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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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12 May 2025 |
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Main ID: |
NCT05078879 |
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Date of registration:
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14/10/2021 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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Empagliflozin as a Treatment for Severe Congenital Neutropenia Due to G6PC3 Deficiency
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Scientific title:
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A Phase 1 Study of Empagliflozin as Treatment for Severe Congenital Neutropenia Due to G6PC3 Deficiency |
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Date of first enrolment:
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November 16, 2021 |
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Target sample size:
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10 |
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Recruitment status: |
Recruiting |
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URL:
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https://clinicaltrials.gov/ct2/show/NCT05078879 |
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Study type:
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Interventional |
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Study design:
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Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
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Phase:
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Phase 1
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Countries of recruitment
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United States
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Contacts
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Name:
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David H McDermott, M.D. |
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Address:
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Telephone:
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Email:
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Affiliation:
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National Institute of Allergy and Infectious Diseases (NIAID) |
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Name:
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OPR Office of Patient Recruitment |
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Address:
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Telephone:
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800-411-1222 |
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Email:
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ccopr@nih.gov |
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Affiliation:
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Name:
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Daniel Velez, R.N. |
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Address:
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Telephone:
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(301) 761-6753 |
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Email:
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daniel.velez@nih.gov |
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Affiliation:
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Key inclusion & exclusion criteria
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- INCLUSION CRITERIA:
1. Aged >=18 years.
2. Documented SCN due to G6PC3 deficiency defined by genetic testing.
3. History of ANC consistently <1000 cells/microL when not treated with G-CSF.
4. Current ANC<1000 cells/microL when not treated with G-CSF.
5. Participants must agree not to become pregnant for the duration of the study.
Study participants must use 2 methods of birth control when engaging in sexual
activities that can result in pregnancy, beginning 30 days before the first
dose of empagliflozin through one month after treatment ends. One method must
be a male or female condom. The other method may be any of the following:
1. Hormonal contraception.
2. Diaphragm or cervical cap with a spermicide.
3. Intrauterine device.
6. Able to provide informed consent.
EXCLUSION CRITERIA:
Individuals meeting any of the following criteria will be excluded from study
participation:
1. Renal failure or eGFR<45 mL/min/1.73 m^2.
2. Type 1 diabetes mellitus.
3. Fasting hypoglycemia (<60 mg/dL).
4. Known hypersensitivity or allergy to any component of empagliflozin.
5. Pregnant.
6. Breastfeeding.
7. Any condition that, in the opinion of the investigator, contraindicates
participation in this study.
Age minimum:
18 Years
Age maximum:
85 Years
Gender:
All
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Health Condition(s) or Problem(s) studied
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Glycogen Metabolism
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Inflammatory Bowel Disease (IBD)
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Crohn's Disease
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Intervention(s)
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Drug: Empagliflozin
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Primary Outcome(s)
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Absolute Neutrophil Count
[Time Frame: End of Treatment at 12 months]
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Safety
[Time Frame: End of Treatment at 12 months]
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Secondary Outcome(s)
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Neutrophil Function
[Time Frame: End of Treatment at 12 months]
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Secondary ID(s)
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000236-I
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10000236
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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