A Phase 2a Open-Label, Multi-Center Study to Evaluate the Safety and Tolerability of Multiple Doses of AT-1501 in Adults With ALS

Full Study Summary:

This is a Phase 2a, multi-center, open label, multiple dose study of AT-1501, a humanized monoclonal antibody antagonist to CD40LG. Approximately 54 adults with ALS will be enrolled into the study in the United States at up to 12 ALS treatment sites.

Three ascending doses of AT-1501 will be administered as an IV infusion to sequentially enrolling cohorts. Each participant will receive 6 bi-weekly (every other week) infusions of AT-1501 over an 11-week period.

The study is estimated to take 19 weeks for participants.

• Eligibility Criteria

  Gender:

  Female, Male

  Minimum Age:

  18

  Maximum Age:

  N/A

  Time since Symptom Onset:

  Time since Diagnosis:

  Can participants use Riluzole?

  Yes

  
  

  Inclusion Criteria:

  1. ALS diagnosed as possible, laboratory supported probable, probable, or definite as defined by revised El Escorial criteria.
  2. FVC (Forced Vital Capacity) equal to or greater than 60%.
  3. No more than 24 months from diagnosis.
  4. Able and willing to give informed consent, follow trial procedures, and make multiple clinical site visits.

  Exclusion Criteria:

  1. Any other central or peripheral nervous system disease that may interfere with the evaluation of ALS or its progression.
  2. Presence of a tracheostomy, or use of permanent assistive ventilation (ventilatory support for 23 hours per day or more).
  3. History of malignancy within the previous 5 years, except for localized non-melanoma skin cancers.
  4. Abnormal function of the immune system resulting from: Clinical conditions affecting the immune system (e.g. HIV infection, agammaglobulinemia), Systemic administration of corticosteroids (PO/IV/IM) at a dose equivalent to 20 mg/day of prednisone for more than 14 consecutive days within 90 days prior to screening, Administration of anti-neoplastic and/or immunomodulating agents (e.g. TNF α antagonists or anti-B cell antibodies) or radiotherapy within 1 year prior to screening.
  5. Recipient of Stem Cell or Gene Therapy.
  6. Positive test for Hepatitis B surface antigen, Hepatitis C antibody, or HIV.
  7. History of deep venous thrombosis or pulmonary embolism.
  8. History of active substance abuse within the past 2 years;
  9. History of stroke, poorly controlled or significant cardiovascular disease, diabetes.

• Site Contact Information

  California Pacific Medical Center
  Research Staff / .(JavaScript must be enabled to view this email address) / 415-600-3758
  San Francisco, California 94109
  United States

  Augusta University
  Research Staff / .(JavaScript must be enabled to view this email address) / 706-721-2681
  Augusta, Georgia 30912
  United States

  University of Indiana
  Research Staff / .(JavaScript must be enabled to view this email address) / 317-963-7382
  Indianapolis, Indiana 46202
  United States

  Johns Hopkins University Medical Center
  Research Staff / .(JavaScript must be enabled to view this email address) / 410-955-8511
  Baltimore, Maryland 21287
  United States