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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.

Register:	ClinicalTrials.gov
Last refreshed on:	10 August 2021
Main ID:	NCT04986735
Date of registration:	27/07/2021
Prospective Registration:	Yes
Primary sponsor:	Hong Kong Children's Hospital
Public title:	Prospective Cohort Study of Children With GSD1b Receiving Empagliflozin
Scientific title:	Evaluation of Safety and Efficacy of Empagliflozin for Neutropenia and Neutrophil Dysfunction in Children With Glycogen Storage Disease Type 1b (GSD1b)
Date of first enrolment:	August 1, 2021
Target sample size:	11
Recruitment status:	Not yet recruiting
URL:	https://clinicaltrials.gov/show/NCT04986735
Study type:	Observational
Study design:
Phase:

Countries of recruitment

Contacts

Contact type:
Name:	Mei Kwun Kwok
Address:
Telephone:
Email:
Affiliation:	Hong Kong Children's Hospital

Contact type:
Name:	Mei Kwun Kwok
Address:
Telephone:	5741 3216 (Secretary)
Email:	kwokmk@ha.org.hk
Affiliation:

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Subject/carers are willing and able to comply with all requirements of the clinical
trial and sign the informed consent

2. Referrer agrees to provide relevant background medical information

Exclusion Criteria:

1. Subject fails to comply with the study protocol

2. Subject has any co-morbidity or condition that could increase the risk of
empagliflozine treatment (e.g. renal failure with eGFR <30 mL/min/1.73m2 or requiring
dialysis, diabetes requiring insulin &/or oral hypoglycemic agents, dyslipidemia
requiring pharmacological intervention)

3. Subject is pregnant, or a sexually active female who does not consent to use effective
contraception during the study

4. History of liver transplantation is NOT an exclusion criterium

Age minimum: 6 Months
Age maximum: 18 Years
Gender: All

Health Condition(s) or Problem(s) studied

Glycogen Storage Disease Type IB

Intervention(s)

Drug: Empagliflozin

Primary Outcome(s)

Efficacy of empagliflozin - usage of granulocyte colony stimulating factor (GCSF) [Time Frame: from the start to the 52nd week of empagliflozin treatment]

Secondary Outcome(s)

Efficacy of empagliflozin - neutrophil number and function [Time Frame: from the start to the 52nd week of empagliflozin treatment]

General metabolic control - GSD1b metabolic & imaging profile, concomitant interventions [Time Frame: from the start to the 52nd week of empagliflozin treatment]

Safety of empagliflozin - prescence of absence of empagliflozin-related side effects [Time Frame: from the start to the 52nd week of empagliflozin treatment]

Efficacy of empagliflozin - biochemical improvement [Time Frame: from the start to the 52nd week of empagliflozin treatment]

Safety of empagliflozin - presence or absence of hypoglycemia [Time Frame: from the start to the 52nd week of empagliflozin treatment]

Efficacy of empagliflozin - bowel manifestations [Time Frame: from the start to the 52nd week of empagliflozin treatment]

Efficacy of empagliflozin - frequency of infections [Time Frame: from the start to the 52nd week of empagliflozin treatment]

General well being - Quality of life [Time Frame: from the start to the 52nd week of empagliflozin treatment]

Secondary ID(s)

HKCH-REC-2021-001

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Ethics review

Results

Results available:
URL:
URL of the protocol:
Date Posted:
Date of completion:
Date of first publication:
Results summary:
Baseline characteristics:
Adverse events:
Outcome measures:
IPD sharing plan:
IPD sharing description:

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