Clinical trial NCT05053035: A Phase 2 Study to Evaluate AL001 in C9orf72-Associated ALS
Official title: A Phase 2, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AL001 in C9orf72-Associated Amyotrophic Lateral Sclerosis
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Amyotrophic Lateral Sclerosis
Lead sponsor: Alector Inc.
Sponsor class: INDUSTRY
First posted: 2021-09-22
Enrollment: 5
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: A phase 2 double-blind, placebo-controlled study of AL001 in participants with C9orf72-associated ALS.

Detailed description: This is a phase 2 double-blind, placebo-controlled trial to test the safety, tolerability, pharmacokinetics, and pharmacodynamics of AL001 in participants with C9orf72-associated Amyotrophic Lateral Sclerosis.

Eligibility criteria: Inclusion Criteria:

* Confirmation of C9orf72 mutation
* Diagnosis of ALS by revised El Escorial criteria
* Time since onset of muscle weakness due to ALS ≤36 months at the time of the Screening Visit
* Slow Vital Capacity (VC) ≥50% of predicted capacity at the time of the Screening Visit
* If taking riluzole, must be on a stable dose of riluzole for at least 30 days prior to the Screening Visit. Riluzole naive participants are allowed.
* If taking edaravone, must have completed at least one cycle of edaravone prior to the Screening Visit and plan to continue edaravone during the study. Edaravone naive participants are allowed.
* Females must not be pregnant, breastfeeding or planning to conceive within the study period. Males must agree to use acceptable contraception
* Capable of providing informed consent at the Screening visit and complying with study procedures throughout the study

Exclusion Criteria:

* Clinically significant, unstable, medical condition (other than ALS)
* Clinically significant heart disease, liver disease or kidney disease
* Cognitive impairment or dementia
* Current uncontrolled hypertension
* History of unresolved cancer
* Any experimental gene therapy
* Any experimental vaccine (any vaccine against COVID-19 either approved or administered under an Emergency Use Authorization is allowed)

Primary outcomes:
measure: Evaluation of Safety and Tolerability of AL001 Measured by Number of Subjects With Adverse Events
timeFrame: 24 weeks
measure: Immunogenicity of AL001
timeFrame: Week 24
measure: Pharmacokinetics (PK) of AL001 in Serum
timeFrame: Week 24
measure: Pharmacokinetics (PK) of AL001 in CSF
timeFrame: Week 24
measure: Change From Baseline in Plasma Progranulin
timeFrame: 24 weeks
measure: Change From Baseline in CSF Progranulin
timeFrame: 24 weeks
Secondary outcomes:
measure: Change From Baseline in Plasma Neurofilament Light Chain
timeFrame: 24 weeks
measure: Change From Baseline in CSF Neurofilament Light Chain
timeFrame: 24 weeks
Other outcomes:
Locations:
city: Tampa
country: United States
facility: University of South Florida
state: Florida
city: Boston
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
city: Saint Louis
country: United States
facility: Washington University School of Medicine
state: Missouri
Study officials:
affiliation: Massachusetts General Hospital
name: Sabrina Paganoni, MD
role: PRINCIPAL_INVESTIGATOR
References: