CTIS2023-505704-30-00
Recruiting
Phase 1
A Phase 1b/2 Open-label Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Exploratory Efficacy Following ABBV-CLS-7262 Administration in Adult and Pediatric Subjects with Vanishing White Matter Disease - M23-523
Overview
- Phase
- Phase 1
- Intervention
- Not specified
- Conditions
- Not specified
- Sponsor
- Calico Life Sciences LLC
- Enrollment
- 14
- Status
- Recruiting
- Last Updated
- last year
Overview
Brief Summary
No summary available.
Investigators
Eligibility Criteria
Inclusion Criteria
- •Males and females \=18 y of age (adult Cohort 1\), \=12 y and \<18 y of age (adolescent Cohort 2\), and \=6 y and \<12 y of age (children Cohort 3\) at the time of Screening., Have VWM disease defined as: a.A clinical diagnosis by a physician experienced in the assessment of VWM disease; AND b.A molecular diagnosis of VWM disease confirmed through biallelic mutation(s) in eIF2B (Note: Subjects can provide source documentation if obtained in the past. If not, molecular diagnosis is required to be confirmed before Screening), AND c.An MRI presentation consistent with VWM disease, as assessed by a neuroradiologist or by a physician experienced in the assessment of VWM disease, Have a designated caregiver who is able to complete the respective caregiver\-centered assessments. The caregiver must be consistently present at visits, including telehealth visits, to report on symptoms and comply with the protocol. The caregiver must be willing to provide informed consent., Subject is willing and able to give informed consent. Where local regulations permit inclusion of participants deemed not able to provide informed consent, a legally authorized representative (LAR) must provide informed consent on the subject’s behalf, and the subject must provide assent, in accordance with the local regulations, guidelines, and Institutional Review Board (IRB), or Independent Ethics Committee (IEC). If the subject becomes cognitively impaired (diminished capacities) during the study and is unable to provide informed consent, the subject should be discontinued from the study unless local regulations permit inclusion of participants deemed not able to provide informed consent. In such case, a LAR must provide informed consent on behalf of the subject and the subject must provide assent as per local regulations, guidelines, and IRB or IEC. Careful consideration will be given to ensure that cognitive impairment/diminished capacity does not limit the subject’s right to withdraw from the study. The LAR and the caregiver can be the same person., Subjects must meet at least 1 of the criteria listed below (a or b): a.Motor criteria as defined below: i.All age groups: (1\)Inability to walk 10 or more steps with or without light support of 2 hands b.Cognitive criteria as defined below (Note: Subjects are not required to complete testing if they meet the motor criteria or if they are able to provide source documentation of prior cognitive testing meeting eligibility criteria obtained in the past 12 months): i.Adults and adolescents \=16 y of age must have: (1\)A perceptual reasoning index \=50 (Wechsler Adult Intelligence Scale \[WAIS IV]) ii.Adolescents and children \=6 y and \<16 y of age must have: (1\)A visuospatial reasoning index \=50 (block design, visual puzzles, Wechsler Intelligence Scale for Children performance \[WISC V]) AND/OR (2\)A fluid reasoning index \=50 (matrix reasoning, figure weights, WISC V), 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
- •Subject who has any clinically significant ECG abnormalities, including QT interval corrected for heart rate using Fridericia’s correction formula (QTcF) of \>450 msec for adult males or \>470 msec for adult females, or \>450 msec for adolescents \=12 y and \<18 y of age (Cohort 2\) and children \=6 y and \<12 y of age (Cohort 3\), Use of any sensitive substrates with narrow therapeutic index as classified by the risk level ‘X’ in the Lexicomp® drug interaction database within 10 days or 5 half\-lives (whichever is longer) prior to Study Day 1\., Current or history of abnormal screening laboratory or imaging results that, in the opinion of the Investigator, are indicative of any significant cardiac, endocrinologic, hematologic, hepatic, immunologic, infectious, metabolic, urologic, pulmonary, gastrointestinal, dermatologic, psychiatric, renal, neurologic, and/or other major disease (other than VWM disease) that would preclude administration of ABBV\-CLS\-7262, Subject who has suicidal ideation at the Screening Visit (V1\). Prior medical history and/or C SSRS may be used to inform the Investigator’s decision, 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, Subjects who, in the opinion of the Investigator, is incapable of completing study\-required visits and procedures to assess primary and secondary endpoints (eg, due to severe comorbid conditions or severity of VWM disease), Pregnant or breastfeeding, Treatment with any other investigational treatment within 30 days or 5 half\-lives (whichever is longer) prior to Study Day 1 and during the study. Current participation in another trial is not permitted unless it is a non\-interventional study and the sole purpose of this study is for long term follow\-up describing clinical features or survival data (registry). Non interventional studies that include biomarker assessments (including, but not limited to, sampling of blood, urine, CSF, or neuroimaging) are not allowed., Based on the metabolism of the compound, the concomitant use of certain inhibitors and inducers of cytochrome P450 enzymes.
Outcomes
Primary Outcomes
Not specified
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