This study is testing whether high-dose Vitamin C is safe and well-tolerated in patients with two inherited red blood cell disorders - Pyruvate Kinase Deficiency (PKD) and Class A Glucose-6-Phosphate Dehydrogenase Deficiency (G6PDA). Both conditions cause red blood cells to break down too quickly, leading to anemia and related complications. Our earlier research showed that a single oral dose of Vitamin C (250 mg, 500 mg, or 750 mg) reduced red blood cell breakdown by approximately 50% within one hour. This study builds on those findings by testing different doses and frequencies of Vitamin C to find the safest and most effective dosing schedule. Participants will take Vitamin C once, twice, or three times daily over a 3-week period, with careful monitoring of blood counts, red blood cell survival, iron levels, and any side effects. The study will first enroll 3 adult patients with PKD at Huntsman Cancer Institute. If the results are safe and promising, the study will be extended to patients with G6PDA deficiency, and eventually to children ages 4 and older at Primary Children's Hospital in Salt Lake City. The goal is to establish a foundation for Vitamin C as a novel therapy to reduce anemia and red blood cell destruction in these rare inherited disorders.
Age range
18 Years
Sex
ALL
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A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
Incidence of Dose-Limiting Toxicities (DLTs)
Timeframe: From first dose of study intervention through the 28-day safety follow-up visit (approximately 7 weeks per participant)
Number of Participants With Treatment-Related Adverse Events as Assessed by NCI CTCAE v5.0
Timeframe: From first dose of study intervention through the 28-day safety follow-up visit (approximately 7 weeks per participant)
Change in Hemoglobin Concentration from Baseline
Timeframe: Baseline (Day 1) through Week 3 of treatment and 28-day safety follow-up visit
Change in Reticulocyte Count from Baseline
Timeframe: Baseline (Day 1) through Week 3 of treatment and 28-day safety follow-up visit
Change in Lactate Dehydrogenase (LDH) from Baseline
Timeframe: Baseline (Day 1) through Week 3 of treatment and 28-day safety follow-up visit
Change in Systolic Blood Pressure from Baseline
Timeframe: Baseline (Day 1) through Week 3 of treatment and 28-day safety follow-up visit
Change in Heart Rate from Baseline
Timeframe: Baseline (Day 1) through Week 3 of treatment and 28-day safety follow-up visit