Clinical trial NCT00637221: Open Label Study Investigating Safety and Efficacy of NPL2009 50 mg - 150 mg on Prepulse Inhibition Tests and Continuous Performance Tasks, Adults With Fragile X Syndrome
Official title: An Open Label Exploratory Study to Investigate the Safety and Effects of NPL-2009 ( 50 mg - 150 mg Single Dose) on Prepulse Inhibition Tests and Continuous Performance Tasks, in Adults With Fragile X Syndrome
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Fragile X Syndrome
Keywords: Safety
Tolerability
Lead sponsor: Neuropharm
Sponsor class: INDUSTRY
First posted: 2008-03-17
Enrollment: 12
Sex: ALL
Minimum age (years): 18
Maximum age (years): 45
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: FACTORIAL
Primary purpose: TREATMENT

Brief summary: This is an open label exploratory study to investigate the safety and effects of a single dose of NPL-2009(50 mg - 150 mg) on Prepulse Inhibition (PPI) Tests and Continuous Performance Tasks (CPT) in adults with Fragile X Syndrome


Eligibility criteria: Inclusion Criteria:

* Male or female patients, 18 to 45 years of age.
* Diagnosis of Fragile X Syndrome.
* Females must demonstrate a negative pregnancy test at screening.
* Females of child-bearing potential must be using a medically accepted means of contraception or must remain abstinent for the duration of the study.
* Each Legally Authorised Representative (LAR, usually parent or caregiver) must have a level of understanding sufficient to provide written informed consent to all required study tests and procedures.
* Each patient must consent/assent (depending on center-specific procedures) to all required study tests and procedures.
* Permitted concomitant medications must be stable for at least 6 weeks prior to enrollment. The following concomitant medications are permitted: psychostimulants, SSRIs, atypical antipsychotics, anticonvulsants which do not have liver inducing effects, clonidine.
* Each patient must be able to swallow the capsules (2, 3 or 4) to be provided in the study.

Exclusion Criteria:

* Current treatment with anticonvulsants known to induce liver enzymes e.g. depakote
* Current treatment with N-methyl-D-aspartate (NMDA) antagonists
* Current treatment with tricyclic antidepressants
* Current treatment with typical antipsychotics
* Current treatment with lithium
* Patients planning to commence cognitive behaviour therapy during the period of the study or those who have begun cognitive behavioural therapy within 6 weeks prior to enrolment.
* History of, or current cardiovascular, renal, hepatic, respiratory and particularly gastrointestinal disease which may interfere with the absorption, distribution, metabolism or excretion of the study medication.
* History of, or current cerebrovascular disease or brain trauma.
* History of, or current significant endocrine disorder, e.g. hypo or hyperthyroidism.
* History of, or current malignancy.
* Presence of psychotic symptoms or lifetime history of schizophrenia, bipolar disorder, or other psychotic disorder, as assessed by the Investigator.
* Current major depressive disorder (patients must be free of the disorder for 3 months prior to enrolment).
* Judged clinically to be at risk of suicide (suicidal ideation, severe depression, or other factors), as assessed by the Investigator.
* Tourette's Disorder.
* Female patients who are either pregnant or nursing.
* Current drug abuse or dependence disorder or dependency in the 3 months prior to enrolment.
* Clinically significant abnormalities in safety laboratory tests, vital signs or EKG, as measured at screening
* Patients with significant hearing and/or visual impairments that may affect their ability to complete the test procedures
* Enrollment in another clinical trial within the previous 30 days

Primary outcomes:
measure: The primary outcome measure for the study is that of safety and subjects will be assessed post-dose at at least hourly intervals for any signs of Adverse Events - up to allowing discharge from the unit at 6 hours post-dose
timeFrame: 7 Days
Secondary outcomes:
measure: Tolerability
timeFrame: 7 days
Other outcomes:
Locations:
city: Sacramento
country: United States
facility: MIND Institute
state: California
city: Chicago
country: United States
facility: Rush University Medical Centre
state: Illinois
Study officials:
affiliation: Neuropharm Ltd
name: Mike Snape, PhD
role: STUDY_DIRECTOR
References: