Trial · NCT04958642

Adrabetadex to Treat Niemann-Pick Type C1 (NPC1) Disease

NCT04958642, Phase 2, Phase 3, Terminated, Mandos LLC; results first posted 2023-08-29

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Adrabetadex to Treat Niemann-Pick Type C1 (NPC1) Disease. Trials, record NCT04958642. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04958642

On this page

Trial

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

Title: Adrabetadex to Treat Niemann-Pick Type C1 (NPC1) Disease
NCT number: NCT04958642
Status: Terminated
Phases: Phase 2, Phase 3
Enrollment: 66 participants, actual
Lead sponsor: Mandos LLC
Conditions: Niemann-Pick Disease, Type C
Interventions: Adrabetadex
First posted: 2021-07-12
Last updated: 2023-08-29
Other registries' IDs: eudract:2015-002548-15
Results first posted: 2023-08-29
Limitations and caveats: The development program for adrabetadex was discontinued and Part C of the study was terminated by the Sponsor.

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 5 years	An 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 5 years	The Open-label population included participants who received at least 1 dose of adrabetadex during Part C.	Participants	COUNT_OF_PARTICIPANTS

Source ClinicalTrials.gov

Results by group6

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	Any TEAEs	18	Participants
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)	Previously Treated in Phase I	Any TEAEs	13	Participants
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)	Previously Treated in Part A/B	Any TEAEs	34	Participants
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)	Treatment Naive	SAEs	9	Participants
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)	Previously Treated in Phase I	SAEs	7	Participants
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)	Previously Treated in Part A/B	SAEs	20	Participants

Source ClinicalTrials.gov

Participant flow12

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

Period	Milestone	Group	Participants
Overall Study	STARTED	Treatment Naive	18
Overall Study	STARTED	Previously Treated in Phase I	13
Overall Study	STARTED	Previously Treated in Part A/B	35
Overall Study	Received at Least 1 Dose of Study Drug	Treatment Naive	18
Overall Study	Received at Least 1 Dose of Study Drug	Previously Treated in Phase I	13
Overall Study	Received at Least 1 Dose of Study Drug	Previously Treated in Part A/B	35
Overall Study	COMPLETED	Treatment Naive	0
Overall Study	COMPLETED	Previously Treated in Phase I	0
Overall Study	COMPLETED	Previously Treated in Part A/B	0
Overall Study	NOT COMPLETED	Treatment Naive	18

1 to 10 of 12Source ClinicalTrials.gov

See all 12 participant flow

Adverse events978

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	Events
other	Iron deficiency anaemia	Blood and lymphatic system disorders	Treatment Naive	0	18	0
other	Iron deficiency anaemia	Blood and lymphatic system disorders	Previously Treated in Phase I	1	13	1
other	Iron deficiency anaemia	Blood and lymphatic system disorders	Previously Treated in Part A/B	0	35	0
other	Leukopenia	Blood and lymphatic system disorders	Treatment Naive	0	18	0
other	Leukopenia	Blood and lymphatic system disorders	Previously Treated in Phase I	1	13	1
other	Leukopenia	Blood and lymphatic system disorders	Previously Treated in Part A/B	0	35	0
other	Thrombocytopenia	Blood and lymphatic system disorders	Treatment Naive	1	18	1
other	Thrombocytopenia	Blood and lymphatic system disorders	Previously Treated in Phase I	0	13	0
other	Thrombocytopenia	Blood and lymphatic system disorders	Previously Treated in Part A/B	1	35	2
other	Palpitations	Cardiac disorders	Treatment Naive	0	18	0

1 to 10 of 978Source ClinicalTrials.gov

See all 978 adverse events

Adverse event totals9

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Treatment Naive	1	18
Total, all-cause mortality	Previously Treated in Phase I	0	13
Total, all-cause mortality	Previously Treated in Part A/B	1	35
Total, other adverse events	Treatment Naive	18	18
Total, other adverse events	Previously Treated in Phase I	13	13
Total, other adverse events	Previously Treated in Part A/B	34	35
Total, serious adverse events	Treatment Naive	9	18
Total, serious adverse events	Previously Treated in Phase I	7	13
Total, serious adverse events	Previously Treated in Part A/B	20	35

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.

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/NCT04958642

GET https://conductscience.org/api/trials/v1/NCT04958642/events?page=2

API reference For AI assistants