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

P

Psyadon Pharma

Status and phase

Completed
Phase 1

Conditions

Lesch-Nyhan Disease

Treatments

Drug: Ecopipam

Study type

Interventional

Funder types

Industry

Identifiers

NCT01065558
Ecopipam and Lesch-Nyhan

Details and patient eligibility

About

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.

Enrollment

5 patients

Sex

Male

Ages

6 to 65 years old

Volunteers

No Healthy Volunteers

Inclusion and exclusion 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

Trial design

Primary purpose

Treatment

Allocation

N/A

Interventional model

Single Group Assignment

Masking

None (Open label)

5 participants in 1 patient group

Ecopipam 12.5 - 200 mg/day
Experimental group
Description:
Patients were administered ecopipam on an escalated dosing schedule over 11 days starting at 12.5 mg/day and increasing to the maximal tolerated dose or to 200 mg/day.
Treatment:
Drug: Ecopipam

Trial contacts and locations

2

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Data sourced from clinicaltrials.gov

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