Trial Outcomes & Findings for A 2-Period Crossover Study of BPN14770 in Adults Males With Fragile X Syndrome (NCT NCT03569631)
NCT ID: NCT03569631
Last Updated: 2024-12-19
Results Overview
A TEAE was any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. A Serious Adverse Event (SAE) was an AE that met at least 1 of the following criteria: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization for the AE, persistent or significant disability/incapacity or substantial disruption of the ability to conduct normal life functions, congenital anomaly/birth defect (in the child of a participant who was exposed to the study drug), important medical event or reaction. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.
COMPLETED
PHASE2
30 participants
Up to 24 weeks
2024-12-19
Participant Flow
Participant milestones
| Measure |
Sequence A-B
Participants received 25 mg BPN1470 capsules twice daily in Period 1 followed by matching placebo in Period 2.
|
Sequence B-A
Participants received placebo in Period 1 followed by 25 mg BPN1477 capsules twice daily in Period 2.
|
|---|---|---|
|
Period 1 (12 Weeks)
STARTED
|
15
|
15
|
|
Period 1 (12 Weeks)
Received At Least 1 Dose of Study Drug
|
15
|
15
|
|
Period 1 (12 Weeks)
COMPLETED
|
15
|
15
|
|
Period 1 (12 Weeks)
NOT COMPLETED
|
0
|
0
|
|
Period 2 (12 Weeks)
STARTED
|
15
|
15
|
|
Period 2 (12 Weeks)
Received At Least 1 Dose of Study Drug
|
15
|
15
|
|
Period 2 (12 Weeks)
COMPLETED
|
15
|
15
|
|
Period 2 (12 Weeks)
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
A 2-Period Crossover Study of BPN14770 in Adults Males With Fragile X Syndrome
Baseline characteristics by cohort
| Measure |
Sequence A-B
n=15 Participants
Participants received 25 mg BPN1470 capsules twice daily in Period 1 followed by matching placebo in Period 2.
|
Sequence B-A
n=15 Participants
Participants received placebo in Period 1 followed by 25 mg BPN1477 capsules twice daily in Period 2.
|
Total
n=30 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
31.2 years
STANDARD_DEVIATION 6.82 • n=5 Participants
|
32.1 years
STANDARD_DEVIATION 8.00 • n=7 Participants
|
31.6 years
STANDARD_DEVIATION 7.32 • n=5 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
15 Participants
n=5 Participants
|
15 Participants
n=7 Participants
|
30 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
14 Participants
n=5 Participants
|
14 Participants
n=7 Participants
|
28 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 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
|
1 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
3 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
|
14 Participants
n=5 Participants
|
12 Participants
n=7 Participants
|
26 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
|
PRIMARY outcome
Timeframe: Up to 24 weeksPopulation: Safety Population included all participants who were randomized and received at least one dose of study drug. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure.
A TEAE was any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. A Serious Adverse Event (SAE) was an AE that met at least 1 of the following criteria: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization for the AE, persistent or significant disability/incapacity or substantial disruption of the ability to conduct normal life functions, congenital anomaly/birth defect (in the child of a participant who was exposed to the study drug), important medical event or reaction. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.
Outcome measures
| Measure |
BPN14770
n=30 Participants
Participants received 25 mg BPN14770 capsules twice daily for 12 weeks either in Period 1 or Period 2.
|
Placebo
n=30 Participants
Participants received placebo to match 25 mg BPN14770 capsules twice daily for 12 weeks either in Period 1 or Period 2.
|
|---|---|---|
|
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
|
11 Participants
|
8 Participants
|
SECONDARY outcome
Timeframe: Baseline to Week 12Population: Completers population included all randomized participants who completed both periods of treatment with no significant protocol violations.
The NIH-TCB is a battery of extensively validated computer-administered cognitive tests administered on an iPad. The Crystallized Composite score assessment includes Picture Vocabulary and Oral Reading Recognition tests. The NIH toolbox standard score has a mean of 100 and standard deviation (SD) of 15. Higher scores indicate better intellectual abilities.
Outcome measures
| Measure |
BPN14770
n=29 Participants
Participants received 25 mg BPN14770 capsules twice daily for 12 weeks either in Period 1 or Period 2.
|
Placebo
n=30 Participants
Participants received placebo to match 25 mg BPN14770 capsules twice daily for 12 weeks either in Period 1 or Period 2.
|
|---|---|---|
|
Change From Baseline in National Institute of Health Toolbox Cognitive Battery Modified for Intellectual Disabilities Crystallized Cognition Composite (NIH-TCB CCC) Score
|
0.1 units on a scale
Standard Deviation 3.45
|
-0.9 units on a scale
Standard Deviation 7.18
|
SECONDARY outcome
Timeframe: Baseline to Week 12Population: Intent-to-Treat (ITT) population included all randomized participants who received at least one dose of treatment and returned for at least one follow-up visit.
The VAS was used to measure the severity of Daily Functioning skills, which is a specific behavioral symptom targeted in this study. Parents or caregivers marked this behavior on a visual line on a score from 0 to 100, with higher scores indicating better quality of life.
Outcome measures
| Measure |
BPN14770
n=30 Participants
Participants received 25 mg BPN14770 capsules twice daily for 12 weeks either in Period 1 or Period 2.
|
Placebo
n=30 Participants
Participants received placebo to match 25 mg BPN14770 capsules twice daily for 12 weeks either in Period 1 or Period 2.
|
|---|---|---|
|
Change From Baseline Between BPN14770 and Placebo Arm in Visual Analog Scale (VAS) Daily Functioning
|
9.4 millimeters (mm)
Standard Deviation 27.91
|
6.8 millimeters (mm)
Standard Deviation 22.77
|
SECONDARY outcome
Timeframe: Baseline to Week 12Population: Intent-to-Treat (ITT) population included all randomized participants who received at least one dose of treatment and returned for at least one follow-up visit.
The VAS was used to measure the severity of Language skills, which is a specific behavioral symptom targeted in this study. Parents or caregivers marked this behavior on a visual line on a score from 0 to 100, with higher scores indicating better quality of life.
Outcome measures
| Measure |
BPN14770
n=30 Participants
Participants received 25 mg BPN14770 capsules twice daily for 12 weeks either in Period 1 or Period 2.
|
Placebo
n=30 Participants
Participants received placebo to match 25 mg BPN14770 capsules twice daily for 12 weeks either in Period 1 or Period 2.
|
|---|---|---|
|
Change From Baseline Between BPN14770 and Placebo Arm in VAS Language
|
18.2 millimeters (mm)
Standard Deviation 21.47
|
15.4 millimeters (mm)
Standard Deviation 22.07
|
Adverse Events
BPN14770
Placebo
Serious adverse events
| Measure |
BPN14770
n=30 participants at risk
Participants received 25 mg BPN14770 capsules twice daily for 12 weeks either in Period 1 or Period 2.
|
Placebo
n=30 participants at risk
Participants received placebo to match 25 mg BPN14770 capsules twice daily for 12 weeks either in Period 1 or Period 2.
|
|---|---|---|
|
Infections and infestations
Bursitis infective
|
3.3%
1/30 • First dose of study drug up to 24 weeks
Safety Population included all participants who were randomized and received at least one dose of study drug.
|
0.00%
0/30 • First dose of study drug up to 24 weeks
Safety Population included all participants who were randomized and received at least one dose of study drug.
|
Other adverse events
| Measure |
BPN14770
n=30 participants at risk
Participants received 25 mg BPN14770 capsules twice daily for 12 weeks either in Period 1 or Period 2.
|
Placebo
n=30 participants at risk
Participants received placebo to match 25 mg BPN14770 capsules twice daily for 12 weeks either in Period 1 or Period 2.
|
|---|---|---|
|
Gastrointestinal disorders
Diarrhoea
|
0.00%
0/30 • First dose of study drug up to 24 weeks
Safety Population included all participants who were randomized and received at least one dose of study drug.
|
6.7%
2/30 • First dose of study drug up to 24 weeks
Safety Population included all participants who were randomized and received at least one dose of study drug.
|
|
Gastrointestinal disorders
Vomiting
|
10.0%
3/30 • First dose of study drug up to 24 weeks
Safety Population included all participants who were randomized and received at least one dose of study drug.
|
6.7%
2/30 • First dose of study drug up to 24 weeks
Safety Population included all participants who were randomized and received at least one dose of study drug.
|
|
Infections and infestations
Upper respiratory tract infection
|
6.7%
2/30 • First dose of study drug up to 24 weeks
Safety Population included all participants who were randomized and received at least one dose of study drug.
|
10.0%
3/30 • First dose of study drug up to 24 weeks
Safety Population included all participants who were randomized and received at least one dose of study drug.
|
Additional Information
Shionogi Clinical Trials Administrator Clinical Support Help Line
Shionogi
Results disclosure agreements
- Principal investigator is a sponsor employee The sponsor can embargo results from a PI's center until the combined results from the completed study have been published in full or the sponsor confirms there will be no multicenter study publication. Results communications must be provided to the sponsor for review at least 60 days before submission for publication. By written request, the sponsor can extend the embargo up to an additional 60 days. The sponsor cannot require changes to scientific content and cannot further extend the embargo.
- Publication restrictions are in place
Restriction type: OTHER