The goal of this clinical trial is to learn if FT1 is safe and works to treat short bowel syndrome (SBS) in adults. It will also learn about the PK/PD profile of FT1. Researchers will compare FT1 to a placebo (a look-alike substance that contains no drug) to see if FT1 is safe and effective in patients with SBS. Participants will * Receive multiple injections of FT1 or placebo according to weight. * Visit the clinic for assessment.
In this placebo-controlled, double-blind, randomized, crossover phase 2 trial, 8 adult SBS patients are treated with once-weekly FT1 or placebo (1:1) for 5 weeks, followed by a washout period of at least 6 weeks, and then the alternate treatment for a further 5 weeks. Efficacy is evaluated through a 72 hour metabolic balance study conducted at baseline and at the end of each treatment cycle.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
8
General Hospital of Eastern Theater Command
Nanjing, Jiangsu, China
RECRUITINGTreatment-related Adverse Events
To evaluate the adverse events as characterized by type, frequency, severity as graded by the National Cancer Institute Common Terminology Criteria for Adverse Events (CTCAE) version 6.0, timing, seriousness, and relationship to study therapy after administration.
Time frame: From the first administration to study completion, appropriately 5 months.
Changes in fecal wet weight from baseline to the end of treatment
The difference in changes in fecal wet weight in the 72-hour metabolic balance study after treatment compared to baseline
Time frame: At the end of the second cycle (each cycle is 5 weeks, with a washout period of at least 6 weeks between two cycles)
Changes in urine volume from baseline to the end of treatment
The difference in changes in urine volume in the 72-hour metabolic balance study after treatment compared to baseline
Time frame: At the end of the second cycle (each cycle is 5 weeks, with a washout period of at least 6 weeks between two cycles)
The Area Under the Curve from dosing to the time of the last measured concentration (AUC0-t)
Pharmacokinetic parameter
Time frame: Up to 8 days, from Day 29 (the last dose administration) to Day 36 (7 days after the last dose) in each treatment cycle (each cycle is 5 weeks)
Maximum plasma concentration (Cmax)
Pharmacokinetic parameter
Time frame: Up to 8 days, from Day 29 (the last dose administration) to Day 36 (7 days after the last dose) in each treatment cycle (each cycle is 5 weeks)
Changes in L-citrulline levels in plasma
Pharmacodynamic parameter
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Time frame: From Day 1 of the first cycle to the end of the second cycle (each cycle is 5 weeks, with a washout period of at least 6 weeks between two cycles)