Ignite Creation Date:
2024-10-26 @ 3:35 PM
Last Modification Date:
2024-10-26 @ 3:35 PM
Study NCT ID:
NCT06519786
Status:
RECRUITING
Last Update Posted:
None
First Post:
2023-12-27
Brief Title:
Safety and Efficacy of Metformin for Treatment of Cytopenia in Children and Adolescents With Fanconi Anemia
Sponsor:
None
Organization:
None
Study Overview
Official Title:
Safety and Efficacy of Metformin for Treatment of Cytopenia in Children and Adolescents With Fanconi Anemia
Status:
RECRUITING
Status Verified Date:
2023-12
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
Prospective interventional open-label non-randomized controlled trial to assess safety and efficacy of metformin in treating cytopenia in children and adolescents with Fanconi Anemia
Detailed Description:
Fanconi anemia FA is a genetic disease characterized by bone marrow failure cancer susceptibility and developmental abnormalities Allogeneic hematopoietic stem cell transplantation offers curative therapy for hematologic complications of FA
Oxymetholone is commonly used in the management of FA as it improves blood counts red cells and platelets However its use is limited by its high toxicity profile
Metformin is a potential agent that reduces levels of both chromosomal radials and breaks in FA cells and increases the size of the hematopoietic stem cell compartment thus reducing cytopenia in patients with FA
Oxymetholone is commonly used in the management of FA as it improves blood counts red cells and platelets However its use is limited by its high toxicity profile
Metformin is a potential agent that reduces levels of both chromosomal radials and breaks in FA cells and increases the size of the hematopoietic stem cell compartment thus reducing cytopenia in patients with FA
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None