A Study to Evaluate AP-101 in Familial and Sporadic Amyotrophic Lateral Sclerosis (ALS)

October 26, 2020 updated by: AL-S Pharma

A Multicenter, Open Label, Single-Ascending Dose Study to Evaluate Safety, Tolerability, and Pharmacokinetics of AP-101 in Familial and Sporadic Amyotrophic Lateral Sclerosis (ALS)

Single ascending doses of AP-101 will be administered by intravenous (IV) infusion

Study Overview

Status

Unknown

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Anticipated)

18

Phase

  • Phase 1

Contacts and Locations

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

Study Locations

  • Canada
    • Ontario
      • London, Ontario, Canada, N6A 5A5
        • London Health Sciences Centre, University Hospital
      • Toronto, Ontario, Canada, M4N 3M5
        • Sunnybrook Health Sciences Centre, Toronto
    • Quebec
      • Montréal, Quebec, Canada, H3A 2B4
        • Montreal Neurological Institute & Hospital

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

16 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • All participants must adhere to contraception restrictions

  • Female patients of non-childbearing potential due to:

    1. Menopause: spontaneous amenorrhea for at least 12 months not induced by a medical conditions such as anorexia nervosa and not taking medications that induced the amenorrhea (e.g., oral contraceptives, hormones, gonadotropin releasing hormones, anti-estrogens, selective estrogen receptor modulators, or chemotherapy)
    2. Surgical sterilization
  • Have possible, probable, probable laboratory supported or definite and definite familial laboratory-supported ALS in accordance with the El-Escorial criteria
  • Have familial or sporadic ALS.
  • With onset of ALS symptoms, specifically onset of muscle weakness within past 48 months
  • Have slow vital capacity (SVC) of (greater than or equal to) ≥60%
  • 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. If the patient is not able to provide written consent due to aggravation of disease condition, written informed consent may be provided by a legally authorized representative
  • Have venous access sufficient to allow for blood sampling
  • Have clinical laboratory test results within normal reference range for the population or study site, or results with acceptable deviations that are judged to be not clinically significant

Exclusion Criteria:

  • Are currently enrolled in, or discontinued from, within the last 30 days, a clinical trial involving an investigational drug or device or off-label use of a drug or device, or any other type of medical research judged not to be scientifically or medically compatible with this study
  • Have previously completed or withdrawn from this study
  • Have a history or presence of medical illness including, but not limited to, any cognitive, cardiovascular, hepatic, hematological, renal, endocrine, or psychiatric, or any clinically significant laboratory abnormality that indicates a medical problem that would preclude study participation
  • Show evidence of human immunodeficiency virus (HIV) infection and/or positive human HIV antibodies
  • Show evidence of hepatitis C and/or positive hepatitis C antibody
  • Show evidence of hepatitis B and/or positive hepatitis B surface antigen
  • Are women who are lactating.
  • Have undergone a tracheostomy unless it was removed at least 6 months prior
  • Are on feeding tube, unless the insertion of a feeding tube is considered prophylactic
  • Are on nasal intermittent positive pressure ventilation (NIPPV) >4 hours per day or at the discretion of the medical monitor
  • 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: Basic Science
  • Allocation: Non-Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: AP-101: Dose Level 1
Single dose of AP-101
Drug: AP-101
Administered by intravenous infusion (IV)
Experimental: AP-101: Dose Level 2
Single dose of AP-101
Drug: AP-101
Administered by intravenous infusion (IV)
Experimental: AP-101: Dose Level 3
Single dose of AP-101
Drug: AP-101
Administered by intravenous infusion (IV)

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of Participants With One or More Non-Serious Adverse Events (AEs) or Any Serious AEs (SAEs)
Time Frame: Baseline up to day 84
A clinical trial AE is any untoward medical event associated with the use of a drug or drug delivery system in humans, whether or not it is considered related to that drug or drug delivery system
Baseline up to day 84
Number of participants with abnormalities in vital signs, clinical laboratory assessments, physical or neurological examinations, or electrocardiograms (ECGs)
Time Frame: Baseline up to day 84
Vital signs include blood pressure, pulse rate, and body temperature
Baseline up to day 84

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Maximum Observed Drug Concentration (Cmax)
Time Frame: Baseline up to day 84
In serum
Baseline up to day 84
Time of Maximum Drug Concentration (Tmax)
Time Frame: Baseline up to day 84
In serum
Baseline up to day 84
Area Under the Concentration Time Curve (AUC)
Time Frame: Baseline up to day 84
In serum
Baseline up to day 84
Pharmacokinetic Concentrations in Cerebrospinal Fluid (CSF)
Time Frame: Screening, and at either 1 hour, 4 hours, 24 hours, 48 hours, 72 hours, or 168 hours
Taken at screening, and then only one sample per participant post-dose, in the higher level doses
Screening, and at either 1 hour, 4 hours, 24 hours, 48 hours, 72 hours, or 168 hours

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)

October 10, 2019

Primary Completion (Anticipated)

November 2, 2020

Study Completion (Anticipated)

November 2, 2020

Study Registration Dates

First Submitted

May 30, 2019

First Submitted That Met QC Criteria

June 7, 2019

First Posted (Actual)

June 11, 2019

Study Record Updates

Last Update Posted (Actual)

October 27, 2020

Last Update Submitted That Met QC Criteria

October 26, 2020

Last Verified

July 1, 2020

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • AP101-01

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

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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