Trial · NCT02912793
Safety and Efficacy of Intravenous Trappsol Cyclo (HPBCD) in Niemann-Pick Type C Patients
NCT02912793, Phase 1, Phase 2, Completed, Cyclo Therapeutics, Inc.
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Safety and Efficacy of Intravenous Trappsol Cyclo (HPBCD) in Niemann-Pick Type C Patients. Trials, record NCT02912793. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02912793
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Safety and Efficacy of Intravenous Trappsol Cyclo (HPBCD) in Niemann-Pick Type C Patients
- NCT number
- NCT02912793
- Status
- Completed
- Phases
- Phase 1, Phase 2
- Enrollment
- 12 participants, actual
- Lead sponsor
- Cyclo Therapeutics, Inc.
- Conditions
- Niemann-Pick Disease, Type C1
- Interventions
- Hydroxypropyl-beta-cyclodextrin
- First posted
- 2016-09-23
- Last updated
- 2021-08-10
Source ClinicalTrials.gov
Registered outcomes15
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | To evaluate the plasma the Maximum Concentration (C max) of 3 doses of Trappsol by measurement of plasma levels | 0,2,4,6,& 8 hours (h) after the start of the IV infusion of Trappsol and 0.5,1,2,4,8 & 12 h after the end of the infusion | To evaluate plasma PK of Trappsol by comparison of Maximum Concentration (Cmax ) of the three doses |
| Primary | To evaluate the Time to Maximum Concentration ( Tmax) of 3 doses of Trappsol by measurement of plasma levels | 0,2,4,6,& 8h after the start of IV infusion of Trappsol and 0.5,1,2,4,6 &12h after the end of infusion | To evaluate the plasma PK of Trappsol by comparison of the Tmax of three doses |
| Primary | To evaluate the Volume of Distribution of Trappsol by measurement of plasma levels | ),2,4,6 & 8 h after the start of the IV infusion of Trappsol and 0.5,1,2,4,8,&12 h after the end of the infusion | To evaluate the plasma PK of Trappsol by comparison of the Volume of Distribution of three doses |
| Primary | To evaluate the elimination half-life of Trappsol by measurement of plasma levels | 0,2,3,6 & 8h after the start of IV infusion of Trappsol and 0.5,1,2,4,8 &12h after the end of infusion | To evaluate the PK of Trappsol by comparison of the Elimination half-lives of three doses |
| Secondary | Markers of cholesterol metabolism | Screening,Days1,2,3,5,8,Weeks 2,4,8,10,12,14,16,18,20,24,28,32,36,40,44,48 and follow-up | To investigate the effect of 3 different doses of intravenous Trappsol in patients upon serum and lymphocytic markers of cholesterol metabolism in patients with NPC-1 |
| Secondary | CSF levels of HP-β-CD | Pre then 4,8,and 12h after the start of the initial infusion | To evaluate HP-β-CD concentrations in CSF following intravenous administration of Trappsol in patients with NPC-1 to determine if the drug crosses the blood brain barrier |
| Secondary | Number of patients with treatment-related adverse events as assessed by CTCAE ( version 4.03) | Screening,Days1,2,3,4,6,8,Week 2,4,6,8,10,12,14,16,18,20,22,24,26,28,30,32,34,36,38,40,42,44,46,48 and follow-up | Events will be gathered by spontaneous reporting, clinical observation and laboratory tests including standard audiology tests and auditory evoked potential to assess hearing |
| Secondary | Abdominal ultrasound | Baseline 12,24,36 and 48 weeks | Change from baseline in hepatic and splenic morphology |
| Secondary | The proportion of patients with a reduction from baseline in the NIH NPC severity scale | Baseline and 48weeks | Reduction of one point in two or more domains |
| Secondary | Top evaluate the impact of treatment on ataxia | Screening, baseline and weeks 12,18,36 and 48 | Ataxia will be rated using the Scale for the assessment and rating of ataxia (SARA) in patients where age and cognitive function allow it. |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 registered outcomesOutcomes0
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
None in this release.
Results by group0
Each measurement the trial posted, group by group, in the words and numbers it posted.
None in this release.
Participant flow0
How many started, completed or left each group, period by period.
None in this release.
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 totals0
The totals the trial posted for each group.
None in this release.
Papers30
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 comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
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/NCT02912793
GET https://conductscience.org/api/trials/v1/NCT02912793/papers?page=2