Multiple Doses of AT-1501-A201 in Adults With ALS

Phase 2

Completed

• Conditions: Amyotrophic Lateral Sclerosis
• Interventions: Drug: AT-1501

• Registration Number: NCT04322149
• Lead Sponsor: Anelixis Therapeutics, LLC

Brief Summary

This is a Phase 2a, multi-center, open label, multiple dose study of AT-1501, a humanized monoclonal antibody antagonist to CD40 ligand (CD40L). Approximately 54 adults with Amyotrophic Lateral Sclerosis (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.

Detailed Description

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 Timeline

• Study First Submitted: 2020-03-20
• Study First Submitted QC: 2020-03-24
• Study First Posted: 2020-03-26
• Study Start: 2020-10-16
• Primary Completion: 2022-03-24
• Study Completion: 2022-03-24
• Results First Submitted: 2023-05-10
• Status Verified: 2023-06
• Results First Submitted QC: 2023-06-06
• Last Update Submitted: 2023-06-06
• Results First Posted: 2023-06-28
• Last Update Posted: 2023-06-28

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 54

Inclusion Criteria

1. ALS diagnosed as possible, laboratory supported probable, probable, or definite as defined by revised El Escorial criteria
2. ALS Functional Rating Scale - Revised (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. Tumor necrosis factor alpha (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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
AT-1501	AT-1501	4 sequential dose cohorts

Primary Outcome Measures

Name	Time	Method
Safety and Tolerability	Up to 18 Weeks	Incidence of adverse events (AEs) reported as number of TEAEs (Treatment Emergent Adverse Events) having occurred.

Trial Locations

Locations (13)

Augusta University; 🇺🇸 Augusta, Georgia, United States

Texas Neurology, P.A.; 🇺🇸 Dallas, Texas, United States

Barrows Neurological Institute; 🇺🇸 Phoenix, Arizona, United States

California Pacific Medical Center; 🇺🇸 San Francisco, California, United States

University of California Irvine; 🇺🇸 Orange, California, United States

University of Indiana; 🇺🇸 Indianapolis, Indiana, United States

The University of Kansas Medical Center; 🇺🇸 Kansas City, Kansas, United States

Johns Hopkins University Medical Center; 🇺🇸 Baltimore, Maryland, United States

Hospital for Special Surgery (HSS); 🇺🇸 New York, New York, United States

Massachusetts General Hospital; 🇺🇸 Boston, Massachusetts, United States

Providence Brain & Spine Institute; 🇺🇸 Portland, Oregon, United States

Houston Methodist Neurological Institute; 🇺🇸 Houston, Texas, United States

Montreal Neurological Institute and Hospital; 🇨🇦 Montreal, Canada