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SGLT2 Inhibitor

Empagliflozin for Severe Congenital Neutropenia

Phase 1
Recruiting
Led By David H McDermott, M.D.
Research Sponsored by National Institute of Allergy and Infectious Diseases (NIAID)
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Documented SCN due to G6PC3 deficiency defined by genetic testing
Aged >=18 years
Must not have
Renal failure or eGFR<45 mL/min/1.73 m^2
Type 1 diabetes mellitus
Timeline
Screening 3 weeks
Treatment Varies
Follow Up end of treatment at 12 months
Awards & highlights
No Placebo-Only Group

Summary

This trial is studying a drug called empagliflozin to see if it can help people with SCN.

Who is the study for?
Adults over 18 with severe congenital neutropenia (SCN) due to G6PC3 deficiency, who have low white blood cell counts and agree to use two forms of birth control can join. Those with kidney failure, type 1 diabetes, fasting hypoglycemia, pregnancy or known allergy to empagliflozin cannot participate.
What is being tested?
The trial is testing if the drug empagliflozin can increase white blood cells in SCN patients. Participants will take the drug daily for a year and monitor their health at home and through visits including heart tests, liver ultrasounds, possibly colonoscopies or bone marrow biopsies.
What are the potential side effects?
While taking empagliflozin participants might experience side effects related to blood sugar levels like hypoglycemia. They'll be monitored closely during an initial hospital stay and throughout the trial for any other potential side effects.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
My severe congenital neutropenia is due to a G6PC3 deficiency.
Select...
I am 18 years old or older.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
Select...
My kidney function is reduced with an eGFR below 45.
Select...
I have type 1 diabetes.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~end of treatment at 12 months
This trial's timeline: 3 weeks for screening, Varies for treatment, and end of treatment at 12 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Absolute Neutrophil Count
Safety
Secondary study objectives
Neutrophil Function

Side effects data

From 2021 Phase 3 trial • 5988 Patients • NCT03057951
15%
Cardiac failure
7%
Hypotension
7%
Hypertension
7%
Urinary tract infection
6%
Renal impairment
6%
Hyperkalaemia
6%
Fall
5%
Atrial fibrillation
5%
Diabetes mellitus
4%
Hyperuricaemia
4%
Anaemia
3%
Acute kidney injury
3%
Pneumonia
2%
COVID-19
2%
Death
2%
Acute myocardial infarction
2%
Cardiac failure congestive
1%
Basal cell carcinoma
1%
Cardiac failure chronic
1%
Cellulitis
1%
Syncope
1%
Chronic kidney disease
1%
COVID-19 pneumonia
1%
Ischaemic stroke
1%
Coronary artery disease
1%
Chronic obstructive pulmonary disease
1%
Myocardial infarction
1%
Sepsis
1%
Transient ischaemic attack
1%
Angina unstable
1%
Cerebrovascular accident
1%
Angina pectoris
1%
Ventricular tachycardia
100%
80%
60%
40%
20%
0%
Study treatment Arm
Placebo
10 mg Empagliflozin

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups
Experimental Treatment
Group I: Treatment ArmExperimental Treatment1 Intervention
Patients with GCPC3 will receive daily Empagliflozin for 12 months.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Empagliflozin
2017
Completed Phase 4
~181750

Find a Location

Who is running the clinical trial?

National Institute of Allergy and Infectious Diseases (NIAID)Lead Sponsor
3,336 Previous Clinical Trials
5,392,824 Total Patients Enrolled
David H McDermott, M.D.Principal InvestigatorNational Institute of Allergy and Infectious Diseases (NIAID)
3 Previous Clinical Trials
1,928 Total Patients Enrolled

Media Library

Empagliflozin (SGLT2 Inhibitor) Clinical Trial Eligibility Overview. Trial Name: NCT05078879 — Phase 1
Crohn's Disease Research Study Groups: Treatment Arm
Crohn's Disease Clinical Trial 2023: Empagliflozin Highlights & Side Effects. Trial Name: NCT05078879 — Phase 1
Empagliflozin (SGLT2 Inhibitor) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05078879 — Phase 1
~2 spots leftby Oct 2025
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