An Optimal Dose Finding Study of N-Acetylcysteine in Patients With Myeloproliferative Neoplasms
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT05123365|
Recruitment Status : Active, not recruiting
First Posted : November 17, 2021
Last Update Posted : January 10, 2023
- Study Details
- Tabular View
- No Results Posted
- How to Read a Study Record
|Condition or disease||Intervention/treatment||Phase|
|Myeloproliferative Neoplasm MPN Essential Thrombocythemia Polycythemia Vera Myelofibrosis||Drug: N-Acetylcysteine||Phase 1 Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||27 participants|
|Intervention Model:||Sequential Assignment|
|Masking:||None (Open Label)|
|Official Title:||An Optimal Dose Finding Study of N-Acetylcysteine in Patients With Myeloproliferative Neoplasms|
|Actual Study Start Date :||January 3, 2022|
|Estimated Primary Completion Date :||July 31, 2024|
|Estimated Study Completion Date :||November 15, 2025|
Experimental: Dose Level 1 (DL1)
Patients take N-Acetylcysteince 600 mg orally twice daily.
This is the starting dose level for the study.
Other Name: N-AC
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.
Other Name: N-AC
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.
Other Name: N-AC
- 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.
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
|Ages Eligible for Study:||18 Years and older (Adult, Older Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
- ≥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.
- 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).
- 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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05123365
|United States, California|
|Chao Family Comprehensive Cancer Center, University of California, Irvine|
|Orange, California, United States, 92868|
|Principal Investigator:||Angela Fleischman, MD, PhD||Chao Family Comprehensive Cancer Center|
|Responsible Party:||Angela G. Fleischman, Associate Professor, University of California, Irvine|
|Other Study ID Numbers:||
UCI 20-50 [HS# 2021-6930]
2021-6930 ( Other Identifier: University of California, Irvine )
|First Posted:||November 17, 2021 Key Record Dates|
|Last Update Posted:||January 10, 2023|
|Last Verified:||January 2023|
|Studies a U.S. FDA-regulated Drug Product:||Yes|
|Studies a U.S. FDA-regulated Device Product:||No|
Bone Marrow Neoplasms
Neoplasms by Site
Bone Marrow Diseases
Blood Platelet Disorders
Blood Coagulation Disorders
Respiratory System Agents
Free Radical Scavengers
Molecular Mechanisms of Pharmacological Action
Physiological Effects of Drugs