Overview

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

Status:
Recruiting
Trial end date:
2025-03-01
Target enrollment:
0
Participant gender:
All
Summary
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.
Phase:
Phase 1
Accepts Healthy Volunteers?
No
Details
Lead Sponsor:
Calico Life Sciences LLC
Collaborator:
AbbVie
Criteria
Inclusion Criteria:

1. Males and females ≥18 y of age at the time of Screening.

2. Have VWM disease defined as:

1. A clinical diagnosis by a physician; 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. 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.

6. 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.

4. Pregnant or breastfeeding.

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