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

Sponsor

Alector Inc. (Industry)

Overall Status

Active, not recruiting

CT.gov ID

NCT05053035

Collaborator

(none)

Enrollment

Locations

Arms

Anticipated Duration (Months)

3.8

Patients Per Site

0.2

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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

Condition or Disease	Intervention/Treatment	Phase
Amyotrophic Lateral Sclerosis	Drug: AL001 Drug: Placebo	Phase 2

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.

Study Design

Study Type:

Interventional

Anticipated Enrollment :

45 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Masking:

Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Primary Purpose:

Treatment

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

Actual Study Start Date :

Sep 2, 2021

Anticipated Primary Completion Date :

Feb 1, 2023

Anticipated Study Completion Date :

Feb 1, 2023

Arms and Interventions

Arm	Intervention/Treatment
Experimental: AL001 AL001 every 4 weeks	Drug: AL001 Administered via intravenous (IV) infusion
Placebo Comparator: Placebo Placebo every 4 weeks	Drug: Placebo Administered via intravenous (IV) infusion

Arm

Intervention/Treatment

Experimental: AL001

AL001 every 4 weeks

Drug: AL001

Administered via intravenous (IV) infusion

Placebo Comparator: Placebo

Placebo every 4 weeks

Drug: Placebo

Administered via intravenous (IV) infusion

Outcome Measures

Primary Outcome Measures

Evaluation of safety and tolerability of AL001 measured by number of subjects with adverse events [32 weeks]
Incidence of adverse events during the study treatment period
Pharmacokinetics (PK) of AL001 [32 weeks]
Concentration of AL001 at specified time points
Maximum plasma concentration (Cmax) for AL001 [32 weeks]
Evaluate Cmax for concentration of AL001 at specified time points
Area under the curve concentration (AUC) for AL001 [32 weeks]
Evaluate AUC for concentration of AL001 at specified time points
Change from baseline in serum progranulin [32 weeks]
Evaluate serum progranulin levels at pre-specified timepoints
Change from baseline in CSF progranulin [32 weeks]
Evaluate CSF progranulin levels at pre-specified timepoints

Secondary Outcome Measures

Change from baseline in plasma neurofilament light chain [32 weeks]
Evaluate plasma neurofilament light chain levels at pre-specified timepoints
Change from baseline in CSF neurofilament light chain [32 weeks]
Evaluate CSF neurofilament light chain levels at pre-specified timepoints

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts 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)

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Barrow Neurological Instiute	Phoenix	Arizona	United States	85013
2	University of California, San Francisco	San Francisco	California	United States	94117
3	University of Colorado	Aurora	Colorado	United States	80045
4	Mayo Clinic Florida	Jacksonville	Florida	United States	32224
5	University of South Florida	Tampa	Florida	United States	33612
6	Indiana University	Indianapolis	Indiana	United States	46202
7	Johns Hopkins University	Baltimore	Maryland	United States	21205
8	Massachusetts General Hospital	Boston	Massachusetts	United States	02114
9	University of Michigan	Ann Arbor	Michigan	United States	48109
10	Washington University School of Medicine	Saint Louis	Missouri	United States	63110
11	Jefferson University	Philadelphia	Pennsylvania	United States	19107
12	University of Washington	Seattle	Washington	United States	98195

Sponsors and Collaborators

Alector Inc.

Investigators

Principal Investigator: Sabrina Paganoni, MD, Massachusetts General Hospital

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

Alector Inc.

ClinicalTrials.gov Identifier:

NCT05053035

Other Study ID Numbers:

AL001-ALS-201

First Posted:

Sep 22, 2021

Last Update Posted:

Apr 15, 2022

Last Verified:

Apr 1, 2022

Studies a U.S. FDA-regulated Drug Product:

Yes

Studies a U.S. FDA-regulated Device Product:

Additional relevant MeSH terms:

Motor Neuron Disease

Amyotrophic Lateral Sclerosis

Sclerosis

Study Results

No Results Posted as of Apr 15, 2022