Efficacy and Safety of Empagliflozin in GSD-Ib Patients

NCT05960617 | Unknown Phase 2 DMC

• 1 other identifier: XHEC-C-2023-051-2
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

Brief Summary

Empagliflozin Treatment of GSD-1b patients

Conditions

Glycogen Storage Disease Type IB

Outcome Measures

Primary Outcomes (2)

• 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

• Occurrence of hypoglycemia

  Safety and tolerability of Empaglifozin measured by hypoglycemia

  1 year

Secondary Outcomes (7)

• Number of infections

  1 year

• Inflammatory bowel disease activity

  1 year

• Endoscopic scores of inflammatory bowel disease

  1 year

• Change of triglycerides

  1 year

• Change of total cholesterol

  1 year

Study Arms (1)

Oral administration of Empagliflozin

EXPERIMENTAL

All subjects will have a baseline assessment and be prospectively followed up for 12 months to examine their outcome after receiving empagliflozin.

Drug: Empagliflozin

Interventions

Empagliflozin DRUG

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.

Also known as: Jardiance

Oral administration of Empagliflozin

Eligibility Criteria

• Age: 1 Year - 50 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Patients with glycogen storage disease type Ib (genetically diagnosed) aged ≥ 1 year and ≤ 50 years;
• 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);
• Subjects and their guardians/clients (\< 18 years old) or subjects (≥ 18 years old) signed the informed consent form.

You may not qualify if:

• Patients with chronic kidney disease (eGFR \< 60 ml/min/1.73 m\^2) or cirrhosis (Metavir F4);
• Experiencing symptomatic or severe hypoglycemia within 1 month before the start of this trial;
• Absolute neutrophil count continued ≥ 1.5 × 10\^9/L (≥ 3 tests, each interval ≥ 5 days);
• Current active urinary tract infection (until urine routine twice negative);
• Participating other clinical investigators in the past 1 month;
• Pregnancy, breast-feeding and having a pregnancy plan;
• Presence of contraindications to empagliflozin therapy (hypersensitivity to empagliflozin, current or history of gangrene, history of recurrent urinary or genital infections);
• Patients who are not suitable for participating in the clinical investigator or with low compliance in the investigator 's opinion.

Sponsors & Collaborators

• Xinhua Hospital, Shanghai Jiao Tong University School of Medicine: lead

Study Sites (1)

Xinhua Hospital, Shanghai Jiao Tong University School of Medicine

Shanghai, Shanghai Municipality, 200092, China

RECRUITING

MeSH Terms

Conditions

Glycogen Storage Disease IB

Interventions

empagliflozin

Study Officials

• Wenjuan Qiu, MD PhD

  Xinhua Hospital, Shanghai Jiao Tong University School of Medicine

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Wenjuan Qiu, MD PhD

CONTACT

+86-21-25076466; qiuwenjuan@xinhuamed.com.cn

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 5, 2023
• First Posted: July 27, 2023
• Study Start: July 15, 2023
• Primary Completion: June 30, 2024
• Study Completion: December 31, 2024
• Last Updated: July 27, 2023

Record last verified: 2023-06

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)