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Quick Info
Status
Ongoing, But Not Recruiting
Phase
1
Trial Type
Non-Randomized, open label
Treatment Type
IV Drug: AP-101
Randomization
Open label trial
Enrollment
18
Start Date
10/10/2019
Sponsor
Contact Information
Locations
Canada, Ontario
London Health Sciences Centre, University Hospital, London, ON, N6A 5A5, Canada
Sunnybrook Health Sciences Centre, Toronto, Toronto, ON, M4N 3M5, Canada
Canada, Quebec
Montreal Neurological Institute & Hospital, Montréal, QC, H3A 2B4, Canada
Enrollment Criteria
Breathing Ability
Percent lung function (FVC) or (SVC)
≥60% SVC
Months Since Onset
Number of months since first symptoms of ALS.
≤ 2 years
Non-Invasive Ventilation (NIV)
Can PALS use a BiPAP in the trial?
unknown
Diaphragm Pacer (DPS)
Can PALS use a DPS in the trial?
unknown
Edaravone Usage
Can a PALS use edaravone (Radicut/Radicava) while enrolled in the trial?
Yes
Open Label
Yes
Update Notes
No longer recruiting, site updates
10/27/2020
Study contact information added.
7/10/2020
Updated recruitment status.
6/25/2020
Moved to Ongoing, Not Recruiting status Removed Primary Contact Removed Calgary Location All Locations moved to not recruiting status
3/26/2020
Montreal Location now recruiting.
3/19/2020
No significant updates
1/30/2020
Updated recruitment status
1/23/2020
No significant updates
12/4/2019
updated recruitment status
10/18/2019
No significant updates
9/18/2019
No significant updates.
7/31/2019
No significant updates
7/1/2019
No significant updates
7/1/2019
Trial added
6/12/2019

Other Information

Purpose
Single ascending doses of AP-101 will be administered by intravenous (IV) infusion
Eligibility
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
Details
Collaborator(s)
  • AL-S Pharma
Trial Protocol as Published on Clinicaltrials.gov
NCT03981536 (First Published: 5/30/2019)