Trial · NCT03569631
A 2-Period Crossover Study of BPN14770 in Adults Males With Fragile X Syndrome
NCT03569631, Phase 2, Completed, Tetra Discovery Partners; results first posted 2024-12-19
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. A 2-Period Crossover Study of BPN14770 in Adults Males With Fragile X Syndrome. Trials, record NCT03569631. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03569631
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A 2-Period Crossover Study of BPN14770 in Adults Males With Fragile X Syndrome
- NCT number
- NCT03569631
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 30 participants, actual
- Lead sponsor
- Tetra Discovery Partners
- Conditions
- Fragile X Syndrome, FXS, Fra(X) Syndrome
- Interventions
- BPN14770, Placebo
- First posted
- 2018-06-26
- Last updated
- 2024-12-19
- Results first posted
- 2024-12-19
Source ClinicalTrials.gov
Registered outcomes4
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Up to 24 weeks | 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. |
| Secondary | Change From Baseline in National Institute of Health Toolbox Cognitive Battery Modified for Intellectual Disabilities Crystallized Cognition Composite (NIH-TCB CCC) Score | Baseline to Week 12 | 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. |
| Secondary | Change From Baseline Between BPN14770 and Placebo Arm in Visual Analog Scale (VAS) Daily Functioning | Baseline to Week 12 | 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. |
| Secondary | Change From Baseline Between BPN14770 and Placebo Arm in VAS Language | Baseline to Week 12 | 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. |
Source ClinicalTrials.gov
Outcomes4
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Up to 24 weeks | 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. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Change From Baseline in National Institute of Health Toolbox Cognitive Battery Modified for Intellectual Disabilities Crystallized Cognition Composite (NIH-TCB CCC) Score | Baseline to Week 12 | Completers population included all randomized participants who completed both periods of treatment with no significant protocol violations. | units on a scale | MEAN |
| SECONDARY | Change From Baseline Between BPN14770 and Placebo Arm in Visual Analog Scale (VAS) Daily Functioning | Baseline to Week 12 | 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. | millimeters (mm) | MEAN |
| SECONDARY | Change From Baseline Between BPN14770 and Placebo Arm in VAS Language | Baseline to Week 12 | 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. | millimeters (mm) | MEAN |
Source ClinicalTrials.gov
Results by group8
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | BPN14770 | 11 | Participants | ||
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Placebo | 8 | Participants | ||
| Change From Baseline in National Institute of Health Toolbox Cognitive Battery Modified for Intellectual Disabilities Crystallized Cognition Composite (NIH-TCB CCC) Score | BPN14770 | 0.1 | Standard Deviation 3.45 | units on a scale | |
| Change From Baseline in National Institute of Health Toolbox Cognitive Battery Modified for Intellectual Disabilities Crystallized Cognition Composite (NIH-TCB CCC) Score | Placebo | -0.9 | Standard Deviation 7.18 | units on a scale | |
| Change From Baseline Between BPN14770 and Placebo Arm in Visual Analog Scale (VAS) Daily Functioning | BPN41770 | 9.4 | Standard Deviation 27.91 | millimeters (mm) | |
| Change From Baseline Between BPN14770 and Placebo Arm in Visual Analog Scale (VAS) Daily Functioning | Placebo | 6.8 | Standard Deviation 22.77 | millimeters (mm) | |
| Change From Baseline Between BPN14770 and Placebo Arm in VAS Language | BPN41770 | 18.2 | Standard Deviation 21.47 | millimeters (mm) | |
| Change From Baseline Between BPN14770 and Placebo Arm in VAS Language | Placebo | 15.4 | Standard Deviation 22.07 | millimeters (mm) |
Source ClinicalTrials.gov
Participant flow16
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Period 1 (12 Weeks) | STARTED | Sequence A-B | 15 | |
| Period 1 (12 Weeks) | STARTED | Sequence B-A | 15 | |
| Period 1 (12 Weeks) | Received At Least 1 Dose of Study Drug | Sequence A-B | 15 | |
| Period 1 (12 Weeks) | Received At Least 1 Dose of Study Drug | Sequence B-A | 15 | |
| Period 1 (12 Weeks) | COMPLETED | Sequence A-B | 15 | |
| Period 1 (12 Weeks) | COMPLETED | Sequence B-A | 15 | |
| Period 1 (12 Weeks) | NOT COMPLETED | Sequence A-B | 0 | |
| Period 1 (12 Weeks) | NOT COMPLETED | Sequence B-A | 0 | |
| Period 2 (12 Weeks) | STARTED | Sequence A-B | 15 | |
| Period 2 (12 Weeks) | STARTED | Sequence B-A | 15 |
1 to 10 of 16Source ClinicalTrials.gov
See all 16 participant flowAdverse events8
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Diarrhoea | Gastrointestinal disorders | BPN14770 | 0 | 30 | |
| other | Diarrhoea | Gastrointestinal disorders | Placebo | 2 | 30 | |
| other | Vomiting | Gastrointestinal disorders | BPN14770 | 3 | 30 | |
| other | Vomiting | Gastrointestinal disorders | Placebo | 2 | 30 | |
| other | Upper respiratory tract infection | Infections and infestations | BPN14770 | 2 | 30 | |
| other | Upper respiratory tract infection | Infections and infestations | Placebo | 3 | 30 | |
| serious | Bursitis infective | Infections and infestations | BPN14770 | 1 | 30 | |
| serious | Bursitis infective | Infections and infestations | Placebo | 0 | 30 |
Source ClinicalTrials.gov
Adverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | BPN14770 | 0 | 30 |
| Total, all-cause mortality | Placebo | 0 | 30 |
| Total, other adverse events | BPN14770 | 5 | 30 |
| Total, other adverse events | Placebo | 6 | 30 |
| Total, serious adverse events | BPN14770 | 1 | 30 |
| Total, serious adverse events | Placebo | 0 | 30 |
Source ClinicalTrials.gov
Papers2
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons3
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| BPN14770 vs Placebo: deaths from any cause | Trial comparisons |
| BPN14770 vs Placebo: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Trial comparisons |
| BPN14770 vs Placebo: serious adverse events | Trial comparisons |
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT03569631
GET https://conductscience.org/api/trials/v1/NCT03569631/registered