NCT03202498

Safety and Tolerability of Yaq-001 in Patients With Cirrhosis

Safety and Tolerability of Yaq-001 in Patients With Cirrhosis ("CARBALIVE-SAFETY")

• Status: Terminated
• Phase: N/A
• Study type: Interventional
• Enrollment: 31 (actual)
• Sponsor: Yaqrit Ltd · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

In patients with cirrhosis (scarring of the liver), bacterial fragments leak from the gut into the blood and cause harm. This study looks into a new way to lower the leakage of bacterial fragments into the blood. Yaq-001 is a new type of carbon that in previous laboratory studies has been shown to have the ability to bind these bacterial fragments and so confine them to the gut. The purpose of this clinical trial is to test the product Yaq-001 for the first time in patients with cirrhosis. This trial will assess if the treatment with Yaq-001 is safe, is well tolerated, and if it helps improve the overall health status of the cirrhotic patients. Candidate patients must be at least 18 years old and have a clinical diagnosis of cirrhosis for any cause. Only postmenopausal women or with surgical sterilisation are eligible. Additional inclusion and exclusion criteria of medical nature will be determined with the investigator at the screening visit, by means of standard care routines plus an additional test to assess the bowel transit time. Eligible patients will be randomly grouped to receive standard care treatment plus Yaq-001, or standard treatment plus placebo (non-active treatment). The use of placebo is necessary to better understand how safe and tolerable Yaq-001 really is. The treatment lasts for 12 weeks. During treatment, the patient will be visited by a study doctor 5 times. At all the visits the patients will undergo a routine physical examination, electrocardiogram, collection of blood and urine samples. On three occasions the patients will be asked to provide additional samples of blood, urine and stool for analysis outside the hospital. 56 patients from 9 hospitals in UK, France, Italy, Portugal, Spain and Switzerland will participate in this study.

Detailed description

First-in-human clinical investigation with Yaq-001. This is a multicentre, randomized, double blinded, placebo controlled trial to intended to evaluate safety and tolerability of oral administration of Yaq-001 therapy in two dosing cohorts. 56 cirrhotic patients with diuretic-responsive ascites will be enrolled. Patients will be randomized to two dosing cohorts. Cohort 1 (1:1 randomization) * Standard medical treatment + Yaq-001 (4 g/ day) - n= 14. * Standard medical treatment + placebo-control (placebo for 4 g of Yaq-001/ day) - n= 14. Cohort 2 (1:1 randomization) * Standard medical treatment + Yaq-001 (8 g/ day) - n= 14. * Standard medical treatment + placebo-control (placebo for 8 g of Yaq-001/ day) - n= 14. Study patients will be dosed daily with Yaq-001 (or an equivalent quantity of placebo) for 12 weeks. Assessments of DSMB will take place after 4 and 12 weeks. Investigational centres specialized in the management of patients with liver cirrhosis will participate in the study. For each patient, the study duration will be up to 17 weeks, including the screening (up to 4 weeks), treatment (12 weeks) and 7-day follow up period. The total study duration is estimated to be approximately 6 months from screening of first patient until study completion of the last patient. This project has received funding from the European Union's Horizon 2020 research and innovation programme under grant agreement No 634579.

Conditions

• Liver Cirrhosis

Interventions

Type	Name	Description
DEVICE	4g Yaq-001	Study patients will be dosed daily with 4g of product Yaq-001 for a period of 12 weeks. The product will be provided as beads packed in individual sachets intended each for one oral administration. For each patient, the study duration will be up to 17 weeks, including the screening (up to 4 weeks), treatment (12 weeks) and 7-day follow up period.
OTHER	4g Placebo	Study patients will be dosed daily with a quantity of placebo equivalent to 4g of product Yaq-001 for a period of 12 weeks. The product will be provided as beads packed in individual sachets intended each for one oral administration. For each patient, the study duration will be up to 17 weeks, including the screening (up to 4 weeks), treatment (12 weeks) and 7-day follow up period.
DEVICE	8g Yaq-001	Study patients will be dosed daily with 8g of product Yaq-001 for a period of 12 weeks. The product will be provided as beads packed in individual sachets intended each for one oral administration. For each patient, the study duration will be up to 17 weeks, including the screening (up to 4 weeks), treatment (12 weeks) and 7-day follow up period.
OTHER	8g Placebo	Study patients will be dosed daily with a quantity of placebo equivalent to 8g of product Yaq-001 for a period of 12 weeks. The product will be provided as beads packed in individual sachets intended each for one oral administration. For each patient, the study duration will be up to 17 weeks, including the screening (up to 4 weeks), treatment (12 weeks) and 7-day follow up period.

Timeline

• Start date: 2019-02-28
• Primary completion: 2020-03-26
• Completion: 2020-03-26
• First posted: 2017-06-28
• Last updated: 2020-08-25

Locations

9 sites across 6 countries: France, Italy, Portugal, Spain, Switzerland, United Kingdom