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Trial record 1 of 487 for:    Amyotrophic Lateral Sclerosis
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A Study to Evaluate AP-101 in Familial and Sporadic Amyotrophic Lateral Sclerosis (ALS)

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ClinicalTrials.gov Identifier: NCT03981536
Recruitment Status : Not yet recruiting
First Posted : June 11, 2019
Last Update Posted : July 1, 2019
Sponsor:
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)
Estimated Study Start Date : July 2019
Estimated Primary Completion Date : January 2020
Estimated Study Completion Date : January 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

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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:

  • 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.
  • Are on feeding tube
  • 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 ClinicalTrials.gov identifier (NCT number): NCT03981536


Contacts
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Contact: Study Director: AL-S Pharma SA 3176517036 choruspharma@lists.lilly.com

Locations
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Canada, Alberta
Department of Clinical Neurosciences, Heritage Medical Research Clinic, University of Calgary Not yet recruiting
Calgary, Alberta, Canada, T2N 4N1
Contact: Lawrence Korngut       korngut@gmail.com   
Contact: Janet Patrillo    403-210-7006    japetril@ucalgary.ca   
Principal Investigator: Lawrence Korngut         
Kaye Edmonton Clinic, University of Alberta Not yet recruiting
Edmonton, Alberta, Canada, T6G 1Z1
Contact: Wendy Johnston    780-407-5000    wendyj@ualberta.ca   
Contact: Shelley Wrona       wrona@ualberta.ca   
Principal Investigator: Wendy Johnston         
Canada, Ontario
London Health Sciences Centre, University Hospital Not yet recruiting
London, Ontario, Canada, N6A 5A5
Contact: Chisten Shoesmith    519-685-8500    Christen.Shoesmith@lhsc.on.ca   
Contact: Christine Piechowicz       Christine.Piechowicz@lhsc.on.ca   
Principal Investigator: Christen Shoesmith         
Sunnybrook Health Sciences Centre, Toronto Not yet recruiting
Toronto, Ontario, Canada, M4N 3M5
Contact: Lorne Zinman    416-480-6100    lorne.zinman@sunnybrook.ca   
Contact: Shirley Pham    416-480-6100    Shirley.Pham@sunnybrook.ca   
Principal Investigator: Lorne Zinman         
Canada, Quebec
Montreal Neurological Institute & Hospital Not yet recruiting
Montréal, Quebec, Canada, H3A 2B4
Contact: Maxime Berube    514-398-6644    maximeberube@sympatico.ca   
Contact: Kristiana Salmon       kristiana.salmon@mcgill.ca   
Principal Investigator: Maxime Berube         
Sponsors and Collaborators
AL-S Pharma
Investigators
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Study Director: Study Director AL-S Pharma SA

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Responsible Party: AL-S Pharma
ClinicalTrials.gov Identifier: NCT03981536     History of Changes
Other Study ID Numbers: AP101-01
First Posted: June 11, 2019    Key Record Dates
Last Update Posted: July 1, 2019
Last Verified: June 2019

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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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Sclerosis
Amyotrophic Lateral Sclerosis
Motor Neuron Disease
Pathologic Processes
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases