Back to results

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

Primary Purpose

Amyotrophic Lateral Sclerosis

Status

Terminated

Phase

Phase 2

Locations

United States

Study Type

Interventional

Intervention

AL001

Placebo

About this trial

This is an interventional treatment trial for Amyotrophic Lateral Sclerosis

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept 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)

Sites / Locations

University of South Florida
Massachusetts General Hospital
Washington University School of Medicine

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

AL001

Placebo

Arm Description

AL001 every 4 weeks

Placebo every 4 weeks

Outcomes

Primary Outcome Measures

Evaluation of safety and tolerability of AL001 measured by number of subjects with adverse events

Incidence of adverse events during the study treatment period

Pharmacokinetics (PK) of AL001

Concentration of AL001 at specified time points

Maximum plasma concentration (Cmax) for AL001

Evaluate Cmax for concentration of AL001 at specified time points

Area under the curve concentration (AUC) for AL001

Evaluate AUC for concentration of AL001 at specified time points

Change from baseline in serum progranulin

Evaluate serum progranulin levels at pre-specified timepoints

Change from baseline in CSF progranulin

Evaluate CSF progranulin levels at pre-specified timepoints

Secondary Outcome Measures

Change from baseline in plasma neurofilament light chain

Evaluate plasma neurofilament light chain levels at pre-specified timepoints

Change from baseline in CSF neurofilament light chain

Evaluate CSF neurofilament light chain levels at pre-specified timepoints

Full Information

NCT ID

NCT05053035

First Posted

September 1, 2021

Last Updated

June 14, 2023

Sponsor

Alector Inc.

1. Study Identification

Unique Protocol Identification Number

NCT05053035

Brief Title

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

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

Study Type

Interventional

2. Study Status

Record Verification Date

June 2023

Overall Recruitment Status

Terminated

Why Stopped

The Sponsor is considering a subsequent study in ALS, potentially with different inclusion/exclusion criteria.

Study Start Date

September 2, 2021 (Actual)

Primary Completion Date

October 28, 2022 (Actual)

Study Completion Date

October 28, 2022 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Alector Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

Yes

5. Study Description

Brief Summary

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

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Amyotrophic Lateral Sclerosis

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Parallel Assignment

Masking

ParticipantCare ProviderInvestigatorOutcomes Assessor

Allocation

Randomized

Enrollment

5 (Actual)

8. Arms, Groups, and Interventions

Arm Title

AL001

Arm Type

Experimental

Arm Description

AL001 every 4 weeks

Arm Title

Placebo

Arm Type

Placebo Comparator

Arm Description

Placebo every 4 weeks

Intervention Type

Drug

Intervention Name(s)

AL001

Intervention Description

Administered via intravenous (IV) infusion

Intervention Type

Drug

Intervention Name(s)

Placebo

Intervention Description

Administered via intravenous (IV) infusion

Primary Outcome Measure Information:

Title

Evaluation of safety and tolerability of AL001 measured by number of subjects with adverse events

Description

Incidence of adverse events during the study treatment period

Time Frame

32 weeks

Title

Pharmacokinetics (PK) of AL001

Description

Concentration of AL001 at specified time points

Time Frame

32 weeks

Title

Maximum plasma concentration (Cmax) for AL001

Description

Evaluate Cmax for concentration of AL001 at specified time points

Time Frame

32 weeks

Title

Area under the curve concentration (AUC) for AL001

Description

Evaluate AUC for concentration of AL001 at specified time points

Time Frame

32 weeks

Title

Change from baseline in serum progranulin

Description

Evaluate serum progranulin levels at pre-specified timepoints

Time Frame

32 weeks

Title

Change from baseline in CSF progranulin

Description

Evaluate CSF progranulin levels at pre-specified timepoints

Time Frame

32 weeks

Secondary Outcome Measure Information:

Title

Change from baseline in plasma neurofilament light chain

Description

Evaluate plasma neurofilament light chain levels at pre-specified timepoints

Time Frame

32 weeks

Title

Change from baseline in CSF neurofilament light chain

Description

Evaluate CSF neurofilament light chain levels at pre-specified timepoints

Time Frame

32 weeks

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

Eligibility 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)

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Sabrina Paganoni, MD

Organizational Affiliation

Massachusetts General Hospital

Official's Role

Principal Investigator

Facility Information:

Facility Name

University of South Florida

City

Tampa

State/Province

Florida

ZIP/Postal Code

33612

Country

United States

Facility Name

Massachusetts General Hospital

City

Boston

State/Province

Massachusetts

ZIP/Postal Code

02114

Country

United States

Facility Name

Washington University School of Medicine

City

Saint Louis

State/Province

Missouri

ZIP/Postal Code

63110

Country

United States

12. IPD Sharing Statement

Learn more about this trial

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

We'll reach out to this number within 24 hrs