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

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

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

Study Overview

• Status: Completed
• Conditions: Fragile X Syndrome
• Intervention / Treatment: Drug: NPL-2009

• Study Type: Interventional
• Enrollment (Actual): 12
• Phase: Phase 2; Phase 1

Contacts and Locations

Study Locations

• United States
  • California
    • Sacramento, California, United States, 95817
      • MIND institute
  • Illinois
    • Chicago, Illinois, United States, 60612
      • Rush university Medical Centre

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 45 years (Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Factorial Assignment
• Masking: None (Open Label)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
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	7 Days

Secondary Outcome Measures

Outcome Measure	Time Frame
Tolerability	7 days

Collaborators and Investigators

• Sponsor: Neuropharm
• Investigators: Study Director: Mike Snape, PhD, Neuropharm Ltd

Study record dates

Study Major Dates

• Study Start: March 1, 2008
• Primary Completion (Actual): April 1, 2008
• Study Completion (Actual): April 1, 2008

Study Registration Dates

• First Submitted: March 3, 2008
• First Submitted That Met QC Criteria: March 10, 2008
• First Posted (Estimate): March 17, 2008

Study Record Updates

• Last Update Posted (Estimate): April 27, 2012
• Last Update Submitted That Met QC Criteria: April 26, 2012
• Last Verified: April 1, 2012

More Information

Terms related to this study

• Keywords: Safety; Tolerability
• Additional Relevant MeSH Terms: Pathologic Processes; Nervous System Diseases; Neurologic Manifestations; Neurobehavioral Manifestations; Disease; Congenital Abnormalities; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Mental Retardation, X-Linked; Intellectual Disability; Heredodegenerative Disorders, Nervous System; Chromosome Disorders; Sex Chromosome Disorders; Syndrome; Fragile X Syndrome
• Other Study ID Numbers: NPL-2009-2-FEN-001