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Combination Therapy of Deferiprone and N-Acetylcysteine for Treating Negative Symptoms in Schizophrenia (IronMan)

9. august 2026 oppdatert av: Amit Lotan
In following guidelines for studying negative symptoms in schizophrenia, 60 patients aged 18-40, diagnosed with schizophrenia or schizoaffective disorder during the last ten years, will receive a combination of the iron chelator DFP plus NAC or a matching placebo plus NAC combination, as add-on to their maintenance antipsychotic drug (APD) treatment, for a duration of 36 weeks. Prior to initiation of treatment, all participants will undergo baseline MRI scan. A follow-up MRI will be conducted at the end of the 36-week study period, or upon early withdrawal if applicable, to evaluate the effect of the add-on treatment on iron dyshomeostasis and morphology.

Studieoversikt

Detaljert beskrivelse

The present study is designed as an interventional trial over a 36-week period per patient. The target populThe present study is designed as an interventional trial over a 36-week period per patient. The target population consists of patients with schizophrenia (age 18-40 years), up to ten years since onset of positive symptoms. The overall objective of the study is to assess the safety and efficacy of DFP-NAC combination add-on in patients with schizophrenia, compared to NAC-only add-on (control group). This study is designed to test the hypothesis that, in patients with prominent negative symptoms, adjunctive treatment with DFP-NAC combination will demonstrate a favorable safety profile and produce clinical improvements in negative and cognitive domains of schizophrenia beyond those offered by addition of NAC alone.ation consists of patients with schizophrenia (age 18-40 years), up to ten years since onset of positive symptoms. The overall objective of the study is to assess the safety and efficacy of DFP-NAC combination add-on in patients with schizophrenia, compared to NAC-only add-on (control group). This study is designed to test the hypothesis that, in patients with prominent negative symptoms, adjunctive treatment with DFP-NAC combination will demonstrate a favorable safety profile and produce clinical improvements in negative and cognitive domains of schizophrenia beyond those offered by addition of NAC alone.

Studietype

Intervensjonell

Registrering (Antatt)

80

Fase

  • Fase 2

Kontakter og plasseringer

Denne delen inneholder kontaktinformasjon for de som utfører studien, og informasjon om hvor denne studien blir utført.

Studiekontakt

Studiesteder

      • Jerusalem, Israel
        • Hadassah Medical Organization
        • Ta kontakt med:
      • Jerusalem, Israel
        • Jerusalem Mental Health Center
        • Ta kontakt med:

Deltakelseskriterier

Forskere ser etter personer som passer til en bestemt beskrivelse, kalt kvalifikasjonskriterier. Noen eksempler på disse kriteriene er en persons generelle helsetilstand eller tidligere behandlinger.

Kvalifikasjonskriterier

Alder som er kvalifisert for studier

  • Voksen

Tar imot friske frivillige

Ja

Beskrivelse

Inclusion criteria:

  1. Aged 18-55 years.
  2. Diagnosis of schizophrenia/schizoaffective disorder according to the Diagnostic and Statistical Manual of Mental Disorders- Fifth Edition (DSM-5) criteria. The diagnostic assessment will be conducted using the Structured Clinical Interview for DSM-5 Clinical Trials Version (SCID-5-CT).
  3. Adequate antipsychotic treatment for ≥ 4 months prior to screening (excluding clozapine use).
  4. Stable dose of antipsychotic medication for ≥ 8 weeks prior to screening (excluding clozapine use).
  5. When relevant, stable dose of antidepressants, mood stabilizers and benzodiazepines/Z-drugs for ≥ 8 weeks prior to screening.
  6. Screening and baseline PANSS total score ranging from 60 to 120.
  7. Sum ≥ 20 for the 7 items in the Negative Symptoms subscale of the PANSS.
  8. At least two items in the PANSS Negative Symptoms subscale scored ≥ 4.
  9. Sum < 20 for the 7 items in the Positive Symptoms subscale of the PANSS.
  10. Score ≤ 5 for each item in the Positive Symptoms subscale of the PANSS.
  11. Calgary Depression Schizophrenia Scale (CDSS) score of ≤ 6.
  12. Persistent and predominant negative symptoms for ≥ 6 months, in the opinion of the investigator
  13. The subject exhibits clinical stability in both positive and negative symptoms of schizophrenia over the past 6 months, as assessed by the treating psychiatrist and supported by documentation in the medical record.
  14. No incarceration in prison or acute crisis intervention due to symptom exacerbation within 6 months of screening. The subject must be considered psychiatrically stable in the opinion of the investigator.
  15. For males or postmenopausal women, either of the following:

    1. Serum ferritin ≥30 ng/mL at screening. OR
    2. Serum ferritin 15-29 ng/mL at screening, provided that:

    i. An evaluation of potential underlying causes as well as potentially comorbid deficiencies has been completed, including assessment of folate, vitamin B12, celiac antibodies and H. pylori infection, and, in patients over 40 years old or with positive family history of colorectal cancer, also an appropriate colorectal screening test, alongside other tests deemed as relevant by the investigator; ii. Identified clinically significant causes have been appropriately managed; iii. Repeated ferritin assessment prior to randomization remains within the range of 15-29 ng/mL; and iv. Oral iron supplementation is initiated according to the study protocol.

  16. For premenopausal women: Serum ferritin ≥15 ng/mL at screening.
  17. Screening serum hemoglobin ≥ 13g/dL for males, ≥ 12g/dL for females
  18. Use of contraceptives in women of childbearing age.
  19. Ability to provide informed consent, confirmed by a non-study psychiatrist. When a subject is under guardianship, both patient assent and guardian consent are required. Obtaining written informed consent, dated and signed, is mandatory prior to the initiation of any procedures related to the clinical trial.
  20. Ability to safely undergo MRI scanning, assessed by a non-study psychiatrist.
  21. In the Investigator's opinion, the subject can understand the nature of the trial, comply with study drug administration and protocol procedures or has a guardian able to assist.
  22. Availability of a reliable caregiver or other responsible person (e.g., family member, social worker, nurse) to support treatment adherence and provide collateral information for rating scales.

Exclusion criteria:

  1. Primary DSM-5 diagnosis other than schizophrenia or schizoaffective disorder in the 12 months prior to screening, according to SCID-5-CT.
  2. Subjects diagnosed with Autistic Spectrum Disorder (ASD).
  3. Subjects diagnosed with Intellectual Developmental Disorder.
  4. Patients who received clozapine within the 6 months preceding the screening.
  5. Patients with a history of relapsing neutropenia (Absolute Neutrophile Count (ANC) < 1,500 cells/µL)
  6. Screening ANC ≤ 2,000 cells/µL.
  7. Non-compliance during run-in period (percent adherence ≤ 80%, as examined by pill count).
  8. Improvement > 20% in PANSS total score or PANSS negative subscale during the run-in period.
  9. Suicide attempt or serious suicidal behavior within the past 12 months.
  10. Risk for suicidal behavior during the study as determined by the investigator's clinical assessment and Columbia-Suicide Severity Rating Scale (C-SSRS).
  11. The subject has a history of substance use disorder or any illicit drug use (other than nicotine) within the past 3 years.
  12. Positive urine drug screen for drugs of abuse (cocaine, methadone, amphetamines, cannabinoids, opiates, and barbiturates).
  13. Risk of violent behavior in the opinion of the Investigator.
  14. Known hypersensitivity to deferiprone or N-Acetylcysteine.
  15. Pregnancy or intention to become pregnant during the study duration.
  16. Female patients who are lactating.
  17. ECT treatment within 18 weeks prior to screening and study entry.
  18. Patient with substantially confounding extrapyramidal symptoms (according to screening SAS, BARS, AIMS score).
  19. Conditions that are not suitable for partaking in an MRI scan, including metal implants and incompatible devices.
  20. Subjects with BMI ≤ 18 or ≥ 40.
  21. Participation in another clinical trial involving investigational drugs within 3 months prior to screening.
  22. Clinically significant general medical conditions (neurological, cardiovascular, respiratory, gastrointestinal, renal, hepatic, hematologic, oncologic) that could interfere with participation.
  23. Major active contagious disease.
  24. Patients with clinically significant abnormalities in hematology, blood chemistry, ECG or physical examination, not resolved by the Baseline visit, that can interfere with study participation.
  25. Any condition that, in the investigator's judgment, may compromise subject safety or interfere with study assessments.

Studieplan

Denne delen gir detaljer om studieplanen, inkludert hvordan studien er utformet og hva studien måler.

Hvordan er studiet utformet?

Designdetaljer

  • Primært formål: Behandling
  • Tildeling: Randomisert
  • Intervensjonsmodell: Parallell tildeling
  • Masking: Firemannsrom

Våpen og intervensjoner

Deltakergruppe / Arm
Intervensjon / Behandling
Placebo komparator: Placebo + N-Acetylcysteine
This drug is given to both groups.
Aktiv komparator: Deferiprone + N-Acetylcysteine
This drug is given to both groups.
This intervention has not been used before on Schizophrenia or Schizoaffective patients with predominant negative symptoms.

Hva måler studien?

Primære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Change in Total PANSS Score
Tidsramme: 36 Weeks (LOCF)
Change from Baseline to Week 36 in the PANSS (Positive and Negative Syndrome Scale) total score. PANSS total score range: 30 to 210 (30 items, each rated 1-7, so minimum 30 and maximum 210). Higher scores indicate more severe symptoms.
36 Weeks (LOCF)

Sekundære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
PANSS Marder Negative Symptom Factor Score
Tidsramme: 12, 24 and 36 weeks
Change from Baseline in the PANSS Marder Negative Symptom Factor Score (NSF)
12, 24 and 36 weeks
PANSS Positive Subscale Score
Tidsramme: 12, 24 and 36 weeks
Change from Baseline in the PANSS Positive sub-scale score
12, 24 and 36 weeks
PANSS Negative Subscale Score
Tidsramme: 12, 24 and 36 weeks
Change from Baseline in the PANSS Negative sub-scale score
12, 24 and 36 weeks
PANSS General Psychopathological Subscale Score
Tidsramme: 12, 24 and 36 weeks
Change from Baseline in the PANSS General Psychopathological sub-scale score
12, 24 and 36 weeks
BNSS (Brief Negative Symptom Scale) Score
Tidsramme: 12, 24 and 36 weeks
Change from Baseline in the BNSS (Brief Negative Symptom Scale) score
12, 24 and 36 weeks
BACS (Brief Cognitive Assessment in Schizophrenia) Score
Tidsramme: 12, 24 and 36 weeks
Change from Baseline in the BACS (Brief Cognitive Assessment in Schizophrenia) score
12, 24 and 36 weeks
Reported adverse events (descriptive)
Tidsramme: From enrollment to the end of treatment at 36 weeks
Safety and tolerability assessment
From enrollment to the end of treatment at 36 weeks
Weight (kg)
Tidsramme: From enrollment to the end of treatment at 36 weeks
Safety and tolerability assessment
From enrollment to the end of treatment at 36 weeks
Height (cm)
Tidsramme: From enrollment to the end of treatment at 36 weeks
Safety and tolerability assessment
From enrollment to the end of treatment at 36 weeks
BMI (Body Mass Index, kg/cm^2)
Tidsramme: From enrollment to the end of treatment at 36 weeks
Safety and tolerability assessment
From enrollment to the end of treatment at 36 weeks
Blood pressure (mm Hg)
Tidsramme: From enrollment to the end of treatment at 36 weeks
Safety and tolerability assessment
From enrollment to the end of treatment at 36 weeks
Heart rate (BPM)
Tidsramme: From enrollment to the end of treatment at 36 weeks
Safety and tolerability assessment
From enrollment to the end of treatment at 36 weeks
Electrocardiogram (ECG)
Tidsramme: From enrollment to the end of treatment at 36 weeks
Safety and tolerability assessment
From enrollment to the end of treatment at 36 weeks
Complete Blood Count (CBC)
Tidsramme: From enrollment to the end of treatment at 36 weeks
Safety and tolerability assessment
From enrollment to the end of treatment at 36 weeks
Serum creatinine
Tidsramme: From enrollment to the end of treatment at 36 weeks
kidney function test, mg/dL, Safety and tolerability assessment
From enrollment to the end of treatment at 36 weeks
Serum alanine transaminase
Tidsramme: From enrollment to the end of treatment at 36 weeks
ALT, liver function test, U/L, Safety and tolerability assessment
From enrollment to the end of treatment at 36 weeks
Serum ferritin
Tidsramme: From enrollment to the end of treatment at 36 weeks
iron store index, ng/mL, Safety and tolerability assessment
From enrollment to the end of treatment at 36 weeks
Simpson-Angus Extrapyramidal Rating Scale (SAS)
Tidsramme: From enrollment to the end of treatment at 36 weeks
10 items, each 0-4, Safety and tolerability assessment
From enrollment to the end of treatment at 36 weeks
Barnes Akathisia Rating Scale (BARS)
Tidsramme: From enrollment to the end of treatment at 36 weeks
Global clinical assessment of akathisia (0-5), Safety and tolerability assessment
From enrollment to the end of treatment at 36 weeks
Abnormal Involuntary Movement Scale (AIMS)
Tidsramme: From enrollment to the end of treatment at 36 weeks
Items counted: 7, Per-item range: 0-4, Total (sum) range: 0-28, Safety and tolerability assessment
From enrollment to the end of treatment at 36 weeks
Columbia-Suicide Severity Rating Scale (C-SSRS)
Tidsramme: From enrollment to the end of treatment at 36 weeks
Ideation severity: 0-5, Ideation intensity (sum): 0-25, Behavior: categorical (yes/no; counts), Safety and tolerability assessment
From enrollment to the end of treatment at 36 weeks
Udvalg for Kliniske Undersøgelser Side Effect Rating Scale (UKU-SERS )
Tidsramme: From enrollment to the end of treatment at 36 weeks
Per item range: 0-3 (0 = none, 1 = mild, 2 = moderate, 3 = marked/severe), Safety and tolerability assessment
From enrollment to the end of treatment at 36 weeks
Discontinuation-Emergent Signs and Symptoms (DESS)
Tidsramme: From enrollment to the end of treatment at 36 weeks
43-item checklist: each symptom is scored 0/1 (absent/present), total range 0-43; Safety and tolerability assessment
From enrollment to the end of treatment at 36 weeks
Quantitative MRI - Proton Density (PD)
Tidsramme: baseline, 36 weeks
PD is normalized to derive the water fraction (WF); WF yields the Macromolecular Tissue Volume (MTV). These reflect the ratio of water to non water tissue and act as biomarkers of atrophy or density
baseline, 36 weeks
Quantitative MRI - R1 (Longitudinal Relaxation Rate)
Tidsramme: Baseline, 36 weeks
R1 is sensitive to myelin, iron and water content. R1 and MTV are acquired using the same variable flip angle or/and MP2RAGE sequences, which can also include MT and multi echo acquisitions for R2* and QSM
Baseline, 36 weeks
Quantitative MRI - Magnetization Transfer (MT)
Tidsramme: Baseline, 36 weeks
MT modeling estimates the macromolecular contribution to R1, yielding MTsat. Combined with R1 and MTV, this enables R1sat and MTVsat estimation, providing contrast related to saturated vs. unsaturated water pools
Baseline, 36 weeks
Quantitative MRI - R2*
Tidsramme: Baseline, 36 weeks
R2* is influenced by magnetic field inhomogeneities and is sensitive to iron deposition and other sources
Baseline, 36 weeks
Quantitative MRI - Quantitative Susceptibility Mapping (QSM)
Tidsramme: Baseline, 36 weeks
QSM estimates tissue magnetic susceptibility from R2* data and is sensitive to iron, calcium and related tissue properties
Baseline, 36 weeks
Quantitative MRI - R2 (Transverse Relaxation Rate)
Tidsramme: Baseline, 36 weeks
R2 reflects tissue integrity and is influenced by myelin and iron. Multi-component fitting allows estimation of Myelin Water Fraction (MWF)
Baseline, 36 weeks
Quantitative MRI - R1-R2 Relaxivity
Tidsramme: Baseline, 36 weeks
The voxel-wise slope of R1 vs. R2* within an ROI (R1-R2* relaxivity) can reflect specific iron forms
Baseline, 36 weeks
Quantitative MRI - Tissue Relaxivity (MTV Dependency)
Tidsramme: Baseline, 36 weeks
Parameter-MTV slope (MDM: Multidimensional Dependency on MTV) reflects lipid and macromolecular composition
Baseline, 36 weeks
Quantitative MRI - Diffusion MRI
Tidsramme: Baseline, 36 weeks
Mean Diffusivity (MD)
Baseline, 36 weeks
Quantitative MRI - Diffusion MRI
Tidsramme: Baseline, 36 weeks
Fractional Anisotropy (FA)
Baseline, 36 weeks
Quantitative MRI - G-Ratio Mapping
Tidsramme: Baseline, 36 weeks
Combines diffusion (axon density) and qMRI (myelin) data to estimate the g-ratio: the ratio of axon to fiber diameter
Baseline, 36 weeks
Quantitative MRI - Macroscopic Morphometry
Tidsramme: Baseline, 36 weeks
Cortical volume
Baseline, 36 weeks
Quantitative MRI - Neuromelanin-Sensitive MRI (NM-MRI)
Tidsramme: Baseline, 36 weeks
T1-weighted or MT-enhanced sequences detect neuromelanin in the substantia nigra and locus coeruleus; used to assess catecholaminergic neuron integrity
Baseline, 36 weeks
Magnetic Resonance Spectroscopy (MRS)
Tidsramme: Baseline, 36 weeks
Estimates concentrations of brain metabolites
Baseline, 36 weeks

Andre resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Proportion of patients showing a PANSS response of ≥30% reduction
Tidsramme: 12, 24 and 36 weeks
Proportion of patients showing a PANSS response of ≥30% reduction in corrected PANSS total score (PANSS total minus 30)
12, 24 and 36 weeks
CDSS (Calgary Depression Scale for Schizophrenia) Scale
Tidsramme: 12, 24 and 36 weeks
Change from Baseline in CDSS (Calgary Depression Scale for Schizophrenia) scale
12, 24 and 36 weeks
CGI-Schizophrenia (CGI-SCH) : Negative Symptoms Severity of Illness sub-scale
Tidsramme: 12, 24 and 36 weeks
Change from Baseline in the CGI-Schizophrenia (CGI-SCH) Scale : Negative Symptoms - Severity of Illness sub-scale; Scale: 1-7; 1 = Normal, not ill; 7 = Among the most extremely ill patients
12, 24 and 36 weeks
CGI-Schizophrenia (CGI-SCH) : Overall Severity of Illness sub-scale
Tidsramme: 12, 24 and 36 weeks
Change from Baseline in the CGI-Schizophrenia (CGI-SCH) Scale : Overall Severity of Illness sub-scale; Scale: 1-7; 1 = Normal, not ill; 7 = Among the most extremely ill patients
12, 24 and 36 weeks
CGI-Schizophrenia (CGI-SCH) : Overall Degree of Change sub-scale
Tidsramme: 12, 24 and 36 weeks
Change from Baseline in the CGI-Schizophrenia (CGI-SCH) Scale : Overall Degree of Change sub-scale; Scale: 1-7, 1 = Very much improved, 4 = No change, 7 = Very much worse
12, 24 and 36 weeks
Personal and Social Performance (PSP) Scale
Tidsramme: 12, 24 and 36 weeks
Change from Baseline in Personal and Social Performance (PSP) scale
12, 24 and 36 weeks
Self-evaluation of Negative Symptoms (SNS) Scale
Tidsramme: 12, 24 and 36 weeks
Change from Baseline in the Self-evaluation of Negative Symptoms (SNS) scale
12, 24 and 36 weeks

Samarbeidspartnere og etterforskere

Det er her du vil finne personer og organisasjoner som er involvert i denne studien.

Publikasjoner og nyttige lenker

Den som er ansvarlig for å legge inn informasjon om studien leverer frivillig disse publikasjonene. Disse kan handle om alt relatert til studiet.

Generelle publikasjoner

Studierekorddatoer

Disse datoene sporer fremdriften for innsending av studieposter og sammendragsresultater til ClinicalTrials.gov. Studieposter og rapporterte resultater gjennomgås av National Library of Medicine (NLM) for å sikre at de oppfyller spesifikke kvalitetskontrollstandarder før de legges ut på det offentlige nettstedet.

Studer hoveddatoer

Studiestart (Antatt)

1. september 2026

Primær fullføring (Antatt)

1. desember 2028

Studiet fullført (Antatt)

1. desember 2029

Datoer for studieregistrering

Først innsendt

20. november 2025

Først innsendt som oppfylte QC-kriteriene

9. august 2026

Først lagt ut (Faktiske)

12. august 2026

Oppdateringer av studieposter

Sist oppdatering lagt ut (Faktiske)

12. august 2026

Siste oppdatering sendt inn som oppfylte QC-kriteriene

9. august 2026

Sist bekreftet

1. august 2026

Mer informasjon

Begreper knyttet til denne studien

Plan for individuelle deltakerdata (IPD)

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UBESLUTTE

Legemiddel- og utstyrsinformasjon, studiedokumenter

Studerer et amerikansk FDA-regulert medikamentprodukt

Nei

Studerer et amerikansk FDA-regulert enhetsprodukt

Nei

produkt produsert i og eksportert fra USA

Nei

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