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

• ClinicalTrials.gov Identifier: NCT04948645
• Recruitment Status: Active, not recruiting
• First Posted: July 2, 2021
• Last Update Posted: January 25, 2023
• Sponsor: Calico Life Sciences LLC
• Collaborator: AbbVie
• Information provided by (Responsible Party): Calico Life Sciences LLC

Study Description

Brief 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.

Condition or disease	Intervention/treatment	Phase
ALS; Amyotrophic Lateral Sclerosis	Drug: ABBV-CLS-7262; Drug: Placebo	Phase 1

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 31 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Triple (Participant, Care Provider, Investigator)
• Primary Purpose: Treatment
• 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
• Actual Study Start Date: September 22, 2021
• Actual Primary Completion Date: January 12, 2023
• Estimated Study Completion Date: December 2025

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 :

1. Safety and Tolerability [ Time Frame: Baseline Up to Approximately Day 28 ]
   Number of patients with treatment-related adverse events as assessed by CTCAE v4.03
2. Pharmacokinetics [ Time Frame: Baseline Up to Approximately Day 28 ]
   Maximum Plasma Concentration [Cmax]
3. Pharmacokinetics [ Time Frame: Baseline Up to Approximately Day 28 ]
   Area Under the Curve [AUC]

Secondary Outcome Measures :

1. CSF Pharmacokinetics [ Time Frame: Baseline Up to Approximately Day 28 ]
   Concentration at steady state in CSF
2. Safety and Tolerability [ Time Frame: Baseline Up to Approximately Week 156 ]
   Number of patients with treatment-related adverse events as assessed by CTCAE v4.03

Eligibility Criteria

• Ages Eligible for Study: 18 Years to 80 Years (Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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
• 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

Contacts and Locations

Locations

United States, California

UC Irvine Health ALS and Neuromuscular Center

Irvine, California, United States, 92868

Cedars-Sinai Medical Center

Los Angeles, California, United States, 90048

Forbes Norris MDA/ALS Research and Treatment Center

San Francisco, California, United States, 94109

United States, Florida

Mayo Clinic

Jacksonville, Florida, United States, 32224

United States, Maryland

Johns Hopkins ALS Clinical Trials Unit

Baltimore, Maryland, United States, 21205

United States, Massachusetts

Healey & AMG Center for ALS Research

Boston, Massachusetts, United States, 02114

United States, Minnesota

Mayo Clinic

Rochester, Minnesota, United States, 55905

Canada, Alberta

University of Calgary - Heritage Medical Research Clinic

Calgary, Alberta, Canada, T2V1P9

University of Alberta

Edmonton, Alberta, Canada, T6G 2G3

Canada, New Brunswick

Stan Cassidy Centre for Rehabilitation

Fredericton, New Brunswick, Canada, E38 0C7

Canada, Ontario

London Health Sciences Centre

London, Ontario, Canada, N6A585

Sunnybrook Research Institute

Toronto, Ontario, Canada, M4N 3M5

Canada, Quebec

Centre Hospitalier de l'Universite de Montreal (CHUM)

Montreal, Quebec, Canada, H2L 4M1

Montreal Neurological Institute and Hospital

Montreal, Quebec, Canada, H3A 2B4

Sponsors and Collaborators

Calico Life Sciences LLC

AbbVie

More Information

• Responsible Party: Calico Life Sciences LLC
• ClinicalTrials.gov Identifier: NCT04948645
• Other Study ID Numbers: M20-405
• First Posted: July 2, 2021
• Last Update Posted: January 25, 2023
• Last Verified: January 2023

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No
• Product Manufactured in and Exported from the U.S.: Yes

Additional relevant MeSH terms:

Motor Neuron Disease; Amyotrophic Lateral Sclerosis; Sclerosis; Pathologic Processes; Neurodegenerative Diseases; Nervous System Diseases; Neuromuscular Diseases; Spinal Cord Diseases; Central Nervous System Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Metabolic Diseases