Clinical Trial Results:
A phase I/II safety and tolerability dose escalation study following the autologous infusion of expanded adult haemopoietic stem cells to patients with liver insufficiency
Summary
|
|
EudraCT number |
2005-001222-83 |
Trial protocol |
GB |
Global end of trial date |
30 Apr 2009
|
Results information
|
|
Results version number |
v1(current) |
This version publication date |
24 Oct 2019
|
First version publication date |
24 Oct 2019
|
Other versions |
|
Summary report(s) |
End of Study Report |
Trial Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
|
|||
Trial identification
|
|||
Sponsor protocol code |
HHSC/001
|
||
Additional study identifiers
|
|||
ISRCTN number |
- | ||
US NCT number |
NCT00655707 | ||
WHO universal trial number (UTN) |
- | ||
Sponsors
|
|||
Sponsor organisation name |
Imperial College London
|
||
Sponsor organisation address |
South Kensingston Campus, London, United Kingdom, SW7 2AZ
|
||
Public contact |
Professor Nagy Habib, Imperial College London, +44 2033138574 , nagy.habib@imperial.ac.uk
|
||
Scientific contact |
Professor Nagy Habib, Imperial College London, +44 2033138574 , nagy.habib@imperial.ac.uk
|
||
Paediatric regulatory details
|
|||
Is trial part of an agreed paediatric investigation plan (PIP) |
No
|
||
Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
|
||
Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
|
||
Results analysis stage
|
|||
Analysis stage |
Final
|
||
Date of interim/final analysis |
21 Apr 2010
|
||
Is this the analysis of the primary completion data? |
Yes
|
||
Primary completion date |
30 Apr 2009
|
||
Global end of trial reached? |
Yes
|
||
Global end of trial date |
30 Apr 2009
|
||
Was the trial ended prematurely? |
No
|
||
General information about the trial
|
|||
Main objective of the trial |
To determine the maximum tolerated dose of expanded autologous cells derived from a subset of CD34+ stem cells when infused into either the hepatic artery or the portal vein. The trial will also seek to determine clinical improvement or deterioration by measurement of clinical parameters such as liver function tests.
|
||
Protection of trial subjects |
None
|
||
Background therapy |
- | ||
Evidence for comparator |
- | ||
Actual start date of recruitment |
31 Jan 2006
|
||
Long term follow-up planned |
No
|
||
Independent data monitoring committee (IDMC) involvement? |
No
|
||
Population of trial subjects
|
|||
Number of subjects enrolled per country |
|||
Country: Number of subjects enrolled |
United Kingdom: 5
|
||
Worldwide total number of subjects |
5
|
||
EEA total number of subjects |
5
|
||
Number of subjects enrolled per age group |
|||
In utero |
0
|
||
Preterm newborn - gestational age < 37 wk |
0
|
||
Newborns (0-27 days) |
0
|
||
Infants and toddlers (28 days-23 months) |
0
|
||
Children (2-11 years) |
0
|
||
Adolescents (12-17 years) |
0
|
||
Adults (18-64 years) |
5
|
||
From 65 to 84 years |
0
|
||
85 years and over |
0
|
|
|||||||
Recruitment
|
|||||||
Recruitment details |
Patient were recruited between 2006 and 2009 at Hammersmith Hospital | ||||||
Pre-assignment
|
|||||||
Screening details |
The study was based on the hypothesis that the CD34+ cell population in granulocyte colony-stimulating factor (G-CSF)-mobilised blood contains a subpopulation of cells with the potential for regenerating damaged tissue. 5 participants were recruited. | ||||||
Period 1
|
|||||||
Period 1 title |
Autologous CD34+ cells (overall period)
|
||||||
Is this the baseline period? |
Yes | ||||||
Allocation method |
Not applicable
|
||||||
Blinding used |
Not blinded | ||||||
Arms
|
|||||||
Arm title
|
Autologous CD34+ cells | ||||||
Arm description |
Patients with chronic liver failure who fitted the study inclusion/exclusion criteria were admitted to the ward and were given 520 μg granulocyte-colony stimulating factor by subcutaneous injection for five consecutive days to increase the number of circulating CD34+ cells. Leukapheresis was performed on Day 5. The leukapheresis product was transferred to the GCP laboratory where CD34+ cells were immunoselected using the CliniMacs device (Miltenyi Biotech). The CD34+ cells were then transferred to the patient via the hepatic artery (2 patients) or portal vein (3 patients) in the Imaging Department. | ||||||
Arm type |
Experimental | ||||||
Investigational medicinal product name |
Autologous CD34+ cells
|
||||||
Investigational medicinal product code |
|||||||
Other name |
|||||||
Pharmaceutical forms |
Concentrate for concentrate for solution for infusion
|
||||||
Routes of administration |
Other use
|
||||||
Dosage and administration details |
Patients with chronic liver failure who fitted the study inclusion/exclusion criteria were admitted to the ward and were given 520 μg granulocyte-colony stimulating factor by subcutaneous injection for five consecutive days to increase the number of circulating CD34+ cells. Leukapheresis was performed on Day 5. The leukapheresis product was transferred to the GCP laboratory where CD34+ cells were immunoselected using the CliniMacs device (Miltenyi Biotech). The CD34+ cells were then transferred to the patient via the hepatic artery (2 patients) or portal vein (3 patients) in the Imaging Department.
1 x 10^6 (3 patients) and 2 x 10^8 cells (2 patients)
|
||||||
|
|
|||||||||||||||||||||||||||||||||||||
Baseline characteristics reporting groups
|
|||||||||||||||||||||||||||||||||||||
Reporting group title |
Autologous CD34+ cells
|
||||||||||||||||||||||||||||||||||||
Reporting group description |
- | ||||||||||||||||||||||||||||||||||||
|
|
|||
End points reporting groups
|
|||
Reporting group title |
Autologous CD34+ cells
|
||
Reporting group description |
Patients with chronic liver failure who fitted the study inclusion/exclusion criteria were admitted to the ward and were given 520 μg granulocyte-colony stimulating factor by subcutaneous injection for five consecutive days to increase the number of circulating CD34+ cells. Leukapheresis was performed on Day 5. The leukapheresis product was transferred to the GCP laboratory where CD34+ cells were immunoselected using the CliniMacs device (Miltenyi Biotech). The CD34+ cells were then transferred to the patient via the hepatic artery (2 patients) or portal vein (3 patients) in the Imaging Department. |
|
|||||||
End point title |
Number of Patients Who Tolerated the Maximum Dose [1] | ||||||
End point description |
Tolerated 1 x 10^6 or 2 x 10^8 cells
|
||||||
End point type |
Primary
|
||||||
End point timeframe |
12 months
|
||||||
Notes [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point. Justification: Low number of participants for statistical analyses |
|||||||
|
|||||||
No statistical analyses for this end point |
|
|||||||
End point title |
Number of participants without specific treatment related side effect [2] | ||||||
End point description |
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.
|
||||||
End point type |
Primary
|
||||||
End point timeframe |
12 months
|
||||||
Notes [2] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point. Justification: Low number of participants for statistical analyses |
|||||||
|
|||||||
No statistical analyses for this end point |
|
|||||||||||||||||||||||||||||||||||||||||||||
Adverse events information
|
|||||||||||||||||||||||||||||||||||||||||||||
Timeframe for reporting adverse events |
12 months
|
||||||||||||||||||||||||||||||||||||||||||||
Assessment type |
Systematic | ||||||||||||||||||||||||||||||||||||||||||||
Dictionary used for adverse event reporting
|
|||||||||||||||||||||||||||||||||||||||||||||
Dictionary name |
MedDRA | ||||||||||||||||||||||||||||||||||||||||||||
Dictionary version |
10
|
||||||||||||||||||||||||||||||||||||||||||||
Reporting groups
|
|||||||||||||||||||||||||||||||||||||||||||||
Reporting group title |
All participants
|
||||||||||||||||||||||||||||||||||||||||||||
Reporting group description |
- | ||||||||||||||||||||||||||||||||||||||||||||
|
|||||||||||||||||||||||||||||||||||||||||||||
Frequency threshold for reporting non-serious adverse events: 0% | |||||||||||||||||||||||||||||||||||||||||||||
|
|
|||
Substantial protocol amendments (globally) |
|||
Were there any global substantial amendments to the protocol? No | |||
Interruptions (globally) |
|||
Were there any global interruptions to the trial? No | |||
Limitations and caveats |
|||
Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data. | |||
None reported |