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

Study Purpose:

This is a Phase 2a, multi-center, open label, multiple dose study of AT-1501, a humanized monoclonal antibody antagonist to CD40L. Approximately 54 adults with ALS will be enrolled into the study in the United States and Canada at approximately 13 ALS treatment sites.

Participants will be enrolled into one of four ascending doses.

Study Status:

Not recruiting

Disease:

Amyotrophic Lateral Sclerosis

Study Type:

Interventional

Type of Intervention:

Drug

Intervention Name:

AT-1501

Placebo:

No

Phase:

Phase 2

Study Chair(s)/Principal Investigator(s):

Jeff Bornstein, MD, Eledon Pharmaceuticals

Clinicaltrials.gov ID:

NCT04322149

Neals Affiliated?

No

Coordinating Center Contact Information

Full Study Summary:

This is a Phase 2a, multi-center, open label, multiple dose study of AT-1501, a humanized monoclonal antibody antagonist to CD40L. Approximately 54 adults with ALS will be enrolled into the study in the United States and Canada at approximately 13 ALS treatment sites.

Four 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.

Study Sponsor:

Anelixis Therapeutics, LLC

Participant Duration:

19 weeks

Estimated Enrollment:

54

Estimated Study Start Date:

10 / 16 / 2020

Estimated Study Completion Date:

03 / 24 / 2022

Posting Last Modified Date:

07 / 14 / 2022

Date Study Added to neals.org:

03 / 26 / 2020

Minimum Age:

18 Years

Maximum Age:

N/A

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. ALSFRS-R Aggregate score of 37 or greater

3. No more than 24 months from diagnosis

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 ╬▒ 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

Barrows Neurological Institute

Phoenix, Arizona 85013
United States

University of California Irvine

Orange, California 92868
United States

California Pacific Medical Center

San Francisco, California 94109
United States

Augusta University

Augusta, Georgia 30912
United States

University of Indiana

Indianapolis, Indiana 46202
United States

The University of Kansas Medical Center

Kansas City, Kansas 66160
United States

Johns Hopkins University Medical Center

Baltimore, Maryland 21287
United States

Massachusetts General Hospital

Boston, Massachusetts 02114
United States

Hospital for Special Surgery (HSS)

New York, New York 10021
United States

Providence Brain & Spine Institute

Portland, Oregon 97213
United States

Texas Neurology, P.A.

Dallas, Texas 75206
United States

Houston Methodist Neurological Institute

Houston, Texas 77030
United States

Montreal Neurological Institute and Hospital

Montreal H3A 2B4
Canada