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

Study Purpose

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

Recruitment Criteria

Accepts Healthy Volunteers Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms	No
Study Type An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes. An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes. Searching Both is inclusive of interventional and observational studies.	Interventional
Eligible Ages	18 Years and Over
Gender	All

More 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.

Trial Details

Trial ID: This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.	NCT05123365
Phase Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans. Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data. Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs. Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.	Phase 1/Phase 2
Lead Sponsor The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.	University of California, Irvine
Principal Investigator The person who is responsible for the scientific and technical direction of the entire clinical study.	Angela Fleischman, MD, PhD
Principal Investigator Affiliation	Chao Family Comprehensive Cancer Center
Agency Class Category of organization(s) involved as sponsor (and collaborator) supporting the trial.	Other
Overall Status	Recruiting
Countries	United States
Conditions The disease, disorder, syndrome, illness, or injury that is being studied.	Myeloproliferative Neoplasm, MPN, Essential Thrombocythemia, Polycythemia Vera, Myelofibrosis

Additional Details

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).

Arms & Interventions

Arms

Experimental: Dose Level 1 (DL1)

Patients take N-Acetylcysteince 600 mg orally twice daily. This is the starting dose level for the study.

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.

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.

Interventions

Drug: - N-Acetylcysteine

Given PO

Contact a Trial Team

If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.

University of California, Irvine, Irvine, California

Status

Recruiting

Address

University of California, Irvine

Irvine, California, 92617

Site Contact

Angela G Fleischman, MD PhD

[email protected]

949-999-2400

Orange, California