Finding the best dose of N-Acetylcysteine for patients with blood disorders
An Optimal Dose Finding Study of N-Acetylcysteine in Patients With Myeloproliferative Neoplasms
This study is testing the best dose of N-Acetylcysteine to see if it can help people with certain blood disorders feel better while they continue their usual treatments.
Quick facts
| Phase | Phase1; Phase2 |
|---|---|
| Study type | Interventional |
| Enrollment | 27 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | University of California, Irvine Academic / other |
| Drugs / interventions | ruxolitinib, fedratinib |
| Locations | 2 sites (Irvine, California and 1 other locations) |
| Trial ID | NCT05123365 on ClinicalTrials.gov |
What this trial studies
This clinical trial is an open-label phase I/II study aimed at determining the optimal biological dose of N-acetylcysteine (N-AC) for patients diagnosed with myeloproliferative neoplasms, including essential thrombocythemia, polycythemia vera, and myelofibrosis. Participants will be evaluated based on their response to N-AC while continuing their current MPN treatments. The study will assess the safety and efficacy of N-AC in improving symptoms associated with these blood disorders.
Who should consider this trial
Good fit: Ideal candidates for this study are adults aged 18 and older with a confirmed diagnosis of essential thrombocythemia, polycythemia vera, or myelofibrosis who meet specific eligibility criteria.
Not a fit: Patients who have recently used interferon-alpha, JAK inhibitors, or N-acetylcysteine may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide a new treatment option that improves symptoms and quality of life for patients with myeloproliferative neoplasms.
How similar studies have performed: While there have been studies on N-acetylcysteine for various conditions, this specific approach for myeloproliferative neoplasms is novel and has not been extensively tested.
Eligibility criteria
Show full inclusion / exclusion 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 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.
Where this trial is running
Irvine, California and 1 other locations
- University of California, Irvine — Irvine, California, United States (Recruiting)
- Chao Family Comprehensive Cancer Center, University of California, Irvine — Orange, California, United States (Active_not_recruiting)
Study contacts
- Principal investigator: Angela Fleischman, MD, PhD — Chao Family Comprehensive Cancer Center
- Study coordinator: Angela Fleischman, MD, PhD
- Email: agf@hs.uci.edu
- Phone: (714) 456-8000
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.