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

Sponsor

Calico Life Sciences LLC (Industry)

Overall Status

Recruiting

CT.gov ID

NCT05757141

Collaborator

AbbVie (Industry)

Enrollment

Location

Arm

Anticipated Duration (Months)

0.3

Patients Per Site Per Month

Study Details

Study Description

Brief 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.

Condition or Disease	Intervention/Treatment	Phase
Vanishing White Matter Disease	Drug: ABBV-CLS-7262	Phase 1

Study Design

Study Type:

Interventional

Anticipated Enrollment :

8 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

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

Anticipated Study Start Date :

Mar 1, 2023

Anticipated Primary Completion Date :

Mar 1, 2025

Anticipated Study Completion Date :

Mar 1, 2025

Arms and Interventions

Arm	Intervention/Treatment
Experimental: ABBV-CLS-7262	Drug: ABBV-CLS-7262 ABBV-CLS-7262 is an eIF2B activator

Arm

Intervention/Treatment

Experimental: ABBV-CLS-7262

Drug: ABBV-CLS-7262

ABBV-CLS-7262 is an eIF2B activator

Outcome Measures

Primary Outcome Measures

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

Secondary Outcome Measures

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

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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.