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

• ClinicalTrials.gov Identifier: NCT04322149
• Recruitment Status: Completed
• First Posted: March 26, 2020
• Results First Posted: June 28, 2023
• Last Update Posted: June 28, 2023
• Sponsor: Anelixis Therapeutics, LLC
• Information provided by (Responsible Party): Eledon Pharmaceuticals ( Anelixis Therapeutics, LLC )

Tracking Information

• First Submitted Date: March 20, 2020
• First Posted Date: March 26, 2020
• Results First Submitted Date: May 10, 2023
• Results First Posted Date: June 28, 2023
• Last Update Posted Date: June 28, 2023
• Actual Study Start Date: October 16, 2020
• Actual Primary Completion Date: March 24, 2022 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: June 6, 2023)

• Safety and Tolerability [ Time Frame: Up to 18 Weeks ]
  Incidence of adverse events (AEs) reported as number of participants with at least one TEAE (Treatment Emergent Adverse Event) and at least one SAE (Serious Adverse Event).
• Safety and Tolerability [ Time Frame: Up to 18 Weeks ]
  Incidence of adverse events (AEs) reported as number of TEAEs (Treatment Emergent Adverse Events) having occurred.

Original Primary Outcome Measures (submitted: March 24, 2020)

Safety and tolerability [ Time Frame: Approx. 5 Months ]

Incidence of adverse events (AEs) as characterized by, changes in physical examination, vital signs, ECG, laboratory parameters, and C-SSRS from baseline

• Current Secondary Outcome Measures: Not Provided
• Original Secondary Outcome Measures: Not Provided
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Multiple Doses of AT-1501-A201 in Adults With ALS
• Official Title: A Phase 2a Open-Label, Multi-Center Study to Evaluate the Safety and Tolerability of Multiple Doses of AT-1501 in Adults With ALS

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 Type: Interventional
• Study Phase: Phase 2
• Study Design: Allocation: N/A; Intervention Model: Sequential Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: Amyotrophic Lateral Sclerosis

Intervention

Drug: AT-1501

AT-1501 monoclonal antibody targeting CD40L given as an IV infusion

Study Arms

Experimental: AT-1501

4 sequential dose cohorts

Intervention: Drug: AT-1501

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: March 24, 2020): 54
• Original Estimated Enrollment: Same as current
• Actual Study Completion Date: March 24, 2022
• Actual Primary Completion Date: March 24, 2022 (Final data collection date for primary outcome measure)

Eligibility Criteria

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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Canada, United States

Administrative Information

• NCT Number: NCT04322149
• Other Study ID Numbers: AT-1501-A201
• Has Data Monitoring Committee: Yes

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

• IPD Sharing Statement: Not Provided
• Current Responsible Party: Eledon Pharmaceuticals ( Anelixis Therapeutics, LLC )
• Original Responsible Party: Anelixis Therapeutics, Inc.
• Current Study Sponsor: Anelixis Therapeutics, LLC
• Original Study Sponsor: Anelixis Therapeutics, Inc.
• Collaborators: Not Provided

Investigators

• Study Chair: Jeff Bornstein, MD; Eledon Pharmaceuticals

• PRS Account: Eledon Pharmaceuticals
• Verification Date: June 2023