NCT05078879

Empagliflozin as a Treatment for Severe Congenital Neutropenia Due to G6PC3 Deficiency

Completed Phase 1 Last updated 26 June 2025

What this trial tests

Phase 1 trial testing Empagliflozin in Crohn's Disease in 4 participants. Completed in 21 May 2025.

Timeline

16 November 2021

Primary endpoint
2 May 2025

21 May 2025

Quick facts

Lead sponsor	National Institute of Allergy and Infectious Diseases (NIAID)
Phase	Phase 1
Status	Completed
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	4
Start date	16 November 2021
Primary completion	2 May 2025
Estimated completion	21 May 2025
Sites	1 location across United States

Drugs / interventions tested

• Empagliflozin (empagliflozin) — full drug profile →

Conditions studied

• Crohn's Disease — all drugs for Crohn's Disease →
• Glycogen Metabolism — all drugs for Glycogen Metabolism →
• Inflammatory Bowel Disease (IBD) — all drugs for Inflammatory Bowel Disease (IBD) →

Sponsor

National Institute of Allergy and Infectious Diseases (NIAID)

Who can join

Adults 18 to 85, any sex, with Crohn's Disease or Glycogen Metabolism. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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.

Publications & conference data

2 peer-reviewed publications reference this trial (live from Europe PMC):

1. The paradox of autoimmunity and autoinflammation in inherited neutrophil disorders - in search of common patterns.
   Krzyzanowski D, Oszer A, Madzio J, Zdunek M, et al · · 2023 · cited 2× · PMID 37350970 · DOI 10.3389/fimmu.2023.1128581
2. Established and emerging non-cellular therapies in inherited bone marrow failure syndromes.
   Janczar S, Urbański B, Ussowicz M, Mlynarski W. · · 2026 · PMID 41859097 · DOI 10.3389/fimmu.2026.1773574