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

This is an interventional basic science trial for Amyotrophic Lateral Sclerosis focused on measuring Familial Amyotrophic Lateral Sclerosis, Sporadic Amyotrophic Lateral Sclerosis Read More

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• Have previously completed or withdrawn from this study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