Hemoglobin Disorder, Sickle Cell Anemia, Sickle Cell Disease
Conditions
Keywords
Metformin, Sickle Cell Anemia, Sickle Cell Disease, Hemoglobinopathies, Hemoglobin Disorder, Blood Disease, Hemoglobin Disease
Brief summary
The purpose of this study is to determine whether metformin is effective in the treatment for sickle cell anemia (SCA).
Detailed description
This is a dose escalation, pilot study for subjects with sickle cell anemia (SCA) disease to determine if metformin has a beneficial effect on the treatment and quality of life of SCA patients.
Interventions
DRUGMetformin
Metfomin will be taken daily. The metformin dose will be increased during two time points per subject if protocol dose escalation criteria is met.
BEHAVIORALQuestionnaires
Questionnaires will be completed to assess the impact quality of life
Sponsors
Pfizer
Baylor College of Medicine
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
10 Years to 60 Years
Healthy volunteers
No
Inclusion criteria
1. Confirmed diagnosis of sickle cell anemia 2. Age greater than or equal to 10 and less than or equal to 60 years of age. 3. If on hydroxyurea, fetal hemoglobin less than 20% at a stable dose (mg/kg) determined by the primary hematology provider over at least four months. 4. Creatinine less than or equal to 1.4 mg/dL and estimated glomerular filtration rate greater than 45 ml/min/1.73 m2 5. Liver function tests (specifically ALT and conjugated bilirubin) less than or equal to 4 times upper limits of normal.
Exclusion criteria
1. Failure to meet inclusion criteria 2. Simple or chronic red blood cell transfusion therapy in the last 3 months OR a HbA level greater than 5% in SCA patients 3. Refusal to use medically effective birth control if female and sexually active. 4. If on hydroxyurea, not at stable dose of hydroxyurea for a minimum of 4 months (temporary exclusion). 5. Creatinine greater than 1.4mg/dL 6. Liver function tests (ALT and conjugated bilirubin) greater than 4 times upper limits of normal.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change in Fetal Hemoglobin (HbF) Percentage (SCA) or Change in Total Hemoglobin (Hb) | 1 Year | Change in HbF percentage (%) or total Hb will be assessed by comparing baseline values to on treatment values per subject and will be summarized. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in Laboratory Values | 1 Year | Evaluation and percentage of change in numeric values of total blood count, liver function, HbF levels, whole blood viscosity, and percent dense red blood cells will be evaluated per subject over the duration of the study and summarized. |
| Impact on Quality of Life | 1 Year | Evaluation of subject's change in quality of life will be assessed per subject per study questionnaire(s) over the duration of the study and summarized. |
| Variability of Hemoglobin Response | 1 Year | Evaluation of hematological variability of fetal hemoglobin induction will be assessed per subject per genetic analysis and summarized. |
| Evaluation of RNA Sequencing | 1 Year | Evaluate expression changes in paired samples collected before and on metformin through RNA sequencing. |
Countries
United States
Outcome results
None listed