Glycogen Storage Disease Type IB Clinical Trial
Official title:
Efficacy and Safety of Empagliflozin in Patients With Glycogen Storage Disease Type Ib
Empagliflozin Treatment of GSD-1b patients
Status | Recruiting |
Enrollment | 10 |
Est. completion date | December 31, 2024 |
Est. primary completion date | June 30, 2024 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 1 Year to 50 Years |
Eligibility | 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. |
Country | Name | City | State |
---|---|---|---|
China | Xinhua Hospital, Shanghai Jiao Tong University School of Medicine | Shanghai | Shanghai |
Lead Sponsor | Collaborator |
---|---|
Xinhua Hospital, Shanghai Jiao Tong University School of Medicine |
China,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Change from Baseline in Absolute neutrophil count at 1 year | Efficacy of Empaglifozin measured by the change in absolute neutrophil count after 12 months of treatment compared to the period before study | 1 year | |
Primary | Occurrence of hypoglycemia | Safety and tolerability of Empaglifozin measured by hypoglycemia | 1 year | |
Secondary | Number of infections | Efficacy of Empaglifozin measured by the number of respiratory tract, skin, and urinary tract infections | 1 year | |
Secondary | Inflammatory bowel disease activity | Measured as classical Crohn 's disease activity index (CDAI) for adults (range from 0 to 600; remission <150; mildly active disease 150-219; moderately active disease 220- 450; severely active disease = 450) or pediatric Crohn' s disease activity index (PCDAI) for children (range from 0 to 100; remission <10; mildly active disease 10-27.5; moderately active disease 30-37.5; severely active disease 40-100) after 3, 6, 9, and 12 months of treatment compared to the period before study | 1 year | |
Secondary | Endoscopic scores of inflammatory bowel disease | Measured as difference in Crohn 's Disease Simplified Endoscopic Score (SES-CD) (range from 0 to 17; remission 0-2; mild endoscopic activity 3-6; moderate endoscopic activity 7-15; severe endoscopic activity >15) before and after 1 year of empagliflozin treatment | 1 year | |
Secondary | Change of triglycerides | Measured as change of triglycerides (mmol/L) compared to the period before study | 1 year | |
Secondary | Change of total cholesterol | Measured as change of total cholesterol (mmol/L) compared to the period before study | 1 year | |
Secondary | Change of lactate | Measured as change of lactate (mmol/L) compared to the period before study | 1 year | |
Secondary | Change of uric acid | Measured as change of uric acid (mmol/L) compared to the period before study | 1 year |
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