A Phase 1 Study to Investigate the Safety and Pharmacokinetics of ABBV-CLS-7262 in Patients With Amyotrophic Lateral Sclerosis
Study Details
Study Description
Brief Summary
ABBV-CLS-7262 is an investigational drug being researched for the treatment of Amyotrophic Lateral Sclerosis. This is a 48-week, 2-part study. Part 1 will be a 4-week, randomized, double-blind, placebo-controlled study; Part 2 will be a 44-week active treatment extension (ATE) during which all subjects will receive ABBV-CLS-7262.
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 |
|---|---|---|
|
Phase 1 |
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Active Comparator: ABBV-CLS-7262 LOW DOSE
|
Drug: ABBV-CLS-7262
Oral
|
| Active Comparator: ABBV-CLS-7262 MEDIUM DOSE
|
Drug: ABBV-CLS-7262
Oral
|
| Active Comparator: ABBV-CLS-7262 HIGH DOSE
|
Drug: ABBV-CLS-7262
Oral
|
| Placebo Comparator: PLACEBO
|
Drug: Placebo
Oral
|
Outcome Measures
Primary Outcome Measures
- Safety and Tolerability [Baseline Up to Approximately Day 28]
Number of patients with treatment-related adverse events as assessed by CTCAE v4.03
- Pharmacokinetics [Baseline Up to Approximately Day 28]
Maximum Plasma Concentration [Cmax]
- Pharmacokinetics [Baseline Up to Approximately Day 28]
Area Under the Curve [AUC]
Secondary Outcome Measures
- CSF Pharmacokinetics [Baseline Up to Approximately Day 28]
Concentration at steady state in CSF
- Safety and Tolerability [Baseline Up to Approximately Week 48]
Number of patients with treatment-related adverse events as assessed by CTCAE v4.03
Eligibility Criteria
Criteria
Inclusion Criteria:
-
Must have an identified, reliable caregiver
-
Confirmed diagnosis of Familial ALS or Sporadic ALS
-
First ALS symptoms occurred ≤36 months before screening
-
Able to swallow solids
-
No known active COVID-19 infection at screening
-
Vital capacity ≥50% predicted value (for sex, age, ethnic origin, and height) at screening
-
Subjects must be a) naïve to, or b) off riluzole (Rilutek), or c) on a stable dose
30 days prior to Baseline visit to enter study
- Subjects must be a) naïve to, or b) off edaravone (Radicava), or c) have completed 2 treatment cycles prior to the Baseline visit.
Exclusion Criteria:
-
History of dementia/severe cognitive problems at screening
-
Use of riluzole (Rilutek®) if dose has NOT been stable for > 30 days prior to Baseline visit
-
History of clinically significant medical conditions (other than ALS) or any other reason, including any physical, psychological, or psychiatric condition that, in the opinion of the Investigator, would compromise the safety or interfere with the subject's participation in the study, or would make the subject an unsuitable candidate to receive study drug, or would put the subject at risk by participating in the study.
-
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 that would preclude administration of ABBV-CLS-7262.
-
If female, is known to be pregnant, breastfeeding, considering becoming pregnant, or donating/banking eggs during the study or within 30 days or >5 half-lives (whichever is longer) after the last dose of study drug
-
If male, plans to donate sperm or father a child during the study or within 30 days after the last dose of study drug.
-
Known to have received any investigational product within 30 days or 5 half-lives of the drug (whichever is longer) prior to the first dose of study drug or is currently enrolled in another clinical study.
-
History of ABBV-CLS-7262 use prior to participation in this study
-
Recent (within 6 months prior to Screening) history of drug or alcohol abuse
-
Previous participation in a stem cell clinical study
-
Current or anticipated use of diaphragmatic pacing during the study period
-
Tracheostomy or use of non-invasive ventilatory support ≥22 hours a day
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | UC Irvine Health ALS and Neuromuscular Center | Irvine | California | United States | 92868 |
| 2 | Forbes Norris MDA/ALS Research and Treatment Center | San Francisco | California | United States | 94109 |
| 3 | Mayo Clinic | Jacksonville | Florida | United States | 32224 |
| 4 | Healey & AMG Center for ALS Research | Boston | Massachusetts | United States | 02114 |
| 5 | Mayo Clinic | Rochester | Minnesota | United States | 55905 |
| 6 | University of Calgary - Heritage Medical Research Clinic | Calgary | Alberta | Canada | T2V1P9 |
| 7 | University of Alberta | Edmonton | Alberta | Canada | T6G 2G3 |
| 8 | Stan Cassidy Centre for Rehabilitation | Fredericton | New Brunswick | Canada | E38 0C7 |
| 9 | London Health Sciences Centre | London | Ontario | Canada | N6A585 |
| 10 | Sunnybrook Research Institute | Toronto | Ontario | Canada | M4N 3M5 |
| 11 | Centre Hospitalier de l'Universite de Montreal (CHUM) | Montreal | Quebec | Canada | H2L 4M1 |
| 12 | Montreal Neurological Institute and Hospital | Montreal | Quebec | Canada | H3A 2B4 |
Sponsors and Collaborators
- Calico Life Sciences LLC
- AbbVie
Investigators
None specified.Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- M20-405