Systemic SclerosisPhase 1NCT00010335
Pilot Study of Total Body Irradiation in Combination With Cyclophosphamide, Anti-thymocyte Globulin, and Autologous CD34-Selected Peripheral Blood Stem Cell Transplantation in Children With Refractory Autoimmune Disorders
A Phase 1 study testing Stem Cell Transplantation and CD34 selection in people with Systemic Sclerosis and Systemic Lupus Erythematosus, run by Fred Hutchinson Cancer Center at 1 site in United States.
Trial summary
OBJECTIVES: I. Determine the safety and long term complications of total body irradiation in combination with cyclophosphamide, anti-thymocyte globulin, and autologous CD34-selected peripheral blood stem cell (PBSC) transplantation in children with refractory autoimmune disorders. II. Read more about this study
- Who is it for?
- Ages 2 to 18 years
- Conditions the study is for
- Systemic Sclerosis, Systemic Lupus Erythematosus, Dermatomyositis
- What is being tested?
- Stem Cell Transplantation and CD34 selection
- Type of treatment
- Medical device, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 1 siteUnited States
- Study dates
- Nov 2000to May 2011
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
PROTOCOL ENTRY CRITERIA:
--Disease Characteristics--
- Diagnosis of 1 of the following based on American College of Rheumatology (ACR) Criteria: Severe juvenile rheumatoid arthritis (systemic onset or polyarticular course) Juvenile systemic lupus erythematosus Systemic sclerosis Dermatomyositis
- Refractory to standard or aggressive therapy OR unacceptable toxicity from standard therapy
- Reasonable expectation of possible improvement as evidenced by a good potential for rehabilitation therapy and adequate social factors
- No serious CNS damage that would preclude significant functional recovery
--Prior/Concurrent Therapy--
- Chemotherapy: At least 4 weeks since prior methotrexate or cyclophosphamide
- Endocrine therapy: At least 4 weeks since prior intra-arterial steroids Juvenile rheumatoid arthritis patients should continue steroids without taper throughout mobilization and harvest of stem cells If receiving corticosteroids, must be continued without taper
Other:
- At least 4 weeks since prior anti-inflammatory agents such as non-steroidal anti-inflammatory drugs (NSAIDs) or sulfasalazine
- At least 4 weeks since prior cyclosporine, tacrolimus, mycophenolate mofetil, azathioprine, penicillamine, or etanercept
--Patient Characteristics--
- Life expectancy: At least 30 days
- Hematopoietic: Absolute neutrophil count at least 1,000/mm3 OR Platelet count at least 100,000/mm3 No bone marrow aspirate or biopsy consistent with production defect (depletion of neutrophil precursors or megakaryocytes) No myelodysplasia
- Hepatic: Bilirubin no greater than 2.5 mg/dL AST no greater than 300 U/L on two sequential tests No severe liver dysfunction within past month No active hepatitis A, B, or C
- Renal: No end-stage glomerulonephritis or renal disease Creatinine clearance at least 40 mL/min
- Cardiovascular: No uncontrolled malignant arrhythmia No New York Heart Association class III or IV congestive heart failure Ejection fraction at least 50%
- Pulmonary: DLCO at least 45% (DLCO at least 70% for patients with pulmonary disease caused by documented processes other than primary autoimmune disorder, such as infectious pneumonia or aspiration pneumonia) No severe pulmonary hypertension (PAP greater than 50) without potential for significant improvement
Other:
- No medical or psychosocial reasons that would make hematopoietic stem cell collection intolerable
- No increased anesthetic risks
- No fever higher than 39 degrees C
- No positive serology for toxoplasmosis
- No active life threatening infection not responsive to therapy
- No other disease or organ dysfunction that would limit survival
- No known hypersensitivity to murine or equine proteins
- No known primary immunodeficiency disease HIV negative
Trial locations and contacts
1 site in 1 country.
United States1 site
Fred Hutchinson Cancer Research Center
Seattle, Washington, 98109
About this study
Official title: A Pilot Study of High-Dose Immunosuppression Followed by Infusion of CD34-Selected Autologous or Syngeneic Peripheral Blood Stem Cells for Treatment of Refractory Autoimmune Disorders
OBJECTIVES: I. Determine the safety and long term complications of total body irradiation in combination with cyclophosphamide, anti-thymocyte globulin, and autologous CD34-selected peripheral blood stem cell (PBSC) transplantation in children with refractory autoimmune disorders.
II. Determine the efficacy of this treatment regimen in these patients. III. Determine the reconstitution of immunity after autologous CD34-selected PBSC transplantation in these patients.
IV. Determine engraftment of autologous CD34-selected PBSC in these patients.
Read the detailed description
PROTOCOL OUTLINE: This is a multicenter study. Patients receive filgrastim (G-CSF) subcutaneously daily until peripheral blood stem cell (PBSC) collection is completed. CD34+ cells are separated from the rest of the PBSCs.
Patients undergo total body irradiation twice daily on days -5 and -4. Patients receive anti-thymocyte globulin IV on days -5, -3, -1, 1, 3, and 5 and cyclophosphamide IV on days -3 and -2. CD34-selected PBSCs are reinfused on day 0. Patients receive G-CSF IV daily beginning on day 0 and continuing until blood counts recover.
Patients are followed annually for 5 years and then every 5 years thereafter.
- Conditions
- Systemic Sclerosis, Systemic Lupus Erythematosus, Dermatomyositis, Juvenile Rheumatoid Arthritis, Autoimmune Diseases
- Being tested
- Stem Cell Transplantation, CD34 selection
- Keywords
- arthritis & connective tissue diseases, dermatomyositis, immunologic disorders and infectious disorders, juvenile rheumatoid arthritis, rare disease, systemic lupus erythematosus, systemic sclerosis
- Medical subject headings
- Scleroderma, Systemic, Lupus Erythematosus, Systemic, Dermatomyositis, Arthritis, Juvenile, Autoimmune Diseases, Arthritis, Connective Tissue Diseases, Immune System Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Stem Cell TransplantationProcedure or surgery
Participants will receive a stem cell transplantation along with irradiation and the drugs anti-thymocyte globulin, cyclophosphamide, and filgrastim as noted in the text of this record.
CD34 selectionDevice
CD34+ cells are separated from the rest of the peripheral blood stem cells.
| Study group | Role | What they receive |
|---|---|---|
| 1 | Experimental | Participants will receive a stem cell transplant. Receives: Procedure: Stem Cell Transplantation; Device: CD34 selection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Mortality
Measured: Annually for 5 years and then every 5 years thereafter
Secondary outcomes (1)
Immune reconstitution, engraftment, efficacy, late-effects
Measured: Annually for 5 years and then every 5 years thereafter
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Feb 2001 (estimated)
- Study startNov 2000
- Primary completionJul 2010
- Study completionMay 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Systemic SclerosisStudies of the same condition, open and under way.
- More trials run by Fred Hutchinson Cancer CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- Fred Hutchinson Cancer Center
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Ann Woolfrey, Study chair, Fred Hutchinson Cancer CenterCarol A. Wallace, MD, Principal investigator, Fred Hutchinson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00010335
- Sponsor's protocol ID
- 1353.00
- Other IDs
- FHCRC-1353.00, 199/15575
- Status last verified
- Mar 2015
- First submitted
- 2 Feb 2001
- Last updated
- 6 Mar 2015
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 6 Mar 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pilot Study of High-Dose Immunosuppression Followed by Infusion of CD34-Selected Autologous or Syngeneic Peripheral Blood Stem Cells for Treatment of Refractory Autoimmune Disorders
- ClinicalTrials.gov ID
- NCT00010335
- Acronym / sponsor ID
- 1353.00
- Conditions
- Systemic Sclerosis, Systemic Lupus Erythematosus, Dermatomyositis, Juvenile Rheumatoid Arthritis, Autoimmune Diseases
- Keywords
- arthritis & connective tissue diseases, dermatomyositis, immunologic disorders and infectious disorders, juvenile rheumatoid arthritis, rare disease, systemic lupus erythematosus, systemic sclerosis
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 6 participants (actual)
- Lead sponsor
- Fred Hutchinson Cancer CenterUniversity, hospital or other
- Study start
- Nov 2000
- Primary completion
- Jul 2010 (actual)
- Study completion
- May 2011 (actual)
- First posted
- 2 Feb 2001 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
OBJECTIVES: I. Determine the safety and long term complications of total body irradiation in combination with cyclophosphamide, anti-thymocyte globulin, and autologous CD34-selected peripheral blood stem cell (PBSC) transplantation in children with refractory autoimmune disorders.
II. Determine the efficacy of this treatment regimen in these patients. III. Determine the reconstitution of immunity after autologous CD34-selected PBSC transplantation in these patients.
IV. Determine engraftment of autologous CD34-selected PBSC in these patients.
Detailed description
PROTOCOL OUTLINE: This is a multicenter study. Patients receive filgrastim (G-CSF) subcutaneously daily until peripheral blood stem cell (PBSC) collection is completed. CD34+ cells are separated from the rest of the PBSCs.
Patients undergo total body irradiation twice daily on days -5 and -4. Patients receive anti-thymocyte globulin IV on days -5, -3, -1, 1, 3, and 5 and cyclophosphamide IV on days -3 and -2. CD34-selected PBSCs are reinfused on day 0. Patients receive G-CSF IV daily beginning on day 0 and continuing until blood counts recover.
Patients are followed annually for 5 years and then every 5 years thereafter.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 1 | Experimental | Procedure: Stem Cell Transplantation, Device: CD34 selectionParticipants will receive a stem cell transplant. |
Who may qualify
- Ages
- 2 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Stem Cell TransplantationParticipants will receive a stem cell transplantation along with irradiation and the drugs anti-thymocyte globulin, cyclophosphamide, and filgrastim as noted in the text of this record. | Procedure or surgery | 1 |
| CD34 selectionCD34+ cells are separated from the rest of the peripheral blood stem cells. | Device | 1 |
Primary outcome
Mortality
Time frame: Annually for 5 years and then every 5 years thereafter
1 secondary outcome
Immune reconstitution, engraftment, efficacy, late-effects
Time frame: Annually for 5 years and then every 5 years thereafter
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Mar 2015.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Ann Woolfrey study chair
Fred Hutchinson Cancer Center
Carol A. Wallace, MD principal investigator
Fred Hutchinson Cancer Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Stem Cell Transplantation and CD34 selection in people with Systemic Sclerosis and Systemic Lupus Erythematosus, run by Fred Hutchinson Cancer Center at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 6 Mar 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Mortality (time frame: Annually for 5 years and then every 5 years thereafter). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Mar 2015.