Amyotrophic Lateral SclerosisPhase 2NCT04322149

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

A Phase 2 study testing AT-1501 in people with Amyotrophic Lateral Sclerosis, run by Anelixis Therapeutics, LLC at 13 sites in 2 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial 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. Read more about this study

Who is it for?: Ages 18 years and older
Conditions the study is for: Amyotrophic Lateral Sclerosis
What is being tested?: AT-1501
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 54 participants
Where: 13 sitesin 2 countries
Study dates: 16 Oct 2020to 24 Mar 2022
Phase: Phase 2
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 18 years and older
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

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

Who cannot join

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,

Show all 12Show fewer

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

Trial locations and contacts

13 sites in 2 countries.

Country

City, postcode or hospital

13 sites in 2 countries

United States12 sites

Augusta University
Augusta, Georgia, 30912
Johns Hopkins University Medical Center
Baltimore, Maryland, 21287
Massachusetts General Hospital
Boston, Massachusetts, 02114
Texas Neurology, P.A.
Dallas, Texas, 75206
Houston Methodist Neurological Institute
Houston, Texas, 77030
University of Indiana
Indianapolis, Indiana, 46202
The University of Kansas Medical Center
Kansas City, Kansas, 66160
Hospital for Special Surgery (HSS)
New York, New York, 10021
University of California Irvine
Orange, California, 92868
Barrows Neurological Institute
Phoenix, Arizona, 85013
Providence Brain & Spine Institute
Portland, Oregon, 97213
California Pacific Medical Center
San Francisco, California, 94109

Other trials in United States

Canada1 site

Montreal Neurological Institute and Hospital
Montreal, H3A 2B4

Other trials in Canada

About this study

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

Participants will be enrolled into one of four ascending doses.

Read the 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.

Conditions: Amyotrophic Lateral Sclerosis
Being tested: AT-1501
Keywords: ALS, Lou Gehrig's Disease, motor neuron disease, motor neuron degeneration, AT-1501, humanized blocking antibody to CD40LG, CD40L inhibitor, monoclonal antibody
Medical subject headings: Amyotrophic Lateral Sclerosis, Motor Neuron Disease

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

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

Study groups in NCT04322149
Study group	Role	What they receive
AT-1501	Experimental	4 sequential dose cohorts Receives: Drug: AT-1501

What is being measured

The primary outcome is the main question the study is designed to answer.

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

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

16 Oct 202024 Mar 2022

First posted26 Mar 2020
Study start16 Oct 2020
Primary completion24 Mar 2022
Study completion24 Mar 2022

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (1)Papers the sponsor has linked to this study.
Other trials for Amyotrophic Lateral SclerosisStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 2
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Sequential
Masking: None (open label)
Enrolment: 54 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT04322149
Sponsor's protocol ID: AT-1501-A201
Status last verified: Jun 2023
First submitted: 20 Mar 2020
Last updated: 28 Jun 2023
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No

Publications

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.
PubMed 39480764

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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Safety, Tolerability, and Pharmacodynamic Exploration of Orelabrutinib in Patients With Amyotrophic Lateral Sclerosis (ALS)
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All Amyotrophic Lateral Sclerosis trials

Study at a glance

From the registry record, last updated by the sponsor on 28 Jun 2023. Recruitment status is as the sponsor reported it: confirm with the study team.

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
ClinicalTrials.gov ID: NCT04322149
Acronym / sponsor ID: AT-1501-A201
Conditions: 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
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 54 participants (actual)
Lead sponsor: Anelixis Therapeutics, LLCIndustry (pharma, biotech, devices)
Study start: 16 Oct 2020 (actual)
Primary completion: 24 Mar 2022 (actual)
Study completion: 24 Mar 2022 (actual)
First posted: 26 Mar 2020 (actual)
Results: Posted 28 Jun 2023

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Participants will be enrolled into one of four ascending doses.

Detailed description

The study is estimated to take 19 weeks for participants.

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Sequential
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
AT-1501	Experimental	Drug: AT-15014 sequential dose cohorts

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (3 listed)

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

Key exclusion criteria (12 listed)

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:
Recipient of Stem Cell or Gene Therapy

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
AT-1501AT-1501 monoclonal antibody targeting CD40L given as an IV infusion	Drug	AT-1501

Primary outcome

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.

Results and safety

Results posted to the registry on 28 Jun 2023, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
AT-1501 1.0 mg/kg	9	8
AT-1501 2.0 mg/kg	9	7
AT-1501 4.0 mg/kg	18	18
AT-1501 8.0 mg/kg	18	16

Primary outcome: Safety and Tolerability

Time frame: Up to 18 Weeks. Number. Unit: participants

Measure	AT-1501 1.0 mg/kg n = 9	AT-1501 2.0 mg/kg n = 9	AT-1501 4.0 mg/kg n = 18	AT-1501 8.0 mg/kg n = 18
Number of Subjects with at Least One TEAE	8	8	10	16
Number of Subjects with at Least One SAE	0	1	0	0

Population analysed: Full analysis set. All enrolled patients.

Primary outcome: Safety and Tolerability

Time frame: Up to 18 Weeks. Number. Unit: TEAEs

Measure	AT-1501 1.0 mg/kg n = 9	AT-1501 2.0 mg/kg n = 9	AT-1501 4.0 mg/kg n = 18	AT-1501 8.0 mg/kg n = 18
Value	30	66	54	67

Population analysed: Full analysis set. All enrolled patients.

Adverse events

Time frame: All serious adverse events were collected from the time of informed consent to 4 weeks after a patient's last dose of investigational product. All adverse events were collected from the first administration of investigational product until completion of the final visit, up to 15 weeks.. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
AT-1501 1.0 mg/kg	0 of 9 (0%)	8 of 9 (88.9%)	0 of 9
AT-1501 2.0 mg/kg	1 of 9 (11.1%)	8 of 9 (88.9%)	0 of 9
AT-1501 4.0 mg/kg	0 of 18 (0%)	10 of 18 (55.6%)	0 of 18
AT-1501 8.0 mg/kg	0 of 18 (0%)	16 of 18 (88.9%)	0 of 18

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 28 Jun 2023.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 39480764Perrin 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.

Related trials for Amyotrophic Lateral Sclerosis

RecruitingPhase 1/2
Study is to Assess the Safety and Tolerability of VTx-002 in Participants With ALS
Amyotrophic Lateral Sclerosis
Not yet recruiting
A Study on the Addition of Alsum30 for the Treatment of Amyotrophic Lateral Sclerosis
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Active, not recruiting
Functional Outcomes and Control Using Synchron BCI - Australia
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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Jeff Bornstein, MD study chair

Eledon Pharmaceuticals

13 sites in 2 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing AT-1501 in people with Amyotrophic Lateral Sclerosis, run by Anelixis Therapeutics, LLC at 13 sites in 2 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 3 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 28 Jun 2023. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 13 sites in United States, Canada. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 28 Jun 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Safety and Tolerability (time frame: Up to 18 Weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04322149, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 Jun 2023.