Trial · NCT02603926
Treatment of Fragile-X Associated Tremor/Ataxia Syndrome (FXTAS) With Allopregnanolone
NCT02603926, Phase 2, Completed, Randi J. Hagerman, MD; results first posted 2018-12-06
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Treatment of Fragile-X Associated Tremor/Ataxia Syndrome (FXTAS) With Allopregnanolone. Trials, record NCT02603926. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02603926
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Treatment of Fragile-X Associated Tremor/Ataxia Syndrome (FXTAS) With Allopregnanolone
- NCT number
- NCT02603926
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 6 participants, actual
- Lead sponsor
- Randi J. Hagerman, MD
- Conditions
- Fragile X-associated Tremor/Ataxia Syndrome
- Interventions
- Allopregnanolone
- First posted
- 2015-11-13
- Last updated
- 2018-12-06
- Results first posted
- 2018-12-06
- Limitations and caveats
This preliminary trial was conducted to obtain evidence of treatment benefits in a small sample, and thus the study was not powered to detect statistical significance while controlling for multiple comparisons. There was also no placebo group.
Source ClinicalTrials.gov
Registered outcomes4
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | California Verbal Learning Test II (CVLT2) Trial 1-5 Free Recall Total Raw Score | Baseline/pre-treatment and 14 weeks/post-treatment | California Verbal Learning Test II (CVLT2) is an assessment measuring working memory. Trials 1-5 measure the total number of words remembered after 5 repeated trials and are summed to generate a raw score (called Trial 1-5 Free Recall Total Raw Score) ranging from 0 to 80, with higher scores reflecting better working memory. Mean and standard deviation for raw score at baseline/pre-treatment and at 14 weeks/post-treatment are presented here. |
| Secondary | Behavioral Dyscontrol Scale - 2 (BDS-2) Total Score | Baseline/pre-treatment and 14 weeks/post-treatment | The BDS-2 is a validated 9-item assessment measuring the ability to regulate purposeful, goal-directed activity and to engage in activities of daily living, with focus on motor items. Each of the 9 items is scored on a scale of 0 to 3, resulting in a summed total score ranging from 0 to 27. Higher scores reflect fewer errors and stronger ability to regulate motor activities. Mean and standard deviation for total score at baseline/pre-treatment and at 14 weeks/post-treatment are presented here. |
| Secondary | CATSYS Dot-to-Dot Tremor Intensity (CATSYS DTD TI) | Baseline/pre-treatment and 14 weeks/post-treatment | The CATSYS system is a portable device recording various measures of neuromotor control, including tremor. The CATSYS Dot-to-Dot Tremor Intensity (DTD TI) protocol quantifies tremor by having a participant hold a tremor pen as they would an ordinary pen, with the elbow joint bent at a right angle and free of body contact, and the pen positioned approximately 4 inches from the navel. Subjects are instructed to use the pen first to tap the center of two circular stickers, approximately 0.5 inch in diameter, placed on opposite ends of the bottom portion of the computer monitor; then, subjects are instructed to trace a line across the table using the tremor pen. The pen is connected to a computer with sensors that measure tremor intensity (TI) in units of meters per second (m/s). Larger values reflect greater tremor intensity. Mean right-hand and left-hand TI and standard deviation at baseline/pre-treatment and at 14 weeks/post-treatment are reported here. |
| Secondary | Hippocampal Volume, as Measured by Structural MRI | Baseline/pre-treatment and 14 weeks/post-treatment | Patients will undergo structural Magnetic Resonance Imaging (MRI) at baseline/pre-treatment and at 14 weeks/post-treatment. The MRI is interpreted by a trained clinician and hippocampal volume in cubic centimeters is measured and recorded. Larger values reflect greater volumes of the hippocampus, and greater hippocampal volume post-treatment may be indicative of increased neurogenesis. Mean hippocampal volume and standard deviation at baseline/pre-treatment and post-treatment is reported here. |
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 | California Verbal Learning Test II (CVLT2) Trial 1-5 Free Recall Total Raw Score | Baseline/pre-treatment and 14 weeks/post-treatment | Primary outcome measure (CVLT2) was completed for all enrolled subjects. | score on a scale | MEAN |
| SECONDARY | Behavioral Dyscontrol Scale - 2 (BDS-2) Total Score | Baseline/pre-treatment and 14 weeks/post-treatment | BDS-2 was completed for all enrolled subjects. | score on a scale | MEAN |
| SECONDARY | CATSYS Dot-to-Dot Tremor Intensity (CATSYS DTD TI) | Baseline/pre-treatment and 14 weeks/post-treatment | CATSYS DTD TI was completed by all subjects. | meters per second (m/s) | MEAN |
| SECONDARY | Hippocampal Volume, as Measured by Structural MRI | Baseline/pre-treatment and 14 weeks/post-treatment | 1 subject was unable to undergo structural MRI procedure at baseline and post-treatment. | cubic centimeters | MEAN |
Source ClinicalTrials.gov
Results by group10
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| California Verbal Learning Test II (CVLT2) Trial 1-5 Free Recall Total Raw Score | Allopregnanolone | Baseline/Pre-Treatment | 34.0 | Standard Deviation 7.5 | score on a scale |
| California Verbal Learning Test II (CVLT2) Trial 1-5 Free Recall Total Raw Score | Allopregnanolone | 14 Weeks/Post-Treatment | 33.5 | Standard Deviation 6.3 | score on a scale |
| Behavioral Dyscontrol Scale - 2 (BDS-2) Total Score | Allopregnanolone | Baseline/Pre-Treatment | 12.7 | Standard Deviation 3.1 | score on a scale |
| Behavioral Dyscontrol Scale - 2 (BDS-2) Total Score | Allopregnanolone | 14 Weeks/Post-Treatment | 15.3 | Standard Deviation 3.8 | score on a scale |
| CATSYS Dot-to-Dot Tremor Intensity (CATSYS DTD TI) | Allopregnanolone | Baseline/Pre-Treatment, Right-Hand | 0.87 | Standard Deviation 0.49 | meters per second (m/s) |
| CATSYS Dot-to-Dot Tremor Intensity (CATSYS DTD TI) | Allopregnanolone | Baseline/Pre-Treatment, Left-Hand | 0.74 | Standard Deviation 0.29 | meters per second (m/s) |
| CATSYS Dot-to-Dot Tremor Intensity (CATSYS DTD TI) | Allopregnanolone | 14 Weeks/Post-Treatment, Right-Hand | 0.79 | Standard Deviation 0.59 | meters per second (m/s) |
| CATSYS Dot-to-Dot Tremor Intensity (CATSYS DTD TI) | Allopregnanolone | 14 Weeks/Post-Treatment, Left-Hand | 0.65 | Standard Deviation 0.26 | meters per second (m/s) |
| Hippocampal Volume, as Measured by Structural MRI | Allopregnanolone | Baseline/Pre-Treatment | 5.87 | Standard Deviation 1.00 | cubic centimeters |
| Hippocampal Volume, as Measured by Structural MRI | Allopregnanolone | 14 Weeks/Post-Treatment | 5.84 | Standard Deviation 1.18 | cubic centimeters |
Source ClinicalTrials.gov
Participant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Allopregnanolone | 6 | |
| Overall Study | COMPLETED | Allopregnanolone | 6 | |
| Overall Study | NOT COMPLETED | Allopregnanolone | 0 |
Source ClinicalTrials.gov
Adverse events0
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
None in this release.
Adverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Allopregnanolone | 0 | 6 |
| Total, other adverse events | Allopregnanolone | 0 | 6 |
| Total, serious adverse events | Allopregnanolone | 0 | 6 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
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 comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests1
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
| Test | Named as |
|---|---|
| Verbal Learning | Primary outcome |
Source ConductCognition
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/NCT02603926
GET https://conductscience.org/api/trials/v1/NCT02603926/registered