Actively Recruiting
An Optimal Dose Finding Study of N-Acetylcysteine in Patients With Myeloproliferative Neoplasms
Led by University of California, Irvine · Updated on 2026-03-10
27
Participants Needed
2
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are studying the best dose of N-acetylcysteine (N-AC) for patients with myeloproliferative neoplasms (MPN), including essential thrombocythemia, polycythemia vera, and myelofibrosis. This phase I/II open-label trial aims to find the optimal biological dose of N-AC to help manage symptoms related to these blood disorders. Participants will receive oral N-acetylcysteine starting at 600 mg twice daily. If this dose is well tolerated, the dose will increase in steps to 1200 mg and then 1800 mg twice daily. The study monitors safety and symptom improvement at each dose level to determine the best dose. Treatment duration is up to 8 weeks. During the study, participants will undergo blood tests and symptom score assessments to monitor effects and side effects. Researchers will track the optimal biological dose and measure symptom reduction using the MPN-SAF Total Symptom Score over about 9 weeks. The trial includes monitoring for side effects and adherence to treatment and contraception requirements, with participation lasting through treatment and follow-up periods.
CONDITIONS
Brief Title
An Optimal Dose Finding Study of N-Acetylcysteine in Patients With Myeloproliferative Neoplasms
Who Can Participate
Eligibility Criteria
You may qualify if you...
- 18 years of age or older
- Diagnosed with essential thrombocythemia, polycythemia vera, or myelofibrosis according to 2016 WHO criteria
- Have not taken interferon-alpha or a JAK inhibitor for MPN treatment in the past 28 days
- May continue current MPN treatments such as aspirin, hydroxyurea, or anagrelide
- Have not taken N-Acetylcysteine or preparations containing it in the past 28 days
- Baseline MPN-SAF Total Symptom Score of 10 or higher at enrollment
- Peripheral blast count less than 10% during screening
- Free of other active or metastatic cancers except localized skin cancer
- Able to provide blood samples and complete symptom assessments
- Agree to use effective contraception during the study and for 60 days after last dose if of childbearing potential, including partners
You will not qualify if you...
- Eastern Cooperative Oncology Group (ECOG) score of 3 or higher
- Currently pregnant or planning pregnancy during the study period
- Currently breastfeeding
- Known uncontrolled active viral or bacterial infection
- Significant major organ impairment including:
- Serum creatinine clearance under 50 ml/min
- Bilirubin over 1.5 mg/dl except Gilbert's disease, or ALT/AST more than twice normal or liver cirrhosis
- Platelet count below 100 x 10^9/L
- Hemoglobin below 10 g/dL
- Absolute neutrophil count below 0.75 x 10^9/L
- Known allergic reaction to N-Acetylcysteine
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 8 weeks
Participants take N-Acetylcysteine orally at escalating dose levels to determine the optimal biological dose. Treatment continues until disease progression, toxicity, delay of treatment, or withdrawal, up to 8 weeks.
Weekly visits for up to 8 weeks
Trial Site Locations
Total: 2 locations
1
University of California, Irvine
Irvine, California, United States, 92617
Actively Recruiting
2
Chao Family Comprehensive Cancer Center, University of California, Irvine
Orange, California, United States, 92868
Active, Not Recruiting
Research Team
A
Angela Fleischman, MD, PhD
U
University of California Irvine Medical Center
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
3
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