Health TrialsbyHealthPanel

Autologous Stem Cell Transplantation for Crohn's Disease

ConditionCrohn's Disease
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age10-60
SponsorPaul Szabolcs

About this trial

The objective of this study is to evaluate the safety and effectiveness of administering high-dose chemotherapy followed by infusion of autologous CD34-selected peripheral blood stem cells (PBSC) in pediatric and adult patients with severe Crohn's disease.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Male or female, 10 through 60 years old, inclusive at time of informed consent.

Patients who have had prior surgery and subsequent severe recurrent disease in spite of aggressive maintenance therapy, necessitating consideration of further extensive surgical resections.

Patients who have diffuse small bowel and colonic disease and who are refractory to aggressive medical treatment, and not eligible for treatment using a surgical approach without the risk of precipitating short bowel syndrome and dependence of parenteral nutrition or who have other conditions that preclude surgery

Patients with a persistently high Harvey Bradshaw Index (HBI) (>6), CDAI (>250) or Pediatric CD Activity Index (PCDAI>45) (44) score or those in the lower, moderate range (HBI ≤ 6), (CDAI < 250), (PCDAI 30-45), but who are dependent on daily doses of corticosteroids, that are unable to be withdrawn, and aggressive medical treatment to maintain moderate disease status.

Disqualifiers

Patients who have not been treated with adequate dosing of 6-MP, 5-ASA products and metronidazole.

Patients who achieved a sustained, corticosteroid free response to anti-TNF alpha therapy, anti-integrin therapy or anti-IL12/23 therapy after a 4 month course of treatment.

Toxic megacolon, intestinal perforation

Conjugated bilirubin > 2.0 mg/dL.

Trial design

Design model

Single group

Treatments tested in this trial

  • autologous CD34-selected peripheral blood stem cells transplant

    Biological/Vaccine

    high-dose immunotherapy followed by infusion of autologous CD34-selected peripheral blood stem cells (PBSC)

  • Alemtuzumab

    Drug

    Transplant conditioning

  • ATG

    Drug

    Transplant conditioning

  • Melphalan

    Drug

    Transplant conditioning

  • Thiotepa

    Drug

    Transplant conditioning

  • Rituximab

    Drug

    Transplant conditioning

  • Cyclophosphamide

    Drug

    Mobilization

  • G-CSF

    Drug

    Mobilization

  • Mesna

    Drug

    Mobilization

Treatment groups

20 Participants
are divided into 1 treatment group
Group A: 1Experimental treatment 9 interventions

Trial outcomes

Primary outcomes

1

Number of participants with regimen-related toxicities.

Time frame
From baseline to 24 months post bone marrow transplant
2

Number of participants with life-threatening infections.

Time frame
From baseline to 24 months post bone marrow transplant
3

Change and duration in the Harvey Bradshaw Index (HBI).

Time frame
Change from Baseline to 24 months post Bone Marrow Transplant
4

Change and duration in the Crohn's Disease Activity Index (CDAI).

Time frame
Change from Baseline to 24 months post Bone Marrow Transplant

Secondary outcomes

1

Number of days it takes for Absolute Neutrophil Count (ANC) to reach greater than 500.

Time frame
3 consecutive days once ANC is greater than 500.
2

Number of days it takes for Platelet count to reach greater than 20,000/mm3

Time frame
From baseline to 24 months post Bone Marrow Transplant.
3

Number of days it takes for T cell Recovery

Time frame
24 months post Bone Marrow Transplant
4

Number of participants who have long term cardiac complications

Time frame
24 months post Bone Marrow Transplant

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Paul Szabolcs

Lead sponsor

University of Pittsburgh

Sponsor institution

Source ClinicalTrials.gov
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