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

NCT03981536 | Unknown Phase 1 DMC

• 1 other identifier: AP101-01
• Study Type: interventional
• Target: 18
• Locations: 1 country
• Sites: 3

Brief Summary

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

Conditions

Amyotrophic Lateral Sclerosis

Keywords

Familial Amyotrophic Lateral Sclerosis; Sporadic Amyotrophic Lateral Sclerosis

Outcome Measures

Primary Outcomes (2)

• 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

• 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 Outcomes (4)

• Maximum Observed Drug Concentration (Cmax)

  Baseline up to day 84

• Time of Maximum Drug Concentration (Tmax)

  Baseline up to day 84

• Area Under the Concentration Time Curve (AUC)

  Baseline up to day 84

• Pharmacokinetic Concentrations in Cerebrospinal Fluid (CSF)

  Screening, and at either 1 hour, 4 hours, 24 hours, 48 hours, 72 hours, or 168 hours

Study Arms (3)

AP-101: Dose Level 1

EXPERIMENTAL

Single dose of AP-101

Drug: AP-101

AP-101: Dose Level 2

EXPERIMENTAL

Single dose of AP-101

Drug: AP-101

AP-101: Dose Level 3

EXPERIMENTAL

Single dose of AP-101

Drug: AP-101

Interventions

AP-101 DRUG

Administered by intravenous infusion (IV)

AP-101: Dose Level 1; AP-101: Dose Level 2; AP-101: Dose Level 3

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• All participants must adhere to contraception restrictions
• Female patients of non-childbearing potential due to:
• 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)
• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• AL-S Pharma: lead

Study Sites (3)

London Health Sciences Centre, University Hospital

London, Ontario, N6A 5A5, Canada

Location

Sunnybrook Health Sciences Centre, Toronto

Toronto, Ontario, M4N 3M5, Canada

Location

Montreal Neurological Institute & Hospital

Montreal, Quebec, H3A 2B4, Canada

Location

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis; Amyotrophic lateral sclerosis 1

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Study Director

  AL-S Pharma SA

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: BASIC SCIENCE
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 30, 2019
• First Posted: June 11, 2019
• Study Start: October 10, 2019
• Primary Completion: November 2, 2020
• Study Completion: November 2, 2020
• Last Updated: October 27, 2020

Record last verified: 2020-07

Locations

CA (3)