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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT05757141
Other study ID # M23-523
Secondary ID
Status Recruiting
Phase Phase 1/Phase 2
First received
Last updated
Start date March 13, 2023
Est. completion date October 2026

Study information

Verified date May 2024
Source Calico Life Sciences LLC
Contact Calico Call Center-English
Phone 1-833-250-9660
Email vwminfo@mylocalstudy.com
Is FDA regulated No
Health authority
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.

Study Design


Related Conditions & MeSH terms


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
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