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Recruiting NCT05123365

An Optimal Dose Finding Study of N-Acetylcysteine in Patients With Myeloproliferative Neoplasms

Phase I / Phase II Interventional Myeloproliferative Neoplasm MPN Essential Thrombocythemia Polycythemia Vera

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: N-Acetylcysteine.
Who it may be relevant to
Registry conditions: Myeloproliferative Neoplasm, MPN, Essential Thrombocythemia, Polycythemia Vera. Basic parameters: from 18 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Overview

This is a phase I/II study evaluating the optimal dose of N-acetylcysteine (N-AC) in patients with myeloproliferative neoplasms (MPN).

Detailed description

This is a phase I/II open-label clinical trial determining the optimal biological dose (OBD) of N-acetylcysteine in subjects with myeloproliferative neoplasms. These are subjects who have a diagnosis of essential thrombocythemia (ET), polycythemia vera (PV), or myelofibrosis (MF).

Interventions

  • Drug N-Acetylcysteine
    Given PO

Primary outcome measures

  • Optimal Biological Dose (OBD) of N-Acetylcysteine [Time frame: From the start date of treatment until 7 days after completion of treatment or removal of treatment due to disease progression, toxicity, delay of treatment, or withdrawal of treatment, whichever came first, up to 8 weeks.]
  • Proportion of subjects who achieve 30% reduction of MPN-SAF Total symptom score (MPN-TSS) [Time frame: 7 days prior to beginning treatment until end of treatment, average of 9 weeks.]

Eligibility criteria

Inclusion criteria

  • ≥18 years of age
  • Have a diagnosis of essential thrombocythemia (ET), polycythemia vera (PV), or myelofibrosis (MF) according to the 2016 WHO criteria
  • Has not taken interferon-alpha or a JAK inhibitor (such as ruxolitinib or fedratinib) for treatment of MPN in the past 28 days before enrollment.
  • May continue on current MPN treatment, including aspirin, hydroxyurea, or anagrelide. Therapeutic phlebotomies should continue per the patient's usual regimen.
  • Has not taken N-Acetylcysteine (N-AC) or preparations containing N-AC in the past 28 days before enrollment.
  • Baseline MPN-TSS score of ≥ 10 at the time of enrollment.
  • Peripheral blast count <10% during Screening.
  • Free of other active or metastatic malignancies other than localized skin cancer.
  • Amenable to blood draws and symptom assessments.
  • Agree to the use of contraceptives. Female subjects of childbearing potential and their male partners, or male subjects who have female partners of childbearing potential, should both use an effective contraception method during the study and continue to use contraception for 60 days after the last dose of study drug.

Exclusion criteria

  • Eastern Cooperative Oncology Group (ECOG) questionnaire score of ≥3
  • Currently pregnant or planning on being pregnant within the study period.
  • Currently breastfeeding.
  • Known uncontrolled active viral or bacterial infection.
  • Significant impairment of major organ function defined as
  • Serum creatinine clearance less than 50 ml/min (calculated with Cockroft-Gault formula).
  • Bilirubin more than 1.5 mg/dl except for Gilbert's disease. ALT or AST more than 2X upper normal limit or has radiologic evidence of liver cirrhosis.
  • Platelets < 100 × 10\^9/L
  • Hgb < 10 g/dL
  • ANC < 0.75 × 10\^9/L
  • Known history of allergic reaction to N-AC.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
Non-randomized
Model
Sequential
Masking
Open label
Primary purpose
Treatment

Study locations

United States · 2 centers
  • University of California, Irvine — Irvine
  • Chao Family Comprehensive Cancer Center, University of California, Irvine — Orange

Identifiers

NCT: NCT05123365 · 20216930 · UCI 20-50

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗