Single ascending doses of AP-101 will be administered by intravenous (IV) infusion
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- Have previously completed or withdrawn from this study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