Fragile X SyndromePhase 1/2NCT00637221
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
A Phase 1/2 study testing NPL-2009 in people with Fragile X Syndrome, run by Neuropharm at 2 sites in United States.
Trial 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 Read more about this study
- Who is it for?
- Ages 18 to 45 years
- Conditions the study is for
- Fragile X Syndrome
- What is being tested?
- NPL-2009
- How many people
- 12 participants
- Where
- 2 sitesUnited States
- Study dates
- Mar 2008to Apr 2008
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 45 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- 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.
Who cannot join
- 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.
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- 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
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Rush University Medical Centre
Chicago, Illinois, 60612
MIND Institute
Sacramento, California, 95817
About this study
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
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
- Being tested
- NPL-2009
- Keywords
- Safety, Tolerability
- Medical subject headings
- Fragile X Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NPL-2009Drug
Single doses of either 50mg, 100 mg or 150 mg NPL-2009
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 7 Days
Secondary outcomes (1)
Tolerability
Measured: 7 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Mar 2008 (estimated)
- Study startMar 2008
- Primary completionApr 2008
- Study completionApr 2008
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Fragile X SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Factorial
- Masking
- None (open label)
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Neuropharm
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Mike Snape, PhD, Study director, Neuropharm Ltd
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00637221
- Sponsor's protocol ID
- NPL-2009-2-FEN-001
- Status last verified
- Apr 2012
- First submitted
- 3 Mar 2008
- Last updated
- 27 Apr 2012
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 27 Apr 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT00637221
- Acronym / sponsor ID
- NPL-2009-2-FEN-001
- Conditions
- Fragile X Syndrome
- Keywords
- Safety, Tolerability
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 12 participants (actual)
- Lead sponsor
- NeuropharmIndustry (pharma, biotech, devices)
- Study start
- Mar 2008
- Primary completion
- Apr 2008 (actual)
- Study completion
- Apr 2008 (actual)
- First posted
- 17 Mar 2008 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Factorial
- Masking
- None (open label)
Who may qualify
- Ages
- 18 to 45 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (8 listed)
- 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.
Key exclusion criteria (19 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| NPL-2009Single doses of either 50mg, 100 mg or 150 mg NPL-2009 | Drug | – |
Primary outcome
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
Time frame: 7 Days
1 secondary outcome
Tolerability
Time frame: 7 days
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Apr 2012.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Mike Snape, PhD study director
Neuropharm Ltd
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing NPL-2009 in people with Fragile X Syndrome, run by Neuropharm at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 19 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 27 Apr 2012. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome 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 (time frame: 7 Days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Apr 2012.