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

Primary Purpose

Amyotrophic Lateral Sclerosis

Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
AT-1501
Sponsored by
Anelixis Therapeutics, LLC
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Amyotrophic Lateral Sclerosis focused on measuring ALS, Lou Gehrig's Disease, motor neuron disease, motor neuron degeneration, AT-1501, humanized blocking antibody to CD40LG, CD40L inhibitor, monoclonal antibody

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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 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

Sites / Locations

  • Barrows Neurological Institute
  • University of California Irvine
  • California Pacific Medical Center
  • Augusta University
  • University of Indiana
  • The University of Kansas Medical Center
  • Johns Hopkins University Medical Center
  • Massachusetts General Hospital
  • Hospital for Special Surgery (HSS)
  • Providence Brain & Spine Institute
  • Texas Neurology, P.A.
  • Houston Methodist Neurological Institute
  • Montreal Neurological Institute and Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

AT-1501

Arm Description

4 sequential dose cohorts

Outcomes

Primary Outcome Measures

Safety and Tolerability
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
Incidence of adverse events (AEs) reported as number of TEAEs (Treatment Emergent Adverse Events) having occurred.

Secondary Outcome Measures

Full Information

First Posted
March 20, 2020
Last Updated
June 6, 2023
Sponsor
Anelixis Therapeutics, LLC
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1. Study Identification

Unique Protocol Identification Number
NCT04322149
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
Study Type
Interventional

2. Study Status

Record Verification Date
June 2023
Overall Recruitment Status
Completed
Study Start Date
October 16, 2020 (Actual)
Primary Completion Date
March 24, 2022 (Actual)
Study Completion Date
March 24, 2022 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Anelixis Therapeutics, LLC

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Amyotrophic Lateral Sclerosis
Keywords
ALS, Lou Gehrig's Disease, motor neuron disease, motor neuron degeneration, AT-1501, humanized blocking antibody to CD40LG, CD40L inhibitor, monoclonal antibody

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Sequential Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
54 (Actual)

8. Arms, Groups, and Interventions

Arm Title
AT-1501
Arm Type
Experimental
Arm Description
4 sequential dose cohorts
Intervention Type
Drug
Intervention Name(s)
AT-1501
Intervention Description
AT-1501 monoclonal antibody targeting CD40L given as an IV infusion
Primary Outcome Measure Information:
Title
Safety and Tolerability
Description
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).
Time Frame
Up to 18 Weeks
Title
Safety and Tolerability
Description
Incidence of adverse events (AEs) reported as number of TEAEs (Treatment Emergent Adverse Events) having occurred.
Time Frame
Up to 18 Weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: ALS diagnosed as possible, laboratory supported probable, probable, or definite as defined by revised El Escorial criteria ALS Functional Rating Scale - Revised (ALSFRS-R) Aggregate score of 37 or greater No more than 24 months from diagnosis Exclusion Criteria: Any other central or peripheral nervous system disease that may interfere with the evaluation of ALS or its progression Presence of a tracheostomy, or use of permanent assistive ventilation (ventilatory support for 23 hours per day or more) History of malignancy within the previous 5 years, except for localized non-melanoma skin cancers 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. Recipient of Stem Cell or Gene Therapy Positive test for Hepatitis B surface antigen, Hepatitis C antibody, or HIV. History of deep venous thrombosis or pulmonary embolism History of active substance abuse within the past 2 years History of stroke, poorly controlled or significant cardiovascular disease, diabetes
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Jeff Bornstein, MD
Organizational Affiliation
Eledon Pharmaceuticals
Official's Role
Study Chair
Facility Information:
Facility Name
Barrows Neurological Institute
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85013
Country
United States
Facility Name
University of California Irvine
City
Orange
State/Province
California
ZIP/Postal Code
92868
Country
United States
Facility Name
California Pacific Medical Center
City
San Francisco
State/Province
California
ZIP/Postal Code
94109
Country
United States
Facility Name
Augusta University
City
Augusta
State/Province
Georgia
ZIP/Postal Code
30912
Country
United States
Facility Name
University of Indiana
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46202
Country
United States
Facility Name
The University of Kansas Medical Center
City
Kansas City
State/Province
Kansas
ZIP/Postal Code
66160
Country
United States
Facility Name
Johns Hopkins University Medical Center
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21287
Country
United States
Facility Name
Massachusetts General Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02114
Country
United States
Facility Name
Hospital for Special Surgery (HSS)
City
New York
State/Province
New York
ZIP/Postal Code
10021
Country
United States
Facility Name
Providence Brain & Spine Institute
City
Portland
State/Province
Oregon
ZIP/Postal Code
97213
Country
United States
Facility Name
Texas Neurology, P.A.
City
Dallas
State/Province
Texas
ZIP/Postal Code
75206
Country
United States
Facility Name
Houston Methodist Neurological Institute
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
Montreal Neurological Institute and Hospital
City
Montreal
ZIP/Postal Code
H3A 2B4
Country
Canada

12. IPD Sharing Statement

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

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