Trial · NCT02124083

Phase 1/2 Study of Vorinostat Therapy in Niemann-Pick Disease, Type C1

NCT02124083, Phase 1, Phase 2, Completed, Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD); results first posted 2018-02-22

3 sourcestrials-2026-11-12-0.5.0

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ConductScience Library. Phase 1/2 Study of Vorinostat Therapy in Niemann-Pick Disease, Type C1. Trials, record NCT02124083. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02124083

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Phase 1/2 Study of Vorinostat Therapy in Niemann-Pick Disease, Type C1
NCT number: NCT02124083
Status: Completed
Phases: Phase 1, Phase 2
Enrollment: 12 participants, actual
Lead sponsor: Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Conditions: Neimann-Pick Disease
Interventions: Vorinostat
First posted: 2014-04-28
Last updated: 2018-02-22
Results first posted: 2018-02-22

Source ClinicalTrials.gov

Registered outcomes6

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Number of Participants With Tolerabilty of 200 mg Vorinostat in Niemann-Pick Disease, Type C1	3 months	The number of Niemann-Pick Disease, type C1 patients completing 3 month 200 mg phase
Primary	Number of Participants With Tolerabilty of 400 mg Vorinostat in Niemann-Pick Disease, Type C1	3 months	The number of Niemann-Pick Disease, type C1 patients completing 3 month 400 mg phase
Secondary	Biochemical Efficacy as Measured by Serum Cathepsin D	6 months	Serum concentration of Cathepsin D. Cathepsin D is an aspartyl protease involved in protein catabolism and tissue remodeling. Cathepsin D normal range = 220-515 ng/ml.
Secondary	Biochemical Efficacy as Measured by Serum Cathepsin D	Baseline	Serum concentration of Cathepsin D. Cathepsin D is an aspartyl protease involved in protein catabolism and tissue remodeling. Cathepsin D normal range = 220-515 ng/ml.
Secondary	Biochemical Efficacy as Measured by Serum LGALS3	6 months	Serum concentration of LGALS3 (galectin-3). Galectin-3 is a carbohydrate-binding lectin whose expression is associated with inflammatory cells including macrophages, neutrophils, and mast cells. LGALS3 normal range = 1.4-5.3 ng/ml.
Secondary	Biochemical Efficacy as Measured by Serum LGALS3	Baseline	Serum concentration of LGALS3 (galectin-3). Galectin-3 is a carbohydrate-binding lectin whose expression is associated with inflammatory cells including macrophages, neutrophils, and mast cells. LGALS3 normal range = 1.4-5.3 ng/ml.

Source ClinicalTrials.gov

Outcomes6

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
SECONDARY	Biochemical Efficacy as Measured by Serum Cathepsin D	6 months	All participants completing both 200 and 400 mg phases	ng/mL	MEAN
SECONDARY	Biochemical Efficacy as Measured by Serum Cathepsin D	Baseline	All participants who started study	ng/mL	MEAN
SECONDARY	Biochemical Efficacy as Measured by Serum LGALS3	6 months	All participants completing both 200 and 400 mg phases	ng/mL	MEAN
SECONDARY	Biochemical Efficacy as Measured by Serum LGALS3	Baseline	All participants who started study	ng/mL	MEAN
PRIMARY	Number of Participants With Tolerabilty of 200 mg Vorinostat in Niemann-Pick Disease, Type C1	3 months	All participants who started study	Participants	NUMBER
PRIMARY	Number of Participants With Tolerabilty of 400 mg Vorinostat in Niemann-Pick Disease, Type C1	3 months	All participants completing 200 mg phase and starting 400 mg phase	Participants	NUMBER

Source ClinicalTrials.gov

Results by group6

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Value	Spread	Units
Biochemical Efficacy as Measured by Serum Cathepsin D	Vorinostat	625.3	Standard Deviation 183.8	ng/mL
Biochemical Efficacy as Measured by Serum Cathepsin D	Vorinostat	648.2	Standard Deviation 266	ng/mL
Biochemical Efficacy as Measured by Serum LGALS3	Vorinostat	6.565	Standard Deviation 4.775	ng/mL
Biochemical Efficacy as Measured by Serum LGALS3	Vorinostat	6.667	Standard Deviation 2.303	ng/mL
Number of Participants With Tolerabilty of 200 mg Vorinostat in Niemann-Pick Disease, Type C1	Vorinostat	12		Participants
Number of Participants With Tolerabilty of 400 mg Vorinostat in Niemann-Pick Disease, Type C1	Vorinostat	11		Participants

Source ClinicalTrials.gov

Participant flow3

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	Vorinostat	12
Overall Study	COMPLETED	Vorinostat	11
Overall Study	NOT COMPLETED	Vorinostat	1

Source ClinicalTrials.gov

Adverse events46

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
other	Tympanic membrane perforation	Ear and labyrinth disorders	Vorinostat	1	12
other	Constipation	Gastrointestinal disorders	Vorinostat	1	12
other	Dry mouth	Gastrointestinal disorders	Vorinostat	2	12
other	Dysphagia	Gastrointestinal disorders	Vorinostat	1	12
other	Gastrostomy tube site complication	Gastrointestinal disorders	Vorinostat	1	12
other	Reflux gastritis	Gastrointestinal disorders	Vorinostat	1	12
other	Salivary hypersecretion	Gastrointestinal disorders	Vorinostat	1	12
other	Vomiting	Gastrointestinal disorders	Vorinostat	2	12
other	Fatigue	General disorders	Vorinostat	4	12
other	Irritability	General disorders	Vorinostat	1	12

1 to 10 of 46Source ClinicalTrials.gov

See all 46 adverse events

Adverse event totals3

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Vorinostat	0	12
Total, other adverse events	Vorinostat	11	12
Total, serious adverse events	Vorinostat	5	12

Source ClinicalTrials.gov

Papers4

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as	Source
11111100	BACKGROUND	PubMed
17003072	BACKGROUND	PubMed
23622394	BACKGROUND	PubMed
34389161	DERIVED	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 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.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

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/NCT02124083

GET https://conductscience.org/api/trials/v1/NCT02124083/events?page=2

API reference For AI assistants