Trial · NCT01751802

Ecopipam Treatment of Self-Injurious Behavior in Subjects With Lesch-Nyhan Disease

NCT01751802, Phase 3, Terminated, Emalex Biosciences Inc.; results first posted 2015-10-08

3 sourcestrials-2026-11-12-0.4.0

Cite this record

ConductScience Library. Ecopipam Treatment of Self-Injurious Behavior in Subjects With Lesch-Nyhan Disease. Trials, record NCT01751802. Release trials-2026-11-12-0.4.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01751802

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Trial

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

Title: Ecopipam Treatment of Self-Injurious Behavior in Subjects With Lesch-Nyhan Disease
NCT number: NCT01751802
Status: Terminated
Phases: Phase 3
Enrollment: 9 participants, actual
Lead sponsor: Emalex Biosciences Inc.
Conditions: Lesch-Nyhan Disease, Self-injurious Behavior
Interventions: Ecopipam, Placebo
First posted: 2012-12-18
Last updated: 2024-04-22
Results first posted: 2015-10-08

Source ClinicalTrials.gov

Registered outcomes3

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

List	Outcome	Time frame	Description
Primary	Behavior Problems Inventory - Self-Injurious Behavior Subscale	Baseline, end of period 1 (6 weeks), end of period 2 (12 weeks), end of period 3 (18 weeks),	The primary endpoint is the BPI (SIB subscales - total for frequency and severity) as assessed by the caregiver. BPI Self-Injurious Behavior Subscale ranges from 0 to 45, with higher scores indicating more self-injurious behavior.
Secondary	Effect of Ecopipam Withdrawal and Maintenance	Baseline, 6 weeks, 12 weeks, 18 weeks	The secondary objectives of this study are to assess the effect of withdrawal and maintenance of ecopipam's effects in subjects with LND. Measured by the number of participants whose score changes significantly from baseline on ecopipam or placebo
Secondary	Safety Summary of Ecopipam in Patients With Lesch-Nyhan Disease: Total Number of Serious and Non-Serious Adverse Events Experienced During 3 Double-blind Crossover Periods	Total duration over which participants recieved double-blind ecopipam or placebo, up to 6 or 12 weeks	An additional objective of the study is to assess the safety of ecopipam in subjects with LND for up to 52 weeks. Total number of serious and non-serious adverse events experienced by participants while receiving ecopipam or placebo. For additional detail, see Adverse Events

Source ClinicalTrials.gov

Outcomes3

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

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Behavior Problems Inventory - Self-Injurious Behavior Subscale	Baseline, end of period 1 (6 weeks), end of period 2 (12 weeks), end of period 3 (18 weeks),	All participants who completed the BPI-Self Injurious Behavior survey for 3 or more time points	scores on a scale	MEAN
SECONDARY	Effect of Ecopipam Withdrawal and Maintenance	Baseline, 6 weeks, 12 weeks, 18 weeks	0 participants analyzed because data are not reliable
SECONDARY	Safety Summary of Ecopipam in Patients With Lesch-Nyhan Disease: Total Number of Serious and Non-Serious Adverse Events Experienced During 3 Double-blind Crossover Periods	Total duration over which participants recieved double-blind ecopipam or placebo, up to 6 or 12 weeks	All participants who received at least one dose	adverse events	NUMBER

Source ClinicalTrials.gov

Results by group20

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

Outcome	Group	Category	Value	Units
Behavior Problems Inventory - Self-Injurious Behavior Subscale	Subject #1: Ecopipam Then Placebo Then Ecopipam	BPI Self-Injurious Behavior Subscale, Baseline	26	scores on a scale
Behavior Problems Inventory - Self-Injurious Behavior Subscale	Subject #2: Ecopipam Then Placebo Then Ecopipam	BPI Self-Injurious Behavior Subscale, Baseline	10	scores on a scale
Behavior Problems Inventory - Self-Injurious Behavior Subscale	Subject #3: Placebo Then Ecopipam Then Placebo	BPI Self-Injurious Behavior Subscale, Baseline	78	scores on a scale
Behavior Problems Inventory - Self-Injurious Behavior Subscale	Subject #4: Placebo Then Ecopipam Then Placebo	BPI Self-Injurious Behavior Subscale, Baseline	27	scores on a scale
Behavior Problems Inventory - Self-Injurious Behavior Subscale	Subject #1: Ecopipam Then Placebo Then Ecopipam	BPI Self-Injurious Behavior Subscale, 6 weeks	24.1	scores on a scale
Behavior Problems Inventory - Self-Injurious Behavior Subscale	Subject #2: Ecopipam Then Placebo Then Ecopipam	BPI Self-Injurious Behavior Subscale, 6 weeks	4.3	scores on a scale
Behavior Problems Inventory - Self-Injurious Behavior Subscale	Subject #3: Placebo Then Ecopipam Then Placebo	BPI Self-Injurious Behavior Subscale, 6 weeks	58.3	scores on a scale
Behavior Problems Inventory - Self-Injurious Behavior Subscale	Subject #4: Placebo Then Ecopipam Then Placebo	BPI Self-Injurious Behavior Subscale, 6 weeks	22.6	scores on a scale
Behavior Problems Inventory - Self-Injurious Behavior Subscale	Subject #1: Ecopipam Then Placebo Then Ecopipam	BPI Self-Injurious Behavior Subscale, 12 weeks	25.2	scores on a scale
Behavior Problems Inventory - Self-Injurious Behavior Subscale	Subject #2: Ecopipam Then Placebo Then Ecopipam	BPI Self-Injurious Behavior Subscale, 12 weeks	11.6	scores on a scale

1 to 10 of 20Source ClinicalTrials.gov

See all 20 results by group

Participant flow12

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

Period	Milestone	Group	Participants
Overall Study	STARTED	Ecopipam Then Placebo Then Ecopipam	5
Overall Study	STARTED	Placebo Then Ecopipam Then Placebo	4
Overall Study	Completed Period 1	Ecopipam Then Placebo Then Ecopipam	2
Overall Study	Completed Period 1	Placebo Then Ecopipam Then Placebo	4
Overall Study	Completed Period 2	Ecopipam Then Placebo Then Ecopipam	2
Overall Study	Completed Period 2	Placebo Then Ecopipam Then Placebo	1
Overall Study	Completed Period 3	Ecopipam Then Placebo Then Ecopipam	2
Overall Study	Completed Period 3	Placebo Then Ecopipam Then Placebo	1
Overall Study	COMPLETED	Ecopipam Then Placebo Then Ecopipam	1
Overall Study	COMPLETED	Placebo Then Ecopipam Then Placebo	2

1 to 10 of 12Source ClinicalTrials.gov

See all 12 participant flow

Adverse events60

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	Abdominal pain	Gastrointestinal disorders	Ecopipam	2	9	2
other	Abdominal pain	Gastrointestinal disorders	Placebo	0	6	0
other	Agitation	Psychiatric disorders	Ecopipam	2	9	2
other	Agitation	Psychiatric disorders	Placebo	0	6	0
other	Anxiety	Psychiatric disorders	Ecopipam	1	9	1
other	Anxiety	Psychiatric disorders	Placebo	1	6	1
other	Body temperature increased	Investigations	Ecopipam	1	9	1
other	Body temperature increased	Investigations	Placebo	0	6	0
other	Bronchospasm	Respiratory, thoracic and mediastinal disorders	Ecopipam	0	9	0
other	Bronchospasm	Respiratory, thoracic and mediastinal disorders	Placebo	1	6	1

1 to 10 of 60Source ClinicalTrials.gov

See all 60 adverse events

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Ecopipam
Total, all-cause mortality	Placebo
Total, other adverse events	Ecopipam	8	9
Total, other adverse events	Placebo	2	6
Total, serious adverse events	Ecopipam	2	9
Total, serious adverse events	Placebo	1	6

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 comparisons1

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

Comparison
Ecopipam vs Placebo: serious adverse events

Source 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/NCT01751802

GET https://conductscience.org/api/trials/v1/NCT01751802/events?page=2

API reference For AI assistants