Trial Outcomes & Findings for Within Subject Crossover Study of Cognitive Effects of Neflamapimod in Early-Stage Huntington Disease (NCT NCT03980938)
NCT ID: NCT03980938
Last Updated: 2022-04-06
Results Overview
Change from baseline of latency during the learning phase of vMWM (hidden platform training) in the neflamapimod first group compared to placebo first group
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
15 participants
Primary outcome timeframe
Baseline and 10 Weeks
Results posted on
2022-04-06
Participant Flow
Participant milestones
| Measure |
Neflamapimod First
neflamapimod in Treatment Period 1, placebo in Treatment Period 2
neflamapimod: 40 mg neflamapimod hard gelatin capsules, taken twice daily with food.
Placebo: hard gelatin capsules containing excipients only, weight- and size-matched; taken twice daily with food.
|
Placebo First
placebo in Treatment Period 1, neflamapimod in Treatment Period 2
Placebo: hard gelatin capsules containing excipients only, weight- and size-matched; taken twice daily with food.
neflamapimod: 40 mg neflamapimod hard gelatin capsules, taken twice daily with food.
|
|---|---|---|
|
Treatment Period 1
STARTED
|
7
|
8
|
|
Treatment Period 1
COMPLETED
|
6
|
7
|
|
Treatment Period 1
NOT COMPLETED
|
1
|
1
|
|
Treatment Period 2
STARTED
|
3
|
2
|
|
Treatment Period 2
COMPLETED
|
1
|
0
|
|
Treatment Period 2
NOT COMPLETED
|
2
|
2
|
Reasons for withdrawal
| Measure |
Neflamapimod First
neflamapimod in Treatment Period 1, placebo in Treatment Period 2
neflamapimod: 40 mg neflamapimod hard gelatin capsules, taken twice daily with food.
Placebo: hard gelatin capsules containing excipients only, weight- and size-matched; taken twice daily with food.
|
Placebo First
placebo in Treatment Period 1, neflamapimod in Treatment Period 2
Placebo: hard gelatin capsules containing excipients only, weight- and size-matched; taken twice daily with food.
neflamapimod: 40 mg neflamapimod hard gelatin capsules, taken twice daily with food.
|
|---|---|---|
|
Treatment Period 1
Study Terminated
|
1
|
1
|
|
Treatment Period 2
Study Terminated
|
2
|
2
|
Baseline Characteristics
Within Subject Crossover Study of Cognitive Effects of Neflamapimod in Early-Stage Huntington Disease
Baseline characteristics by cohort
| Measure |
Neflamapimod First
n=7 Participants
neflamapimod in Treatment Period 1, placebo in Treatment Period 2
neflamapimod: 40 mg neflamapimod hard gelatin capsules, taken twice daily with food.
Placebo: hard gelatin capsules containing excipients only, weight- and size-matched; taken twice daily with food.
|
Placebo First
n=8 Participants
placebo in Treatment Period 1, neflamapimod in Treatment Period 2
Placebo: hard gelatin capsules containing excipients only, weight- and size-matched; taken twice daily with food.
neflamapimod: 40 mg neflamapimod hard gelatin capsules, taken twice daily with food.
|
Total
n=15 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
5 Participants
n=93 Participants
|
7 Participants
n=4 Participants
|
12 Participants
n=27 Participants
|
|
Age, Categorical
>=65 years
|
2 Participants
n=93 Participants
|
1 Participants
n=4 Participants
|
3 Participants
n=27 Participants
|
|
Age, Continuous
|
51 years
STANDARD_DEVIATION 12.26 • n=93 Participants
|
54.5 years
STANDARD_DEVIATION 9.68 • n=4 Participants
|
52.87 years
STANDARD_DEVIATION 10.7 • n=27 Participants
|
|
Sex: Female, Male
Female
|
5 Participants
n=93 Participants
|
5 Participants
n=4 Participants
|
10 Participants
n=27 Participants
|
|
Sex: Female, Male
Male
|
2 Participants
n=93 Participants
|
3 Participants
n=4 Participants
|
5 Participants
n=27 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
7 Participants
n=93 Participants
|
8 Participants
n=4 Participants
|
15 Participants
n=27 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
|
Race (NIH/OMB)
Asian
|
2 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
2 Participants
n=27 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
|
Race (NIH/OMB)
White
|
5 Participants
n=93 Participants
|
8 Participants
n=4 Participants
|
13 Participants
n=27 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
|
Region of Enrollment
United Kingdom
|
7 participants
n=93 Participants
|
8 participants
n=4 Participants
|
15 participants
n=27 Participants
|
|
Body Mass Index (BMI)
|
26.3 kg/m2
STANDARD_DEVIATION 5.4 • n=93 Participants
|
27.61 kg/m2
STANDARD_DEVIATION 6.15 • n=4 Participants
|
27 kg/m2
STANDARD_DEVIATION 5.65 • n=27 Participants
|
PRIMARY outcome
Timeframe: Baseline and 10 WeeksPopulation: Participants who had analyzable results at Baseline and Week 10 of Treatment Period 1 (13 participants)
Change from baseline of latency during the learning phase of vMWM (hidden platform training) in the neflamapimod first group compared to placebo first group
Outcome measures
| Measure |
Neflamapimod First
n=6 Participants
neflamapimod in Treatment Period 1, placebo in Treatment Period 2
neflamapimod: 40 mg neflamapimod hard gelatin capsules, taken twice daily with food.
Placebo: hard gelatin capsules containing excipients only, weight- and size-matched; taken twice daily with food.
|
Placebo First
n=7 Participants
placebo in Treatment Period 1, neflamapimod in Treatment Period 2
Placebo: hard gelatin capsules containing excipients only, weight- and size-matched; taken twice daily with food.
neflamapimod: 40 mg neflamapimod hard gelatin capsules, taken twice daily with food.
|
|---|---|---|
|
Change in Latency During the Learning Phase of Virtual Morris Water Maze Test (vMWM)
|
-6.92 seconds
Standard Deviation 3.814
|
-14.05 seconds
Standard Deviation 12.680
|
Adverse Events
Neflamapimod First
Serious events: 0 serious events
Other events: 4 other events
Deaths: 0 deaths
Placebo First
Serious events: 0 serious events
Other events: 5 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Neflamapimod First
n=7 participants at risk
neflamapimod in Treatment Period 1, placebo in Treatment Period 2
neflamapimod: 40 mg neflamapimod hard gelatin capsules, taken twice daily with food.
Placebo: hard gelatin capsules containing excipients only, weight- and size-matched; taken twice daily with food.
|
Placebo First
n=8 participants at risk
placebo in Treatment Period 1, neflamapimod in Treatment Period 2
Placebo: hard gelatin capsules containing excipients only, weight- and size-matched; taken twice daily with food.
neflamapimod: 40 mg neflamapimod hard gelatin capsules, taken twice daily with food.
|
|---|---|---|
|
Infections and infestations
Common Cold
|
42.9%
3/7 • Number of events 3 • Up to 37 weeks. AEs occurring from when the subject signed the ICF until up to 30 days after the last dose were collected. Any AEs occurring before the start of treatment (i.e., before the first dose of the investigational product) were recorded in the medical history.
Any sign, symptom, or disease present before starting the treatment period were only considered AEs if they worsen after starting the treatment period. All-Cause Mortality was not monitored/assessed.
|
0.00%
0/8 • Up to 37 weeks. AEs occurring from when the subject signed the ICF until up to 30 days after the last dose were collected. Any AEs occurring before the start of treatment (i.e., before the first dose of the investigational product) were recorded in the medical history.
Any sign, symptom, or disease present before starting the treatment period were only considered AEs if they worsen after starting the treatment period. All-Cause Mortality was not monitored/assessed.
|
|
Gastrointestinal disorders
Diarrhea
|
0.00%
0/7 • Up to 37 weeks. AEs occurring from when the subject signed the ICF until up to 30 days after the last dose were collected. Any AEs occurring before the start of treatment (i.e., before the first dose of the investigational product) were recorded in the medical history.
Any sign, symptom, or disease present before starting the treatment period were only considered AEs if they worsen after starting the treatment period. All-Cause Mortality was not monitored/assessed.
|
12.5%
1/8 • Number of events 1 • Up to 37 weeks. AEs occurring from when the subject signed the ICF until up to 30 days after the last dose were collected. Any AEs occurring before the start of treatment (i.e., before the first dose of the investigational product) were recorded in the medical history.
Any sign, symptom, or disease present before starting the treatment period were only considered AEs if they worsen after starting the treatment period. All-Cause Mortality was not monitored/assessed.
|
|
Gastrointestinal disorders
Nausea
|
0.00%
0/7 • Up to 37 weeks. AEs occurring from when the subject signed the ICF until up to 30 days after the last dose were collected. Any AEs occurring before the start of treatment (i.e., before the first dose of the investigational product) were recorded in the medical history.
Any sign, symptom, or disease present before starting the treatment period were only considered AEs if they worsen after starting the treatment period. All-Cause Mortality was not monitored/assessed.
|
12.5%
1/8 • Number of events 1 • Up to 37 weeks. AEs occurring from when the subject signed the ICF until up to 30 days after the last dose were collected. Any AEs occurring before the start of treatment (i.e., before the first dose of the investigational product) were recorded in the medical history.
Any sign, symptom, or disease present before starting the treatment period were only considered AEs if they worsen after starting the treatment period. All-Cause Mortality was not monitored/assessed.
|
|
Infections and infestations
Sores
|
0.00%
0/7 • Up to 37 weeks. AEs occurring from when the subject signed the ICF until up to 30 days after the last dose were collected. Any AEs occurring before the start of treatment (i.e., before the first dose of the investigational product) were recorded in the medical history.
Any sign, symptom, or disease present before starting the treatment period were only considered AEs if they worsen after starting the treatment period. All-Cause Mortality was not monitored/assessed.
|
12.5%
1/8 • Number of events 1 • Up to 37 weeks. AEs occurring from when the subject signed the ICF until up to 30 days after the last dose were collected. Any AEs occurring before the start of treatment (i.e., before the first dose of the investigational product) were recorded in the medical history.
Any sign, symptom, or disease present before starting the treatment period were only considered AEs if they worsen after starting the treatment period. All-Cause Mortality was not monitored/assessed.
|
|
Infections and infestations
Sinusitis
|
0.00%
0/7 • Up to 37 weeks. AEs occurring from when the subject signed the ICF until up to 30 days after the last dose were collected. Any AEs occurring before the start of treatment (i.e., before the first dose of the investigational product) were recorded in the medical history.
Any sign, symptom, or disease present before starting the treatment period were only considered AEs if they worsen after starting the treatment period. All-Cause Mortality was not monitored/assessed.
|
12.5%
1/8 • Number of events 1 • Up to 37 weeks. AEs occurring from when the subject signed the ICF until up to 30 days after the last dose were collected. Any AEs occurring before the start of treatment (i.e., before the first dose of the investigational product) were recorded in the medical history.
Any sign, symptom, or disease present before starting the treatment period were only considered AEs if they worsen after starting the treatment period. All-Cause Mortality was not monitored/assessed.
|
|
Psychiatric disorders
Increased Anxiety
|
0.00%
0/7 • Up to 37 weeks. AEs occurring from when the subject signed the ICF until up to 30 days after the last dose were collected. Any AEs occurring before the start of treatment (i.e., before the first dose of the investigational product) were recorded in the medical history.
Any sign, symptom, or disease present before starting the treatment period were only considered AEs if they worsen after starting the treatment period. All-Cause Mortality was not monitored/assessed.
|
12.5%
1/8 • Number of events 1 • Up to 37 weeks. AEs occurring from when the subject signed the ICF until up to 30 days after the last dose were collected. Any AEs occurring before the start of treatment (i.e., before the first dose of the investigational product) were recorded in the medical history.
Any sign, symptom, or disease present before starting the treatment period were only considered AEs if they worsen after starting the treatment period. All-Cause Mortality was not monitored/assessed.
|
|
Gastrointestinal disorders
Abdominal Pain
|
0.00%
0/7 • Up to 37 weeks. AEs occurring from when the subject signed the ICF until up to 30 days after the last dose were collected. Any AEs occurring before the start of treatment (i.e., before the first dose of the investigational product) were recorded in the medical history.
Any sign, symptom, or disease present before starting the treatment period were only considered AEs if they worsen after starting the treatment period. All-Cause Mortality was not monitored/assessed.
|
12.5%
1/8 • Number of events 1 • Up to 37 weeks. AEs occurring from when the subject signed the ICF until up to 30 days after the last dose were collected. Any AEs occurring before the start of treatment (i.e., before the first dose of the investigational product) were recorded in the medical history.
Any sign, symptom, or disease present before starting the treatment period were only considered AEs if they worsen after starting the treatment period. All-Cause Mortality was not monitored/assessed.
|
|
Nervous system disorders
Headache
|
14.3%
1/7 • Number of events 1 • Up to 37 weeks. AEs occurring from when the subject signed the ICF until up to 30 days after the last dose were collected. Any AEs occurring before the start of treatment (i.e., before the first dose of the investigational product) were recorded in the medical history.
Any sign, symptom, or disease present before starting the treatment period were only considered AEs if they worsen after starting the treatment period. All-Cause Mortality was not monitored/assessed.
|
0.00%
0/8 • Up to 37 weeks. AEs occurring from when the subject signed the ICF until up to 30 days after the last dose were collected. Any AEs occurring before the start of treatment (i.e., before the first dose of the investigational product) were recorded in the medical history.
Any sign, symptom, or disease present before starting the treatment period were only considered AEs if they worsen after starting the treatment period. All-Cause Mortality was not monitored/assessed.
|
|
Infections and infestations
Urinary Tract Infection
|
14.3%
1/7 • Number of events 1 • Up to 37 weeks. AEs occurring from when the subject signed the ICF until up to 30 days after the last dose were collected. Any AEs occurring before the start of treatment (i.e., before the first dose of the investigational product) were recorded in the medical history.
Any sign, symptom, or disease present before starting the treatment period were only considered AEs if they worsen after starting the treatment period. All-Cause Mortality was not monitored/assessed.
|
0.00%
0/8 • Up to 37 weeks. AEs occurring from when the subject signed the ICF until up to 30 days after the last dose were collected. Any AEs occurring before the start of treatment (i.e., before the first dose of the investigational product) were recorded in the medical history.
Any sign, symptom, or disease present before starting the treatment period were only considered AEs if they worsen after starting the treatment period. All-Cause Mortality was not monitored/assessed.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place