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

Recruitment & 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Have previously completed or withdrawn from this study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

Study & Design

Arm && Interventions

Primary Outcome Measures

Name	Time	Method
Number of Participants With One or More Non-Serious Adverse Events (AEs) or Any Serious AEs (SAEs)	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)	Baseline up to day 84	Vital signs include blood pressure, pulse rate, and body temperature

Secondary Outcome Measures

Name	Time	Method
Area Under the Concentration Time Curve (AUC)	Baseline up to day 84	In serum
Maximum Observed Drug Concentration (Cmax)	Baseline up to day 84	In serum
Pharmacokinetic Concentrations in Cerebrospinal Fluid (CSF)	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
Time of Maximum Drug Concentration (Tmax)	Baseline up to day 84	In serum

Recruitment & Eligibility