Clinical trial
Efficacy and Safety of Empagliflozin in Patients With Glycogen Storage Disease Type Ib
Name
XHEC-C-2023-051-2
Description
Empagliflozin Treatment of GSD-1b patients
Trial arms
Trial start
2023-07-15
Estimated PCD
2024-06-30
Trial end
2024-12-31
Status
Recruiting
Phase
Early phase I
Treatment
Empagliflozin
Oral administration of Empagliflozin:
The starting dose was 0.3 mg/kg/day in 2 divided doses for 3 months. If the subject had an absolute neutrophil count \> 1.0 × 10\^9/L and clinical improvement (decreased number of infections and/or decreased IBD activity within 3 months), the maintenance dose was maintained.
If the subject had an absolute neutrophil count \< 1.0 × 10\^9/L but clinical improvement (decreased number of infections and/or decreased IBD activity within 3 months), the maintenance dose was maintained and reassessed 1 month later.
If the subject had an absolute neutrophil count \< 1.0 × 10\^9/L and no clinical improvement (no change in number of infections and/or no change in IBD activity within 3 months), the dose was increased by 0.1 mg/kg/day and reassessed 3 months later.
Assessments were then performed every 3 months with the same dose modification criteria as above.
Arms:
Oral administration of Empagliflozin
Other names:
Jardiance
Size
10
Primary endpoint
Change from Baseline in Absolute neutrophil count at 1 year
1 year
Occurrence of hypoglycemia
1 year
Eligibility criteria
Inclusion Criteria:
1. Patients with glycogen storage disease type Ib (genetically diagnosed) aged ≥ 1 year and ≤ 50 years;
2. Patients meet the diagnostic criteria for Crohn's disease (CD) based on Expert consensus on the diagnosis and treatment of inflammatory bowel disease in Chinese children (2019) or Consensus opinion on the diagnosis and treatment of inflammatory bowel disease in China (2018), or patients meet the diagnostic criteria for recurrent respiratory tract infection based on Clinical diagnosis and treatment for recurrent respiratory tract infection in Chinese children (2022);
3. Subjects and their guardians/clients (\< 18 years old) or subjects (≥ 18 years old) signed the informed consent form.
Exclusion Criteria:
1. Patients with chronic kidney disease (eGFR \< 60 ml/min/1.73 m\^2) or cirrhosis (Metavir F4);
2. Experiencing symptomatic or severe hypoglycemia within 1 month before the start of this trial;
3. Absolute neutrophil count continued ≥ 1.5 × 10\^9/L (≥ 3 tests, each interval ≥ 5 days);
4. Current active urinary tract infection (until urine routine twice negative);
5. Participating other clinical investigators in the past 1 month;
6. Pregnancy, breast-feeding and having a pregnancy plan;
7. Presence of contraindications to empagliflozin therapy (hypersensitivity to empagliflozin, current or history of gangrene, history of recurrent urinary or genital infections);
8. Patients who are not suitable for participating in the clinical investigator or with low compliance in the investigator 's opinion.
Protocol
{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE2'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 10, 'type': 'ESTIMATED'}}
Updated at
2023-07-27
1 organization
1 product
1 indication
Product
EmpagliflozinIndication
Glycogen Storage Disease Type 1B