Trial Outcomes & Findings for A Phase I/II Safety and Tolerability Dose Escalation Study of Autologous Stem Cells to Patients With Liver Insufficiency (NCT NCT00655707)

NCT ID: NCT00655707

Last Updated: 2019-10-31

Results Overview

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

Recruitment status

COMPLETED

Study phase

PHASE1/PHASE2

Target enrollment

5 participants

Primary outcome timeframe

12 months

Results posted on

2019-10-31

Participant Flow

The planned dose escalation design did not work due to low participants number. All participants received similar dose of Autologous expanded CD34+ cells .

Participant milestones

Participant milestones
Measure
Treatment
Autologous CD34+ cells Autologous Expanded CD34+ Haemopoietic cells: Autologous expanded CD34+ cells obtained by leukapheresis on a single occasion by infusion into the hepatic artery or portal vein.
Overall Study
STARTED
5
Overall Study
COMPLETED
5
Overall Study
NOT COMPLETED
0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Race and Ethnicity were not collected from any participant.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Autologous CD34+ Cells
n=5 Participants
Patients enrolled in the study underwent 5 days of treatment with G-CSF to mobilise CD34+ cells, followed by leukapheresis to obtain CD34+ cells.
Age, Categorical
<=18 years
0 Participants
n=5 Participants
Age, Categorical
Between 18 and 65 years
5 Participants
n=5 Participants
Age, Categorical
>=65 years
0 Participants
n=5 Participants
Age, Continuous
49.4 years
n=5 Participants
Sex: Female, Male
Female
1 Participants
n=5 Participants
Sex: Female, Male
Male
4 Participants
n=5 Participants

PRIMARY outcome

Timeframe: 12 months

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

Outcome measures

Outcome measures
Measure
Autologous CD34+ Cells
n=5 Participants
Autologous Expanded CD34+ Haemopoietic cells: Autologous expanded CD34+ cells obtained by leukapheresis on a single occasion by infusion into the hepatic artery or portal vein.
Number of Patients Who Tolerated the Maximum Dose
5 Participants

PRIMARY outcome

Timeframe: 12 months

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.

Outcome measures

Outcome measures
Measure
Autologous CD34+ Cells
n=5 Participants
Autologous Expanded CD34+ Haemopoietic cells: Autologous expanded CD34+ cells obtained by leukapheresis on a single occasion by infusion into the hepatic artery or portal vein.
Number of Participants Without Specific Treatment Related Side Effect
5 Participants

Adverse Events

Autologous CD34+ Cells

Serious events: 1 serious events
Other events: 5 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
Autologous CD34+ Cells
n=5 participants at risk
Autologous Expanded CD34+ Haemopoietic cells: Autologous expanded CD34+ cells obtained by leukapheresis on a single occasion by infusion into the hepatic artery or portal vein.
Renal and urinary disorders
Urinary Tract Infection
20.0%
1/5 • Number of events 1 • 12 months

Other adverse events

Other adverse events
Measure
Autologous CD34+ Cells
n=5 participants at risk
Autologous Expanded CD34+ Haemopoietic cells: Autologous expanded CD34+ cells obtained by leukapheresis on a single occasion by infusion into the hepatic artery or portal vein.
Immune system disorders
Fever
80.0%
4/5 • Number of events 4 • 12 months
Surgical and medical procedures
Pain
80.0%
4/5 • Number of events 4 • 12 months
Gastrointestinal disorders
Nausea
100.0%
5/5 • Number of events 5 • 12 months
Hepatobiliary disorders
Jaundice
20.0%
1/5 • Number of events 4 • 12 months
Hepatobiliary disorders
Ascites
60.0%
3/5 • Number of events 3 • 12 months

Additional Information

Professor Nagy Habib

ImperialC

Phone: +442033138574

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place