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Phase 2 N=5 Treatment

A Phase I/II Safety and Tolerability Dose Escalation Study of Autologous Stem Cells to Patients With Liver Insufficiency

Liver Disease

Enrolled (actual)
5
Serious AEs
20.0%
Results posted
Oct 2019
Primary outcome: Primary: Number of Patients Who Tolerated the Maximum Dose — 5 Participants

Study Design & Population

Study type
Interventional
Phase
Phase 2
Interventions
Autologous Expanded CD34+ Haemopoietic cells (Biological)
Age
Adult, Older Adult · 20+ yrs
Sex
All
Sponsor
Imperial College London
Primary completion
Apr 2009

Outcome Measures

OutcomeResultp-value
PRIMARY
Number of Patients Who Tolerated the Maximum Dose
5
PRIMARY
Number of Participants Without Specific Treatment Related Side Effect
5

Summary

This is a prospective dose escalation study of the administration of adult human stem cells in patients with end stage liver failure. Successive groups of two patients will receive ascending doses of autologous adult human stem cells starting at 1x10[9] cells. Following expansion in an approved Good Manufacturing Practice (GMP) facility the cells will be infused into either the hepatic artery or portal vein of research participants. The aim of this trial is to determine the maximum tolerated dose of autologous adult stem cells when infused into either the hepatic artery or the portal vein. The maximum dose that would be given would be 5x10 to the ten [10]. To assess improvement in liver function as measured by serological and biochemical analysis and determine whether there are any symptomatic improvements as reported by the patients.

Eligibility Criteria

Inclusion Criteria

  • Male or female patients aged from 20 to 65 years of age
  • Evidence of chronic liver insufficiency
  • The presence of abnormal serum albumin and/or bilirubin and/or prothrombin time
  • Patient is unlikely to receive a liver transplant
  • Has a World Health Organisation (WHO) performance score of less than 2
  • Has a life expectancy of at least 3 months
  • Ability to give written consent
  • Women of childbearing potential may be included, but must use a reliable and appropriate contraceptive method

Exclusion Criteria

  • Patients below the age of 20 or above the age of 65 years
  • Pregnant or lactating women
  • Patients with recent recurrent GI bleeding or spontaneous bacterial peritonitis
  • Patients with evidence of HIV or other life threatening infection
  • Patients unable to give written consent
  • Patients with a history of hypersensitivity to Granulocyte-Colony Stimulating Factor (F-CSF)
  • Patients who have been included in any other clinical trial within the previous month
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT00655707). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.

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