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

March 6, 2026 updated by: Angela G. Fleischman, University of California, Irvine
This is a phase I/II study evaluating the optimal dose of N-acetylcysteine (N-AC) in patients with myeloproliferative neoplasms (MPN).

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

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).

Study Type

Interventional

Enrollment (Estimated)

27

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

  • Name: University of California Irvine Medical Center

Study Contact Backup

  • Name: Angela Fleischman, MD, PhD
  • Phone Number: (714) 456-8000
  • Email: agf@hs.uci.edu

Study Locations

  • United States
    • California
      • Irvine, California, United States, 92617
        • Recruiting
        • University of California, Irvine
        • Contact:
          • Angela G Fleischman, MD PhD
          • Phone Number: (949) 999-2400
          • Email: agf@uci.edu
      • Orange, California, United States, 92868
        • Active, not recruiting
        • Chao Family Comprehensive Cancer Center, University of California, Irvine

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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

    1. Serum creatinine clearance less than 50 ml/min (calculated with Cockroft-Gault formula).
    2. 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.
    3. Platelets < 100 × 10^9/L
    4. Hgb < 10 g/dL
    5. ANC < 0.75 × 10^9/L
  • Known history of allergic reaction to N-AC.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Sequential Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Dose Level 1 (DL1)

Patients take N-Acetylcysteince 600 mg orally twice daily.

This is the starting dose level for the study.
Drug: N-Acetylcysteine
Given PO
Other Names:
  • N-AC
Experimental: Dose Level 2 (DL2)

Patients take N-Acetylcysteince 1200 mg orally twice daily.

If DL1 is well tolerated, the next cohort will progress to this dose level.
Drug: N-Acetylcysteine
Given PO
Other Names:
  • N-AC
Experimental: Dose Level 3 (DL3)

Patients take N-Acetylcysteince 1800 mg orally twice daily.

If DL2 is well tolerated, the next cohort will progress to this dose level.
Drug: N-Acetylcysteine
Given PO
Other Names:
  • N-AC

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
Determination of the optimal biological dose (OBD) will be utilized to evaluate the safety and tolerability of N-AC as a treatment for patients with MPN. Optimal biological dose is defined as the therapeutic dose that possesses the highest efficacy probability while inducing acceptable toxicity
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.
MPN-SAF Total symptom score (MPN-TSS) is a validated tool to objectively measure the burden of symptoms associated with MPN. Baseline TSS will be defined as the average of the daily TSS of 7 consecutive days immediately prior to beginning N-AC. The end of study MPN-TSS will be defined as the average of the daily TSS of 7 consecutive days during week 8.
7 days prior to beginning treatment until end of treatment, average of 9 weeks.

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

University of California, Irvine

Investigators

  • Principal Investigator: Angela Fleischman, MD, PhD, Chao Family Comprehensive Cancer Center

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

January 3, 2022

Primary Completion (Estimated)

November 15, 2026

Study Completion (Estimated)

November 15, 2026

Study Registration Dates

First Submitted

November 5, 2021

First Submitted That Met QC Criteria

November 5, 2021

First Posted (Actual)

November 17, 2021

Study Record Updates

Last Update Posted (Actual)

March 10, 2026

Last Update Submitted That Met QC Criteria

March 6, 2026

Last Verified

March 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 20216930
  • UCI 20-50 (Other Identifier: CFCCC)

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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