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

• Register: ClinicalTrials.gov
• Last refreshed on: 26 June 2023
• Main ID: NCT05053035
• Date of registration: 01/09/2021
• Prospective Registration: Yes
• Primary sponsor: Alector Inc.
• Public title: A Phase 2 Study to Evaluate AL001 in C9orf72-Associated ALS
• Scientific 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
• Date of first enrolment: September 2, 2021
• Target sample size: 5
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT05053035
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Sabrina Paganoni, MD
• Affiliation: Massachusetts General Hospital

Key inclusion & exclusion 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)



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Amyotrophic Lateral Sclerosis

Intervention(s)

Drug: Placebo

Drug: AL001

Primary Outcome(s)

Maximum plasma concentration (Cmax) for AL001 [Time Frame: 32 weeks]

Pharmacokinetics (PK) of AL001 [Time Frame: 32 weeks]

Change from baseline in serum progranulin [Time Frame: 32 weeks]

Change from baseline in CSF progranulin [Time Frame: 32 weeks]

Evaluation of safety and tolerability of AL001 measured by number of subjects with adverse events [Time Frame: 32 weeks]

Area under the curve concentration (AUC) for AL001 [Time Frame: 32 weeks]

Secondary Outcome(s)

Change from baseline in plasma neurofilament light chain [Time Frame: 32 weeks]

Change from baseline in CSF neurofilament light chain [Time Frame: 32 weeks]

Secondary ID(s)

AL001-ALS-201

Source(s) of Monetary Support

Please refer to primary and secondary sponsors