Kuvan in People With Schizophrenia and Schizoaffective Disorder

NCT01706965 | Completed Phase 1 Results

• 1 other identifier: 6320
• Study Type: interventional
• Target: 27
• Locations: 1 country
• Sites: 1

Brief Summary

Rater blinded trial of six weeks of Kuvan vs. multivitamin in 60 outpatients with schizophrenia or schizoaffective disorder. The aims are to evaluate an anticipated clinical response to Kuvan treatment including negative symptom and cognitive deficits, evaluate safety of Kuvan treatment for schizophrenic patients and evaluate the relationship of changes in plasma Kuvan levels and efficacy outcomes.

Conditions

Schizophrenia; Schizoaffective Disorder

Outcome Measures

Primary Outcomes (2)

• Final Positive and Negative Symptom Scale (PANSS)

  This is a is a 30 item rating scale widely used in the assessment of schizophrenia ranging from 30-210. Higher scores are worse

  Baseline (start of Kuvan) and at six weeks of treatment

• Final MATRICS Cognitive Battery

  MATRICS assessing 7 domains (Speed of Processing, Attention/Vigilance, Working Memory, Verbal Learning, Visual Learning, Reasoning and Problem Solving, and Social Cognition. Raw scores are converted into a composite T-score (normative mean = 50; standard deviation = 10), where higher values indicated less impairment.

  Baseline to week 6

Study Arms (2)

Kuvan

EXPERIMENTAL

Kuvan once-daily dosing initiated with 10mg/kg for the first week followed by 20mg/kg for the remaining weeks of the study

Drug: Kuvan

Multivitamin

ACTIVE COMPARATOR

Daily multivitamin tablet

Drug: Multivitamin

Interventions

Kuvan DRUG

20mg/kg using a daily dosing schedule. To increase tolerability, treatment will be initiated at 10mg/kg for the first week of the study. Given the short length of the trial, no dose adjustments will be made for changes in weight

Also known as: Sapropterin dihydrochloride

Kuvan

Multivitamin DRUG

Multi-vitamin will be used as an active control in this study. Will be dosed daily

Multivitamin

Eligibility Criteria

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

You may qualify if:

• Male and female outpatients with schizophrenia or schizoaffective disorder
• Ages 18-64
• A score of 45 or greater on PANSS
• All oral and depot antipsychotics (with the exception of clozapine) are allowable. Patients must be on their antipsychotic medication for 3 months and stable on dose of antipsychotic and adjunctive medications for 2 weeks prior to study entry. If a patient is on depot medication, they must be stable in dose for 2 months

You may not qualify if:

• Organic brain disorder, including epilepsy; mental retardation; or a medical condition whose pathology or treatment would likely alter the presentation or treatment of schizophrenia
• Participated in any investigational study or taken an investigational drug within 30 days
• Current Diagnostic and Statistical Manual of Mental Disorders, 4th edition (DSM-IV) diagnosis of drug/alcohol abuse in last month and current DSM-IV diagnosis drug/alcohol dependence in last 6 months. Subjects must have a negative drug screen at baseline (with one retest allowed for suspected false positive based on clinical judgement of the investigator)
• Diagnosis of any known BH4 deficiency disorder (other than schizophrenia or schizoaffective disorder), including dopa-responsive dystonia,phenylketonuria (PKU), and autism
• Current treatment with clozapine
• In the investigator's judgment, a significant risk of suicide or violent behavior
• Current use of levodopa and nitric oxide-mediated vasorelaxation or oral minoxidil
• Women will be excluded if they are pregnant, lactating, or not either surgically-sterile or using appropriate methods of birth control. Women must agree to continue using applicable birth control throughout the trial. All women of child-bearing potential must have a negative urine pregnancy test at the screening visit and visit 2 (1 week before beginning study medication)
• Absolute neutrophil count below 2.0 on screening
• Any contraindication or allergic reaction to previous multi-vitamin or unwillingness to stop use of current multi-vitamin during study

Sponsors & Collaborators

• New York State Psychiatric Institute: lead
• Stanley Medical Research Institute: collaborator

Study Sites (1)

New York State Psychiatric Institute

New York, New York, 10032, United States

Location

MeSH Terms

Conditions

Schizophrenia; Psychotic Disorders

Interventions

sapropterin; Geritol

Condition Hierarchy (Ancestors)

Schizophrenia Spectrum and Other Psychotic Disorders; Mental Disorders

Results Point of Contact

• Title: Marlene Carlson
• Organization: New York State Psychiatric Insititute

marlene.carlson@nyspi.columbia.edu

646-774-8436

Study Officials

• Jeffrey A Lieberman, M.D.

  New York State Psychiatric

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 10, 2012
• First Posted: October 15, 2012
• Study Start: October 1, 2012
• Primary Completion: June 1, 2016
• Study Completion: June 1, 2016
• Last Updated: September 13, 2017
• Results First Posted: September 13, 2017

Record last verified: 2017-08

Locations

US (1)