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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05053035
Recruitment Status : Active, not recruiting
First Posted : September 22, 2021
Last Update Posted : April 15, 2022
Sponsor:
Information provided by (Responsible Party):
Alector Inc.

Study Description
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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
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Study Type : Interventional  (Clinical Trial)
Estimated 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 : September 2, 2021
Estimated Primary Completion Date : February 2023
Estimated Study Completion Date : February 2023

Resource links provided by the National Library of Medicine


Arms and Interventions
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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
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Primary Outcome Measures :
  1. Evaluation of safety and tolerability of AL001 measured by number of subjects with adverse events [ Time Frame: 32 weeks ]
    Incidence of adverse events during the study treatment period

  2. Pharmacokinetics (PK) of AL001 [ Time Frame: 32 weeks ]
    Concentration of AL001 at specified time points

  3. Maximum plasma concentration (Cmax) for AL001 [ Time Frame: 32 weeks ]
    Evaluate Cmax for concentration of AL001 at specified time points

  4. Area under the curve concentration (AUC) for AL001 [ Time Frame: 32 weeks ]
    Evaluate AUC for concentration of AL001 at specified time points

  5. Change from baseline in serum progranulin [ Time Frame: 32 weeks ]
    Evaluate serum progranulin levels at pre-specified timepoints

  6. Change from baseline in CSF progranulin [ Time Frame: 32 weeks ]
    Evaluate CSF progranulin levels at pre-specified timepoints


Secondary Outcome Measures :
  1. Change from baseline in plasma neurofilament light chain [ Time Frame: 32 weeks ]
    Evaluate plasma neurofilament light chain levels at pre-specified timepoints

  2. Change from baseline in CSF neurofilament light chain [ Time Frame: 32 weeks ]
    Evaluate CSF neurofilament light chain levels at pre-specified timepoints


Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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)
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05053035


Locations
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United States, Arizona
Barrow Neurological Instiute
Phoenix, Arizona, United States, 85013
United States, California
University of California, San Francisco
San Francisco, California, United States, 94117
United States, Colorado
University of Colorado
Aurora, Colorado, United States, 80045
United States, Florida
Mayo Clinic Florida
Jacksonville, Florida, United States, 32224
University of South Florida
Tampa, Florida, United States, 33612
United States, Indiana
Indiana University
Indianapolis, Indiana, United States, 46202
United States, Maryland
Johns Hopkins University
Baltimore, Maryland, United States, 21205
United States, Massachusetts
Massachusetts General Hospital
Boston, Massachusetts, United States, 02114
United States, Michigan
University of Michigan
Ann Arbor, Michigan, United States, 48109
United States, Missouri
Washington University School of Medicine
Saint Louis, Missouri, United States, 63110
United States, Pennsylvania
Jefferson University
Philadelphia, Pennsylvania, United States, 19107
United States, Washington
University of Washington
Seattle, Washington, United States, 98195
Sponsors and Collaborators
Alector Inc.
Investigators
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Principal Investigator: Sabrina Paganoni, MD Massachusetts General Hospital
More Information
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Responsible Party: Alector Inc.
ClinicalTrials.gov Identifier: NCT05053035    
Other Study ID Numbers: AL001-ALS-201
First Posted: September 22, 2021    Key Record Dates
Last Update Posted: April 15, 2022
Last Verified: April 2022

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Sclerosis
Pathologic Processes
Neurodegenerative Diseases
Nervous System Diseases
 Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases