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

NCT05053035 | Terminated Phase 2 Results DMC FDA Drug

• 1 other identifier: AL001-ALS-201
• Study Type: interventional
• Target: 5
• Locations: 1 country
• Sites: 3

Brief Summary

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

Conditions

Amyotrophic Lateral Sclerosis

Outcome Measures

Primary Outcomes (6)

• Evaluation of Safety and Tolerability of AL001 Measured by Number of Subjects With Adverse Events

  Count of participants with adverse events during the study treatment period

  24 weeks

• Immunogenicity of AL001

  Count of participants positive for Anti-drug Antibodies (ADAs) to AL001 at week 24

  Week 24

• Pharmacokinetics (PK) of AL001 in Serum

  Concentration of AL001 in Serum at week 24

  Week 24

• Pharmacokinetics (PK) of AL001 in CSF

  Concentration of AL001 in Cerebrospinal fluid (CSF) at week 24

  Week 24

• Change From Baseline in Plasma Progranulin

  Evaluate the change from baseline to week 24 in plasma progranulin levels

  24 weeks

• Change From Baseline in CSF Progranulin

  Evaluate the change from baseline to week 24 in Cerebrospinal fluid (CSF) progranulin levels

  24 weeks

Secondary Outcomes (2)

• Change From Baseline in Plasma Neurofilament Light Chain

  24 weeks

• Change From Baseline in CSF Neurofilament Light Chain

  24 weeks

Study Arms (2)

AL001

EXPERIMENTAL

AL001 every 4 weeks

Drug: AL001

Placebo

PLACEBO COMPARATOR

Placebo every 4 weeks

Drug: Placebo

Interventions

AL001 DRUG

Administered via intravenous (IV) infusion

AL001

Placebo DRUG

Administered via intravenous (IV) infusion

Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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)

Sponsors & Collaborators

• Alector Inc.: lead

Study Sites (3)

University of South Florida

Tampa, Florida, 33612, United States

Location

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

Washington University School of Medicine

St Louis, Missouri, 63110, United States

Location

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Results Point of Contact

• Title: Alector Medical Information
• Organization: Alector

medinfo@alector.com

650-826-2454

Study Officials

• Sabrina Paganoni, MD

  Massachusetts General Hospital

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 1, 2021
• First Posted: September 22, 2021
• Study Start: September 2, 2021
• Primary Completion: October 28, 2022
• Study Completion: October 28, 2022
• Last Updated: June 18, 2025
• Results First Posted: June 18, 2025

Record last verified: 2025-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (3)