Ecopipam Treatment of Self-Injurious Behavior in Subjects With Lesch-Nyhan Disease
| Status: | Terminated |
|---|---|
| Conditions: | Psychiatric |
| Therapuetic Areas: | Psychiatry / Psychology |
| Healthy: | No |
| Age Range: | 6 - 70 |
| Updated: | 4/21/2016 |
| Start Date: | December 2012 |
| End Date: | December 2014 |
An Outpatient Phase 3 Efficacy Study of Ecopipam (PSYRX 101) in the Symptomatic Treatment of Self-Injurious Behavior in Subjects With Lesch-Nyhan Disease
The purpose of this research study is to gather scientific information about the
effectiveness and safety of the study drug, Ecopipam (PSYRX 101), for the treatment of
self-injurious behaviors when compared with the effectiveness and safety of placebo
(inactive substance) in subjects with Lesch-Nyhan Disease.
effectiveness and safety of the study drug, Ecopipam (PSYRX 101), for the treatment of
self-injurious behaviors when compared with the effectiveness and safety of placebo
(inactive substance) in subjects with Lesch-Nyhan Disease.
This study will be done in approximately 6 centers in approximately 4 countries, and
approximately 24 subjects will be included. This study is divided into two parts. The first
is a double-blinded portion lasting up to 18 weeks in total. The second portion is an
optional open-label extension and lasts up to 54 weeks total. The total duration of the
study, if you choose to participate in both portions, is anticipated to be up to
approximately 78 weeks.
The first portion of this study is double-blind and assignment to a treatment group is done
randomly. In this study, there are two treatment groups. One group will receive Ecopipam for
one 6-week period and placebo for two 6-week periods, and the other group will receive
Ecopipam for two 6-week periods and placebo for one 6-week period.
Subjects who did not experience any clinically significant side effects during the blinded
portion of the study may be eligible to participate in an open-label extension that may last
up to 54 weeks.
approximately 24 subjects will be included. This study is divided into two parts. The first
is a double-blinded portion lasting up to 18 weeks in total. The second portion is an
optional open-label extension and lasts up to 54 weeks total. The total duration of the
study, if you choose to participate in both portions, is anticipated to be up to
approximately 78 weeks.
The first portion of this study is double-blind and assignment to a treatment group is done
randomly. In this study, there are two treatment groups. One group will receive Ecopipam for
one 6-week period and placebo for two 6-week periods, and the other group will receive
Ecopipam for two 6-week periods and placebo for one 6-week period.
Subjects who did not experience any clinically significant side effects during the blinded
portion of the study may be eligible to participate in an open-label extension that may last
up to 54 weeks.
Inclusion Criteria:
- Subjects must have classic LND as defined by (a) characteristic clinical syndrome
(evidence of overproduction of uric acid, severe generalized dystonia, frequent and
persistent self-injurious behavior (SIB), and cognitive impairment) and (b)
laboratory confirmation for mutation of the HPRT gene or severe deficiency of the
associated enzyme.
- Subjects must have a minimum combined score of 20 on the Behavior Problems Inventory
(BPI) SIB subscales for frequency and severity as assessed by the caregiver.
- Subjects must have a minimum score of 4 on the Physician's Global Impression (PGI)
severity scale.
- Subject must be ≥ 6 years old.
- Subjects must weigh > 10 kg.
Exclusion Criteria:
- Subjects who are currently treated with medications for seizures.
- Subjects who are on neuroleptics or dopamine-depleting agents.
- Subjects with impaired renal function as defined by a serum creatinine >1.5 mg/dL.
We found this trial at
2
sites
