A Phase 1 Study to Investigate the Safety and Pharmacokinetics of ABBV-CLS-7262 in Patients With Amyotrophic Lateral Sclerosis

Amyotrophic Lateral Sclerosis Clinical Trial

Official title:

A Randomized, Double-Blind, Placebo-Controlled Study to Assess Safety, Tolerability, and Pharmacokinetics Following Multiple Doses of ABBV-CLS-7262 in Subjects With Amyotrophic Lateral Sclerosis Followed by an Active Treatment Extension

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT04948645
• Other study ID #: M20-405
• Status: Active, not recruiting
• Phase: Phase 1
• Start date: September 22, 2021
• Est. completion date: December 2025

Study information

• Verified date: May 2024
• Source: Calico Life Sciences LLC
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

ABBV-CLS-7262 is an investigational drug being researched for the treatment of Amyotrophic Lateral Sclerosis. This is an up to 156-week, 2-part study. Part 1 will be a 4-week, randomized, double-blind, placebo-controlled study; Part 2 will be up to a 152-week active treatment extension (ATE) during which all subjects will receive ABBV-CLS-7262.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 31
• Est. completion date: December 2025
• Est. primary completion date: January 12, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 80 Years

Eligibility

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

• If taking concomitant standard-of-care medications approved for the treatment of ALS (or their components), subjects must be on a stable dose of the medication(s) for >30 days prior to Baseline in order to enter the study. For edaravone, a stable dose is defined by having completed 2 treatment cycles prior to Baseline.

Exclusion Criteria:

• History of dementia/severe cognitive problems at screening

• 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.

• Documented active or suspected malignancy or history of any malignancy within the last 5 years except for successfully treated non-melanoma skin cancer or localized carcinoma in situ of the cervix

• 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 for treatment of ALS

• Current or anticipated use of diaphragmatic pacing during the study period

• Tracheostomy or use of non-invasive ventilatory support =22 hours a day

Related Conditions & MeSH terms

• ALS
• Amyotrophic Lateral Sclerosis
• Motor Neuron Disease
• Sclerosis

Intervention

Drug:
• ABBV-CLS-7262
Oral
• Placebo
Oral

Locations

Country	Name	City	State
Canada	University of Calgary - Heritage Medical Research Clinic	Calgary	Alberta
Canada	University of Alberta	Edmonton	Alberta
Canada	Stan Cassidy Centre for Rehabilitation	Fredericton	New Brunswick
Canada	London Health Sciences Centre	London	Ontario
Canada	Centre Hospitalier de l'Universite de Montreal (CHUM)	Montreal	Quebec
Canada	Montreal Neurological Institute and Hospital	Montreal	Quebec
Canada	Sunnybrook Research Institute	Toronto	Ontario
United States	Johns Hopkins ALS Clinical Trials Unit	Baltimore	Maryland
United States	Healey & AMG Center for ALS Research	Boston	Massachusetts
United States	UC Irvine Health ALS and Neuromuscular Center	Irvine	California
United States	Mayo Clinic	Jacksonville	Florida
United States	Cedars-Sinai Medical Center	Los Angeles	California
United States	Mayo Clinic	Rochester	Minnesota
United States	Forbes Norris MDA/ALS Research and Treatment Center	San Francisco	California

Sponsors (2)

Lead Sponsor	Collaborator
Calico Life Sciences LLC	AbbVie

Countries where clinical trial is conducted

United States,  Canada, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Safety and Tolerability	Number of patients with treatment-related adverse events as assessed by CTCAE v4.03	Baseline Up to Approximately Day 28
Primary	Pharmacokinetics	Maximum Plasma Concentration [Cmax]	Baseline Up to Approximately Day 28
Primary	Pharmacokinetics	Area Under the Curve [AUC]	Baseline Up to Approximately Day 28
Secondary	CSF Pharmacokinetics	Concentration at steady state in CSF	Baseline Up to Approximately Day 28
Secondary	Safety and Tolerability	Number of patients with treatment-related adverse events as assessed by CTCAE v4.03	Baseline Up to Approximately Week 156