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

Tracking Information

• First Submitted Date: June 23, 2021
• First Posted Date: July 2, 2021
• Last Update Posted Date: January 25, 2023
• Actual Study Start Date: September 22, 2021
• Actual Primary Completion Date: January 12, 2023 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: June 30, 2021)

• 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
• Pharmacokinetics [ Time Frame: Baseline Up to Approximately Day 28 ]
  Maximum Plasma Concentration [Cmax]
• Pharmacokinetics [ Time Frame: Baseline Up to Approximately Day 28 ]
  Area Under the Curve [AUC]

• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: January 20, 2023)

• CSF Pharmacokinetics [ Time Frame: Baseline Up to Approximately Day 28 ]
  Concentration at steady state in CSF
• 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

Original Secondary Outcome Measures (submitted: June 30, 2021)

• CSF Pharmacokinetics [ Time Frame: Baseline Up to Approximately Day 28 ]
  Concentration at steady state in CSF
• Safety and Tolerability [ Time Frame: Baseline Up to Approximately Week 48 ]
  Number of patients with treatment-related adverse events as assessed by CTCAE v4.03

• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: A Phase 1 Study to Investigate the Safety and Pharmacokinetics of ABBV-CLS-7262 in Patients With Amyotrophic Lateral Sclerosis
• 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
• 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.
• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 1
• Study Design: Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Triple (Participant, Care Provider, Investigator); Primary Purpose: Treatment

Condition

• ALS
• Amyotrophic Lateral Sclerosis

Intervention

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

Study Arms

• Active Comparator: ABBV-CLS-7262 LOW DOSE
  Intervention: Drug: ABBV-CLS-7262
• Active Comparator: ABBV-CLS-7262 MEDIUM DOSE
  Intervention: Drug: ABBV-CLS-7262
• Active Comparator: ABBV-CLS-7262 HIGH DOSE
  Intervention: Drug: ABBV-CLS-7262
• Placebo Comparator: PLACEBO
  Intervention: Drug: Placebo

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Active, not recruiting
• Actual Enrollment (submitted: January 20, 2023): 31
• Original Estimated Enrollment (submitted: June 30, 2021): 30
• Estimated Study Completion Date: December 2025
• Actual Primary Completion Date: January 12, 2023 (Final data collection date for primary outcome measure)

Eligibility 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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years to 80 Years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Canada, United States

Administrative Information

• NCT Number: NCT04948645
• Other Study ID Numbers: M20-405
• Has Data Monitoring Committee: Yes

U.S. FDA-regulated Product

• 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

• IPD Sharing Statement: Not Provided
• Current Responsible Party: Calico Life Sciences LLC
• Original Responsible Party: Same as current
• Current Study Sponsor: Calico Life Sciences LLC
• Original Study Sponsor: Same as current
• Collaborators: AbbVie
• Investigators: Not Provided
• PRS Account: Calico Life Sciences LLC
• Verification Date: January 2023