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

NCT ID: NCT04322149

Last Updated: 2023-06-28

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

54 participants

Study Classification

INTERVENTIONAL

Study Start Date

2020-10-16

Study Completion Date

2022-03-24

Brief Summary

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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

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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.

Conditions

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Amyotrophic Lateral Sclerosis

Study Design

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Allocation Method

NA

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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AT-1501

4 sequential dose cohorts

Group Type EXPERIMENTAL

AT-1501

Intervention Type DRUG

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

Interventions

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AT-1501

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

Intervention Type DRUG

Eligibility Criteria

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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
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Anelixis Therapeutics, LLC

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Jeff Bornstein, MD

Role: STUDY_CHAIR

Eledon Pharmaceuticals

Locations

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Barrows Neurological Institute

Phoenix, Arizona, United States

Site Status

University of California Irvine

Orange, California, United States

Site Status

California Pacific Medical Center

San Francisco, California, United States

Site Status

Augusta University

Augusta, Georgia, United States

Site Status

University of Indiana

Indianapolis, Indiana, United States

Site Status

The University of Kansas Medical Center

Kansas City, Kansas, United States

Site Status

Johns Hopkins University Medical Center

Baltimore, Maryland, United States

Site Status

Massachusetts General Hospital

Boston, Massachusetts, United States

Site Status

Hospital for Special Surgery (HSS)

New York, New York, United States

Site Status

Providence Brain & Spine Institute

Portland, Oregon, United States

Site Status

Texas Neurology, P.A.

Dallas, Texas, United States

Site Status

Houston Methodist Neurological Institute

Houston, Texas, United States

Site Status

Montreal Neurological Institute and Hospital

Montreal, , Canada

Site Status

Countries

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United States Canada

References

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Perrin S, Ladha S, Maragakis N, Rivner MH, Katz J, Genge A, Olney N, Lange D, Heitzman D, Bodkin C, Jawdat O, Goyal NA, Bornstein JD, Mak C, Appel SH, Paganoni S. Safety and tolerability of tegoprubart in patients with amyotrophic lateral sclerosis: A Phase 2A clinical trial. PLoS Med. 2024 Oct 31;21(10):e1004469. doi: 10.1371/journal.pmed.1004469. eCollection 2024 Oct.

Reference Type DERIVED
PMID: 39480764 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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AT-1501-A201

Identifier Type: -

Identifier Source: org_study_id

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