A Study to Evaluate, Safety, Tolerability, Pharmacodynamic (PD) Markers and Pharmacokinetics (PK) of AP-101 in Participants With Amyotrophic Lateral Sclerosis (ALS)

August 2, 2023 updated by: AL-S Pharma

A Phase 2a, Multicenter, Randomized, Double-blind, Placebo-controlled Study to Evaluate Safety, Tolerability, Pharmacodynamic Markers, and Pharmacokinetics of AP-101 in Patients With Familial Amyotrophic Lateral Sclerosis (fALS) and Sporadic Amyotrophic Lateral Sclerosis (sALS)

The purpose of this study is to evaluate the safety, tolerability, PK, and PD of AP-101 in participants with fALS and sALS.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

63

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Belgium
      • Leuven, Belgium, 3000
        • Recruiting
        • Department of Neurology, University Hospitals
        • Principal Investigator:
          • Philip Van Damme
  • Canada
    • Alberta
      • Edmonton, Alberta, Canada, AB T6G 1Z1
        • Recruiting
        • ALS clinic at the Kaye Edmonton Clinic, University of Alberta
        • Principal Investigator:
          • Wendy Johnston
        • Contact:
    • Ontario
      • London, Ontario, Canada, ON N6A 5W9
        • Recruiting
        • London Health Sciences Centre - Victoria Hospital
        • Principal Investigator:
          • Christen Shoesmith
        • Contact:
      • Toronto, Ontario, Canada, M4N 3M5
        • Recruiting
        • ALS Research Sunnybrook Health Sciences Centre
        • Contact:
        • Principal Investigator:
          • Lorne Zinman, Dr
    • Quebec
      • Montréal, Quebec, Canada, H3A 2B4
        • Recruiting
        • Montreal Neurological Institute and Hospital / Dr Genge
        • Contact:
        • Principal Investigator:
          • Rami Massie, Dr
  • Germany
      • Berlin, Germany, 13353
      • Bonn, Germany, 53127
        • Recruiting
        • Deutsches Zentrum für Neurodegenerative Erkrankungen e.V. (DZNE)
        • Principal Investigator:
          • Patrick Weydt
      • Hanover, Germany, 30625
      • Ulm, Germany, 89081
        • Recruiting
        • Ulm University Hospital
        • Contact:
        • Principal Investigator:
          • Albert Ludolph
  • Korea, Republic of
      • Seoul, Korea, Republic of, 04763
        • Recruiting
        • Hanyang University Medical Center
        • Contact:
        • Principal Investigator:
          • SeungHyun Kim
  • Sweden
      • Stockholm, Sweden, 113 61
        • Recruiting
        • Studieenheten Akademiskt specialistcentrum, SLSO
        • Principal Investigator:
          • Caroline Ingre
        • Contact:
          • Phone Number: +46851771231
      • Umeå, Sweden, SE- 901 85
        • Recruiting
        • Norrlands universitetssjukhus/ University Hospital of Northern Sweden (NUS)
        • Contact:
          • Phone Number: +46725487410
        • Principal Investigator:
          • Peter Munch Andersen
  • United States
    • California
      • La Jolla, California, United States, 92037
        • Recruiting
        • UC San Diego, ACTRI
        • Contact:
        • Principal Investigator:
          • John Ravits

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • All participants must adhere to contraception restrictions
  • Female participants of childbearing potential must adhere to contraception restrictions
  • Have possible, clinically probable, clinically probable-laboratory supported or definite familial or sporadic ALS in accordance with the El-Escorial criteria or who have a diagnosis of ALS as defined by the Gold Coast Criteria; progressive motor impairment documented by history or repeated clinical examination, preceded by normal motor development, and presence of upper and lower motor neuron dysfunction in at least 1 body region or lower motor neuron dysfunction in at least 2 body regions and investigations excluding other conditions
  • In familial ALS participants, a confirmed pathogenic superoxide dismutase 1 (SOD1) mutation
  • Onset of symptoms (i.e, weakness) within past 24 months prior to screening, at the time of obtaining informed consent
  • Have slow vital capacity (SVC) of greater than or equal to (> or =) 50 percentage (%) of predicted values. Participants with SVC of <50% of predicted values may be permitted to enter the open-label extension, based on the opinion of the investigator
  • Absence of bilevel positive airway pressure (BiPAP)/proportional assist ventilation (PAV) > 4 hours for symptoms attributable to ALS. Use of a CPAP for pre-existing conditions will be allowed
  • If on riluzole, must be on a stable dose.
  • If on edaravone, must have completed 2 cycles and are expected to remain on the same dose throughout the study
  • Able to provide informed consent which includes compliance with the requirements and restrictions
  • Have venous access sufficient to allow for blood sampling
  • Have clinical laboratory test results within the normal reference range for the population or study site, or results with acceptable deviations that are judged to be not clinically significant by the investigator

Exclusion Criteria:

  • Have participated or currently participating in another clinical trial within 12 weeks of baseline (Day 1)
  • Have undergone a tracheostomy for ALS symptoms
  • Are on nasal intermittent positive pressure ventilation (NIPPV) >4 hours per day for the treatment of ALS related symptoms
  • Have other causes of neuromuscular weakness
  • Have cognitive impairment, severe disease in the cardiovascular, hematological, renal system, neurodegenerative disease, pulmonary disorder, or psychiatric illness
  • Pregnant or nursing women
  • Have been exposed to any antisense treatment targeting SOD1 within 6 months of the baseline visit
  • Have undergone stem cell therapy

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Double

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: AP-101
AP-101 is administered by IV.
Drug: AP-101
Participants receive AP-101 by intravenous infusion (IV).
Placebo Comparator: Placebo
Placebo is administered by IV.
Drug: Placebo
Participants receive placebo by IV.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of Participants With Adverse Events (AEs) and Serious AEs (SAEs)
Time Frame: From start of the study up to Week 51
An AE is any untoward medical occurrence in a participant administered a pharmaceutical product and which does not necessarily have a causal relationship with the study intervention. An AE can therefore be any unfavourable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product. SAE is defined as any untoward medical occurrence that, at any dose: results in death, is life threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent disability/incapacity, is a congenital anomaly/birth defect, other situations.
From start of the study up to Week 51
Number of Participants with Abnormalities in Vital Signs, Clinical Laboratory Assessments, Physical and Neurological Examinations, Electrocardiograms (ECGs)
Time Frame: From start of the study up to Week 51
From start of the study up to Week 51

Secondary Outcome Measures

Outcome Measure
Time Frame
Elimination half-life (t1/2) of AP-101 in Serum
Time Frame: Predose up to Week 51
Predose up to Week 51
Area Under the Drug Concentration-Time Curve (AUC)
Time Frame: Predose up to Week 51
Predose up to Week 51
Concentration at End of Infusion (Cat EOI)
Time Frame: Week 24
Week 24
Change From Baseline in AP-101 Levels in the Cerebrospinal Fluid (CSF) up to Week 24
Time Frame: Baseline, up to Week 24
Baseline, up to Week 24
Change From Baseline in Neurofilament Light Chain and Phospho-Neurofilament Heavy Chain Levels in the Cerebrospinal Fluid (CSF) up to Week 51
Time Frame: Baseline, up to Week 51
Baseline, up to Week 51
Change From Baseline in Neurofilament Light Chain and Phospho-Neurofilament Heavy Chain Levels in Plasma up to Week 51
Time Frame: Baseline, up to Week 51
Baseline, up to Week 51

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

AL-S Pharma

Investigators

  • Study Director: Study Director, AL-S Pharma SA

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

September 2, 2021

Primary Completion (Estimated)

June 28, 2024

Study Completion (Estimated)

January 18, 2025

Study Registration Dates

First Submitted

September 1, 2021

First Submitted That Met QC Criteria

September 1, 2021

First Posted (Actual)

September 9, 2021

Study Record Updates

Last Update Posted (Actual)

August 3, 2023

Last Update Submitted That Met QC Criteria

August 2, 2023

Last Verified

August 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • AP101-02
  • 2020-005971-11 (EudraCT Number)

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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