Eligibility Module
The Eligibility Module contains detailed information about who can participate in the clinical trial. This includes eligibility criteria, age restrictions, gender requirements, healthy volunteer status, and study population descriptions, helping researchers understand who is eligible to participate in the study.
Eligibility Module path is as follows:
Study -> Protocol Section -> Eligibility Module
Eligibility Module
Ignite Creation Date:
2025-12-25 @ 4:01 AM
Ignite Modification Date:
2025-12-25 @ 4:01 AM
NCT ID:
NCT01751802
Eligibility Criteria:
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.
Healthy Volunteers:
False
Sex:
ALL
Minimum Age:
6 Years
Maximum Age:
70 Years
Study:
NCT01751802
Study Brief:
Protocol Section:
NCT01751802