Clinical Trials Logo

Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT03398824
Other study ID # IRB-P00026540
Secondary ID
Status Completed
Phase Phase 2
First received
Last updated
Start date March 29, 2018
Est. completion date October 9, 2020

Study information

Verified date November 2022
Source Boston Children's Hospital
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

This is a single institution, open-label, single arm pilot study of Metformin in patients with Fanconi Anemia (FA) and cytopenias with the primary endpoint of hematologic response. This study will also assess safety, tolerability, and the biologic effects of Metformin in patients with FA.


Recruitment information / eligibility

Status Completed
Enrollment 15
Est. completion date October 9, 2020
Est. primary completion date October 9, 2020
Accepts healthy volunteers No
Gender All
Age group 6 Years to 35 Years
Eligibility Inclusion Criteria: - Age > 6 years and =35 years - Lansky/Karnofsky performance status = 50% for patients =16 years of age and Lansky = 50% for patients <16 years of age (see Appendix A) - Diagnosis requirement - Participants must have a clinical diagnosis of Fanconi Anemia. - Participants must have confirmed diepoxybutane-mitomycin C (DEB/MMC) stress testing to document diagnosis of Fanconi Anemia. - Patients must have at least one of the following cytopenias: Hemoglobin <10g/dL; Platelets <100k/uL; Absolute neutrophil count <1000/uL - Participants must have normal organ function as defined below: - Hepatic Function : Total bilirubin = 1.5 x upper limit of normal for age; alanine aminotransferase (ALT)/aspartate aminotransferase (AST) = 135 U/L - Renal Function: A serum creatinine based on age/gender as follows: Age Maximum Serum Creatinine (mg/dL) Male Female 1. to < 2 years 0.6 0.6 2. to < 6 years 0.8 0.8 6 to < 10 years 1 1 10 to < 13 years 1.2 1.2 13 to < 16 years 1.5 1.4 = 16 years 1.7 1.4 AND • Creatinine clearance = 60 mL/min/1.73 m2 for participants with creatinine levels above institutional normal - Normal cardiac status as documented clinically, otherwise they will need an echocardiogram prior to enrollment - Serum bicarbonate must be >17. - Participants of child-bearing or child-fathering potential must agree to use adequate contraception (hormonal birth control; intrauterine device; double barrier method; or total abstinence) throughout their participation, including up until 30 days after last dose of Metformin. - Patients must be able to swallow pills. - Ability to understand and/or the willingness of the patient (or parent or legally authorized representative, if minor) to provide informed consent, documented using an institutionally approved informed consent procedure Exclusion Criteria: - Patients must not have undergone prior bone marrow transplantation. - Patients must not have very severe aplastic anemia at the time of enrollment which would require bone marrow transplantation (as defined by at least 2 out of the following 3: Absolute Neutrophil Count (ANC) <200k/uL, platelets <20k/uL, absolute reticulocyte count <40k/uL). - Patients must not be taking any other concurrent medications to improve their hematopoiesis such as androgens or growth factors such as Granulocyte colony-stimulating factor (G-CSF), erythropoietin (EPO), or thrombopoietin (TPO) mimetics. There is a one month wash-out period for prior therapies including androgens. - Pregnant participants will not be entered on this study given that the effects of Metformin on the developing human fetus are unknown. - Breastfeeding mothers are not eligible because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with Metformin. - Patients must not have a history of allergic reactions attributed to compounds of similar chemical or biologic composition to Metformin. - Patients must not have uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements. - Patients must not have prior history of symptomatic hypoglycemia over the past year or hypoglycemia with glucose <50mg/dL on screening and baseline laboratory assessments. - Patients must not have type 1 diabetes mellitus. - Patients must abstain from alcohol as part of this study. - Patients must not have a diagnosis of myelodysplastic syndrome or leukemia, or other concurrent malignancy undergoing treatment. - Patients must not have vitamin B12 deficiency. - Patients must not have Glucose-6-Phosphate Dehydrogenase (G6PD) deficiency.

Study Design


Related Conditions & MeSH terms


Intervention

Drug:
metformin HCl
Receive metformin HCl

Locations

Country Name City State
United States Boston Children's Hospital Boston Massachusetts

Sponsors (1)

Lead Sponsor Collaborator
Boston Children's Hospital

Country where clinical trial is conducted

United States, 

Outcome

Type Measure Description Time frame Safety issue
Primary Hematologic Response Hematologic response is defined by specific increases in erythroid, platelet, and neutrophil counts.
Erythroid response: Hemoglobin (Hgb) increase by >1.5g/dL for non-transfusion dependent patients and for patients who are transfusion dependent: Transfusion independence Only transfusions given for Hgb =8g/dL or for symptoms will be counted in the response evaluation
Platelet response: If initially <20k/uL to start, then must increase to >20k/UL with an absolute increase of 100%. If initially =20k/uL to start, then must have an absolute increase of >30k/UL
Neutrophil response: If initially <500/uL to start, then must increase to >500/uL with an absolute increase of > 250/uL. If initially =500/uL to start, then must have an absolute increase of >500/uL
6 months
See also
  Status Clinical Trial Phase
Completed NCT02931071 - Clinical Phase II Trial to Evaluate CD34+ Cells Mobilization and Collection in Patients With Fanconi Anemia for Subsequent Transduction With a Lentiviral Vector Carring FANCA Gene. FANCOSTEM-1 Phase 2
Terminated NCT01319851 - Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation N/A
Recruiting NCT00084695 - Umbilical Cord Blood for Stem Cell Transplantation in Treating Young Patients With Malignant or Nonmalignant Diseases Phase 2
Completed NCT00000603 - Cord Blood Stem Cell Transplantation Study (COBLT) Phase 2
Recruiting NCT05687474 - Baby Detect : Genomic Newborn Screening
Recruiting NCT01146210 - Identification of de Novo Fanconi Anemia in Younger Patients With Newly Diagnosed Acute Myeloid Leukemia N/A
Completed NCT01082133 - Multicenter Transplant Study for Fanconi Anemia Phase 2
Completed NCT00965666 - Pilot Study of Etanercept (Enbrel) in Children With Fanconi Anemia Early Phase 1
Terminated NCT00290628 - Donor Umbilical Cord Blood Transplant in Treating Patients With Hematologic Cancer N/A
Recruiting NCT00027274 - Cancer in Inherited Bone Marrow Failure Syndromes
Terminated NCT01001598 - Safety and Efficacy Trial of Danazol in Patients With Fanconi Anemia or Dyskeratosis Congenita Phase 1/Phase 2
Recruiting NCT03206086 - Eltrombopag for People With Fanconi Anemia Phase 2
Recruiting NCT03579875 - Alpha/Beta TCD HCT in Patients With Inherited BMF Disorders Phase 2
Recruiting NCT05598515 - Time-restricted Feeding to Reduce Inflammation in Fanconi Anemia N/A
Active, not recruiting NCT03476330 - Quercetin Chemoprevention for Squamous Cell Carcinoma in Patients With Fanconi Anemia Phase 2
Terminated NCT05910853 - Whole Blood Biospecimen Collection for Subjects With Fanconi Anemia
Completed NCT03609840 - Study of Thiotepa and TEPA Drug Exposure in Pediatric Hematopoietic Stem Cell Transplant Patients
Completed NCT00479115 - Mobilization and Collection of Peripheral Blood Stem Cells in Patients With Fanconi Anemia Using G-CSF and AMD3100 Phase 1/Phase 2
Completed NCT00352976 - TBI Dose De-escalation for Fanconi Anemia Phase 2/Phase 3
Terminated NCT03600909 - A Study of the Effect of Blood Stem Cell Transplant After Chemotherapy Alone in Patients With Fanconi Anemia Phase 2