Trial · NCT03643562

Niemann-Pick Type C Treatment With Adrabetadex for Symptoms of Brain and Nervous System

NCT03643562, Phase 3, Terminated, Mandos LLC; results first posted 2024-01-17

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Niemann-Pick Type C Treatment With Adrabetadex for Symptoms of Brain and Nervous System. Trials, record NCT03643562. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03643562

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Trial

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

Title: Niemann-Pick Type C Treatment With Adrabetadex for Symptoms of Brain and Nervous System
NCT number: NCT03643562
Status: Terminated
Phases: Phase 3
Enrollment: 27 participants, actual
Lead sponsor: Mandos LLC
Conditions: Niemann-Pick Type C Disease
Interventions: Adrabetadex
First posted: 2018-08-23
Last updated: 2024-01-17
Results first posted: 2024-01-17
Limitations and caveats: This study was terminated early when the Sponsor had determined that the benefit-risk profile of adrabetadex was not favorable and the clinical development program for adrabetadex was discontinued.

Source ClinicalTrials.gov

Registered outcomes1

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

List	Outcome	Time frame	Description
Primary	Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)	Baseline up to Week 134	An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Serious adverse events (SAEs) were defined as death, a life-threatening AE, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or incapacity, a congenital anomaly or birth defect, or an important medical event that jeopardized participant and required medical intervention to prevent 1 of the outcomes listed in this definition. A TEAE was defined as an AE with onset on or after the start of adrabetadex treatment. A summary of other non-serious AEs and all serious AEs, regardless of causality is located in Reported AE section.

Source ClinicalTrials.gov

Outcomes1

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

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)	Baseline up to Week 134	All enrolled participants	Participants	COUNT_OF_PARTICIPANTS

Source ClinicalTrials.gov

Results by group4

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

Outcome	Group	Category	Value	Units
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)	Treatment Naive	All TEAEs	9	Participants
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)	Previously Treated	All TEAEs	17	Participants
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)	Treatment Naive	SAEs	5	Participants
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)	Previously Treated	SAEs	9	Participants

Source ClinicalTrials.gov

Participant flow8

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

Period	Milestone	Group	Participants
Overall Study	STARTED	Treatment Naive	9
Overall Study	STARTED	Previously Treated	18
Overall Study	Received at Least 1 Dose of Study Drug	Treatment Naive	9
Overall Study	Received at Least 1 Dose of Study Drug	Previously Treated	18
Overall Study	COMPLETED	Treatment Naive	1
Overall Study	COMPLETED	Previously Treated	2
Overall Study	NOT COMPLETED	Treatment Naive	8
Overall Study	NOT COMPLETED	Previously Treated	16

Source ClinicalTrials.gov

Adverse events260

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	Tachycardia	Cardiac disorders	Treatment Naive	0	9
other	Tachycardia	Cardiac disorders	Previously Treated	1	18
other	Deafness	Ear and labyrinth disorders	Treatment Naive	0	9
other	Deafness	Ear and labyrinth disorders	Previously Treated	2	18
other	Deafness neurosensory	Ear and labyrinth disorders	Treatment Naive	0	9
other	Deafness neurosensory	Ear and labyrinth disorders	Previously Treated	2	18
other	Hypoacusis	Ear and labyrinth disorders	Treatment Naive	2	9
other	Hypoacusis	Ear and labyrinth disorders	Previously Treated	4	18
other	Middle ear effusion	Ear and labyrinth disorders	Treatment Naive	0	9
other	Middle ear effusion	Ear and labyrinth disorders	Previously Treated	1	18

1 to 10 of 260Source ClinicalTrials.gov

See all 260 adverse events

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Treatment Naive	0	9
Total, all-cause mortality	Previously Treated	0	18
Total, other adverse events	Treatment Naive	9	9
Total, other adverse events	Previously Treated	17	18
Total, serious adverse events	Treatment Naive	5	9
Total, serious adverse events	Previously Treated	9	18

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 comparisons2

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

Comparison	Source
Treatment Naive vs Previously Treated: deaths from any cause	Trial comparisons
Treatment Naive vs Previously Treated: 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.

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/NCT03643562

GET https://conductscience.org/api/trials/v1/NCT03643562/events?page=2

API reference For AI assistants