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

Amyotrophic Lateral Sclerosis Clinical Trial

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)

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT03981536
• Other study ID #: AP101-01
• Status: Active, not recruiting
• Phase: Phase 1
• Start date: October 10, 2019
• Est. completion date: November 2, 2020

Study information

• Verified date: July 2020
• Source: AL-S Pharma
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 18
• Est. completion date: November 2, 2020
• Est. primary completion date: November 2, 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

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

Related Conditions & MeSH terms

• Amyotrophic Lateral Sclerosis
• Motor Neuron Disease
• Sclerosis

Intervention

Drug:
• AP-101
Administered by intravenous infusion (IV)

Locations

Country	Name	City	State
Canada	London Health Sciences Centre, University Hospital	London	Ontario
Canada	Montreal Neurological Institute & Hospital	Montréal	Quebec
Canada	Sunnybrook Health Sciences Centre, Toronto	Toronto	Ontario

Sponsors (1)

Lead Sponsor	Collaborator
AL-S Pharma

Country where clinical trial is conducted

Canada, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of Participants With One or More Non-Serious Adverse Events (AEs) or Any Serious AEs (SAEs)	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
Primary	Number of participants with abnormalities in vital signs, clinical laboratory assessments, physical or neurological examinations, or electrocardiograms (ECGs)	Vital signs include blood pressure, pulse rate, and body temperature	Baseline up to day 84
Secondary	Maximum Observed Drug Concentration (Cmax)	In serum	Baseline up to day 84
Secondary	Time of Maximum Drug Concentration (Tmax)	In serum	Baseline up to day 84
Secondary	Area Under the Concentration Time Curve (AUC)	In serum	Baseline up to day 84
Secondary	Pharmacokinetic Concentrations in Cerebrospinal Fluid (CSF)	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