Empagliflozin as a Treatment for Severe Congenital Neutropenia Due to G6PC3 Deficiency (NCT05078879) | Clinical Trial Compass
CompletedPhase 1
Empagliflozin as a Treatment for Severe Congenital Neutropenia Due to G6PC3 Deficiency
United States4 participantsStarted 2021-11-16
Plain-language summary
Background:
Severe congenital neutropenia (SCN) is an immune system disease. People with SCN do not have enough of a kind of white blood cell called neutrophils. This means they get sick easily from infections. Some drugs to treat SCN have lots of side effects. Researchers want to see if a the drug empagliflozin can help increase the number of neutrophils in a person with SCN.
Objective:
To see if a drug called empagliflozin can help people with SCN.
Eligibility:
Adults aged 18 and older with SCN.
Design:
Participants will be screened with a physical exam, medical history, and blood tests. They may have a pregnancy test.
Participants will have study visits and local lab visits. They will repeat the screening tests. They will have heart and lung function tests. They will have an ultrasound of the liver and spleen. Their skin symptoms will be photographed. They may have consultations with specialists. They may give a stool sample. They may have an optional colonoscopy with tissue sample collection. They may have an optional bone marrow biopsy and aspirate. They may have an optional magnetic resonance imaging scan of their heart.
Participants will be admitted to NIH for 5 7 days. They will start taking the study drug as a pill once daily. They will be monitored for side effects.
Participants will take the study drug at home for 12 months. They will use a fingerstick blood glucose meter to measure blood sugar at home.
Participants may be able to take the study drug through their local doctor after the study ends.
Participation will last for 15 months.
Who can participate
Age range
18 Years – 85 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Aged \>=18 years.
. Documented SCN due to G6PC3 deficiency defined by genetic testing.
. History of ANC consistently \<1000 cells/microL when not treated with G-CSF.
. Current ANC\<1000 cells/microL when not treated with G-CSF.
. 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:
. Hormonal contraception.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Absolute Neutrophil Count
Timeframe: End of Treatment at 12 months
2
Safety
Timeframe: End of Treatment at 12 months
Trial details
NCT IDNCT05078879
SponsorNational Institute of Allergy and Infectious Diseases (NIAID)