Pyruvate Kinase DeficiencyPhase 1G6PDA PKDNCT07612345
High-Dose Vitamin C in G6PDA and Pyruvate Kinase Deficiency: A Safety Study
A Phase 1 study testing Vitamin C in people with Pyruvate Kinase Deficiency and Glucose 6 Phosphate Dehydrogenase Deficiency, run by University of Utah at 1 site in United States.
Trial summary
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). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Pyruvate Kinase Deficiency, Glucose 6 Phosphate Dehydrogenase Deficiency
- What is being tested?
- Vitamin C
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 3 participants
- Where
- 1 siteUnited States
- Study dates
- 15 Jul 2026to 15 Dec 2027
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Confirmed diagnosis of Pyruvate Kinase Deficiency (PKD) or Class A glucose-6-phosphate dehydrogenase (G6PD A) as verified by genetic mutation documentation or enzymatic testing
- 18 years of age or older
- Able to take oral medications either by mouth or via gastrostomy tube
- Willing and able to provide written informed consent prior to any study procedures
- Heterozygous female carrier of G6PDA deficiency
Who cannot join
- Does not have an established diagnosis of Pyruvate Kinase Deficiency (PKD) or Class A Glucose-6-Phosphate Dehydrogenase Deficiency (G6PDA)
- Unable to tolerate oral medications either by mouth or gastrostomy tube
- Currently pregnant or planning to become pregnant during the study period
- Incarcerated individuals
- Unable to provide written informed consent or assent
- Known allergy or hypersensitivity to Vitamin C (ascorbic acid)
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site
University of Utah
Salt Lake City, Utah, 84112
Jihyun Song, PhD, 801-587-4682, jihyun.song@utah.edu
About this study
Official title: Safety and Tolerability of Supratherapeutic Vitamin C Supplementation in Class A Glucose-6-Phosphate Dehydrogenase (G6PD A) Deficiency and Pyruvate Kinase Deficiency (PKD)
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.
Read more
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.
Read the detailed description
Pyruvate kinase deficiency (PKD) and Class A Glucose-6-Phosphate Dehydrogenase deficiency (G6PDA) are inherited red blood cell enzyme disorders characterized by chronic hemolytic anemia. Current treatment options are limited and do not fully address the underlying mechanisms of red blood cell destruction, iron overload, and oxidative stress.
Our preliminary studies demonstrated that oral Vitamin C administration at doses of 250 mg, 500 mg, and 750 mg reduced the rate of hemolysis by approximately 50% within one hour of administration, as measured by end-tidal carbon monoxide corrected for ambient CO (ETCOc) - a validated marker of red blood cell breakdown. These findings provide a strong rationale for evaluating the safety and optimal dosing frequency of Vitamin C supplementation in these patient populations.
Study Design:
This is a Phase I, single-site, sequential within-patient dose escalation study. Three adult PKD patients will be enrolled in the initial cohort, each assigned to a different starting dose:
Patient 1: 250 mg daily → 250 mg twice daily → 250 mg three times daily Patient 2: 500 mg daily → 500 mg twice daily → 500 mg three times daily Patient 3: 750 mg daily → 750 mg twice daily → 750 mg twice daily + 500 mg once daily (to reach the NIH-recommended maximum daily dose of 2,000 mg/day)
Each dosing frequency will be maintained for one week, for a total active treatment period of 3 weeks per participant. Safety and hematologic assessments will be performed at baseline and at each dose adjustment visit (9 assessments per participant).
Assessments:
At each visit, participants will undergo:
ETCOc measurement to assess real-time hemolysis Complete blood count and reticulocyte count Metabolic panel including bilirubin and LDH Iron studies including serum iron, transferrin, ferritin, hepcidin, and erythroferrone Vital signs and adverse event assessment
Study Extension:
Following successful completion of the PKD cohort, the study will be extended to G6PDA patients, including 5 G6PDA males and 5 G6PDA heterozygous females from a well-characterized family. A subsequent extension to pediatric patients ages 4 and older is planned at Primary Children's Hospital in Salt Lake City, pending safety and preliminary efficacy data from the adult cohort.
- Being tested
- Vitamin C
- Keywords
- Pyruvate Kinase Deficiency, Glucose-6-Phosphate Dehydrogenase Deficiency, Antioxidant, Vitamin C, Hemolytic Anemia
- Medical subject headings
- Pyruvate Kinase Deficiency of Red Cells, Glucosephosphate Dehydrogenase Deficiency, Anemia, Hemolytic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Vitamin CDrug
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 group | Role | What they receive |
|---|---|---|
| Low Dose Vitamin C (250 mg) | Experimental | 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 Receives: Drug: Vitamin C |
| Medium Dose Vitamin C (500 mg) | Experimental | 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 Receives: Drug: Vitamin C |
| High Dose Vitamin C (750 mg) | Experimental | 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 Receives: Drug: Vitamin C |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Dose-Limiting Toxicities (DLTs)
Number of participants experiencing Grade 3 or higher adverse events as defined by NCI CTCAE v5.0 that are possibly, probably, or definitely related to oral Vitamin C administration at each dose level (250 mg, 500 mg, and 750 mg) and dosing frequency (once daily, twice daily, and three times daily). Hematologic DLTs include Grade ≥3 hemolysis above patient baseline, Grade ≥3 anemia (hemoglobin <8 g/dL), Grade 4 neutropenia (ANC <0.5 × 10⁹/L), and Grade 4 thrombocytopenia (platelet count <25 × 10⁹/L). Non-hematologic DLTs include Grade 3 acute kidney injury, Grade 3 ALT or AST elevation, and any other Grade 3 or higher toxicity considered related to study intervention.
Measured: 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
Incidence and severity of all adverse events reported during the study, graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE v5.0), including all adverse events possibly, probably, or definitely related to oral Vitamin C administration.
Measured: 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
Change in hemoglobin concentration from baseline to each dose adjustment visit, measured by complete blood count (CBC).
Measured: Baseline (Day 1) through Week 3 of treatment and 28-day safety follow-up visit
Change in Reticulocyte Count from Baseline
Change in reticulocyte count from baseline to each dose adjustment visit, measured by complete blood count (CBC).
Measured: Baseline (Day 1) through Week 3 of treatment and 28-day safety follow-up visit
Change in Lactate Dehydrogenase (LDH) from Baseline
Change in serum LDH levels from baseline to each dose adjustment visit as a marker of hemolysis.
Measured: Baseline (Day 1) through Week 3 of treatment and 28-day safety follow-up visit
Change in Systolic Blood Pressure from Baseline
Change in systolic blood pressure from baseline to each study visit as part of vital signs assessment.
Measured: Baseline (Day 1) through Week 3 of treatment and 28-day safety follow-up visit
Change in Heart Rate from Baseline
Change in heart rate from baseline to each study visit as part of vital signs assessment.
Measured: Baseline (Day 1) through Week 3 of treatment and 28-day safety follow-up visit
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 May 2026
- Study start15 Jul 2026 (estimated)
- Primary completion15 Jul 2027 (estimated)
- Study completion15 Dec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (17)Papers the sponsor has linked to this study.
- Other trials for Pyruvate Kinase DeficiencyStudies of the same condition, open and under way.
- More trials run by University of UtahThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 3 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of Utah
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Jihyun Song, University of Utah
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07612345
- Sponsor's protocol ID
- 00187727
- Acronym
- G6PDA PKD
- Status last verified
- May 2026
- First submitted
- 19 May 2026
- Last updated
- 28 May 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: De-identified individual participant data will be made available upon reasonable request following publication of study results. Data will be stored securely at the Huntsman Cancer Institute and shared in accordance with applicable IRB and institutional data sharing policies.
Publications
Gammal RS, Pirmohamed M, Somogyi AA, Morris SA, Formea CM, Elchynski AL, Oshikoya KA, McLeod HL, Haidar CE, Whirl-Carrillo M, Klein TE, Caudle KE, Relling MV. Expanded Clinical Pharmacogenetics Implementation Consortium Guideline for Medication Use in the Context of G6PD Genotype. Clin Pharmacol Ther. 2023 May;113(5):973-985. doi: 10.1002/cpt.2735. Epub 2022 Sep 24.
PubMed 36049896Mohammed BM, Fisher BJ, Kraskauskas D, Ward S, Wayne JS, Brophy DF, Fowler AA 3rd, Yager DR, Natarajan R. Vitamin C promotes wound healing through novel pleiotropic mechanisms. Int Wound J. 2016 Aug;13(4):572-84. doi: 10.1111/iwj.12484. Epub 2015 Aug 20.
PubMed 26290474Bechara N, Flood VM, Gunton JE. A Systematic Review on the Role of Vitamin C in Tissue Healing. Antioxidants (Basel). 2022 Aug 19;11(8):1605. doi: 10.3390/antiox11081605.
PubMed 36009324Coffey R, Jung G, Olivera JD, Karin G, Pereira RC, Nemeth E, Ganz T. Erythroid overproduction of erythroferrone causes iron overload and developmental abnormalities in mice. Blood. 2022 Jan 20;139(3):439-451. doi: 10.1182/blood.2021014054.
PubMed 34614145Grootendorst S, de Wilde J, van Dooijeweert B, van Vuren A, van Solinge W, Schutgens R, van Wijk R, Bartels M. The Interplay between Drivers of Erythropoiesis and Iron Homeostasis in Rare Hereditary Anemias: Tipping the Balance. Int J Mol Sci. 2021 Feb 23;22(4):2204. doi: 10.3390/ijms22042204.
PubMed 33672223Lal A, Patterson L, Goldrich A, Marsh A. Point-of-care end-tidal carbon monoxide reflects severity of hemolysis in sickle cell anemia. Pediatr Blood Cancer. 2015 May;62(5):912-4. doi: 10.1002/pbc.25447. Epub 2015 Feb 14.
PubMed 25683629Quinn J, Gerber B, Fouche R, Kenyon K, Blom Z, Muthukanagaraj P. Effect of High-Dose Vitamin C Infusion in a Glucose-6-Phosphate Dehydrogenase-Deficient Patient. Case Rep Med. 2017;2017:5202606. doi: 10.1155/2017/5202606. Epub 2017 Nov 26.
PubMed 29317868Mehta JB, Singhal SB, Mehta BC. Ascorbic-acid-induced haemolysis in G-6-PD deficiency. Lancet. 1990 Oct 13;336(8720):944. doi: 10.1016/0140-6736(90)92317-b. No abstract available.
PubMed 1976956McLennan JD, Sparshu S. Returning to Stimulants in Children with Treatment Resistant ADHD: A Case Series. J Can Acad Child Adolesc Psychiatry. 2018 Jan;27(1):50-56. Epub 2018 Jan 1.
PubMed 29375634Padayatty SJ, Katz A, Wang Y, Eck P, Kwon O, Lee JH, Chen S, Corpe C, Dutta A, Dutta SK, Levine M. Vitamin C as an antioxidant: evaluation of its role in disease prevention. J Am Coll Nutr. 2003 Feb;22(1):18-35. doi: 10.1080/07315724.2003.10719272.
PubMed 12569111Carr AC, Maggini S. Vitamin C and Immune Function. Nutrients. 2017 Nov 3;9(11):1211. doi: 10.3390/nu9111211.
PubMed 29099763Juneja D, Jain R, Nasa P. Vitamin C-induced Hemolysis: Meta-summary and Review of Literature. Indian J Crit Care Med. 2022 Feb;26(2):224-227. doi: 10.5005/jp-journals-10071-24111.
PubMed 35712748Gayet RV, Ilia K, Razavi S, Tippens ND, Lalwani MA, Zhang K, Chen JX, Chen JC, Vargas-Asencio J, Collins JJ. Autocatalytic base editing for RNA-responsive translational control. Nat Commun. 2023 Mar 11;14(1):1339. doi: 10.1038/s41467-023-36851-z.
PubMed 36906659Rubinstein MP, Lind NA, Purton JF, Filippou P, Best JA, McGhee PA, Surh CD, Goldrath AW. IL-7 and IL-15 differentially regulate CD8+ T-cell subsets during contraction of the immune response. Blood. 2008 Nov 1;112(9):3704-12. doi: 10.1182/blood-2008-06-160945. Epub 2008 Aug 8.
PubMed 18689546Bahr TM, Agarwal AM, Meznarich JA, Prince WL, Wait TWP, Prchal JT, Christensen RD. Thirty-five males with severe (Class 1) G6PD deficiency (c.637G>T) in a North American family of European ancestry. Blood Cells Mol Dis. 2021 Dec;92:102625. doi: 10.1016/j.bcmd.2021.102625. Epub 2021 Nov 4.
PubMed 34773909
Study at a glance
From the registry record, last updated by the sponsor on 28 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety and Tolerability of Supratherapeutic Vitamin C Supplementation in Class A Glucose-6-Phosphate Dehydrogenase (G6PD A) Deficiency and Pyruvate Kinase Deficiency (PKD)
- ClinicalTrials.gov ID
- NCT07612345
- Acronym / sponsor ID
- G6PDA PKD / 00187727
- Keywords
- Pyruvate Kinase Deficiency, Glucose-6-Phosphate Dehydrogenase Deficiency, Antioxidant, Vitamin C, Hemolytic Anemia
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 3 participants (estimated)
- Lead sponsor
- University of UtahUniversity, hospital or other
- Study start
- 15 Jul 2026 (estimated)
- Primary completion
- 15 Jul 2027 (estimated)
- Study completion
- 15 Dec 2027 (estimated)
- First posted
- 28 May 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
Pyruvate kinase deficiency (PKD) and Class A Glucose-6-Phosphate Dehydrogenase deficiency (G6PDA) are inherited red blood cell enzyme disorders characterized by chronic hemolytic anemia. Current treatment options are limited and do not fully address the underlying mechanisms of red blood cell destruction, iron overload, and oxidative stress.
Our preliminary studies demonstrated that oral Vitamin C administration at doses of 250 mg, 500 mg, and 750 mg reduced the rate of hemolysis by approximately 50% within one hour of administration, as measured by end-tidal carbon monoxide corrected for ambient CO (ETCOc) - a validated marker of red blood cell breakdown. These findings provide a strong rationale for evaluating the safety and optimal dosing frequency of Vitamin C supplementation in these patient populations.
Study Design:
This is a Phase I, single-site, sequential within-patient dose escalation study. Three adult PKD patients will be enrolled in the initial cohort, each assigned to a different starting dose:
Patient 1: 250 mg daily → 250 mg twice daily → 250 mg three times daily Patient 2: 500 mg daily → 500 mg twice daily → 500 mg three times daily Patient 3: 750 mg daily → 750 mg twice daily → 750 mg twice daily + 500 mg once daily (to reach the NIH-recommended maximum daily dose of 2,000 mg/day)
Each dosing frequency will be maintained for one week, for a total active treatment period of 3 weeks per participant. Safety and hematologic assessments will be performed at baseline and at each dose adjustment visit (9 assessments per participant).
Assessments:
At each visit, participants will undergo:
ETCOc measurement to assess real-time hemolysis Complete blood count and reticulocyte count Metabolic panel including bilirubin and LDH Iron studies including serum iron, transferrin, ferritin, hepcidin, and erythroferrone Vital signs and adverse event assessment
Study Extension:
Following successful completion of the PKD cohort, the study will be extended to G6PDA patients, including 5 G6PDA males and 5 G6PDA heterozygous females from a well-characterized family. A subsequent extension to pediatric patients ages 4 and older is planned at Primary Children's Hospital in Salt Lake City, pending safety and preliminary efficacy data from the adult cohort.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Low Dose Vitamin C (250 mg) | Experimental | Drug: Vitamin CParticipants 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 |
| Medium Dose Vitamin C (500 mg) | Experimental | Drug: Vitamin CParticipants 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 |
| High Dose Vitamin C (750 mg) | Experimental | Drug: Vitamin CParticipants 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 |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Confirmed diagnosis of Pyruvate Kinase Deficiency (PKD) or Class A glucose-6-phosphate dehydrogenase (G6PD A) as verified by genetic mutation documentation or enzymatic testing
- 18 years of age or older
- Able to take oral medications either by mouth or via gastrostomy tube
- Willing and able to provide written informed consent prior to any study procedures
- Heterozygous female carrier of G6PDA deficiency
Key exclusion criteria (6 listed)
- Does not have an established diagnosis of Pyruvate Kinase Deficiency (PKD) or Class A Glucose-6-Phosphate Dehydrogenase Deficiency (G6PDA)
- Unable to tolerate oral medications either by mouth or gastrostomy tube
- Currently pregnant or planning to become pregnant during the study period
- Incarcerated individuals
- Unable to provide written informed consent or assent
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Vitamin COral 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. | Drug | High Dose Vitamin C (750 mg), Low Dose Vitamin C (250 mg), Medium Dose Vitamin C (500 mg) |
Primary outcome
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)
Number of participants experiencing Grade 3 or higher adverse events as defined by NCI CTCAE v5.0 that are possibly, probably, or definitely related to oral Vitamin C administration at each dose level (250 mg, 500 mg, and 750 mg) and dosing frequency (once daily, twice daily, and three times daily). Hematologic DLTs include Grade ≥3 hemolysis above patient baseline, Grade ≥3 anemia (hemoglobin <8 g/dL), Grade 4 neutropenia (ANC <0.5 × 10⁹/L), and Grade 4 thrombocytopenia (platelet count <25 × 10⁹/L). Non-hematologic DLTs include Grade 3 acute kidney injury, Grade 3 ALT or AST elevation, and any other Grade 3 or higher toxicity considered related to study intervention.
Number of Participants With Treatment-Related Adverse Events as Assessed by NCI CTCAE v5.0
Time frame: From first dose of study intervention through the 28-day safety follow-up visit (approximately 7 weeks per participant)
Incidence and severity of all adverse events reported during the study, graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE v5.0), including all adverse events possibly, probably, or definitely related to oral Vitamin C administration.
Change in Hemoglobin Concentration from Baseline
Time frame: Baseline (Day 1) through Week 3 of treatment and 28-day safety follow-up visit
Change in hemoglobin concentration from baseline to each dose adjustment visit, measured by complete blood count (CBC).
Change in Reticulocyte Count from Baseline
Time frame: Baseline (Day 1) through Week 3 of treatment and 28-day safety follow-up visit
Change in reticulocyte count from baseline to each dose adjustment visit, measured by complete blood count (CBC).
Change in Lactate Dehydrogenase (LDH) from Baseline
Time frame: Baseline (Day 1) through Week 3 of treatment and 28-day safety follow-up visit
Change in serum LDH levels from baseline to each dose adjustment visit as a marker of hemolysis.
Change in Systolic Blood Pressure from Baseline
Time frame: Baseline (Day 1) through Week 3 of treatment and 28-day safety follow-up visit
Change in systolic blood pressure from baseline to each study visit as part of vital signs assessment.
Change in Heart Rate from Baseline
Time frame: Baseline (Day 1) through Week 3 of treatment and 28-day safety follow-up visit
Change in heart rate from baseline to each study visit as part of vital signs assessment.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 May 2026.
- Background: PubMed 36049896Gammal RS, Pirmohamed M, Somogyi AA, Morris SA, Formea CM, Elchynski AL, Oshikoya KA, McLeod HL, Haidar CE, Whirl-Carrillo M, Klein TE, Caudle KE, Relling MV. Expanded Clinical Pharmacogenetics Implementation Consortium Guideline for Medication Use in the Context of G6PD Genotype. Clin Pharmacol Ther. 2023 May;113(5):973-985. doi: 10.1002/cpt.2735. Epub 2022 Sep 24.
- Background: PubMed 26290474Mohammed BM, Fisher BJ, Kraskauskas D, Ward S, Wayne JS, Brophy DF, Fowler AA 3rd, Yager DR, Natarajan R. Vitamin C promotes wound healing through novel pleiotropic mechanisms. Int Wound J. 2016 Aug;13(4):572-84. doi: 10.1111/iwj.12484. Epub 2015 Aug 20.
- Background: PubMed 36009324Bechara N, Flood VM, Gunton JE. A Systematic Review on the Role of Vitamin C in Tissue Healing. Antioxidants (Basel). 2022 Aug 19;11(8):1605. doi: 10.3390/antiox11081605.
- Background: PubMed 34614145Coffey R, Jung G, Olivera JD, Karin G, Pereira RC, Nemeth E, Ganz T. Erythroid overproduction of erythroferrone causes iron overload and developmental abnormalities in mice. Blood. 2022 Jan 20;139(3):439-451. doi: 10.1182/blood.2021014054.
- Background: PubMed 33672223Grootendorst S, de Wilde J, van Dooijeweert B, van Vuren A, van Solinge W, Schutgens R, van Wijk R, Bartels M. The Interplay between Drivers of Erythropoiesis and Iron Homeostasis in Rare Hereditary Anemias: Tipping the Balance. Int J Mol Sci. 2021 Feb 23;22(4):2204. doi: 10.3390/ijms22042204.
- Background: PubMed 25683629Lal A, Patterson L, Goldrich A, Marsh A. Point-of-care end-tidal carbon monoxide reflects severity of hemolysis in sickle cell anemia. Pediatr Blood Cancer. 2015 May;62(5):912-4. doi: 10.1002/pbc.25447. Epub 2015 Feb 14.
- Background: PubMed 29317868Quinn J, Gerber B, Fouche R, Kenyon K, Blom Z, Muthukanagaraj P. Effect of High-Dose Vitamin C Infusion in a Glucose-6-Phosphate Dehydrogenase-Deficient Patient. Case Rep Med. 2017;2017:5202606. doi: 10.1155/2017/5202606. Epub 2017 Nov 26.
- Background: PubMed 1976956Mehta JB, Singhal SB, Mehta BC. Ascorbic-acid-induced haemolysis in G-6-PD deficiency. Lancet. 1990 Oct 13;336(8720):944. doi: 10.1016/0140-6736(90)92317-b. No abstract available.
- Background: PubMed 29375634McLennan JD, Sparshu S. Returning to Stimulants in Children with Treatment Resistant ADHD: A Case Series. J Can Acad Child Adolesc Psychiatry. 2018 Jan;27(1):50-56. Epub 2018 Jan 1.
- Background: PubMed 12569111Padayatty SJ, Katz A, Wang Y, Eck P, Kwon O, Lee JH, Chen S, Corpe C, Dutta A, Dutta SK, Levine M. Vitamin C as an antioxidant: evaluation of its role in disease prevention. J Am Coll Nutr. 2003 Feb;22(1):18-35. doi: 10.1080/07315724.2003.10719272.
- Background: PubMed 29099763Carr AC, Maggini S. Vitamin C and Immune Function. Nutrients. 2017 Nov 3;9(11):1211. doi: 10.3390/nu9111211.
- Background: PubMed 35712748Juneja D, Jain R, Nasa P. Vitamin C-induced Hemolysis: Meta-summary and Review of Literature. Indian J Crit Care Med. 2022 Feb;26(2):224-227. doi: 10.5005/jp-journals-10071-24111.
- Individual participant dataSharing planned: Yes. De-identified individual participant data will be made available upon reasonable request following publication of study results. Data will be stored securely at the Huntsman Cancer Institute and shared in accordance with applicable IRB and institutional data sharing policies.
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Pediatric Pyruvate Kinase Deficiency, Pediatric Hemolytic Anemia
Active, not recruitingPhase 3
A Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Participants With Pyruvate Kinase Deficiency (PKD) Who Are Not Regularly Transfused, Followed by a 5-Year Extension Period
Pediatric Pyruvate Kinase Deficiency, Pediatric Hemolytic Anemia
RecruitingObservational study
RADeep Multicenter European Epidemiological Platform for Patients Diagnosed With Rare Anemia Disorders (RADs)
Sickle Cell Disease, Thalassemia
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Vitamin C in people with Pyruvate Kinase Deficiency and Glucose 6 Phosphate Dehydrogenase Deficiency, run by University of Utah at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 28 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Jihyun Song, PhD (801-587-4682, jihyun.song@utah.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the 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)). The study lists 7 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT07612345, which also lists 17 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 May 2026.