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

Overview

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

Full Title of Study: “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)”

Study Type

  • Study Type: Interventional
  • Study Design
    • Allocation: Non-Randomized
    • Intervention Model: Parallel Assignment
    • Primary Purpose: Basic Science
    • Masking: None (Open Label)
  • Study Primary Completion Date: November 2, 2020

Interventions

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

Arms, Groups and Cohorts

  • Experimental: AP-101: Dose Level 1
    • Single dose of AP-101
  • Experimental: AP-101: Dose Level 2
    • Single dose of AP-101
  • Experimental: AP-101: Dose Level 3
    • Single dose of AP-101

Clinical Trial Outcome Measures

Primary Measures

  • 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

Secondary Measures

  • 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

Participating in This Clinical Trial

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

Gender Eligibility: All

Minimum Age: 18 Years

Maximum Age: N/A

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • AL-S Pharma
  • Provider of Information About this Clinical Study
    • Sponsor
  • Overall Official(s)
    • Study Director, Study Director, AL-S Pharma SA

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