Velo-cardio-facial SyndromePhase 2NCT01127503
Metyrosine (Demser®) for the Treatment of Psychotic Disorders in Patients With Velocardiofacial Syndrome
A Phase 2 study testing Metyrosine in people with Velo-cardio-facial Syndrome and Psychosis, run by Bausch Health Americas, Inc. at 1 site in United States.
Trial summary
This is an exploratory clinical investigation. The objectives of this study are to evaluate the safety, steady-state pharmacokinetics, and efficacy of metyrosine (Demser®) for the treatment of psychosis in patients with velocardiofacial syndrome (VCFS). Read more about this study
- Who is it for?
- Ages 16 to 65 years
- Conditions the study is for
- Velo-cardio-facial Syndrome, Psychosis
- What is being tested?
- Metyrosine
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 2 participants
- Where
- 1 siteUnited States
- Study dates
- Jun 2010to Sep 2011
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 16 to 65 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Females of childbearing potential cannot be at risk of pregnancy during the study.
- Genetically confirmed diagnosis of VCFS at the time of screening.
- Must have one of the following Diagnostic and Statistical Manual of Mental Disorders, Fourth Edition Text Revision (DSM-IV TR) diagnoses (applicable based upon clinical assessments): schizophrenia, schizoaffective disorder, psychosis not otherwise specified (NOS), bipolar disorder, or mood disorder with psychotic features.
- A total PANSS composite score >65.
- Willing to discontinue psychotropic medications. -
Who cannot join
- Evidence of acute suicidality.
- Known or observed clinically significant cardiovascular, pulmonary, renal, hepatic, or gastrointestinal disorders; other clinically significant psychiatric/neurological and sleep disorders by DSM-IV-TR criteria; endocrine, or hematological or metabolic diseases.
- Full scale IQ of less than 50.
- Pregnancy.
- Not using a reliable means of contraception.
- Systolic blood pressure of ≤110 mm/Hg or ≥160 mm/Hg, diastolic blood pressure ≤60 mm/Hg or ≥90 mm/Hg, or has clinically symptomatic orthostatic changes.
- QTcF > 450 msec, or PR > 250 msec, or QRS > 110 msec on ECG.
- History of seizure disorder. -
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Double-Blind, Placebo-Controlled, Multi-Center, Randomized Trial of the Safety and Efficacy of Metyrosine (Demser®) for the Treatment of Psychotic Disorders in Patients With Velo-Cardio-Facial Syndrome
This is an exploratory clinical investigation. The objectives of this study are to evaluate the safety, steady-state pharmacokinetics, and efficacy of metyrosine (Demser®) for the treatment of psychosis in patients with velocardiofacial syndrome (VCFS).
- Conditions
- Velo-cardio-facial Syndrome, Psychosis
- Being tested
- Metyrosine, Placebo
- Keywords
- Patients with velocardiofacial syndrome and psychosis
- Medical subject headings
- DiGeorge Syndrome, Psychotic Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MetyrosineDrug
Metyrosine (250 mg capsules) were to be used at all dose levels (administered as multiples of that dosing unit). The starting dose was 250 mg/day of metyrosine. Dose escalation was to be carried out weekly for 8 weeks (up to a maximum of 8 capsules/day [2000 mg/day if metyrosine]) with dosage increments of 1 capsule/day per week. Weekly dose escalation was to stop based upon the investigator's assessment of safety, but not efficacy (i.e., dose escalation was to be forced to the maximum of 8 capsules/day assuming acceptable safety and tolerability).
PlaceboDrug
Placebo capsules were identically matched to Metyrosine.
| Study group | Role | What they receive |
|---|---|---|
| Metyrosine | Experimental | Not described Receives: Drug: Metyrosine |
| Placebo | Placebo comparator | Not described Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
To Evaluate the Safety of Metyrosine (Demser®) for the Treatment of Psychosis in Patients With VCFS
Measured: 13 weeks
Secondary outcomes (1)
To Evaluate the Efficacy of Metyrosine (Demser®) for the Treatment of Psychosis in Patients With VCFS
Measured: 13 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 May 2010 (estimated)
- Study startJun 2010
- Primary completionNov 2010
- Study completionSep 2011
Why it stopped: Enrollment, study-design and execution challenges.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Velo-cardio-facial SyndromeStudies of the same condition, open and under way.
- More trials run by Bausch Health Americas, Inc.The sponsor's other registered studies.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- Bausch Health Americas, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Robert J Shprintzen, PhD, Principal investigator, Upstate Medical University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01127503
- Sponsor's protocol ID
- 09-MET-101
- Status last verified
- Nov 2019
- First submitted
- 19 May 2010
- Last updated
- 22 Nov 2019
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Nov 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Double-Blind, Placebo-Controlled, Multi-Center, Randomized Trial of the Safety and Efficacy of Metyrosine (Demser®) for the Treatment of Psychotic Disorders in Patients With Velo-Cardio-Facial Syndrome
- ClinicalTrials.gov ID
- NCT01127503
- Acronym / sponsor ID
- 09-MET-101
- Conditions
- Velo-cardio-facial Syndrome, Psychosis
- Keywords
- Patients with velocardiofacial syndrome and psychosis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Enrollment, study-design and execution challenges.
- Enrolment
- 2 participants (actual)
- Lead sponsor
- Bausch Health Americas, Inc.Industry (pharma, biotech, devices)
- Study start
- Jun 2010
- Primary completion
- Nov 2010 (actual)
- Study completion
- Sep 2011 (actual)
- First posted
- 21 May 2010 (estimated)
- Results
- Posted 8 May 2018
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 exploratory clinical investigation. The objectives of this study are to evaluate the safety, steady-state pharmacokinetics, and efficacy of metyrosine (Demser®) for the treatment of psychosis in patients with velocardiofacial syndrome (VCFS).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Metyrosine | Experimental | Drug: Metyrosine |
| Placebo | Placebo comparator | Drug: Placebo |
Who may qualify
- Ages
- 16 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Females of childbearing potential cannot be at risk of pregnancy during the study.
- Genetically confirmed diagnosis of VCFS at the time of screening.
- Must have one of the following Diagnostic and Statistical Manual of Mental Disorders, Fourth Edition Text Revision (DSM-IV TR) diagnoses (applicable based upon clinical assessments): schizophrenia, schizoaffective disorder, psychosis not otherwise specified (NOS), bipolar disorder, or mood disorder with psychotic features.
- A total PANSS composite score >65.
- Willing to discontinue psychotropic medications. -
Key exclusion criteria (8 listed)
- Evidence of acute suicidality.
- Known or observed clinically significant cardiovascular, pulmonary, renal, hepatic, or gastrointestinal disorders; other clinically significant psychiatric/neurological and sleep disorders by DSM-IV-TR criteria; endocrine, or hematological or metabolic diseases.
- Full scale IQ of less than 50.
- Pregnancy.
- Not using a reliable means of contraception.
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 |
|---|---|---|
| MetyrosineMetyrosine (250 mg capsules) were to be used at all dose levels (administered as multiples of that dosing unit). The starting dose was 250 mg/day of metyrosine. Dose escalation was to be carried out weekly for 8 weeks (up to a maximum of 8 capsules/day [2000 mg/day if metyrosine]) with dosage increments of 1 capsule/day per week. Weekly dose escalation was to stop based upon the investigator's assessment of safety, but not efficacy (i.e., dose escalation was to be forced to the maximum of 8 capsules/day assuming acceptable safety and tolerability). | Drug | Metyrosine |
| PlaceboPlacebo capsules were identically matched to Metyrosine. | Drug | Placebo |
Primary outcome
To Evaluate the Safety of Metyrosine (Demser®) for the Treatment of Psychosis in Patients With VCFS
Time frame: 13 weeks
1 secondary outcome
To Evaluate the Efficacy of Metyrosine (Demser®) for the Treatment of Psychosis in Patients With VCFS
Time frame: 13 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Nov 2019.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
Robert J Shprintzen, PhD principal investigator
Upstate Medical University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Metyrosine in people with Velo-cardio-facial Syndrome and Psychosis, run by Bausch Health Americas, Inc. at 1 site 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 5 inclusion and 8 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: stopped early, as of the sponsor's last update on 22 Nov 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
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?
Yes. Results are posted in the registry (first posted 8 May 2018). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
To Evaluate the Safety of Metyrosine (Demser®) for the Treatment of Psychosis in Patients With VCFS (time frame: 13 weeks). 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 22 Nov 2019.