An Optimal Dose Finding Study of N-Acetylcysteine in Patients With Myeloproliferative Neoplasms
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age18+
SponsorUniversity of California, Irvine
About this trial
This is a phase I/II study evaluating the optimal dose of N-acetylcysteine (N-AC) in patients with myeloproliferative neoplasms (MPN).
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
≥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.
Disqualifiers
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.
Trial design
Design model
Sequential
Treatments tested in this trial
N-Acetylcysteine
DrugGiven PO
Treatment groups
27 Participants
are divided into 3 treatment groupsGroup A: Dose Level 1 (DL1)Experimental treatment 1 intervention
Group B: Dose Level 2 (DL2)Experimental treatment 1 intervention
Group C: Dose Level 3 (DL3)Experimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
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
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.2
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.
Time frame
7 days prior to beginning treatment until end of treatment, average of 9 weeks.Sponsors and contacts
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Source ClinicalTrials.gov