NCT02479776

Brief Summary

The bone marrow mononuclear cell fraction has been used as therapy after myocardial infarction and in dilated cardiomyopathy in adults. The absence of adult co-morbidities may enhance the potential effectiveness of pediatric stem cells.This study is a randomized, crossover, placebo controlled pilot study to primarily determine the safety and feasibility of stem cell intracoronary therapy in children. Secondary end points are MRI measurements and NTproBNP. Ten children (mean age 7.2 years, range 2.2-14.1, 6 male) with dilated cardiomyopathy (NYHA/ Ross Classification 2-4) will be recruited. Bone marrow aspiration MRI and cell injection are performed under the same anaesthetic. Patients will be crossed over at 6 months.

Trial Health

100
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
10

participants targeted

Target at below P25 for not_applicable

Timeline
Completed

Started May 2008

Longer than P75 for not_applicable

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

May 1, 2008

Completed
6.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 1, 2015

Completed
2 months until next milestone

Study Completion

Last participant's last visit for all outcomes

March 1, 2015

Completed
3 months until next milestone

First Submitted

Initial submission to the registry

June 8, 2015

Completed
16 days until next milestone

First Posted

Study publicly available on registry

June 24, 2015

Completed
Last Updated

June 24, 2015

Status Verified

June 1, 2015

Enrollment Period

6.7 years

First QC Date

June 8, 2015

Last Update Submit

June 23, 2015

Conditions

Dilated Cardiomyopathy

Outcome Measures

Primary Outcomes (1)

  • adverse effect

    death , transplant or major adverse effect

    6 months

Secondary Outcomes (2)

  • Blood for NT pro BNP

    6 months

  • MRI volumes

    6 months

Study Arms (2)

Stem cell injection

ACTIVE COMPARATOR

Stem cells are injected and patients crossed over to placebo at 6 months

Biological: stem cell injection

saline injection

PLACEBO COMPARATOR

saline is injected and patients crossed over to active arm at 6 months

Interventions

stem cell injectionBIOLOGICAL

Injection of autologous bone marrow derived stem cells

Stem cell injection

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Age 1-16 years.
  • Diagnosis of dilated cardiomyopathy

You may not qualify if:

  • Age less than 1 year or over 17 years.
  • A need for high dependency or intensive care and congenital heart disease
  • Viral infection that would preclude the use of hospital Cell Therapy Laboratory facilities
  • Active malignancy
  • Unstable cardiac drug therapy

Contact the study team to confirm eligibility.

Sponsors & Collaborators

MeSH Terms

Conditions

Cardiomyopathy, Dilated

Condition Hierarchy (Ancestors)

CardiomegalyHeart DiseasesCardiovascular DiseasesCardiomyopathiesLaminopathiesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Design

Study Type
interventional
Phase
not applicable
Allocation
RANDOMIZED
Masking
SINGLE
Who Masked
OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
CROSSOVER
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

June 8, 2015

First Posted

June 24, 2015

Study Start

May 1, 2008

Primary Completion

January 1, 2015

Study Completion

March 1, 2015

Last Updated

June 24, 2015

Record last verified: 2015-06