Trial Outcomes & Findings for Safety and Tolerability of the D1 Dopamine Receptor Antagonist Ecopipam in Patients With Lesch-Nyhan Disease (NCT NCT01065558)

Results Overview

This study's primary outcome is the safety of ecopipam in Lesch-Nyhan patients as measured by standard clinical laboratory tests. The patients will also be observed and questioned about other side effects, such as whether they feel more or less tired.Standard clinical laboratory tests for liver, kidney and blood function were conducted. The normal ranges for each of these tests were different and are too numerous to be individually listed here. However, if any individual value were to be either three-times greater or lesser than the upper or the lower limit of the test, then that value was considered to have been changed.

Recruitment status

COMPLETED

Study phase

PHASE1

Target enrollment

5 participants

Primary outcome timeframe

Two weeks

Results posted on

2013-04-18

Participant Flow

Patients were recruited by the investigators according to the inclusion and exclusion criteria

No preassignment

Participant milestones

Participant milestones
Measure	Ecopipam (12.5- 200 mg/Day) Patients were given ecopipapm over an 11 day period as follows: day 1 12.5 mg/day day 2-3 25 mg/day day 4-5 50 mg/day day 6-9 100 mg/day day 9-11 200 mg/day Note: Doses were reduced as necessary to a previously tolerated dose if paitents reached doses that were not safely tolerable.
Overall Study STARTED	5
Overall Study COMPLETED	5
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Safety and Tolerability of the D1 Dopamine Receptor Antagonist Ecopipam in Patients With Lesch-Nyhan Disease

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Ecopipam n=5 Participants
Age, Categorical <=18 years	4 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	1 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age Continuous	20.4 years STANDARD_DEVIATION 17.9 • n=5 Participants
Sex: Female, Male Female	0 Participants n=5 Participants
Sex: Female, Male Male	5 Participants n=5 Participants
Region of Enrollment United States	5 participants n=5 Participants

PRIMARY outcome

Timeframe: Two weeks

Population: All patients were analyzed

This study's primary outcome is the safety of ecopipam in Lesch-Nyhan patients as measured by standard clinical laboratory tests. The patients will also be observed and questioned about other side effects, such as whether they feel more or less tired.Standard clinical laboratory tests for liver, kidney and blood function were conducted. The normal ranges for each of these tests were different and are too numerous to be individually listed here. However, if any individual value were to be either three-times greater or lesser than the upper or the lower limit of the test, then that value was considered to have been changed.

Outcome measures

Outcome measures
Measure	Ecopipam Treated Patients n=5 Participants These were patients who had diagnoses Lesch-Nyhan Disease based either on their genetic changes or on changes of specific enzymes in their blood.
Number of Participants With Clinically Significant Changes in Standard Laboratory Tests	0 Participants

SECONDARY outcome

Timeframe: Screening visit and end of study (two weeks)

Population: All patients who received any dose of ecopipam were in the analysis population

Change in the self-injurious subscale of the Behavior Problems Inventory (BPI). the BPI is a well-validate test to evaluate the frequency and severity of a patient's self-injurious behavior. Values range from 0 to 50, and a low score means few/less severe behaviors

Outcome measures

Outcome measures
Measure	Ecopipam Treated Patients n=5 Participants These were patients who had diagnoses Lesch-Nyhan Disease based either on their genetic changes or on changes of specific enzymes in their blood.
Decrease in Self-injurious Behavior at End of Study (Two Weeks After Screening) Compared to Screening	22.5 Change in BPI score Standard Deviation 13.4

Adverse Events

Ecopipam Treated Patients

Serious events: 2 serious events

Other events: 5 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Ecopipam Treated Patients n=5 participants at risk
Renal and urinary disorders Flank Pain	20.0% 1/5 • Number of events 1 • 12 days of active treatment and 28 days follow-up
Infections and infestations Respiratory Infection	20.0% 1/5 • Number of events 1 • 12 days of active treatment and 28 days follow-up

Other adverse events

Other adverse events
Measure	Ecopipam Treated Patients n=5 participants at risk
Nervous system disorders Sedation	60.0% 3/5 • Number of events 3 • 12 days of active treatment and 28 days follow-up
Nervous system disorders Dystonia	40.0% 2/5 • Number of events 2 • 12 days of active treatment and 28 days follow-up
Gastrointestinal disorders Nausea	40.0% 2/5 • Number of events 2 • 12 days of active treatment and 28 days follow-up

Additional Information

Dr. Richard Chipkin

Psyadon Pharmaceuticals

Phone: 301-919-2020

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: LTE60