Overview
Sponsor-declared trial summary
Phase
Therapeutic exploratory (Phase II)
Status
Ended
Participants planned
20
Countries
1
Sites
2
Glycogenosis type Ib is an ultra-rare disease, inherited in an autosomal recessive manner.
The primary objective of the study is to evaluate the safety and tolerability of neutropenia treatment with empagliflozin in patients with GSD Ib
Key facts
- Sponsor
- Instytut Pomnik Centrum Zdrowia Dziecka
- Participant type
- Pediatric, Patients
- Age range
- 0-17 years, 18-64 years, 65+ years
- Gender
- Male and Female
- Therapeutic area
- Phenomena and Processes [G] - Metabolism [G03]
- Trial duration
- 3 Mar 2022 → 3 Apr 2025
- Decision date (initial)
- 2024-12-02
- Transition trial
- Yes
- Low-intervention
- No
- Rare-disease indication
- Yes
- Vulnerable population
- Yes
- Funding sources
- 100 % Agencja Badań Medycznych
External identifiers
- EU CT number
- 2024-512840-52-00
- EudraCT number
- 2021-000580-78
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Safety
The primary objective of the study is to evaluate the safety and tolerability of neutropenia treatment with empagliflozin in patients with GSD Ib
Secondary objectives 1
- The secondary goal is to assess efficacy expressed as restoration of neutrophil numbers and function. The tertiary goal is to reduce/stop filgrastim dosing, thereby reducing the adverse effects associated with filgrastim treatment. The quaternary objective will be to assess metabolic compensation.
Conditions and MedDRA coding
Glycogenosis type Ib is an ultra-rare disease, inherited in an autosomal recessive manner.
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 1
- Inclusion criteria for the study Patients who meet all of the following criteria will be included in the study: - males and females - age of 4 weeks or older - biochemically and/or molecularly diagnosed Ib glycogenosis with neutropenia and/or abnormal NADPH oxidase activity in neutrophils - the expression of voluntary informed consent by the legal representative for children < 13 years of age, the legal representative and the patient for children >=13 years of age, and the patient himself for adult patients.
Exclusion criteria 1
- Exclusion criteria for the study The following criteria exclude a patient from eligibility for the study: - risk of non-cooperation, including the risk of non-adherence to follow-up and non-adherence to dietary recommendations; - chronic kidney disease with eGFR < 60 ml/min/1.73 m2 - urinary tract infection (time criterion at the time of inclusion in the study, pending completion of treatment for UTI), - participation in another clinical trial (withdrawal period: 6 months from the end of participation until the date of signing the Informed Consent Form), - participation in a therapeutic experiment, other than experimental treatment with empagliflozin (grace period: 24 months from the end of participation until the date of signing the Informed Consent Form) - pregnancy, breastfeeding - allergy to the administered drugs - lack of consent to participate in the study.
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 1
- The primary endpoint will be an assessment of the safety and tolerability of empagliflozin, expressed as the type and frequency of adverse reactions throughout the study period. Safety data will be collected at all visits and will be related to possible adverse reactions to empagliflozin as described in the summary of product characteristics.
Secondary endpoints 1
- The secondary objective is to assess efficacy expressed as restoration of neutrophil numbers and function
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 1
Jardiance 10 mg film-coated tablets
PRD1594848 · Product
- Active substance
- Empagliflozin
- Pharmaceutical form
- FILM-COATED TABLET
- Route of administration
- ORAL
- Max daily dose
- 20 mg milligram(s)
- Max total dose
- 20 mg milligram(s)
- Max treatment duration
- 24 Month(s)
- Authorisation status
- Authorised
- ATC code
- A10BK03 — -
- Marketing authorisation
- EU/1/14/930/010
- MA holder
- BOEHRINGER INGELHEIM INTERNATIONAL GMBH
- MA country
- EU
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- Yes
- Modification description
- Aby otrzymać dawki pediatryczne 2,5 mg i 5 mg będzie przeprowadzona mikronizacja tabletek i okapsułkowanie.
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Instytut Pomnik Centrum Zdrowia Dziecka
- Sponsor organisation
- Instytut Pomnik Centrum Zdrowia Dziecka
- Address
- Aleja Dzieci Polskich 20
- City
- Warsaw
- Postcode
- 04-730
- Country
- Poland
Scientific contact point
- Organisation
- Instytut Pomnik Centrum Zdrowia Dziecka
- Contact name
- Mariola Modzelewska
Public contact point
- Organisation
- Instytut Pomnik Centrum Zdrowia Dziecka
- Contact name
- Mariola Modzelewska
Locations
1 EU/EEA country · 2 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Poland | Ended | 20 | 2 |
| Rest of world | — | 0 | — |
Investigational sites
Instytut Pomnik Centrum Zdrowia Dziecka
Klinika Pediatrii, Żywienia i Chorób Metabolicznych, Aleja Dzieci Polskich 20, 04-730, Warsaw
Warszawski Uniwersytet Medyczny
Klinika Chorób Wew., Nadciśnienia Tętniczego i Angiologii WUM SPCSK, ul.Banacha 1a, 02-097 Warszawa, Żwirki i Wigury 61, 02-091, Warszawa
Country notifications
Trial-start, recruitment-start, end and early-termination notifications submitted per Member State
| Country | Trial start | Trial end | Recruitment start | Recruitment end | Early termination |
|---|---|---|---|---|---|
| Poland | 2022-03-03 | 2025-04-03 | 2022-03-07 | 2023-05-29 |
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 8 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | D1_Protoko Badania Klinicznego_2024-512840-52 | 3.1 |
| Recruitment arrangements (for publication) | K_Ustalenia dotyczace rekrutacji | 1 |
| Subject information and informed consent form (for publication) | L1_Dzienniczek Pacjenta | 3.1 |
| Subject information and informed consent form (for publication) | L1_Informacja i Formularz Swiadomej Zgody_pediatryczna_Dorosli | 3 |
| Subject information and informed consent form (for publication) | L1_Informacja i Formularz Swiadomej Zgody_pediatryczna_Pediatryczni | 3 |
| Subject information and informed consent form (for publication) | L1_Informacja i Formularz Swiadomej Zgody_pediatryczna_pow 13 rz | 4 |
| Subject information and informed consent form (for publication) | L1_Karta Identyfikacyjna Pacjenta | 2 |
| Summary of Product Characteristics (SmPC) (for publication) | G_Charakterystyka produktu leczniczego_Jardiance | 1 |
Application history
1 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2024-10-15 | Poland | Acceptable 2024-11-28
|
2024-12-02 |