What side effects are associated with this type of intervention?

Common treatments for Myeloproliferative Neoplasms (MPNs) include N-acetylcysteine (N-AC), chemotherapy agents like busulphan and 6-thioguanine, ruxolitinib, and pegylated interferon alpha-2a. N-AC is generally well-tolerated but may cause gastrointestinal disturbances. Chemotherapy agents can lead to side effects such as neutropenia, leukopenia, and increased risk of infections. Ruxolitinib may cause cytopenias, dizziness, headache, fatigue, and increased susceptibility to infections. Pegylated interferon alpha-2a is associated with leukopenia and mild to moderate flu-like symptoms.

What prior FDA approvals exist?

The FDA has approved several treatments for Myeloproliferative Neoplasms (MPNs), including hydroxyurea, which is commonly used for polycythemia vera and essential thrombocythemia. Anagrelide is another approved drug specifically for thrombocytosis in MPNs, reducing platelet counts effectively. While N-acetylcysteine (N-AC) is being studied for its antioxidant properties and potential benefits in MPNs, it is not yet FDA-approved for this indication. Other treatments like romiplostim and eltrombopag, which are thrombopoietin receptor agonists, have shown efficacy in managing thrombocytopenia but come with concerns about leukemic transformation, particularly in myelodysplastic syndromes.

What other types of research exist?

Promising novel alternatives to N-acetylcysteine for Myeloproliferative Neoplasms patients include JAK inhibitors like ruxolitinib and fedratinib, which target the JAK-STAT pathway commonly mutated in MPNs. Additionally, interferon-alpha and its pegylated forms have shown efficacy in controlling disease progression and symptom burden. Investigational agents such as BET inhibitors and telomerase inhibitors are also being explored in clinical trials and may offer new therapeutic options.

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Antioxidant

N-Acetylcysteine for Myeloproliferative Disorders

Verified Trial

Phase 1 & 2

Waitlist Available

Led By Angela Fleischman, MD, PhD

Research Sponsored by University of California, Irvine

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

≥18 years of age

Have a diagnosis of essential thrombocythemia (ET), polycythemia vera (PV), or myelofibrosis (MF) according to the 2016 WHO criteria

Must not have

Are you currently taking interferon or a Jak inhibitor?

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 7 days prior to beginning treatment until end of treatment, average of 9 weeks.

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing the best dose of N-acetylcysteine for patients with certain types of blood cancers. The medication helps reduce inflammation and protect cells, which might be beneficial for these patients. N-acetylcysteine (NAC) has been studied for its potential to prevent heart damage caused by certain cancer treatments and reduce flare-ups in chronic lung disease.

Who is the study for?

Adults diagnosed with essential thrombocythemia, polycythemia vera, or myelofibrosis can join this trial. They must be on stable MPN treatment and not have used interferon-alpha, JAK inhibitors, or N-Acetylcysteine recently. Participants need a certain symptom score and agree to use contraception. Those with severe allergies to N-AC, poor organ function, low blood counts, active infections or pregnancy are excluded.

What is being tested?

The study is testing the best dose of a drug called N-Acetylcysteine for patients with myeloproliferative neoplasms (MPNs). It's in early stages (phase I/II) to see how much should be given safely while participants continue their usual treatments for MPNs.

What are the potential side effects?

Potential side effects of N-Acetylcysteine may include allergic reactions for those sensitive to it. Since this is a dose-finding study, other specific side effects will be monitored as different doses are tested.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am 18 years old or older.

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I have been diagnosed with ET, PV, or MF according to the latest criteria.

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I have been diagnosed with ET, PV, or MF according to the latest criteria.

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I have no active cancers except for skin cancer that hasn't spread.

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I am willing to have my blood drawn and my symptoms checked regularly.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

Select...

I am not currently taking interferon or a Jak inhibitor.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 7 days prior to beginning treatment until end of treatment, average of 9 weeks.

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~7 days prior to beginning treatment until end of treatment, average of 9 weeks.

This trial's timeline: 3 weeks for screening, Varies for treatment, and 7 days prior to beginning treatment until end of treatment, average of 9 weeks. for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Optimal Biological Dose (OBD) of N-Acetylcysteine

Proportion of subjects who achieve 30% reduction of MPN-SAF Total symptom score (MPN-TSS)

Side effects data

From 2015 Phase 3 trial • 302 Patients • NCT01675661

Headache

Nausea

Diarrhea

Abdominal discomfort

Dyspepsia

Pruritis

Cellulitis

Alcohol abuse

Road traffic accident

Arthropod bite

Gastrointestinal disorder

Abdominal pain

Vomiting

Reflux disease

Dizziness

Insomnia

Rash

Energy increased

Groin Abscess

Flushing

Fatigue

Panic attack

Depression

Blood pressure increased

100%

80%

60%

40%

20%

Study treatment Arm

NAC Plus CM

Placebo Plus CM

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

3Treatment groups

Experimental Treatment

Group I: Dose Level 3 (DL3)Experimental Treatment1 Intervention

Patients take N-Acetylcysteince 1800 mg orally twice daily. If DL2 is well tolerated, the next cohort will progress to this dose level.

Group II: Dose Level 2 (DL2)Experimental Treatment1 Intervention

Patients take N-Acetylcysteince 1200 mg orally twice daily. If DL1 is well tolerated, the next cohort will progress to this dose level.

Group III: Dose Level 1 (DL1)Experimental Treatment1 Intervention

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

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

N-Acetylcysteine

2013

Completed Phase 3

~1470

0 / 6Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Common treatments for Myeloproliferative Neoplasms (MPNs) include antioxidants like N-acetylcysteine (N-AC), which replenishes glutathione levels to reduce oxidative stress, a key factor in disease progression. Hydroxyurea inhibits DNA synthesis to curb cell proliferation, while interferon-alpha modulates the immune response and has antiproliferative effects. These treatments are crucial for managing blood cell counts, alleviating symptoms, and preventing complications such as thrombosis and transformation to acute leukemia.