Trial Outcomes & Findings for A Phase 2 Study to Evaluate AL001 in C9orf72-Associated ALS (NCT NCT05053035)
NCT ID: NCT05053035
Last Updated: 2025-06-18
Results Overview
Count of participants with adverse events during the study treatment period
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
5 participants
Primary outcome timeframe
24 weeks
Results posted on
2025-06-18
Participant Flow
Participant milestones
| Measure |
AL001
AL001 every 4 weeks
AL001: Administered via intravenous (IV) infusion
|
Placebo
Placebo every 4 weeks
Placebo: Administered via intravenous (IV) infusion
|
|---|---|---|
|
Overall Study
STARTED
|
3
|
2
|
|
Overall Study
COMPLETED
|
2
|
2
|
|
Overall Study
NOT COMPLETED
|
1
|
0
|
Reasons for withdrawal
| Measure |
AL001
AL001 every 4 weeks
AL001: Administered via intravenous (IV) infusion
|
Placebo
Placebo every 4 weeks
Placebo: Administered via intravenous (IV) infusion
|
|---|---|---|
|
Overall Study
Withdrawal by Subject
|
1
|
0
|
Baseline Characteristics
A Phase 2 Study to Evaluate AL001 in C9orf72-Associated ALS
Baseline characteristics by cohort
| Measure |
AL001
n=3 Participants
AL001 every 4 weeks
AL001: Administered via intravenous (IV) infusion
|
Placebo
n=2 Participants
Placebo every 4 weeks
Placebo: Administered via intravenous (IV) infusion
|
Total
n=5 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
|
|
Age, Categorical
Between 18 and 65 years
|
2 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
2 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 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
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
3 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
3 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 24 weeksPopulation: Safety population
Count of participants with adverse events during the study treatment period
Outcome measures
| Measure |
AL001
n=3 Participants
AL001 every 4 weeks
AL001: Administered via intravenous (IV) infusion
|
Placebo
n=2 Participants
Placebo every 4 weeks
Placebo: Administered via intravenous (IV) infusion
|
|---|---|---|
|
Evaluation of Safety and Tolerability of AL001 Measured by Number of Subjects With Adverse Events
|
2 Participants
|
2 Participants
|
PRIMARY outcome
Timeframe: Week 24Population: Due to early termination of the study and insufficient sample size, no population-level analysis (within-group and between-group) was performed.
Count of participants positive for Anti-drug Antibodies (ADAs) to AL001 at week 24
Outcome measures
| Measure |
AL001
n=2 Participants
AL001 every 4 weeks
AL001: Administered via intravenous (IV) infusion
|
Placebo
n=2 Participants
Placebo every 4 weeks
Placebo: Administered via intravenous (IV) infusion
|
|---|---|---|
|
Immunogenicity of AL001
|
1 Participants
|
0 Participants
|
PRIMARY outcome
Timeframe: Week 24Population: Due to early termination of the study and insufficient sample size, no population-level analysis (within-group and between-group) was performed.
Concentration of AL001 in Serum at week 24
Outcome measures
| Measure |
AL001
n=2 Participants
AL001 every 4 weeks
AL001: Administered via intravenous (IV) infusion
|
Placebo
Placebo every 4 weeks
Placebo: Administered via intravenous (IV) infusion
|
|---|---|---|
|
Pharmacokinetics (PK) of AL001 in Serum
|
705792.5 ng/mL
Standard Deviation 87387.7916
|
—
|
PRIMARY outcome
Timeframe: Week 24Population: Due to early termination of the study and insufficient sample size, no population-level analysis (within-group and between-group) was performed.
Concentration of AL001 in Cerebrospinal fluid (CSF) at week 24
Outcome measures
| Measure |
AL001
n=2 Participants
AL001 every 4 weeks
AL001: Administered via intravenous (IV) infusion
|
Placebo
Placebo every 4 weeks
Placebo: Administered via intravenous (IV) infusion
|
|---|---|---|
|
Pharmacokinetics (PK) of AL001 in CSF
|
547.5 ng/mL
Standard Deviation 395.2727
|
—
|
PRIMARY outcome
Timeframe: 24 weeksPopulation: Due to early termination of the study and insufficient sample size, no population-level analysis (within-group and between-group) was performed.
Evaluate the change from baseline to week 24 in plasma progranulin levels
Outcome measures
| Measure |
AL001
n=2 Participants
AL001 every 4 weeks
AL001: Administered via intravenous (IV) infusion
|
Placebo
n=2 Participants
Placebo every 4 weeks
Placebo: Administered via intravenous (IV) infusion
|
|---|---|---|
|
Change From Baseline in Plasma Progranulin
|
175 ng/mL
Standard Deviation 2.8284
|
-24.4 ng/mL
Standard Deviation 9.3338
|
PRIMARY outcome
Timeframe: 24 weeksPopulation: Due to early termination of the study and insufficient sample size, no population-level analysis (within-group and between-group) was performed.
Evaluate the change from baseline to week 24 in Cerebrospinal fluid (CSF) progranulin levels
Outcome measures
| Measure |
AL001
n=2 Participants
AL001 every 4 weeks
AL001: Administered via intravenous (IV) infusion
|
Placebo
n=2 Participants
Placebo every 4 weeks
Placebo: Administered via intravenous (IV) infusion
|
|---|---|---|
|
Change From Baseline in CSF Progranulin
|
2.865 ng/mL
Standard Deviation 1.0677
|
-0.385 ng/mL
Standard Deviation 0.3323
|
SECONDARY outcome
Timeframe: 24 weeksPopulation: Due to early termination of the study and insufficient sample size, no population-level analysis (within-group and between-group) was performed.
Evaluate the change from baseline to week 24 in plasma neurofilament light chain levels
Outcome measures
| Measure |
AL001
n=2 Participants
AL001 every 4 weeks
AL001: Administered via intravenous (IV) infusion
|
Placebo
n=2 Participants
Placebo every 4 weeks
Placebo: Administered via intravenous (IV) infusion
|
|---|---|---|
|
Change From Baseline in Plasma Neurofilament Light Chain
|
-0.35 pg/mL
Standard Deviation 3.3234
|
1.9 pg/mL
Standard Deviation 2.8284
|
SECONDARY outcome
Timeframe: 24 weeksPopulation: Due to early termination of the study and insufficient sample size, no population-level analysis (within-group and between-group) was performed.
Evaluate change from baseline to week 24 in Cerebrospinal fluid (CSF) neurofilament light chain levels
Outcome measures
| Measure |
AL001
n=2 Participants
AL001 every 4 weeks
AL001: Administered via intravenous (IV) infusion
|
Placebo
n=2 Participants
Placebo every 4 weeks
Placebo: Administered via intravenous (IV) infusion
|
|---|---|---|
|
Change From Baseline in CSF Neurofilament Light Chain
|
995.5 pg/mL
Standard Deviation 95.4594
|
-1250.5 pg/mL
Standard Deviation 825.1936
|
Adverse Events
AL001
Serious events: 0 serious events
Other events: 2 other events
Deaths: 0 deaths
Placebo
Serious events: 0 serious events
Other events: 2 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
AL001
n=3 participants at risk
AL001 every 4 weeks
AL001: Administered via intravenous (IV) infusion
|
Placebo
n=2 participants at risk
Placebo every 4 weeks
Placebo: Administered via intravenous (IV) infusion
|
|---|---|---|
|
Gastrointestinal disorders
Constipation
|
0.00%
0/3 • 32 Weeks
|
50.0%
1/2 • 32 Weeks
|
|
Gastrointestinal disorders
Diarrhea
|
0.00%
0/3 • 32 Weeks
|
50.0%
1/2 • 32 Weeks
|
|
Gastrointestinal disorders
Dyspepsia
|
33.3%
1/3 • 32 Weeks
|
0.00%
0/2 • 32 Weeks
|
|
Gastrointestinal disorders
Nausea
|
0.00%
0/3 • 32 Weeks
|
50.0%
1/2 • 32 Weeks
|
|
General disorders
Catheter site irritation
|
0.00%
0/3 • 32 Weeks
|
50.0%
1/2 • 32 Weeks
|
|
General disorders
Fatigue
|
0.00%
0/3 • 32 Weeks
|
50.0%
1/2 • 32 Weeks
|
|
General disorders
Medical device site hemorrhage
|
0.00%
0/3 • 32 Weeks
|
50.0%
1/2 • 32 Weeks
|
|
General disorders
Pain
|
0.00%
0/3 • 32 Weeks
|
50.0%
1/2 • 32 Weeks
|
|
General disorders
Pyrexia
|
0.00%
0/3 • 32 Weeks
|
50.0%
1/2 • 32 Weeks
|
|
Infections and infestations
Upper respiratory tract infection
|
33.3%
1/3 • 32 Weeks
|
0.00%
0/2 • 32 Weeks
|
|
Infections and infestations
Urinary tract infection
|
0.00%
0/3 • 32 Weeks
|
50.0%
1/2 • 32 Weeks
|
|
Injury, poisoning and procedural complications
Post lumbar puncture syndrome
|
66.7%
2/3 • 32 Weeks
|
50.0%
1/2 • 32 Weeks
|
|
Injury, poisoning and procedural complications
Procedural pain
|
33.3%
1/3 • 32 Weeks
|
0.00%
0/2 • 32 Weeks
|
|
Injury, poisoning and procedural complications
Stoma site erythema
|
33.3%
1/3 • 32 Weeks
|
0.00%
0/2 • 32 Weeks
|
|
Injury, poisoning and procedural complications
Stoma site hypergranulation
|
0.00%
0/3 • 32 Weeks
|
50.0%
1/2 • 32 Weeks
|
|
Investigations
Hepatic enzyme increased
|
33.3%
1/3 • 32 Weeks
|
0.00%
0/2 • 32 Weeks
|
|
Nervous system disorders
Headache
|
0.00%
0/3 • 32 Weeks
|
50.0%
1/2 • 32 Weeks
|
|
Nervous system disorders
Paraesthesia
|
0.00%
0/3 • 32 Weeks
|
50.0%
1/2 • 32 Weeks
|
|
Renal and urinary disorders
Urinary incontinence
|
0.00%
0/3 • 32 Weeks
|
50.0%
1/2 • 32 Weeks
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
0.00%
0/3 • 32 Weeks
|
50.0%
1/2 • 32 Weeks
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonitis aspiration
|
0.00%
0/3 • 32 Weeks
|
50.0%
1/2 • 32 Weeks
|
|
Respiratory, thoracic and mediastinal disorders
Throat irritation
|
0.00%
0/3 • 32 Weeks
|
50.0%
1/2 • 32 Weeks
|
|
Skin and subcutaneous tissue disorders
Drug eruption
|
0.00%
0/3 • 32 Weeks
|
50.0%
1/2 • 32 Weeks
|
|
Skin and subcutaneous tissue disorders
Rash
|
33.3%
1/3 • 32 Weeks
|
0.00%
0/2 • 32 Weeks
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place