Trial · NCT01725152
Ganaxolone Treatment in Children With Fragile X Syndrome
NCT01725152, Phase 2, Completed, Marinus Pharmaceuticals; results first posted 2023-04-10
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Ganaxolone Treatment in Children With Fragile X Syndrome. Trials, record NCT01725152. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01725152
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Ganaxolone Treatment in Children With Fragile X Syndrome
- NCT number
- NCT01725152
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 59 participants, actual
- Lead sponsor
- Marinus Pharmaceuticals
- Conditions
- Fragile x Syndrome
- Interventions
- Ganaxolone, Placebo
- First posted
- 2012-11-12
- Last updated
- 2023-04-10
- Results first posted
- 2023-04-10
Source ClinicalTrials.gov
Registered outcomes6
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Clinical Global Impression-Improvement (CGI-I) Scale | Week 14 (End of Treatment) | The CGI-I scale is a clinician-rated 7-point scale used to assess how much the participant's illness had improved or worsened relative to a Baseline state at the beginning of the intervention. It was rated as: 1. "very much improved"; 2. "much improved"; 3. "minimally improved"; 4. "no change"; 5. "minimally worse"; 6. "much worse"; 7. "very much worse". Higher scores indicated worse condition. |
| Secondary | Pediatric Anxiety Rating Scale (PARS) Total Score | Week 14 (End of Treatment) | Pediatric Anxiety Rating Scale (PARS) is a clinician administered measure of anxiety in children and adolescents. The PARS is comprised of a 50-item symptom checklist used to determine the presence or absence of specific anxiety symptoms during the prior week and 7 severity/impairment items, each scored from 0 to 5. The score on the 7 items allows the clinician to rate symptom severity and associated impairment on a range from 0 to 35, with higher scores reflecting greater symptom severity and associated impairment. |
| Secondary | Visual Analogue Scale (VAS) | Week 14 (End of Treatment) | A semi-structured VAS design was employed, providing two of the three behaviors to be assessed as anxiety and attention, chosen by caregiver(s) of one additional behavior from a bank of five. This bank included sociability, attention, aggression, language, and hyperactivity/impulsivity. Parents mark on a visual line measuring 10 centimeters (cm) with "worst behavior" at 0 cm and "best behavior" at 10 cm. For each behavior the caregiver is instructed to mark their impression of behavior at Baseline visit and again at each visit. The calculated distance in cm between the baseline and each visit marks thereby demonstrates whether each behavior improved, worsened, or stayed the same during the study, and by how much. Shown here is the least square mean distance and its corresponding standard error in cm from the "worst behavior" side, at baseline. Smaller the value, worser the behavior. |
| Secondary | Anxiety, Depression, and Mood Scale (ADAMS) | Week 14 (End of Treatment) | The ADAMS is a 28-item behavior-based informant instrument rated by the parent(s), legal authorized guardian(s), or consistent caregiver(s). The scale is composed of 5 factors, which addresses Manic/Hyperactive Behavior, Depressed Mood, Social Avoidance, General Anxiety, and Obsessive/Compulsive Behavior. Items are scored on a 0-3 Likert scale that combines frequency and severity ratings (where 0=behavior has not occurred or is not a problem, 3=behavior occurs a lot or is a severe problem). Each subscale score is calculated separately; the scale range for Manic/Hyperactive Behavior is 0-15; for Depressed Mood, 0-21; for Social Avoidance, 0-21; for General Anxiety, 0-21; for Obsessive Behavior, 0-9. There is an overlapped item between Manic/Hypertension Behavior and General Anxiety; hence, the subscale ranges appear to reflect scores for 29 items instead of 28 items. |
| Secondary | Aberrant Behavior Checklist (ABC) | Week 14 (End of Treatment) | The ABC is a 58-item parent rated from 0 (not at all a problem) to 3 (the problem is severe in degree with 6 subscales: Irritability (includes agitation, aggression, and self-injury, 15 items) with range of scores from 0-45; Social Withdrawal/Lethargy (16 items) with range of scores from 0-48; Stereotypy (7 items) with range of scores from 0-21; Hyperactivity (16 items) with range of scores from 0-48; Inappropriate Speech (4 items) with range of scores from 0-12 and Social avoidance (4 items) with range of scores from 0-12. Higher scores indicated greater severity. Social Withdrawal and Lethargy are reporting the same scale and Social Avoidance is a subscale of Social Withdrawal; hence, the subscale ranges appear to reflect scores for 62 items instead of 58 items. |
| Secondary | Swanson, Nolan, and Pelham-IV Questionnaire (SNAP-IV) | Week 14 (End of Treatment) | The SNAP-IV is a revision of the Swanson, Nolan, and Pelham (SNAP) Questionnaire. The SNAP-IV: ADHD Inattention Subscale (items 1-9) scores the intensity of each item during the last seven days on a 0 to 3 scale (0=not at all, 1=just a little, 2=pretty much, 3=very much). Possible scores ranged from 0-27; higher scores indicated a greater intensity. The SNAP-IV ADHD Hyperactivity/Impulsivity Subscale (items 10-18) scores the intensity of each item in the last seven days on a 0 to 3 scale (0=not at all, 1=just a little, 2=pretty much, 3=very much). Possible scores ranged from 0-27; higher scores indicated a greater intensity. SNAP-IV ADHD Combined Scale score (inattention + hyperactivity/impulsivity) ranged from 0-54. A low score of 0 indicates less inattention + hyperactivity/impulsivity. A high score of 54 indicates more inattention + hyperactivity. |
Source ClinicalTrials.gov
Outcomes6
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Clinical Global Impression-Improvement (CGI-I) Scale | Week 14 (End of Treatment) | Intention-to-Treat (ITT) Population consisted of all participants who received medication and who had at least one post-Baseline assessment. Only those participants with data available at the specified data points were analyzed. | Scores on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Pediatric Anxiety Rating Scale (PARS) Total Score | Week 14 (End of Treatment) | ITT Population. Only those participants with data available at the specified data points were analyzed. | Scores on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Visual Analogue Scale (VAS) | Week 14 (End of Treatment) | ITT Population. Only those participants with data available at the specified data points were analyzed | Centimeters (cm) | LEAST_SQUARES_MEAN |
| SECONDARY | Anxiety, Depression, and Mood Scale (ADAMS) | Week 14 (End of Treatment) | ITT Population. Only those participants with data available at the specified data points were analyzed. | Scores on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Aberrant Behavior Checklist (ABC) | Week 14 (End of Treatment) | ITT Population. Only those participants with data available at the specified data points were analyzed. | Scores on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Swanson, Nolan, and Pelham-IV Questionnaire (SNAP-IV) | Week 14 (End of Treatment) | ITT Population. Only those participants with data available at the specified data points were analyzed. | Scores on a scale | LEAST_SQUARES_MEAN |
Source ClinicalTrials.gov
Results by group38
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Clinical Global Impression-Improvement (CGI-I) Scale | Ganaxolone | 3.4 | Standard Error 0.13 | Scores on a scale | |
| Clinical Global Impression-Improvement (CGI-I) Scale | Placebo | 3.5 | Standard Error 0.13 | Scores on a scale | |
| Pediatric Anxiety Rating Scale (PARS) Total Score | Ganaxolone | 8.3 | Standard Error 0.54 | Scores on a scale | |
| Pediatric Anxiety Rating Scale (PARS) Total Score | Placebo | 9.2 | Standard Error 0.54 | Scores on a scale | |
| Visual Analogue Scale (VAS) | Ganaxolone | Severity of Anxiety | 5.6 | Standard Error 0.27 | Centimeters (cm) |
| Visual Analogue Scale (VAS) | Placebo | Severity of Anxiety | 5.0 | Standard Error 0.26 | Centimeters (cm) |
| Visual Analogue Scale (VAS) | Ganaxolone | Severity of Attention | 4.4 | Standard Error 0.24 | Centimeters (cm) |
| Visual Analogue Scale (VAS) | Placebo | Severity of Attention | 3.9 | Standard Error 0.24 | Centimeters (cm) |
| Visual Analogue Scale (VAS) | Ganaxolone | Severity of Target Behavior 3 | 4.4 | Standard Error 0.28 | Centimeters (cm) |
| Visual Analogue Scale (VAS) | Placebo | Severity of Target Behavior 3 | 3.9 | Standard Error 0.27 | Centimeters (cm) |
1 to 10 of 38Source ClinicalTrials.gov
See all 38 results by groupParticipant flow18
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Treatment Period 1 (Week 1 to Week 6) | STARTED | Ganaxolone, Then Placebo | 30 | |
| Treatment Period 1 (Week 1 to Week 6) | STARTED | Placebo, Then Ganaxolone | 29 | |
| Treatment Period 1 (Week 1 to Week 6) | COMPLETED | Ganaxolone, Then Placebo | 26 | |
| Treatment Period 1 (Week 1 to Week 6) | COMPLETED | Placebo, Then Ganaxolone | 29 | |
| Treatment Period 1 (Week 1 to Week 6) | NOT COMPLETED | Ganaxolone, Then Placebo | 4 | |
| Treatment Period 1 (Week 1 to Week 6) | NOT COMPLETED | Placebo, Then Ganaxolone | 0 | |
| Washout Period (2 Weeks) | STARTED | Ganaxolone, Then Placebo | 26 | |
| Washout Period (2 Weeks) | STARTED | Placebo, Then Ganaxolone | 29 | |
| Washout Period (2 Weeks) | COMPLETED | Ganaxolone, Then Placebo | 26 | |
| Washout Period (2 Weeks) | COMPLETED | Placebo, Then Ganaxolone | 29 |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 participant flowAdverse events210
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 | Fatigue | General disorders | Ganaxolone | 29 | 59 | |
| other | Fatigue | General disorders | Placebo | 11 | 54 | |
| other | Condition aggravated | General disorders | Ganaxolone | 10 | 59 | |
| other | Condition aggravated | General disorders | Placebo | 6 | 54 | |
| other | Somnolence | Nervous system disorders | Ganaxolone | 20 | 59 | |
| other | Somnolence | Nervous system disorders | Placebo | 3 | 54 | |
| other | Headache | Nervous system disorders | Ganaxolone | 2 | 59 | |
| other | Headache | Nervous system disorders | Placebo | 6 | 54 | |
| other | Diarrhoea | Gastrointestinal disorders | Ganaxolone | 8 | 59 | |
| other | Diarrhoea | Gastrointestinal disorders | Placebo | 7 | 54 |
1 to 10 of 210Source ClinicalTrials.gov
See all 210 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Ganaxolone | 0 | 59 |
| Total, all-cause mortality | Placebo | 0 | 54 |
| Total, other adverse events | Ganaxolone | 49 | 59 |
| Total, other adverse events | Placebo | 46 | 54 |
| Total, serious adverse events | Ganaxolone | 0 | 59 |
| Total, serious adverse events | Placebo | 0 | 54 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 28764646 | DERIVED |
Source PubMed
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 comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Ganaxolone vs Placebo: deaths from any cause | Trial comparisons |
| Ganaxolone 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/NCT01725152
GET https://conductscience.org/api/trials/v1/NCT01725152/results?page=2