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

Amyotrophic Lateral Sclerosis Clinical Trial

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT05053035
• Other study ID #: AL001-ALS-201
• Status: Terminated
• Phase: Phase 2
• Start date: September 2, 2021
• Est. completion date: October 28, 2022

Study information

• Verified date: June 2023
• Source: Alector Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 5
• Est. completion date: October 28, 2022
• Est. primary completion date: October 28, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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)

Related Conditions & MeSH terms

• Amyotrophic Lateral Sclerosis
• Motor Neuron Disease
• Sclerosis

Intervention

Drug:
• AL001
Administered via intravenous (IV) infusion
• Placebo
Administered via intravenous (IV) infusion

Locations

Country	Name	City	State
United States	Massachusetts General Hospital	Boston	Massachusetts
United States	Washington University School of Medicine	Saint Louis	Missouri
United States	University of South Florida	Tampa	Florida

Sponsors (1)

Lead Sponsor	Collaborator
Alector Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Evaluation of safety and tolerability of AL001 measured by number of subjects with adverse events	Incidence of adverse events during the study treatment period	32 weeks
Primary	Pharmacokinetics (PK) of AL001	Concentration of AL001 at specified time points	32 weeks
Primary	Maximum plasma concentration (Cmax) for AL001	Evaluate Cmax for concentration of AL001 at specified time points	32 weeks
Primary	Area under the curve concentration (AUC) for AL001	Evaluate AUC for concentration of AL001 at specified time points	32 weeks
Primary	Change from baseline in serum progranulin	Evaluate serum progranulin levels at pre-specified timepoints	32 weeks
Primary	Change from baseline in CSF progranulin	Evaluate CSF progranulin levels at pre-specified timepoints	32 weeks
Secondary	Change from baseline in plasma neurofilament light chain	Evaluate plasma neurofilament light chain levels at pre-specified timepoints	32 weeks
Secondary	Change from baseline in CSF neurofilament light chain	Evaluate CSF neurofilament light chain levels at pre-specified timepoints	32 weeks