NCT00692939 · Paul Szabolcs
Autologous Stem Cell Transplantation for Crohn's Disease
What this study is about
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.
View original scientific description
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.
Interventions
BIOLOGICAL
autologous CD34-selected peripheral blood stem cells transplant
high-dose immunotherapy followed by infusion of autologous CD34-selected peripheral blood stem cells (PBSC)
DRUG
Alemtuzumab
Transplant conditioning
DRUG
ATG
Transplant conditioning
DRUG
Melphalan
Transplant conditioning
DRUG
Thiotepa
Transplant conditioning
DRUG
Rituximab
Transplant conditioning
DRUG
Cyclophosphamide
Mobilization
DRUG
G-CSF
Mobilization
DRUG
Mesna
Mobilization
Primary outcome measures
Number of participants with regimen-related toxicities.
Time frame: From baseline to 24 months post bone marrow transplant
Number of participants with life-threatening infections.
Time frame: From baseline to 24 months post bone marrow transplant
Change and duration in the Harvey Bradshaw Index (HBI).
Time frame: Change from Baseline to 24 months post Bone Marrow Transplant
Change and duration in the Crohn's Disease Activity Index (CDAI).
Time frame: Change from Baseline to 24 months post Bone Marrow Transplant
Change and duration in the Pediatric Crohn's Disease Activity Index (PCDAI).
Time frame: Change from Baseline to 24 months post Bone Marrow Transplant
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- Subject and/or guardian must be able to understand and provide informed consent. 2. Male or female, 10 through 60 years old, inclusive at time of informed consent. 3. Examples of subjects for whom stem cell transplant therapy would be appropriate include, but are not limited to:
- 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 ran
Where
- Pittsburgh, Pennsylvania
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Dec 15, 2025 · Source of record for eligibility and locations