Myeloproliferative NeoplasmPhase 1/2NCT05123365
An Optimal Dose Finding Study of N-Acetylcysteine in Patients With Myeloproliferative Neoplasms
A Phase 1/2 study testing N-Acetylcysteine in people with Myeloproliferative Neoplasm and MPN, run by University of California, Irvine at 2 sites in United States.
Trial summary
This is a phase I/II study evaluating the optimal dose of N-acetylcysteine (N-AC) in patients with myeloproliferative neoplasms (MPN). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myeloproliferative Neoplasm, MPN, Essential Thrombocythemia
- What is being tested?
- N-Acetylcysteine
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 27 participants
- Where
- 2 sitesUnited States
- Study dates
- 3 Jan 2022to 15 Nov 2026
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- ≥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.
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- 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.
Who cannot join
- 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
- Serum creatinine clearance less than 50 ml/min (calculated with Cockroft-Gault formula).
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- 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.
- Platelets < 100 × 10^9/L
- Hgb < 10 g/dL
- ANC < 0.75 × 10^9/L
- Known history of allergic reaction to N-AC.
Trial locations and contacts
2 sites in 1 country. Contact the study team or a site near you to ask about taking part.
University of California Irvine Medical Center contact
United States2 sites, 1 recruiting
- Recruiting
University of California, Irvine
Irvine, California, 92617
Angela G Fleischman, MD PhD, (949) 999-2400, agf@uci.edu
- Active, not recruiting
Chao Family Comprehensive Cancer Center, University of California, Irvine
Orange, California, 92868
About this study
This is a phase I/II study evaluating the optimal dose of N-acetylcysteine (N-AC) in patients with myeloproliferative neoplasms (MPN).
Read the detailed description
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).
- Conditions
- Myeloproliferative Neoplasm, MPN, Essential Thrombocythemia, Polycythemia Vera, Myelofibrosis
- Being tested
- N-Acetylcysteine
- Keywords
- MPN, Myeloproliferative Neoplasm, Essential Thrombocytemia, Polycythemia Vera, Myelofibrosis
- Medical subject headings
- Myeloproliferative Disorders, Thrombocythemia, Essential, Polycythemia Vera, Primary Myelofibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
N-AcetylcysteineDrug
Also called N-AC
Given PO
| Study group | Role | What they receive |
|---|---|---|
| Dose Level 1 (DL1) | Experimental | Patients take N-Acetylcysteince 600 mg orally twice daily.
This is the starting dose level for the study. Receives: Drug: N-Acetylcysteine |
| Dose Level 2 (DL2) | Experimental | Patients take N-Acetylcysteince 1200 mg orally twice daily.
If DL1 is well tolerated, the next cohort will progress to this dose level. Receives: Drug: N-Acetylcysteine |
| Dose Level 3 (DL3) | Experimental | Patients take N-Acetylcysteince 1800 mg orally twice daily.
If DL2 is well tolerated, the next cohort will progress to this dose level. Receives: Drug: N-Acetylcysteine |
What is being measured
The primary outcome is the main question the study is designed to answer.
Optimal Biological Dose (OBD) of N-Acetylcysteine
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
Measured: 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)
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.
Measured: 7 days prior to beginning treatment until end of treatment, average of 9 weeks.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Nov 2021
- Study start3 Jan 2022
- Primary completion15 Nov 2026 (estimated)
- Study completion15 Nov 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myeloproliferative NeoplasmStudies of the same condition, open and under way.
- More trials run by University of California, IrvineThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 27 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of California, Irvine
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Angela G. Fleischman, University of California, Irvine
- Investigators
- Angela Fleischman, MD, PhD, Principal investigator, Chao Family Comprehensive Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05123365
- Sponsor's protocol ID
- 20216930
- Other IDs
- UCI 20-50
- Status last verified
- Mar 2026
- First submitted
- 5 Nov 2021
- Last updated
- 10 Mar 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Optimal Dose Finding Study of N-Acetylcysteine in Patients With Myeloproliferative Neoplasms
- ClinicalTrials.gov ID
- NCT05123365
- Acronym / sponsor ID
- 20216930
- Conditions
- Myeloproliferative Neoplasm, MPN, Essential Thrombocythemia, Polycythemia Vera, Myelofibrosis
- Keywords
- MPN, Myeloproliferative Neoplasm, Essential Thrombocytemia, Polycythemia Vera, Myelofibrosis
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 27 participants (estimated)
- Lead sponsor
- University of California, IrvineUniversity, hospital or other
- Study start
- 3 Jan 2022 (actual)
- Primary completion
- 15 Nov 2026 (estimated)
- Study completion
- 15 Nov 2026 (estimated)
- First posted
- 17 Nov 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a phase I/II study evaluating the optimal dose of N-acetylcysteine (N-AC) in patients with myeloproliferative neoplasms (MPN).
Detailed description
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).
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Dose Level 1 (DL1) | Experimental | Drug: N-AcetylcysteinePatients take N-Acetylcysteince 600 mg orally twice daily. This is the starting dose level for the study. |
| Dose Level 2 (DL2) | Experimental | Drug: N-AcetylcysteinePatients take N-Acetylcysteince 1200 mg orally twice daily. If DL1 is well tolerated, the next cohort will progress to this dose level. |
| Dose Level 3 (DL3) | Experimental | Drug: N-AcetylcysteinePatients take N-Acetylcysteince 1800 mg orally twice daily. If DL2 is well tolerated, the next cohort will progress to this dose level. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- ≥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.
Key exclusion criteria (11 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| N-AcetylcysteineAlso: N-ACGiven PO | Drug | Dose Level 1 (DL1), Dose Level 2 (DL2), Dose Level 3 (DL3) |
Primary outcome
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.
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)
Time frame: 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.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Mar 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
University of California Irvine Medical Center contact
Angela Fleischman, MD, PhD principal investigator
Chao Family Comprehensive Cancer Center
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing N-Acetylcysteine in people with Myeloproliferative Neoplasm and MPN, run by University of California, Irvine at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 10 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Angela Fleischman, MD, PhD ((714) 456-8000, agf@hs.uci.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the 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.). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Mar 2026.