Trial · NCT02185651
A Pilot Study of Zavesca® in Patients With Pompe Disease and Infusion Associated Reaction
NCT02185651, Phase 1, Terminated, University of Florida
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. A Pilot Study of Zavesca® in Patients With Pompe Disease and Infusion Associated Reaction. Trials, record NCT02185651. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02185651
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Pilot Study of Zavesca® in Patients With Pompe Disease and Infusion Associated Reaction
- NCT number
- NCT02185651
- Status
- Terminated
- Phases
- Phase 1
- Enrollment
- 2 participants, actual
- Lead sponsor
- University of Florida
- Conditions
- Pompe Disease, Hypersensitivity Reaction
- Interventions
- Zavesca® Prescription
- First posted
- 2014-07-09
- Last updated
- 2018-12-05
Source ClinicalTrials.gov
Registered outcomes3
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Evaluate Pharmacodynamics of ERT with pre-medication Zavesca | Baseline, week 0, week 6 | Change in GAA antibody level from Baseline to week 0: GAA antibody titer is reported at timepoints: pre-infusion, and 6, 12, and 24 hours post-infusion start. Change in GAA antibody level from Baseline to week 6: GAA antibody titer is reported at time points: pre-infusion and post-infusion. |
| Secondary | Evaluate pharmacokinetics of ERT with pre-medication Zavesca® | Baseline, week 0 | Change in ERT half life at baseline (without Zavesca®) compared to week 0 (ERT with pre-medication Zavesca®). Change in Maximum plasma concentration (Cmax) at baseline (without Zavesca®) compared to week 0 (ERT with pre-medication Zavesca®). |
| Secondary | Evaluate biodistribution of ERT with pre-medication Zavesca®. | Week 0, week 7 | Punch muscle biopsy is performed at week 0 and week 7 for biodistribution: Change in glycogen content and muscle fiber morphology is reported. |
Source ClinicalTrials.gov
Outcomes0
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.
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.
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/NCT02185651
GET https://conductscience.org/api/trials/v1/NCT02185651/registered