Phase 1 N=5 Treatment

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

Lesch-Nyhan Disease

Bottom Line

View on ClinicalTrials.gov: NCT01065558 ↗

Enrolled (actual)

Serious AEs

40.0%

Results posted

Apr 2013

Primary outcome: Primary: Number of Participants With Clinically Significant Changes in Standard Laboratory Tests — 0 Participants

Study Design & Population

Study type: Interventional
Phase: Phase 1
Interventions: Ecopipam (Drug)
Age: Pediatric, Adult, Older Adult · 6+ yrs
Sex: Male
Sponsor: Psyadon Pharma
Primary completion: Jan 2011

Outcome Measures

Outcome	Result	p-value
PRIMARY Number of Participants With Clinically Significant Changes in Standard Laboratory Tests	—	—
SECONDARY Decrease in Self-injurious Behavior at End of Study (Two Weeks After Screening) Compared to Screening	22.5	—

Summary

The purpose of this study is to determine the effects of ecopipam in patients with Lesch-Nyhan Disease (LND). Ecopipam specifically blocks the actions of one chemical in the brain that helps nerves talk to one another; ecopipam does this by stopping the chemical (dopamine) from binding to one of its family of receptors (i.e, the D1 family). LND is a very rare genetic disease. The sponsor is doing this study to find out what side effects ecopipam causes in LND patients, and whether ecopipam may be able to relieve the self-injurious behaviors seen in these patients.

Eligibility Criteria

Inclusion Criteria

Diagnosis of Lesch-Nyhan disease with moderate to severe Self- Injurious Behavior and are routinely restrained
Male - 6 years of age and above
Body weight greater than or equal to 44 pounds

Exclusion Criteria

Taking Neuroleptic or Dopamine Depleting medications
Currently treated with medications for depression or seizures
Impaired renal function
Medical conditions that may interfere with completing the study

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT01065558). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.