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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| AT-1501 | Experimental | 4 sequential dose cohorts |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| AT-1501 | Drug | AT-1501 monoclonal antibody targeting CD40L given as an IV infusion |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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). | Up to 18 Weeks |
| Safety and Tolerability | Incidence of adverse events (AEs) reported as number of TEAEs (Treatment Emergent Adverse Events) having occurred. | Up to 18 Weeks |
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Inclusion Criteria:
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:
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
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| Name | Affiliation | Role |
|---|---|---|
| Jeff Bornstein, MD | Eledon Pharmaceuticals | Study Chair |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Barrows Neurological Institute | Phoenix | Arizona | 85013 | United States | ||
| University of California Irvine |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 39480764 | Derived | 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. |
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| ID | Title | Description |
|---|---|---|
| FG000 | AT-1501 1.0 mg/kg | This group received a 1.0 mg/kg dose administered as an 1-hour IV infusion. Each participant received 6 bi-weekly (every other week) infusions of AT-1501 over an 11-week period. |
| FG001 | AT-1501 2.0 mg/kg | This group received a 2.0 mg/kg dose administered as an 1-hour IV infusion. Each participant received 6 bi-weekly (every other week) infusions of AT-1501 over an 11-week period. |
| FG002 | AT-1501 4.0 mg/kg | This group received a 4.0 mg/kg dose administered as an 1-hour IV infusion. Each participant received 6 bi-weekly (every other week) infusions of AT-1501 over an 11-week period. |
| FG003 | AT-1501 8.0 mg/kg | This group received a 8.0 mg/kg dose administered as an 1-hour IV infusion. Each participant received 6 bi-weekly (every other week) infusions of AT-1501 over an 11-week period. |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | AT-1501 1.0 mg/kg | This group received a 1.0 mg/kg dose administered as an 1-hour IV infusion. Each participant received 6 bi-weekly (every other week) infusions of AT-1501 over an 11-week period. |
| BG001 | AT-1501 2.0 mg/kg |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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). | Full analysis set. All enrolled patients. | Posted | Number | participants | Up to 18 Weeks |
|
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.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | AT-1501 1.0 mg/kg | This group received a 1.0 mg/kg dose administered as an 1-hour IV infusion. Each participant received 6 bi-weekly (every other week) infusions of AT-1501 over an 11-week period. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Tonsil cancer | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Headache | Nervous system disorders | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Laura Batterman | Eledon Pharmaceuticals | 949-874-3487 | lbatterman@eledon.com |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Mar 8, 2021 | May 10, 2023 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Sep 30, 2021 | May 10, 2023 | SAP_001.pdf |
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| ID | Term |
|---|---|
| D000690 | Amyotrophic Lateral Sclerosis |
| D016472 | Motor Neuron Disease |
| ID | Term |
|---|---|
| D013118 | Spinal Cord Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D019636 | Neurodegenerative Diseases |
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| Orange |
| California |
| 92868 |
| United States |
| California Pacific Medical Center | San Francisco | California | 94109 | United States |
| Augusta University | Augusta | Georgia | 30912 | United States |
| University of Indiana | Indianapolis | Indiana | 46202 | United States |
| The University of Kansas Medical Center | Kansas City | Kansas | 66160 | United States |
| Johns Hopkins University Medical Center | Baltimore | Maryland | 21287 | United States |
| Massachusetts General Hospital | Boston | Massachusetts | 02114 | United States |
| Hospital for Special Surgery (HSS) | New York | New York | 10021 | United States |
| Providence Brain & Spine Institute | Portland | Oregon | 97213 | United States |
| Texas Neurology, P.A. | Dallas | Texas | 75206 | United States |
| Houston Methodist Neurological Institute | Houston | Texas | 77030 | United States |
| Montreal Neurological Institute and Hospital | Montreal | H3A 2B4 | Canada |
| Withdrawal by Subject |
|
| Undergoing cancer treatment & was unable to return to site to complete EOS visit within window |
|
This group received a 2.0 mg/kg dose administered as an 1-hour IV infusion. Each participant received 6 bi-weekly (every other week) infusions of AT-1501 over an 11-week period.
| BG002 | AT-1501 4.0 mg/kg | This group received a 4.0 mg/kg dose administered as an 1-hour IV infusion. Each participant received 6 bi-weekly (every other week) infusions of AT-1501 over an 11-week period. |
| BG003 | AT-1501 8.0 mg/kg | This group received a 8.0 mg/kg dose administered as an 1-hour IV infusion. Each participant received 6 bi-weekly (every other week) infusions of AT-1501 over an 11-week period. |
| BG004 | Total | Total of all reporting groups |
| Participants |
|
| Age, Continuous | Mean | Standard Deviation | years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| ALSFRS-R Total Score at Screening | The ALS Functional Rating Scale - Revised (ALSFRS-R) measures 12 aspects of physical function, ranging from one's ability to swallow and use utensils to climbing stairs and breathing. Each function is scored from 4 (normal) to 0 (no ability), with a maximum total score of 48 and a minimum total score of 0. Function scores are summed to compute the total score. | Mean | Standard Deviation | units on a scale |
|
| ALSFRS-R Subdomain at Screening | Measure Description: The ALS Functional Rating Scale - Revised (ALSFRS-R) measures 12 aspects of physical function, ranging from one's ability to swallow and use utensils to climbing stairs and breathing. The functions reported individually for this study are dyspnea, orthopnea, and respiratory insufficiency. The functions are scored from 4 (normal) to 0 (no ability). | Mean | Standard Deviation | units on a scale |
|
| ALSFRS-R Total Score at Baseline | The ALS Functional Rating Scale - Revised (ALSFRS-R) measures 12 aspects of physical function, ranging from one's ability to swallow and use utensils to climbing stairs and breathing. Each function is scored from 4 (normal) to 0 (no ability), with a maximum total score of 48 and a minimum total score of 0. Function scores are summed to compute the total score. | Mean | Standard Deviation | units on a scale |
|
| ALSFRS-R Subdomain at Baseline | Measure Description: The ALS Functional Rating Scale - Revised (ALSFRS-R) measures 12 aspects of physical function, ranging from one's ability to swallow and use utensils to climbing stairs and breathing. The functions reported individually for this study are dyspnea, orthopnea, and respiratory insufficiency. The functions are scored from 4 (normal) to 0 (no ability). | Mean | Standard Deviation | units on a scale |
|
| Time Since ALS Symptom Onset | Mean | Standard Deviation | days |
|
| Time Since ALS Diagnosis | Mean | Standard Deviation | days |
|
| OG002 | AT-1501 4.0 mg/kg | This group received a 4.0 mg/kg dose administered as an 1-hour IV infusion. Each participant received 6 bi-weekly (every other week) infusions of AT-1501 over an 11-week period. |
| OG003 | AT-1501 8.0 mg/kg | This group received a 8.0 mg/kg dose administered as an 1-hour IV infusion. Each participant received 6 bi-weekly (every other week) infusions of AT-1501 over an 11-week period. |
|
|
| Primary | Safety and Tolerability | Incidence of adverse events (AEs) reported as number of TEAEs (Treatment Emergent Adverse Events) having occurred. | Full analysis set. All enrolled patients. | Posted | Number | TEAEs | Up to 18 Weeks |
|
|
|
| 0 |
| 9 |
| 0 |
| 9 |
| 8 |
| 9 |
| EG001 | AT-1501 2.0 mg/kg | This group received a 2.0 mg/kg dose administered as an 1-hour IV infusion. Each participant received 6 bi-weekly (every other week) infusions of AT-1501 over an 11-week period. | 0 | 9 | 1 | 9 | 8 | 9 |
| EG002 | AT-1501 4.0 mg/kg | This group received a 4.0 mg/kg dose administered as an 1-hour IV infusion. Each participant received 6 bi-weekly (every other week) infusions of AT-1501 over an 11-week period. | 0 | 18 | 0 | 18 | 10 | 18 |
| EG003 | AT-1501 8.0 mg/kg | This group received a 8.0 mg/kg dose administered as an 1-hour IV infusion. Each participant received 6 bi-weekly (every other week) infusions of AT-1501 over an 11-week period. | 0 | 18 | 0 | 18 | 16 | 18 |
| Dizziness | Nervous system disorders | Systematic Assessment |
|
| Hypoaesthesia | Nervous system disorders | Systematic Assessment |
|
| Somnolence | Nervous system disorders | Systematic Assessment |
|
| Aphasia | Nervous system disorders | Systematic Assessment |
|
| Migraine | Nervous system disorders | Systematic Assessment |
|
| Muscle spasticity | Nervous system disorders | Systematic Assessment |
|
| Neuropathy peripheral | Nervous system disorders | Systematic Assessment |
|
| Paraesthesia | Nervous system disorders | Systematic Assessment |
|
| Speech disorder | Nervous system disorders | Systematic Assessment |
|
| Fatigue | General disorders | Systematic Assessment |
|
| Asthenia | General disorders | Systematic Assessment |
|
| Peripheral swelling | General disorders | Systematic Assessment |
|
| Feeling jittery | General disorders | Systematic Assessment |
|
| Gait disturbance | General disorders | Systematic Assessment |
|
| Malaise | General disorders | Systematic Assessment |
|
| Non-cardiac chest pain | General disorders | Systematic Assessment |
|
| Pain | General disorders | Systematic Assessment |
|
| Swelling | General disorders | Systematic Assessment |
|
| Fall | Injury, poisoning and procedural complications | Systematic Assessment |
|
| Contusion | Injury, poisoning and procedural complications | Systematic Assessment |
|
| Eye contusion | Injury, poisoning and procedural complications | Systematic Assessment |
|
| Hand fracture | Injury, poisoning and procedural complications | Systematic Assessment |
|
| Skin abrasion | Injury, poisoning and procedural complications | Systematic Assessment |
|
| Skin laceration | Injury, poisoning and procedural complications | Systematic Assessment |
|
| Extra-axial haemorrhage | Injury, poisoning and procedural complications | Systematic Assessment |
|
| Foot fracture | Injury, poisoning and procedural complications | Systematic Assessment |
|
| Post lumbar puncture syndrome | Injury, poisoning and procedural complications | Systematic Assessment |
|
| Post-traumatic pain | Injury, poisoning and procedural complications | Systematic Assessment |
|
| Scratch | Injury, poisoning and procedural complications | Systematic Assessment |
|
| Superficial injury of eye | Injury, poisoning and procedural complications | Systematic Assessment |
|
| Muscle spasms | Musculoskeletal and connective tissue disorders | Systematic Assessment |
|
| Arthralgia | Musculoskeletal and connective tissue disorders | Systematic Assessment |
|
| Muscular weakness | Musculoskeletal and connective tissue disorders | Systematic Assessment |
|
| Musculoskeletal stiffness | Musculoskeletal and connective tissue disorders | Systematic Assessment |
|
| Neck pain | Musculoskeletal and connective tissue disorders | Systematic Assessment |
|
| Joint range of motion decreased | Musculoskeletal and connective tissue disorders | Systematic Assessment |
|
| Joint swelling | Musculoskeletal and connective tissue disorders | Systematic Assessment |
|
| Muscle twitching | Musculoskeletal and connective tissue disorders | Systematic Assessment |
|
| Soft tissue swelling | Musculoskeletal and connective tissue disorders | Systematic Assessment |
|
| Tendonitis | Musculoskeletal and connective tissue disorders | Systematic Assessment |
|
| Nausea | Gastrointestinal disorders | Systematic Assessment |
|
| Abdominal discomfort | Gastrointestinal disorders | Systematic Assessment |
|
| Constipation | Gastrointestinal disorders | Systematic Assessment |
|
| Dysphagia | Gastrointestinal disorders | Systematic Assessment |
|
| Vomiting | Gastrointestinal disorders | Systematic Assessment |
|
| Abdominal pain upper | Gastrointestinal disorders | Systematic Assessment |
|
| Diarrhoea | Gastrointestinal disorders | Systematic Assessment |
|
| Lip swelling | Gastrointestinal disorders | Systematic Assessment |
|
| Salivary hypersecretion | Gastrointestinal disorders | Systematic Assessment |
|
| Depression | Psychiatric disorders | Systematic Assessment |
|
| Affect lability | Psychiatric disorders | Systematic Assessment |
|
| Anxiety | Psychiatric disorders | Systematic Assessment |
|
| Confusional state | Psychiatric disorders | Systematic Assessment |
|
| Disorientation | Psychiatric disorders | Systematic Assessment |
|
| Mental status changes | Psychiatric disorders | Systematic Assessment |
|
| Middle insomnia | Psychiatric disorders | Systematic Assessment |
|
| Suicidal ideation | Psychiatric disorders | Systematic Assessment |
|
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
|
| Cough | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
|
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
|
| Dysphonia | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
|
| Epistaxis | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
|
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
|
| Orthopnoea | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
|
| Productive cough | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
|
| Respiratory tract congestion | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
|
| Rhinitis allergic | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
|
| Throat clearing | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
|
| Nasopharyngitis | Infections and infestations | Systematic Assessment |
|
| Candida infection | Infections and infestations | Systematic Assessment |
|
| Cystitis | Infections and infestations | Systematic Assessment |
|
| Fungal infection | Infections and infestations | Systematic Assessment |
|
| Herpes zoster | Infections and infestations | Systematic Assessment |
|
| Sinusitis | Infections and infestations | Systematic Assessment |
|
| Upper respiratory tract infection | Infections and infestations | Systematic Assessment |
|
| Urinary tract infection | Infections and infestations | Systematic Assessment |
|
| Actinic keratosis | Skin and subcutaneous tissue disorders | Systematic Assessment |
|
| Dermatitis contact | Skin and subcutaneous tissue disorders | Systematic Assessment |
|
| Hyperhidrosis | Skin and subcutaneous tissue disorders | Systematic Assessment |
|
| Pruritus | Skin and subcutaneous tissue disorders | Systematic Assessment |
|
| Rash | Skin and subcutaneous tissue disorders | Systematic Assessment |
|
| Blood pressure increased | Investigations | Systematic Assessment |
|
| Cardiac murmur | Investigations | Systematic Assessment |
|
| Weight decreased | Investigations | Systematic Assessment |
|
| Micturition urgency | Renal and urinary disorders | Systematic Assessment |
|
| Pollakiuria | Renal and urinary disorders | Systematic Assessment |
|
| Urinary incontinence | Renal and urinary disorders | Systematic Assessment |
|
| Atrial fibrillation | Cardiac disorders | Systematic Assessment |
|
| Nodal rhythm | Cardiac disorders | Systematic Assessment |
|
| Eye swelling | Eye disorders | Systematic Assessment |
|
| Periorbital oedema | Eye disorders | Systematic Assessment |
|
| Decreased appetite | Metabolism and nutrition disorders | Systematic Assessment |
|
| Hyponatraemia | Metabolism and nutrition disorders | Systematic Assessment |
|
| Malignant melanoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | Systematic Assessment |
|
| Prostatitis | Reproductive system and breast disorders | Systematic Assessment |
|
| Vulvovaginal pruritus | Reproductive system and breast disorders | Systematic Assessment |
|
| Flushing | Vascular disorders | Systematic Assessment |
|
| Hypertension | Vascular disorders | Systematic Assessment |
|
| Tinnitus | Ear and labyrinth disorders | Systematic Assessment |
|
| Seasonal allergy | Immune system disorders | Systematic Assessment |
|
PI will not submit any results of the Study until after the first to occur: publication of the trial results, notification by Sponsor the trial submission is no longer planned, or the 18 month anniversary of the completion or early termination of the trial. A copy of any proposed publication or disclosure of the results of the Study will be given to Sponsor for review at least 30 days prior to the date of submission for publication (including abstracts) or of public disclosure
| D057177 | TDP-43 Proteinopathies |
| D009468 | Neuromuscular Diseases |
| D057165 | Proteostasis Deficiencies |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |