An Outpatient Phase 3 Efficacy Study of Ecopipam (PSYRX 101) in the Symptomatic Treatment of Self-Injurious Behavior in Subjects with Lesch-Nyhan Disease (LND).

Recruitment & Eligibility

Status: Authorised-recruitment may be ongoing or finished

Sex: All

Target Recruitment: 24

Inclusion Criteria

1. Subjects must have classic LND as defined by (a) characteristic clinical syndrome (evidence of overproduction of uric acid, severe generalized dystonia, frequent and persistent SIB, and cognitive impairment) and (b) laboratory confirmation for mutation of the HPRT gene or severe deficiency of the associated enzyme.
2. Subjects must have a minimum combined score of 20 on the BPI SIB subscales for frequency and severity as assessed by the caregiver.
3. Subjects must have a minimum score of 4 on the Physician?s Global Impression (PGI) severity scale.
4. Subjects must be ?6 years old.
5. Subject must have a body weight greater than 10 kg.
6. Although females with LND are extremely rare, they are occasionally encountered. For a woman to be eligible she must be postmenopausal (>12 months without menses) or surgically sterile (ie, by hysterectomy and/or bilateral oophorectomy) or must be using effective contraception (ie, oral contraceptives, intrauterine device, double barrier method of condom and spermicide) and agree to continue use of contraception for the duration of their participation in the study. Women of childbearing potential must agree to use contraception for 30 days after their last dose of study drug.
7. Sexually active male subjects must use a barrier method of contraception during the study and agree to continue the use of male contraception for at least 30 days after the last dose of study drug.
8. Subject and/or legal guardian must execute a written Informed Consent and/or Assent.
Are the trial subjects under 18? yes
Number of subjects for this age range: 24
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 1
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. Subjects who have participated in a clinical trial (other than PSY101) and have received an investigational drug within 30 days prior to the start of the study.
2. Subjects who are on neuroleptics or dopamine depleting agents.
3. Subjects who are currently treated with medications for seizures
4. Subjects with impaired renal function as defined by a serum creatinine >1.5 mg/dL.
5. Subjects with medical comorbidities that may interfere with completion of the study.
6. Women of childbearing potential who are currently pregnant or lactating.

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To assess the efficacy of ecopipam to reduce self-injurious behaviors (SIB) in adults and children with Lesch-Nyhan Disease (LND) in an outpatient setting.;Secondary Objective: To assess the effect of withdrawal, maintenance, and safety of ecopipam in subjects with Lesch-Nyhan Disease (LND).;Primary end point(s): Behavior Problems Inventory (BPI) SIB subscales for frequency and severity as assessed by the caregiver.;Timepoint(s) of evaluation of this end point: The caregiver will complete the BPI each week.

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): Vital signs, Caregiver Global Impression (CGI) improvement, adverse events (AEs).;Timepoint(s) of evaluation of this end point: Baseline, once during each tratment period (1-3), once during follow-up, twice during open-label treatment period, once at the end of the study.

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