Trial Outcomes & Findings for Safety and Efficacy Study of Stem Cell Transplantation to Treat Dilated Cardiomyopathy (NCT NCT00629018)

NCT ID: NCT00629018

Last Updated: 2015-05-12

Results Overview

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 110 participants
• Primary outcome timeframe: 5 years
• Results posted on: 2015-05-12

Participant Flow

This study consisted of an open-label randomized study design conducted at the Advanced Heart Failure and Transplantation Center at University Medical Center Ljubljana in collaboration with the Methodist DeBakey Heart Center and Stanford University.

Patients with acute multi-organ failure or history of haematologic neoplasms were not included.

Participant milestones

Measure	SC Group 55 patients were randomized to CD34+ cell transplantation (SC group). In the SC group, peripheral CD34+cells were mobilized by G-CSF and collected via apheresis. Patients underwent myocardial scintigraphy and CD34+ cells were injected in the artery supplying the segments with reduced viability	Control Group 55 patients were randomized to standard medical therapy, without stem cell injection.
Overall Study STARTED	55	55
Overall Study COMPLETED	55	55
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Safety and Efficacy Study of Stem Cell Transplantation to Treat Dilated Cardiomyopathy

Baseline characteristics by cohort

Measure	SC Group n=55 Participants 55 patients were randomized to CD34+ cell transplantation (SC group). In the SC group, peripheral CD34+cells were mobilized by G-CSF and collected via apheresis. Patients underwent myocardial scintigraphy and CD34+ cells were injected in the artery supplying the segments with reduced viability	Control Group n=55 Participants 55 patients were randomized to standard medical therapy, without stem cell injection.	Total n=110 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	53 Participants n=5 Participants	52 Participants n=7 Participants	105 Participants n=5 Participants
Age, Categorical >=65 years	2 Participants n=5 Participants	3 Participants n=7 Participants	5 Participants n=5 Participants
Age, Continuous	53 years STANDARD_DEVIATION 8 • n=5 Participants	55 years STANDARD_DEVIATION 7 • n=7 Participants	54 years STANDARD_DEVIATION 9 • n=5 Participants
Sex: Female, Male Female	10 Participants n=5 Participants	11 Participants n=7 Participants	21 Participants n=5 Participants
Sex: Female, Male Male	45 Participants n=5 Participants	44 Participants n=7 Participants	89 Participants n=5 Participants
Region of Enrollment Slovenia	55 participants n=5 Participants	55 participants n=7 Participants	110 participants n=5 Participants

PRIMARY outcome

Timeframe: 5 years

Population: The minimal sample size for the study was calculated using a pre-specified power of 90% and P value of 0.05.

Outcome measures

Measure	SC Group n=55 Participants 55 patients were randomized to CD34+ cell transplantation (SC group). In the SC group, peripheral CD34+cells were mobilized by G-CSF and collected via apheresis. Patients underwent myocardial scintigraphy and CD34+ cells were injected in the artery supplying the segments with reduced viability	Control Group n=55 Participants 55 patients were randomized to standard medical therapy, without stem cell injection.
Heart Failure Mortality	8 participants	19 participants

PRIMARY outcome

Timeframe: 5 years

Left ventricular ejection fraction measured by echocardiography

Outcome measures

Measure	SC Group n=55 Participants 55 patients were randomized to CD34+ cell transplantation (SC group). In the SC group, peripheral CD34+cells were mobilized by G-CSF and collected via apheresis. Patients underwent myocardial scintigraphy and CD34+ cells were injected in the artery supplying the segments with reduced viability	Control Group n=55 Participants 55 patients were randomized to standard medical therapy, without stem cell injection.
Changes in Left Ventricular Ejection Fraction Baseline	24.3 Percentage of ejection Standard Deviation 6.5	25.7 Percentage of ejection Standard Deviation 4.1
Changes in Left Ventricular Ejection Fraction 5 years	30.0 Percentage of ejection Standard Deviation 5.1	23.3 Percentage of ejection Standard Deviation 4.2

SECONDARY outcome

Timeframe: 5 years

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 6 months

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 6 months

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 5 years

Outcome measures

Outcome data not reported

Adverse Events

SC Group

Serious events: 8 serious events

Other events: 2 other events

Deaths: 0 deaths

Control Group

Serious events: 19 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Measure	SC Group n=55 participants at risk 55 patients were randomized to CD34+ cell transplantation (SC group). In the SC group, peripheral CD34+cells were mobilized by G-CSF and collected via apheresis. Patients underwent myocardial scintigraphy and CD34+ cells were injected in the artery supplying the segments with reduced viability	Control Group n=55 participants at risk 55 patients were randomized to standard medical therapy, without stem cell injection.
Cardiac disorders Death	14.5% 8/55	34.5% 19/55

Other adverse events

Measure	SC Group n=55 participants at risk 55 patients were randomized to CD34+ cell transplantation (SC group). In the SC group, peripheral CD34+cells were mobilized by G-CSF and collected via apheresis. Patients underwent myocardial scintigraphy and CD34+ cells were injected in the artery supplying the segments with reduced viability	Control Group n=55 participants at risk 55 patients were randomized to standard medical therapy, without stem cell injection.
Cardiac disorders Non-sustained ventricular tachycardia	3.6% 2/55	0.00% 0/55

Additional Information

Prof. Dr. Bojan Vrtovec

UMC Ljubljana

Phone: +3861 522 2844

Email: bvrtovec@stanford.edu

Results disclosure agreements

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