CLS-7262 Administration in Adult and Pediatric Subjects With Vanishing White Matter Disease

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05757141
• Other study ID #: M23-523
• Status: Recruiting
• Phase: Phase 1/Phase 2
• Start date: March 13, 2023
• Est. completion date: October 2026

Study information

• Verified date: April 2024
• Source: Calico Life Sciences LLC
• Contact: Calico Call Center-English
• Phone: 1-833-250-9660
• Email: vwminfo[@]mylocalstudy.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

ABBV-CLS-7262 is an investigational drug being researched for the treatment of Vanishing White Matter disease in adult and pediatric subjects. This is a 96-week, open-label, multiple cohort study enrolling adults and pediatric subjects with Vanishing White Matter disease who are 6 years or older. Subjects will attend regular visits during the course of the study and complete medical assessments, blood tests, checking for side effects, and completing questionnaires.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 14
• Est. completion date: October 2026
• Est. primary completion date: October 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Years and older

Eligibility

Inclusion Criteria:

1. Males and females =6 years of age at the time of Screening.

2. Have VWM disease defined as:

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

2. A molecular diagnosis of VWM disease confirmed through mutation(s) in eIF2B, and

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

3. Have a designated caregiver who is able to complete the respective caregiver-centered assessments.

4. Signed and dated informed consent provided by the subject, or from a legally authorized representative (LAR) if subject is incapable to consent themselves.

5. Subjects must meet functional criteria related to ambulation and cognition; specific details can be provided by the Study physician.

6. 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.

7. 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:

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

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

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

4. Pregnant or breastfeeding.

5. Treatment with any other investigational treatment within 30 days or 5 half-lives (whichever is longer) prior to Baseline.

Related Conditions & MeSH terms

• Leukoencephalopathies
• Vanishing White Matter Disease

Intervention

Drug:
• ABBV-CLS-7262
Active Drug: ABBV-CLS-7262 - Oral

Locations

Country	Name	City	State
Netherlands	Amsterdam UMC - Department of Neurology	Amsterdam
United States	Massachusetts General Hospital	Boston	Massachusetts
United States	University of Utah Hospital	Salt Lake City	Utah

Sponsors (2)

Lead Sponsor	Collaborator
Calico Life Sciences LLC	AbbVie

Countries where clinical trial is conducted

United States,  Netherlands, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Primary	Plasma Concentration	Maximum Plasma Concentration [Cmax]	Baseline up to approximately Week 96
Secondary	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 100