Trial Outcomes & Findings for Multiple Doses of AT-1501-A201 in Adults With ALS (NCT NCT04322149)

Last Updated: 2023-06-28

Results Overview

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).

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

54 participants

Primary outcome timeframe

Up to 18 Weeks

Results posted on

2023-06-28

Participant Flow

Participant milestones

Participant milestones
Measure	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.	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.	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.	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.
Overall Study STARTED	9	9	18	18
Overall Study COMPLETED	8	7	18	16
Overall Study NOT COMPLETED	1	2	0	2

Reasons for withdrawal

Reasons for withdrawal
Measure	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.	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.	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.
Overall Study Adverse Event	1	1	0
Overall Study Withdrawal by Subject	0	0	2
Overall Study Undergoing cancer treatment & was unable to return to site to complete EOS visit within window	0	1	0

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	AT-1501 1.0 mg/kg n=9 Participants 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.	AT-1501 2.0 mg/kg n=9 Participants 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.	AT-1501 4.0 mg/kg n=18 Participants 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.	AT-1501 8.0 mg/kg n=18 Participants 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.	Total n=54 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants
Age, Categorical Between 18 and 65 years	7 Participants n=5 Participants	6 Participants n=7 Participants	15 Participants n=5 Participants	11 Participants n=4 Participants	39 Participants n=21 Participants
Age, Categorical >=65 years	2 Participants n=5 Participants	3 Participants n=7 Participants	3 Participants n=5 Participants	7 Participants n=4 Participants	15 Participants n=21 Participants
Age, Continuous	61.2 years STANDARD_DEVIATION 6.5 • n=5 Participants	59 years STANDARD_DEVIATION 14.76 • n=7 Participants	56.4 years STANDARD_DEVIATION 8.53 • n=5 Participants	60.3 years STANDARD_DEVIATION 11.11 • n=4 Participants	59 years STANDARD_DEVIATION 10.29 • n=21 Participants
Sex: Female, Male Female	3 Participants n=5 Participants	2 Participants n=7 Participants	6 Participants n=5 Participants	8 Participants n=4 Participants	19 Participants n=21 Participants
Sex: Female, Male Male	6 Participants n=5 Participants	7 Participants n=7 Participants	12 Participants n=5 Participants	10 Participants n=4 Participants	35 Participants n=21 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	3 Participants n=4 Participants	3 Participants n=21 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	9 Participants n=5 Participants	9 Participants n=7 Participants	18 Participants n=5 Participants	15 Participants n=4 Participants	51 Participants n=21 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants	0 Participants n=4 Participants	2 Participants n=21 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants
Race (NIH/OMB) White	9 Participants n=5 Participants	8 Participants n=7 Participants	17 Participants n=5 Participants	18 Participants n=4 Participants	52 Participants n=21 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants
ALSFRS-R Total Score at Screening	41.1 units on a scale STANDARD_DEVIATION 3.02 • n=5 Participants	40.2 units on a scale STANDARD_DEVIATION 1.92 • n=7 Participants	39.7 units on a scale STANDARD_DEVIATION 3.69 • n=5 Participants	39.2 units on a scale STANDARD_DEVIATION 2.65 • n=4 Participants	39.9 units on a scale STANDARD_DEVIATION 3.0 • n=21 Participants
ALSFRS-R Subdomain at Screening Dyspnea	4 units on a scale STANDARD_DEVIATION 0 • n=5 Participants	3.8 units on a scale STANDARD_DEVIATION 0.44 • n=7 Participants	3.7 units on a scale STANDARD_DEVIATION 0.59 • n=5 Participants	3.8 units on a scale STANDARD_DEVIATION 0.43 • n=4 Participants	3.8 units on a scale STANDARD_DEVIATION 0.46 • n=21 Participants
ALSFRS-R Subdomain at Screening Orthopnea	4.0 units on a scale STANDARD_DEVIATION 0 • n=5 Participants	3.8 units on a scale STANDARD_DEVIATION 0.67 • n=7 Participants	3.9 units on a scale STANDARD_DEVIATION 0.32 • n=5 Participants	3.9 units on a scale STANDARD_DEVIATION 0.24 • n=4 Participants	3.9 units on a scale STANDARD_DEVIATION 0.35 • n=21 Participants
ALSFRS-R Subdomain at Screening Respiratory Insufficiency	4.0 units on a scale STANDARD_DEVIATION 0 • n=5 Participants	4.0 units on a scale STANDARD_DEVIATION 0 • n=7 Participants	3.9 units on a scale STANDARD_DEVIATION 0.47 • n=5 Participants	3.7 units on a scale STANDARD_DEVIATION 0.57 • n=4 Participants	3.9 units on a scale STANDARD_DEVIATION 0.44 • n=21 Participants
ALSFRS-R Total Score at Baseline	40.4 units on a scale STANDARD_DEVIATION 3.17 • n=5 Participants	40.0 units on a scale STANDARD_DEVIATION 2.0 • n=7 Participants	39.6 units on a scale STANDARD_DEVIATION 3.99 • n=5 Participants	38.6 units on a scale STANDARD_DEVIATION 3.96 • n=4 Participants	39.5 units on a scale STANDARD_DEVIATION 3.56 • n=21 Participants
ALSFRS-R Subdomain at Baseline Dyspnea	4.0 units on a scale STANDARD_DEVIATION 0 • n=5 Participants	3.7 units on a scale STANDARD_DEVIATION 0.50 • n=7 Participants	3.6 units on a scale STANDARD_DEVIATION 0.85 • n=5 Participants	3.8 units on a scale STANDARD_DEVIATION 0.55 • n=4 Participants	3.7 units on a scale STANDARD_DEVIATION 0.62 • n=21 Participants
ALSFRS-R Subdomain at Baseline Orthopnea	4.0 units on a scale STANDARD_DEVIATION 0 • n=5 Participants	3.8 units on a scale STANDARD_DEVIATION 0.67 • n=7 Participants	3.8 units on a scale STANDARD_DEVIATION 0.38 • n=5 Participants	3.9 units on a scale STANDARD_DEVIATION 0.24 • n=4 Participants	3.9 units on a scale STANDARD_DEVIATION 0.37 • n=21 Participants
ALSFRS-R Subdomain at Baseline Respiratory Insufficiency	4.0 units on a scale STANDARD_DEVIATION 0 • n=5 Participants	4.0 units on a scale STANDARD_DEVIATION 0 • n=7 Participants	3.9 units on a scale STANDARD_DEVIATION 0.47 • n=5 Participants	3.7 units on a scale STANDARD_DEVIATION 0.59 • n=4 Participants	3.9 units on a scale STANDARD_DEVIATION 0.45 • n=21 Participants
Time Since ALS Symptom Onset	610.2 days STANDARD_DEVIATION 340.20 • n=5 Participants	647.2 days STANDARD_DEVIATION 388.67 • n=7 Participants	705.0 days STANDARD_DEVIATION 580.30 • n=5 Participants	631.4 days STANDARD_DEVIATION 431.02 • n=4 Participants	655.1 days STANDARD_DEVIATION 457.45 • n=21 Participants
Time Since ALS Diagnosis	251.9 days STANDARD_DEVIATION 241.52 • n=5 Participants	101.6 days STANDARD_DEVIATION 75.32 • n=7 Participants	244.8 days STANDARD_DEVIATION 204.40 • n=5 Participants	219.1 days STANDARD_DEVIATION 174.57 • n=4 Participants	231.5 days STANDARD_DEVIATION 188.57 • n=21 Participants

PRIMARY outcome

Timeframe: Up to 18 Weeks

Population: Full analysis set. All enrolled patients.

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).

Outcome measures

Outcome measures
Measure	AT-1501 1.0 mg/kg n=9 Participants 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.	AT-1501 2.0 mg/kg n=9 Participants 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. 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.	AT-1501 4.0 mg/kg n=18 Participants 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.	AT-1501 8.0 mg/kg n=18 Participants 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.
Safety and Tolerability Number of Subjects with at Least One SAE	0 participants	1 participants	0 participants	0 participants
Safety and Tolerability Number of Subjects with at Least One TEAE	8 participants	8 participants	10 participants	16 participants

PRIMARY outcome

Timeframe: Up to 18 Weeks

Population: Full analysis set. All enrolled patients.

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

Outcome measures

Outcome measures
Measure	AT-1501 1.0 mg/kg n=9 Participants 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.	AT-1501 2.0 mg/kg n=9 Participants 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. 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.	AT-1501 4.0 mg/kg n=18 Participants 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.	AT-1501 8.0 mg/kg n=18 Participants 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.
Safety and Tolerability	30 TEAEs	66 TEAEs	54 TEAEs	67 TEAEs

Adverse Events

AT-1501 1.0 mg/kg

Serious events: 0 serious events

Other events: 8 other events

Deaths: 0 deaths

AT-1501 2.0 mg/kg

Serious events: 1 serious events

Other events: 8 other events

Deaths: 0 deaths

AT-1501 4.0 mg/kg

Serious events: 0 serious events

Other events: 10 other events

Deaths: 0 deaths

AT-1501 8.0 mg/kg

Serious events: 0 serious events

Other events: 16 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	AT-1501 1.0 mg/kg n=9 participants at risk 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.	AT-1501 2.0 mg/kg n=9 participants at risk 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.	AT-1501 4.0 mg/kg n=18 participants at risk 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.	AT-1501 8.0 mg/kg n=18 participants at risk 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.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Tonsil cancer	0.00% 0/9 • 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.	11.1% 1/9 • 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.	0.00% 0/18 • 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.	0.00% 0/18 • 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

Additional Information

Laura Batterman

Eledon Pharmaceuticals

Phone: 949-874-3487

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee 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
Publication restrictions are in place

Restriction type: OTHER