RecruitingPhase 1Phase 2NCT04224558

Stem Cell Transplantation in Crohn's Disease

Autologous Hematopoietic Stem Cell Transplantation for Refractory Crohn's Disease


Sponsor

Cedars-Sinai Medical Center

Enrollment

15 participants

Start Date

Nov 15, 2019

Study Type

INTERVENTIONAL

Conditions

Summary

Unfortunately, some patients with Crohn's disease (CD) fail to respond to the best clinical treatments and some only experience temporary benefit. For severe Crohn's disease, there is an experimental treatment called "high dose immunoablation" followed by autologous hematopoietic stem cell transplantation (HSCT). This study removes over active lymphocytes (immunoablation) and replaces them using blood stem cells that have been taken from the patient's own body. The aim of the study is to reset or reprogram the patient's immune system to its state prior to diagnosis.


Eligibility

Min Age: 13 YearsMax Age: 28 Years

Plain Language Summary

Simplified for easier understanding

This study is testing whether stem cell transplantation can help young people (aged 13–28) with severe Crohn's disease — a chronic inflammatory bowel condition — that has not responded to multiple standard treatments. **You may be eligible if...** - You are between 13 and 28 years old - You have a confirmed diagnosis of active Crohn's disease - Your disease has not responded to at least three immunosuppressive treatments (such as azathioprine, methotrexate, infliximab, or adalimumab) - Surgery is not an appropriate option for you, or you risk developing short bowel syndrome from surgery - Your disease is active and flaring despite ongoing treatment **You may NOT be eligible if...** - You are pregnant or unwilling to use contraception - You have active, uncontrolled infection - Your health would make stem cell transplantation too risky - You are unwilling or unable to give informed consent and commit to intensive follow-up Talk to your doctor to see if this trial is right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGMesna

Stem Cell Mobilization: Infused according to institutional guidelines; Post-PBSC Infusion Conditioning: Mesna provided with Cytoxan according to institutional protocol.

DRUGCyclophosphamide

Stem Cell Mobilization: Cyclophosphamide (CY) infused intravenously over 1 hour: 50 mg/kg (25 mg/kg/day on 2 consecutive days)

DRUGFilgrastim

Stem Cell Mobilization: Filgrastim (G-CSF) 10 mcg/kg SC will start 5 days after the last dose of CY and will end the day before the last leukapheresis; Post-PBSC Infusion Conditioning: Filgrastim administered intravenously 5 mcg/kg IV starting day + 5, continue until ANC of \>1000/μL

PROCEDUREApheresis catheter placement

Subjects will require placement of an Apheresis catheter by Intervention Radiologists on the day of collection of stem cells.

PROCEDURELeukapheresis

Leukapheresis will be performed on a continuous flow separator machine according to institutional guidelines to target 3-8 x 10\^6 CD34+ cells/kg body weight.

DRUGFludarabine

Preparative/Conditioning Regime Fludarabine given as 30 mg/m2 per dose x 4 days, beginning on day -6.

DRUGMethylprednisolone

Preparative/Conditioning Regime r-ATG pre-medication according to institutional guidelines

DRUGDiphenhydramine

Preparative/Conditioning Regime r-ATG premedication according to institutional guidelines

DRUGAcetaminophen

Preparative/Conditioning Regime r-ATG premedication according to institutional guidlines

DRUGanti-thymocyte globulin (rabbit)

Preparative/Conditioning Regime r-ATG administered intravenously: 2.5 mg/kg/dose IV over 6 hours on specified days (day -6,-4,-2); ); total 3 doses=7.5 mg/kg.

DRUGlymphocyte immune globulin

Preparative/Conditioning Regime In patients who develop severe allergic reactions to rATG (Thymoglobulin), it may be substituted by horse ATG (hATG, ATGAM, Pharmacia \& Upjohn, Kalamazoo, MI). The recommended dose of hATG is 25 mg/kg/day for 3 doses.

BIOLOGICALPeripheral Blood Stem Cell Infusion

PBSC (peripheral blood stem cell) infusion on day 0 as per institutional guidelines.

DRUGCytoxan

Post-PBSC Infusion Conditioning Cytoxan infused intravenously: 50mg/kg/day x 2 days. Infused over 2 hours with adequate hydration or according to institutional guidelines.


Locations(1)

Cedars-Sinai Medical Center

Los Angeles, California, United States

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NCT04224558


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