Schizophrenia MedDRA version: 20.0 Level: PT Classification code 10039626 Term: Schizophrenia System Organ Class: 10037175 - Psychiatric disorders
Conditions
Interventions
Product Name: Vafidemstat
Product Code: ORY-2001
Pharmaceutical Form: Capsule, hard
INN or Proposed INN: VAFIDEMSTAT
CAS Number: 1357247-95-0
Concentration unit: mg milligram(s)
Concentration type: eq
Sponsors
Oryzon Genomics, S.A.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Male or female subject 18-50 years of age. 2. The participant has schizophrenia, according to DSM-5™ as confirmed by the Mini International Neuropsychiatric Interview (MINI) Version 7.0.2 at Screening. 3. Documented DSM-5™ diagnostic criteria for schizophrenia greater than one (1) year, but less than five (5) years from Screening. 4. The participant has persistent, predominant negative symptoms (PNS) of schizophrenia, and minimal positive symptoms. 5. Subject is stable in terms of positive and negative symptoms of schizophrenia over the last 2 months. 6. The participant is currently an outpatient and has not been hospitalized within the last 2 months for an acute exacerbation of their schizophrenia or symptom worsening. In addition, the subject’s treatment must be stable and include no more than one atypical antipsychotic. 7. Stable in their regimen of background therapy other than for psychiatric indications, as per the Summary of Product Characteristics (SmPC) for concomitant medications at the Screening visit. 8. Body mass index (BMI) of at least 18.5 kg/m2, but no more than 35 kg/m2. 9. Subject is considered by the investigator to be reliable and willing and able to adhere to the prohibitions, restrictions and requirements specified in this protocol. 10. Otherwise, healthy and medically stable based on medical history. 11. Able to read and write fluently and must have adequate hearing and visual acuity to complete the required testing outlined in this protocol. 12. Negative Covid-19 test (PCR, antigen test or serology) at Screening (only applicable if Covid-19 precautions are still in force by the time of the Screening Visit). 13. The subject has a study partner/caregiver who, in the investigator’s judgement, has frequent and sufficient contact with the subject. 14. The subject has a stable living environment for > 6 months before the Screening visit, as confirmed by study partner/caregiver. 15. Fertile male and female subjects must use highly efficient contraception, from the Screening visit until 30 days after last dose of the IMP. 16. Female subjects of childbearing potential must have a negative urine pregnancy test at screening and baseline. 17. Signed informed consent by the subject and the subjects´ study partner/caregiver Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 100 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Failure to perform screening or baseline procedures. 2. Inadequate response in the level of psychotic symptoms during more than one documented treatment trial with an adequate dose of an antipsychotic drug prescribed for an adequate time (i.e., at least lasting for 6 weeks) within 2 years prior to the Screening Visit. 3. Any current diagnosis of psychiatric disorder other than schizophrenia. 4. DSM-5 diagnosis of neurodevelopmental disorders. 5. Current DSM-5 diagnosis of conduct disorders. 6. Current DSM-5 diagnosis of panic disorder or post-traumatic stress disorder (PTSD). 7. Current diagnosis or a history of substance use disorder according to DSM-5™ criteria within 6 months prior to the Screening Visit. 8. Use of illicit drugs for at least one week before Screening and subjects unwilling to abstain from use of these substances during the study. 9. Suicide attempt or significant risk of suicide within 6-months prior to the Screening visit or the period between Screening and Baseline visit, defined as a “yes” to suicidal ideation questions 4 or 5, or answering “yes” to suicidal behavior on the Columbia-Suicide Severity Rating Scale. 10. The subject has started formal cognitive or behavioral therapy or systematic psychotherapy within 3 months prior to Screening or plans to start such therapy during the study. 11. The subject has a previous or current diagnosis of neuroleptic malignant syndrome. 12. The subject has been treated with and is resistant to clozapine according to the investigator’s judgement. 13. Hospitalization or medication change for any reason 2 months prior to the Screening visit or during the Screening period that makes the subject medically or mentally unsuitable for trial participation. 14. Clinically significant, advanced, or unstable disease that is likely to result in rapid deterioration of the subject’s condition or affect their safety during the study. 15. Positive results for tuberculosis, Human Immunodeficiency Virus (HIV), Hepatitis C or Hepatitis B serology obtained at the Screening Visit. 16. Uncontrolled hypo- or hyperthyroidism at Screening Visit, based on laboratory parameters. 17. Clinically significant infection within the previous 30-days (e.g., persistent or acute infection such as a urinary tract infection or upper respiratory infection). 18. Chronic drug intake of specific forbidden medication as described in the clinical study protocol. 19. Electroconvulsive therapy (ECT) or transcranial magnetic stimulation (TMS) in the past 3 months before the screening visit, and no use of these treatments is allowed throughout the trial. 20. Any regular intake of medications acting directly on central nervous system that investigator considers relevant to the study. 21. Member or immediate family of the study personnel or subordinate to any of the study personnel. 22. Enrollment in another investigational study or intake of investigational drug within the previous 3 months. 23. Any condition that in the opinion of the investigator makes the subject unsuitable for inclusion in the study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: •To assess the effect of vafidemstat on negative symptoms of schizophrenia in adult patients •To assess the effect of vafidemstat on cognitive impairment associated with schizophrenia (CIAS) in adult patients;Secondary Objective: •To assess the effect of vafidemstat on positive symptoms of schizophrenia in adult patients •To assess the effect of vafidemstat on functional impairment in adult schizophrenia patients •To evaluate vafidemstat safety in adult schizophrenia patients •To evaluate the effect of vafidemstat on the use of health care services in adult schizophrenia patients •To evaluate the effect of vafidemstat on the stable background antipsychotic medication in adult schizophrenia patients.;Primary end point(s): • To evaluate the change from baseline to week 24 on the PANSS Factor Score for Negative Symptoms (PANSS-FSNS) • To evaluate the change from baseline to week 24 on the Brief Assessment in Cognition in Schizophrenia (BACS);Timepoint(s) of evaluation of this end point: 24 weeks | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): • To evaluate the change over time on the PANSS Factor Score for Negative Symptoms (FSNS) • To evaluate the change over time on the BACS • To evaluate the difference from baseline to week 24, as well as change over time on the following: a) PANSS – Positive Symptoms Scale b) Clinical Global Impression – Severity (CGI-S) for Negative Symptoms c) Personal and Social Performance Scale (PSP) To evaluate the following safety endpoints throughout the study, from baseline to week 28: a) Number, frequency, and severity of Treatment Emergent Adverse Events (TEAEs) b) Number, frequency, and severity of Serious TEAEs c) Number and percentage of withdrawn subjects due to TEAEs d) Use of concomitant medications e) Frequency of physical examination parameters, vital signs, and ECG parameters of potential clinical concern f) Frequency of clinical laboratory parameters of potential clinical concern g) Columbia – Suicide Severity Rating Scale (C-SSRS);Timepoint(s) of evaluation of this end point: 24-28 weeks | — |
Countries
Spain
Contacts
Public ContactSonia Gutierrez
Oryzon Genomics, S.A.
Outcome results
None listed