Back to search results

Optimal Dose Finding Study of N-Acetylcysteine for Myeloproliferative Neoplasms

This study is looking for the best dose of N-Acetylcysteine for people with myeloproliferative neoplasms (MPN). MPN is a group of blood cancers that includes essential thrombocythemia (ET), polycythemia vera (PV), and myelofibrosis (MF). The study will test different doses of N-Acetylcysteine, given by mouth, to see which dose works best. Researchers will measure the optimal biological dose (OBD) of N-Acetylcysteine and see if symptoms improve, specifically if 30% of participants have a reduction in their MPN-SAF Total Symptom Score. You may be able to join if you are at least 18 years old and have been diagnosed with ET, PV, or MF. The study is currently unclear about its recruitment status and plans to enroll 27 participants.

Study design
This is a Phase I/II open-label study, meaning both you and your doctors will know what treatment you are receiving. It aims to find the best dose of N-Acetylcysteine and plans to enroll 27 participants.
What's involved
Treatment will last up to 8 weeks, or longer if you continue to benefit. Symptom scores will be measured for an average of 9 weeks.
Compensation
Not stated in the trial record.
Follow-up
The study will track the optimal biological dose until 7 days after treatment completion or removal. Symptom reduction will be measured until the end of treatment.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT05123365

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

Recruiting
PHASE1Ages 18+InterventionalTreatment
University of California, Irvine
~27 participants
Updated 2026-03-10 on ClinicalTrials.gov
What's tested:N-Acetylcysteine

At a glance

Recruiting sites
1 of 2 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Optimal Biological Dose (OBD) of N-Acetylcysteine
Measured over 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.
+1 more outcome measured

Conditions

Myeloproliferative Neoplasm
MPN
Essential Thrombocythemia
Polycythemia Vera
Myelofibrosis

Explore related trials

Essential ThrombocythemiaMyelofibrosisPolycythemia Vera

Where it's being run

2 sites across 1 states
California2

Study leadership

  • Angela Fleischman, MD, PhD · PRINCIPAL_INVESTIGATOR · Chao Family Comprehensive Cancer Center

Who to contact

Angela Fleischman, MD, PhD
[email protected]
(714) 456-8000
Email the study team

Opens a ready-to-send draft in your own email app — review before sending.

Do you actually qualify for this trial?

Add a private profile and we'll compare every criterion below against your situation — and tell you which ones are met, uncertain, or excluding.

Check eligibility for this trial ~2 min · HIPAA-protected · delete anytime
Eligibility criteria

Inclusion

≥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

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
Known history of allergic reaction to N-AC.

What this trial measures

  • Optimal Biological Dose (OBD) of N-AcetylcysteineFrom 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

  • Proportion of subjects who achieve 30% reduction of MPN-SAF Total symptom score (MPN-TSS)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.