Recruiting
Phase 1
Phase 2

N-Acetylcysteine

Sponsor:

UCLA

Code:

NCT05123365

Conditions

Myeloproliferative Neoplasm

MPN

Essential Thrombocythemia

Polycythemia Vera

Myelofibrosis

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Interventions

N-Acetylcysteine

Study Details

Brief summary:

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

Conditions

Myeloproliferative Neoplasm

MPN

Essential Thrombocythemia

Polycythemia Vera

Myelofibrosis

Study ID

NCT05123365

Start date

Jan 3, 2022

Status verified date

Mar, 2026

Completion date

Nov 15, 2026

Anticipated

Primary completion date

Nov 15, 2026

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

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 Design

Enrollment

27 participants

Anticipated

Allocation

Non randomized

Intervention Model

Sequential

Primary purpose

Treatment

Interventions and Outcome Measures

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

N-Acetylcysteine

Given PO

Primary outcome measure

  • 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. ]
  • 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. ]

Central Contacts and Locations

Central contacts

Angela Fleischman, MD, PhD

(714) 456-8000agf@hs.uci.edu

Locations

University of California, Irvine

Recruiting

Irvine, California, United States, 92617

Contacts

Angela G Fleischman, MD PhD

(949) 999-2400agf@uci.edu

More Information

Sponsor

University of California, Irvine

Last update posted

Mar 10, 2026

Last verified

Mar, 2026

Keywords

  • MPN
  • Myeloproliferative Neoplasm
  • Essential Thrombocytemia
  • Polycythemia Vera
  • Myelofibrosis

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by University of California, Irvine on 2026-03-10.