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

NCT00637221 | Completed Phase 1 DMC

• 1 other identifier: NPL-2009-2-FEN-001
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 2

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

Conditions

Fragile X Syndrome

Keywords

Safety; Tolerability

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (1)

• Tolerability

  7 days

Interventions

NPL-2009 DRUG

Single doses of either 50mg, 100 mg or 150 mg NPL-2009

Eligibility Criteria

• Age: 18 Years - 45 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Neuropharm: lead

Study Sites (2)

MIND Institute

Sacramento, California, 95817, United States

Location

Rush University Medical Centre

Chicago, Illinois, 60612, United States

Location

MeSH Terms

Conditions

Fragile X Syndrome

Condition Hierarchy (Ancestors)

X-Linked Intellectual Disability; Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Sex Chromosome Disorders; Chromosome Disorders; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Heredodegenerative Disorders, Nervous System

Study Officials

• Mike Snape, PhD

  Neuropharm Ltd

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: FACTORIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 3, 2008
• First Posted: March 17, 2008
• Study Start: March 1, 2008
• Primary Completion: April 1, 2008
• Study Completion: April 1, 2008
• Last Updated: April 27, 2012

Record last verified: 2012-04

Locations

US (2)