Safety and Efficacy of Empagliflozin in GSD1b Patients Wi... | NCT04930627

Studies
Safety and Efficacy of Empagliflozin in GSD1b Patients With Neutropenia

NCT04930627

Sponsor

Children's Memorial Health Institute, Poland

Status

Unknown status

Last Update Posted

Jun 23, 2021Actual

Enrollment

20Estimated

Phase

Phase 2

Conditions

Glucose 6 Phosphatase Deficiency

Interventions

Empagliflozin

Countries

Poland

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

ID	Type	Description	Link
2021-000580-78	EudraCT Number

Status Module

Record Verification Date

Jun 2021

Overall Recruitment Status or Expanded Access Status

Unknown status

Last Known Status

Not yet recruiting

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Jul 2021Estimated

Primary Completion Date

Jun 2024Estimated

Completion Date

Mar 2025Estimated

First Submitted Date

Jun 11, 2021

First Submission Date that Met QC Criteria

Jun 11, 2021

First Posted Date

Jun 18, 2021Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Jun 18, 2021

Last Update Posted Date

Jun 23, 2021Actual

Sponsor/Collaborators Module

Responsible Party, by Official Title

Dariusz Rokicki, Pricipal Investigator, Children's Memorial Health Institute, PolandPrincipal Investigator

Lead Sponsor

Children's Memorial Health Institute, PolandOTHER

Collaborators

Name	Class
Department of Internal Medicine, Hypertension and Vascular Diseases, The Medical University of Warsaw	UNKNOWN

Oversight Module

Has Data Monitoring Committee (DMC)

Not provided

Is FDA Regulated Drug

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

FDAAA801 Violation

Not provided

Description Module

Brief Summary

Treatment of neutropenia of Glycogenosis type 1b patients with empagliflozin

Detailed Description

Symptoms of glycogen storage disease type Ib (GSD Ib) include - among others - hypoglycemia, hepatomegaly and neutropenia with concomitant neutrophil dysfunction, which results in recurrent bacterial and fungal infections, and inflammatory bowel disease.

At present filgrastim is the only available drug to treat neutropenia in GSD Ib patients; it stimulates neutrophil production, but doesn't restore their function. Part of GSD Ib patients doesn't respond to filgrastim treatment. The latest research results showed, that neutropenia and neutrophil dysfunction in GSD Ib patients are results of extensive accumulation of 1,5-anhydroglucitol-phosphate. Empagliflozin, a SGLT2 inhibitor, inhibits renal glucose and 1,5-anhydroglucitol reabsorption and is an effective and safe method of treatment of neutropenia in this group of patients. Empagliflozin (Jardiance®) is a drug, which is registered in Poland to treat type II diabetes in adults. The aim of our study is to evaluate the efficacy and safety of neutropenia in patients with GSD Ib with empagliflozin (Jardiance®).

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 2

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

20Estimated

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
oral administration of Empagliflozin	Experimental		Drug: Empagliflozin

Interventions

Name	Type	Description	Arm Group Labels	Other Names
Empagliflozin	Drug	dosis depending on body weight: <20 kg 5 mg 1x/day; 20-40 kg 2 x 5 mg; >40 kg 2 x 10 mg	oral administration of Empagliflozin

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Empaglifozin safety and tolerability measured by occurrence of adverse reactions	Empaglifozin saftey and tolerability measured by occurrence of adverse reactions	2 years

Secondary Outcomes

Measure	Description	Time Frame
Efficacy of neutropenia treatment measured as percentage of the patients	who achieved >500 neutrophils/ml for at least 6 months with normalization of oxidative burst with decrease of bacterial and fungal infections compared to the period before study with decrease of hospitalization number with decrease of the number of defecation, gingival sores, and calprotectin average concentration in stool

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

Minimum age 4 weeks old female Or Male
GSD1b confirmed by genetic analysis with neutropenia and/or reduced respiratory burst
Informed consent signed by the parents/assigns, and the recipient (>13 years old)

Exclusion Criteria:

Risk of non-compliance
Chronic renal diseases (eGFR < 60 ml/min/1,73 m2)
Active urinary tract infection (temporal criterion, up to recovery)
Participation in another clinical trial (minimum 6 months from the end of participation until the date of signing the Informed Consent Form)
Participation in therapeutic experiment, in addition to the experimental treatment with empagliflozin (minimum 12 months from the end of participation until the date of signing the Informed Consent Form)
Pregnancy, breastfeeding
Allergy to Empagliflozin
Lack of informed consent

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

4 Weeks

Maximum Age

Not provided

Standard Ages

ChildAdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Not provided

Locations

Facility	Status	City	State	ZIP	Country	Contacts
The Children's Memorial Health Institute		Warsaw		04-730	Poland

Condition Browse Module

MeSH Terms

ID	Term
D005953	Glycogen Storage Disease Type I

Ancestor Terms

ID	Term
D006008	Glycogen Storage Disease
D002239	Carbohydrate Metabolism, Inborn Errors
D008661	Metabolism, Inborn Errors
D030342	Genetic Diseases, Inborn

Browse Leaves

Not provided

Browse Branches

Not provided

Intervention Browse Module

MeSH Terms

ID	Term
C570240	empagliflozin

Ancestor Terms

Not provided

Browse Leaves

Not provided

Browse Branches

Not provided