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出境医 / 临床实验 / A Study to Evaluate AP-101 in Familial and Sporadic Amyotrophic Lateral Sclerosis (ALS)

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

Study Description
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

Study Design
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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
Arms and Interventions
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)
Outcome Measures
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


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:

  • 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
Contacts and Locations

Locations
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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
Investigators
Layout table for investigator information
Study Director: Study Director AL-S Pharma SA
Tracking Information
First Submitted Date  ICMJE May 30, 2019
First Posted Date  ICMJE June 11, 2019
Last Update Posted Date October 27, 2020
Actual Study Start Date  ICMJE October 10, 2019
Estimated Primary Completion Date November 2, 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: June 7, 2019)
  • 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
  • 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
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: June 7, 2019)
  • Maximum Observed Drug Concentration (Cmax) [ Time Frame: Baseline up to day 84 ]
    In serum
  • Time of Maximum Drug Concentration (Tmax) [ Time Frame: Baseline up to day 84 ]
    In serum
  • Area Under the Concentration Time Curve (AUC) [ Time Frame: Baseline up to day 84 ]
    In serum
  • 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
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Study to Evaluate AP-101 in Familial and Sporadic Amyotrophic Lateral Sclerosis (ALS)
Official Title  ICMJE 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)
Brief Summary Single ascending doses of AP-101 will be administered by intravenous (IV) infusion
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Basic Science
Condition  ICMJE Amyotrophic Lateral Sclerosis
Intervention  ICMJE Drug: AP-101
Administered by intravenous infusion (IV)
Study Arms  ICMJE
  • Experimental: AP-101: Dose Level 1
    Single dose of AP-101
    Intervention: Drug: AP-101
  • Experimental: AP-101: Dose Level 2
    Single dose of AP-101
    Intervention: Drug: AP-101
  • Experimental: AP-101: Dose Level 3
    Single dose of AP-101
    Intervention: Drug: AP-101
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Active, not recruiting
Estimated Enrollment  ICMJE
 (submitted: June 7, 2019) 		18
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE November 2, 2020
Estimated Primary Completion Date November 2, 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Canada
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03981536
Other Study ID Numbers  ICMJE AP101-01
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party AL-S Pharma
Study Sponsor  ICMJE AL-S Pharma
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Study Director AL-S Pharma SA
PRS Account AL-S Pharma
Verification Date July 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP

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