MedTrace Logo

Pilot Study of Stem Cell Transplantation for Children and Young Adults With Refractory Crohn's Disease.

NCT02225795 · Status: WITHDRAWN · Phase: NA · Type: INTERVENTIONAL

Last updated 2018-02-07

No results posted yet for this study

Summary

This is a study for people with Crohn's Disease (CD) that are not responsive to standard treatment. CD is a chronic disease with an auto-immune component that goes away and relapses over the years and causes lifelong impairment of health and quality of life. Regardless of the therapy used some patients remain seriously ill with active disease after multiple therapeutic options have been exhausted. There is currently no drug that will cure CD. Drug treatment is focused on controlling symptoms. Another treatment is to perform surgery but again this does not lead to cure and is often linked to infection, short gut syndrome problems and psycho-social and cosmetic issues. Therefore, a treatment that does not involve surgery or long-term drug treatment may be beneficial especially to young adults.

Hematopoietic stem cell transplantation (HSCT) has been of value in other auto-immune diseases and it is possible that it could be of value in CD. This is a pilot study to determine if HSCT is safe and effective for children and young adults with severe CD. For this study the stem cells will come from the patient. This is called an autologous transplant. The patient will undergo collection and storage of his/her peripheral blood stem cells (PBSC). The patient will be given drugs to move (or mobilize) the stem cells from his/her bone marrow into his/her blood where they will be collected on a machine called apheresis (similar to dialysis.) The cells will be stored and given back to the patient about 1 month after collection.

Conditions

Interventions

OTHER

Hematopoietic stem cell transplantation

Immuno-ablative regimen for HSCT: Day Treatment * 6 r-ATG 2 mg/kg * 5 r-ATG3 2 mg/kg * 4 r-ATG3 2 mg/kg * 3 Cyclophosphamide with Mesna * 2 Cyclophosphamide with Mesna * 1 REST 0 PBSC infusion * 3 Cyclophosphamide with Mesna * 4 Cyclophosphamide with Mesna * 6 Start GCSF

Sponsors & Collaborators

  • TriStar Health

    lead OTHER

Principal Investigators

  • Haydar Frangoul, MD · TriStar Health

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
10 Years
Max Age
30 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2014-08-31
Primary Completion
2019-08-31
Completion
2019-12-31

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02225795 on ClinicalTrials.gov