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

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE2

Total Enrollment

5 participants

Study Classification

INTERVENTIONAL

Study Start Date

2021-09-02

Study Completion Date

2022-10-28

Brief Summary

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

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Detailed Description

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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.

Conditions

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Amyotrophic Lateral Sclerosis

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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AL001

AL001 every 4 weeks

Group Type EXPERIMENTAL

AL001

Intervention Type DRUG

Administered via intravenous (IV) infusion

Placebo

Placebo every 4 weeks

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Administered via intravenous (IV) infusion

Interventions

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AL001

Administered via intravenous (IV) infusion

Intervention Type DRUG

Placebo

Administered via intravenous (IV) infusion

Intervention Type DRUG

Eligibility Criteria

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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)

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No