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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

Timeline

Screening 3 weeks

Treatment Varies

Follow Up end of treatment at 12 months

Awards & highlights

Study 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.Check my eligibility

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.See study design

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.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ end of treatment at 12 months

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

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Absolute Neutrophil Count

Safety

Secondary outcome measures

Neutrophil Function

Side effects data

From 2021 Phase 3 trial • 5988 Patients • NCT03057951

15%

Cardiac failure

Hypertension

Urinary tract infection

Hypotension

Hyperkalaemia

Fall

Renal impairment

Atrial fibrillation

Diabetes mellitus

Anaemia

Hyperuricaemia

Acute kidney injury

Pneumonia

Acute myocardial infarction

COVID-19

Cardiac failure congestive

Death

Myocardial infarction

Cardiac failure chronic

Angina pectoris

Angina unstable

Ventricular tachycardia

COVID-19 pneumonia

Cellulitis

Sepsis

Chronic kidney disease

Coronary artery disease

Chronic obstructive pulmonary disease

Basal cell carcinoma

Cerebrovascular accident

Ischaemic stroke

Syncope

Transient ischaemic attack

100%

80%

60%

40%

20%

Study treatment Arm

Placebo

10 mg Empagliflozin

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

0 / 2Eligibility criteria met

Find a Location

Who is running the clinical trial?

National Institute of Allergy and Infectious Diseases (NIAID)Lead Sponsor

3,269 Previous Clinical Trials

5,481,477 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

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

To what extent has participation been secured in this research endeavor?

"Affirmative. Clinicaltrials.gov confirms that recruitment for this study is ongoing, having first been posted on November 16th 2021 and last modified August 20th 2022. This clinical trial necessitates the enrolling of 10 individuals across one site."

Answered by AI

What other investigations have investigated the efficacy of Empagliflozin?

"As of now, 49 clinical trials investigating the effects of Empagliflozin are underway. Out of those studies, 13 have advanced to Phase 3. Although most tests for this medication take place in Quilmes, Maryland, there are over 1000 research sites worldwide conducting these experiments."

Answered by AI

What health concerns does Empagliflozin typically address?

"The conventional use of empagliflozin is in the treatment of cardiovascular ailments. However, it can also help reduce mortality and treat type 2 diabetes mellitus, as well as other heart-related issues such as congestive heart failure."

Answered by AI

To what extent is Empagliflozin innocuous for people?

"Analysts from Power have assigned Empagliflozin a safety rating of 1 on their scale due to the limited evidence available that supports its efficacy and security."

Answered by AI

Is this research a pioneer in its field?

"Ever since 2018, Empagliflozin has been under clinical scrutiny. Initiated by Boehringer Ingelheim with a trial of 175 patients in 2018, the drug ultimately earned its Phase 3 approval. Nowadays, there are 49 ongoing trials occurring across 237 cities and 43 countries."

Answered by AI

Are there any vacant positions available for participants of this research trial?

"That is correct. Clinicaltrials.gov's information reveals that recruitment for this experiment, which was posted on November 16th 2021, has just begun. At the current moment, 10 patients must be recruited from one site only."

Answered by AI

Recent research and studies

g6pc3Journal

A Syndrome with Congenital Neutropenia and Mutations In<i>g6pc3</i>

Boztug, Kaan, Giridharan Appaswamy, Angel Ashikov, Alejandro A. Schäffer, Ulrich Salzer, Jana Diestelhorst, Manuela Germeshausen, et al.. 2009. “A Syndrome with Congenital Neutropenia and Mutations Ing6pc3”. New England Journal of Medicine. Massachusetts Medical Society. doi:10.1056/nejmoa0805051.

Current Opinion in HematologyJournal

Neutropenia in Glycogen Storage Disease Ib

Dale, David C., Audrey Anna Bolyard, Tracy Marrero, Merideth L. Kelley, Vahagn Makaryan, Emily Tran, Jamie Leung, et al.. 2019. “Neutropenia in Glycogen Storage Disease Ib”. Current Opinion in Hematology. Ovid Technologies (Wolters Kluwer Health). doi:10.1097/moh.0000000000000474.

Orphanet Journal of Rare DiseasesJournal

A Clinical and Molecular Review of Ubiquitous Glucose-6-phosphatase Deficiency Caused by G6PC3 Mutations

Banka, Siddharth, and William G Newman. 2013. “A Clinical and Molecular Review of Ubiquitous Glucose-6-phosphatase Deficiency Caused by G6PC3 Mutations”. Orphanet Journal of Rare Diseases. Springer Science and Business Media LLC. doi:10.1186/1750-1172-8-84.

clinicaltrials.govStudy