Amyotrophic Lateral Sclerosis
Conditions
Keywords
Familial Amyotrophic Lateral Sclerosis, Sporadic Amyotrophic Lateral Sclerosis
Brief summary
Single ascending doses of AP-101 will be administered by intravenous (IV) infusion
Interventions
DRUGAP-101
Administered by intravenous infusion (IV)
Sponsors
AL-S Pharma
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
BASIC_SCIENCE
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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medical research judged not to be scientifically or medically compatible with this study * Have previously completed or withdrawn from this study * Have a history or presence of medical illness including, but not limited to, any cognitive, cardiovascular, hepatic, hematological, renal,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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| Maximum Observed Drug Concentration (Cmax) | Baseline up to day 84 | In serum |
| Time of Maximum Drug Concentration (Tmax) | Baseline up to day 84 | In serum |
| Area Under the Concentration Time Curve (AUC) | 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 |
Countries
Canada
Outcome results
None listed