A Phase 2 Study to Evaluate AL001 in C9orf72-Associated ALS

Alector

Status and phase: Terminated; Phase 2

Conditions: Amyotrophic Lateral Sclerosis

Treatments: Drug: Placebo; Drug: AL001

Study type: Interventional

Funder types: Industry

Identifiers

NCT05053035

AL001-ALS-201

Details and patient eligibility

About

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

Full 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.

Enrollment: 5 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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)

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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5 participants in 2 patient groups, including a placebo group

AL001

Experimental group

Description:

AL001 every 4 weeks

Treatment:

Drug: AL001

Placebo

Placebo Comparator group

Description:

Placebo every 4 weeks

Treatment:

Drug: Placebo