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A Study to Evaluate AP-101 in Familial and Sporadic Amyotrophic Lateral Sclerosis (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. Identifier: NCT03981536
Recruitment Status : Unknown
Verified July 2020 by AL-S Pharma.
Recruitment status was:  Active, not recruiting
First Posted : June 11, 2019
Last Update Posted : October 27, 2020
Information provided by (Responsible Party):
AL-S Pharma

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

Condition or disease Intervention/treatment Phase
Amyotrophic Lateral Sclerosis Drug: AP-101 Phase 1

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 18 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Basic Science
Official Title: 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)
Actual Study Start Date : October 10, 2019
Estimated Primary Completion Date : November 2, 2020
Estimated Study Completion Date : November 2, 2020

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)

Primary Outcome Measures :
  1. 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

  2. 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

Secondary Outcome Measures :
  1. Maximum Observed Drug Concentration (Cmax) [ Time Frame: Baseline up to day 84 ]
    In serum

  2. Time of Maximum Drug Concentration (Tmax) [ Time Frame: Baseline up to day 84 ]
    In serum

  3. Area Under the Concentration Time Curve (AUC) [ Time Frame: Baseline up to day 84 ]
    In serum

  4. 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

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

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 identifier (NCT number): NCT03981536

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Canada, Ontario
London Health Sciences Centre, University Hospital
London, Ontario, Canada, N6A 5A5
Sunnybrook Health Sciences Centre, Toronto
Toronto, Ontario, Canada, M4N 3M5
Canada, Quebec
Montreal Neurological Institute & Hospital
Montréal, Quebec, Canada, H3A 2B4
Sponsors and Collaborators
AL-S Pharma
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Study Director: Study Director AL-S Pharma SA
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Responsible Party: AL-S Pharma Identifier: NCT03981536    
Other Study ID Numbers: AP101-01
First Posted: June 11, 2019    Key Record Dates
Last Update Posted: October 27, 2020
Last Verified: July 2020

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