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

NCT00655707 | Completed Phase 1 Results DMC

• 1 other identifier: HHSC/001
• Study Type: interventional
• Target: 5
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Liver Disease

Keywords

Adult stem cell therapy

Outcome Measures

Primary Outcomes (2)

• Number of Patients Who Tolerated the Maximum Dose

  To determine the maximum tolerated dose of autologous adult stem cells when infused into either the hepatic artery or the portal vein.

  12 months

• Number of Participants Without Specific Treatment Related Side Effect

  To assess the safety of ascending doses of autologous adult stem cells when introduced into either the hepatic artery or the portal vein and to determine the maximum tolerated dose of stem cells.

  12 months

Study Arms (1)

Autologous CD34+ cells

EXPERIMENTAL

Autologous Cluster Designation 34+(CD34+) cells

Biological: Autologous Expanded CD34+ Haemopoietic cells

Interventions

Autologous Expanded CD34+ Haemopoietic cells BIOLOGICAL

Autologous expanded CD34+ cells obtained by leukapheresis on a single occasion by infusion into the hepatic artery or portal vein. Patient numbers 01 and 02 will receive 1x109 cells Patient numbers 03 and 04 will receive 1x1010 cells Patient numbers 05 and 06 will receive 2x1010 cells Patient numbers 07 and 08 will receive 5x1010 cells

Autologous CD34+ cells

Eligibility Criteria

• Age: 20 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Imperial College London: lead

Study Sites (1)

Imperial College Healthcare Trust

London, W12 0HS, United Kingdom

Location

MeSH Terms

Conditions

Liver Diseases

Condition Hierarchy (Ancestors)

Digestive System Diseases

Limitations and Caveats

This was not a randomised study and although the results were encouraging only a small number of patients were treated.

Results Point of Contact

• Title: Professor Nagy Habib
• Organization: ImperialC

nagy.habib@imperial.ac.uk

+442033138574

Study Officials

• Nagy Habib, ChM FRCS

  Imperial College London

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 4, 2008
• First Posted: April 10, 2008
• Study Start: January 1, 2006
• Primary Completion: April 1, 2009
• Study Completion: April 1, 2009
• Last Updated: October 31, 2019
• Results First Posted: October 31, 2019

Record last verified: 2019-10

Data Sharing

• IPD Sharing: Will not share

Locations

GB (1)