A Phase Ib/IIa Study Evaluating the Safety and Tolerability of Vitamin C in Patients With Intermediate or High Risk Myelodysplastic Syndrome With TET2 Mutations
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|ClinicalTrials.gov Identifier: NCT03433781|
Recruitment Status : Suspended (It is on hold given changes planned for the protocol.)
First Posted : February 15, 2018
Last Update Posted : October 4, 2019
|Condition or disease||Intervention/treatment||Phase|
|Myelodysplastic Syndromes||Drug: 50 gm CIVI/24 hours x 5 days every 4 week||Phase 1 Phase 2|
This study will enroll patients with intermediate or high risk myelodysplastic syndrome. All patients will receive at least 1 cycle of treatment (4 weeks). Patients with clinical benefit (CR,PR, or SD) then will undergo a second 4-week cycle of treatment.
The primary objectives of this study are:
- Evaluate the safety and toxicity of high dose Vitamin C
- Estimate the proportion of Myelodysplastic syndrome (MDS) patients with Ten-eleven translocation-2, (TET2) mutations who exhibit a biological response defined as maintaing a vtamin C serum concentration of ≥1mM over the treatment cycle.
The secondary objectives are:
- Estimate the clinical efficacy, namely objectiveresponse rate (ORR). [including complete response (CR) and partial response (PR)] duration of response (DOR) and progression-free survival (PFS) as defined in the IWG (International Working Group) response criteria in myelodisplasia.
- Evaluate the pharmacokinetic profile (PK) of Vitamin C as hypomethylating or demethylating agent
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||18 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase Ib/IIa Study Evaluating the Safety and Tolerability of Vitamin C in Patients With Intermediate or High Risk Myelodysplastic Syndrome With TET2 Mutations|
|Actual Study Start Date :||May 1, 2018|
|Estimated Primary Completion Date :||July 1, 2025|
|Estimated Study Completion Date :||July 1, 2026|
Experimental: Absorbic Acid
All patients will receive at least 1 cycle of treatment (4 weeks). Patients with clinical benefit (CR,PR, or SD) then will undergo a second 4-week cycle of treatment.
Drug: 50 gm CIVI/24 hours x 5 days every 4 week
Each one ml solution will contain 500 mg ascorbic acid in the addition of disodium edetate 0.25mg, sodium hydroxide 110 mg in water with a pH in the range of 5.5 to 7.0 adjusted with sodium bicarbonate and sodium hydroxide. Vitamin C will be dispensed continuously by a computerized ambulatory drug deliver (CADD) pump, which will bereplaced every 24 hours.
- Measure of serum bioavailability of Vitamin C in Myelodysplastic syndrome (MDS) patients with TET2 mutations [ Time Frame: 6 Months ]Weekly serum anion gap as vitamin C can be associated with elevated anion gapacidosis. If anion gap is elevated (>11mEq/L) then we will hold the study drug for one week and recheck anion gap. If still elevated (>11mEq/L), we will withdraw the patient from the study, If not elevated (≤11mEq/L) we will resume vitamin C treatment.
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): NCT03433781
|United States, Florida|
|University of Miami Miller School of Medicine -Sylvester Cancer Center|
|Miami, Florida, United States, 33136|
|United States, New York|
|New York University School of Medicine|
|New York, New York, United States, 10016|
|Principal Investigator:||Mohammad M Abdul Hay, MD||NYU Langone Health|