High-Dose Vitamin C in G6PDA and Pyruvate Kinase Deficiency: A Safety Study

Conditions

• Glucose 6 Phosphate Dehydrogenase Deficiency
• Pyruvate Kinase Deficiency

Eligibility Criteria

Sex

ALL

Age

18 Years - N/A

Healthy Volunteers

Not accepted

Interventions

• Vitamin C — DRUG
  Oral pharmacological grade Vitamin C (Ascorbic Acid) administered at escalating doses and frequencies over a 3-week period. Three dose levels are evaluated: 250 mg, 500 mg, and 750 mg, administered once daily (QD), twice daily (BID), or three times daily (TID) depending on the assigned arm. The maximum daily dose does not exceed 2,000 mg/day, consistent with the NIH-recommended maximum daily dose.

Study Details

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.

Key Dates

Start date

Jul 15, 2026

Status verified

May 2026

Primary completion

Jul 15, 2027

Completion

Dec 15, 2027

Study Design

Enrollment

3 participants (estimated)

Allocation

NON_RANDOMIZED

Intervention model

SEQUENTIAL

Primary purpose

TREATMENT

Arms

• Experimental: Low Dose Vitamin C (250 mg)
  Participants receive oral Vitamin C at 250 mg once daily during Week 1, 250 mg twice daily during Week 2, and 250 mg three times daily during Week 3
• Experimental: Medium Dose Vitamin C (500 mg)
  Participants receive oral Vitamin C at 500 mg once daily during Week 1, 500 mg twice daily during Week 2, and 500 mg three times daily during Week 3
• Experimental: High Dose Vitamin C (750 mg)
  Participants receive oral Vitamin C at 750 mg once daily during Week 1, 750 mg twice daily during Week 2, and 750 mg twice daily plus 500 mg once daily during Week 3, to reach the NIH-recommended maximum daily dose of 2,000 mg/day

Primary Outcome Measure

Incidence of Dose-Limiting Toxicities (DLTs) [ Time Frame: From first dose of study intervention through the 28-day safety follow-up visit (approximately 7 weeks per participant) ]

Central Contacts

• Jihyun Song, PhD
  801-587-4682
  [email protected]
• Josef T. Prchal, MD
  801-213-6013
  [email protected]

Locations (1)

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