Systemic SclerodermaPhase 2NCT01047072
Low-Dose Conditioning Followed by Donor Stem Cell Transplant in Treating Patients With Severe Systemic Sclerosis
A Phase 2 study testing fludarabine phosphate, total-body irradiation and tacrolimus in people with Systemic Scleroderma, run by Fred Hutchinson Cancer Research Center/University of Washington Cancer Consortium at 1 site in United States.
Trial summary
The purpose of the study is to see how well reduced intensity conditioning followed by a stem cell transplant from a donor (allogeneic) works in treating patients with severe systemic sclerosis. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Systemic Scleroderma
- What is being tested?
- fludarabine phosphate, total-body irradiation and tacrolimus
- Type of treatment
- Pill or oral medicine, Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- Start not givento Sep 2017
- Phase
- Phase 2
- 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 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients with severe SSc as defined by the American College of Rheumatology and at high-risk for a fatal outcome based on the following prognostic factors from groups 1-5:
- Group 1: Patients with 1) both a. and b. below; and 2) at least one of c., d. or e:
- a. Diffuse cutaneous scleroderma with skin score of >= 16 (modified Rodnan skin scale)
- b. Duration of systemic sclerosis =< 5 years from the onset of first non-Raynaud's symptom
- c. Presence of interstitial lung disease with FVC or DLCOcorr =< 70% of predicted and evidence of alveolitis (abnormal bronchoalveolar lavage [BAL] or high resolution chest computed tomography [CT] scan)
- d. Left heart failure with left ventricular ejection Fraction (LVEF) < 50% or pericardial effusion (mild-moderate) or 2nd or 3rd Atrial-Ventricular (AV) block; Myocardial disease not secondary to SSc must be excluded by a cardiologist
Show all 22Show fewer
- e. History of SSc-related renal disease that is not active at the time of screening; History of scleroderma hypertensive renal crisis is included in this criterion
- Group 2: Patients will have progressive pulmonary disease as the primary indication for transplant as defined by a decrease in the FVC or DLCO by 15 percent or greater in the previous 12-month period. In addition, patients may have either less skin involvement than group 1 (mRSS < 16) if they have a history of diffuse cutaneous disease and the FVC or DLCOcorr is < 70% or both FVC and DLCOcorr >= 70% if they have diffuse cutaneous disease (mRSS > 16) at screening for the study; Patients must also have evidence of alveolitis as defined by abnormal chest CT or BAL
- Group 3: Have progressive active SSc after prior autologous HCT based on the presence of progressive pulmonary disease; This will be defined by a decrease in the FVC since prior autologous transplant by 10 percent or greater, or DLCO since prior autologous transplant by 15 percent or greater in addition to evidence of alveolitis as defined by chest CT changes or BAL; If patients had prior autologous HCT on the SCOT clinical trial, they must have failed based on the defined study endpoints and be approved by the protocol principal investigator (PI)
- Group 4: Patients who meet group 1 inclusion criteria but have FVC or DLCO < 70% plus have had an adverse event to cyclophosphamide preventing its further use (i.e., hemorrhagic cystitis, leucopenia with WBC, 2000 or ANC < 1000 or platelet count < 100,000 and other adverse events)
- Group 5: Diffuse scleroderma with disease duration =< 2 years since development of first sign of skin thickening plus modified Rodnan skin score >= 25 plus ESR > 25 mm/1st hour and/or Hb < 11 g/dL not explained by causes other than active scleroderma.
- Unless patients have a DLCOcorr less than 45%, patients must have failed either oral or intravenous cyclophosphamide regimen defined as:
- IV cyclophosphamide administration for > 6 months or a total cumulative IV dose of 6 g/m^2, or
- oral cyclophosphamide administration for > 6 months regardless of dose, or
- combination of oral and IV cyclophosphamide for > 6 months independent of dose
- Patient must have a sibling who is a) HLA-identical and b) could serve as a donor of a peripheral blood stem cell graft to be placed on the transplant arm or an unrelated donor matched at HLA-A, B, C, DRB1 and DQB1. Patients without an HLA-identical sibling or an HLA-matched unrelated donor that meet the above criteria will be placed on the non-transplant arm
- DONOR:
- The donor must be an HLA-identical sibling of the patient or an HLA-matched unrelated donor.
- If the donor has reached the age of assent, then they must have completed the local institutional review board (IRB) assent process.
- Donor must consent to G-CSF administration and to leukapheresis for HSC collection.
- Donor must have adequate veins for leukapheresis or agree to placement of central venous catheter (femoral, subclavian).
- Age 12-75 years; Pediatric donors must be > 50 kg body weight.
Who cannot join
- Eligible for the NIH-sponsored randomized clinical trial (SCOT)
- Fertile men or women unwilling to use contraceptive techniques during and for 12 months or until immunosuppression is discontinued following transplantation
- Evidence of ongoing active infection
- Pregnancy
- Subjects with pulmonary, cardiac, hepatic, or renal impairment that would limit their ability to receive therapy and compromise their survival. This includes but is not restricted to, subjects with any of the following:
- Severe pulmonary dysfunction defined as:
Show all 28Show fewer
- A hemoglobin corrected DLCOcorr < 30% or FVC < 40% of predicted; or
- O2 saturation < 92% at rest without supplemental oxygen; or
- PO2 < 70 mmHg or pCO2 > 50 mmHg without supplemental oxygen
- Significant uncontrolled pulmonary hypertension defined as:
- Pulmonary artery peak systolic pressure > 45 mmHg by echocardiogram and mean pulmonary artery pressure by right heart catheterization exceeding 32 mmHg at rest or 42 mm Hg during exercise; or
- New York Heart Association (NYHA)/World Health Organization (WHO) classification for pulmonary hypertension, Class III or IV
- Cardiac: Uncontrolled clinically significant arrhythmias; clinical evidence of significant cardiac disease (NYHA Class III or IV); LVEF < 40% by echocardiogram
- Significant renal pathology, defined as:
- Estimated CrCl < 60 mL/min (using Cockcroft-Gault formula based on actual body weight) or serum creatinine > 2.0 mg/dL OR
- Active, untreated SSc renal crisis at the time of enrollment
- Active hepatitis or liver biopsy evidence of cirrhosis or periportal fibrosis. Total bilirubin > 2.5 x the upper limit of normal (and not related to Gilbert's syndrome) and/or Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) > 4 x the upper limit of normal
- Patients with poorly controlled hypertension
- Patients whose life expectancy is severely limited by illness other than autoimmune disease
- DONOR:
- Identical twin of patient
- Female donors who are pregnant (positive B-HCG) or breastfeeding
- Infection with human immunodeficiency virus (HIV) or active viral hepatitis (B or C)
- Known allergy to G-CSF
- Current serious systemic illness
- Uncontrolled bacterial, viral or fungal infection (currently taking medication and progression of clinical symptoms)
- Donors receiving experimental therapy or investigational agents
- Donors with cancer other than treated basal cell or carcinoma in situ of cervix; Cancer treated with curative intent > 2 years previous will be reviewed on a case-by-case basis by a Protocol Chair or Medical Monitor
Trial locations and contacts
1 site in 1 country.
United States1 site
Fred Hutchinson Cancer Research Center/University of Washington Cancer Consortium
Seattle, Washington, 98109
About this study
Official title: Phase II Clinical Trial of Allogeneic Hematopoietic Cell Transplantation After Nonmyeloablative Conditioning for Patients With Severe Systemic Sclerosis
The purpose of the study is to see how well reduced intensity conditioning followed by a stem cell transplant from a donor (allogeneic) works in treating patients with severe systemic sclerosis. In an allogeneic stem cell transplant procedure, stem cells are taken from a healthy donor and transplanted into the patient. Stem cells can be donated by a family member or an unrelated donor who is a complete tissue type match.
Read the detailed description
PRIMARY OBJECTIVES:
- To evaluate the overall effects of allogeneic hematopoietic cell transplantation (HCT) in systemic sclerosis (SSc).
II. To assess the potential efficacy, in terms of event-free survival (EFS) at 2 (part 1) and 5 (part 2) years, of allogeneic HCT as treatment for patients with severe SSc.
SECONDARY OBJECTIVES:
- To evaluate the rate of complications of allogeneic HCT after nonmyeloablative conditioning including the incidence of graft rejection, regimen-related toxicities, severe acute and chronic graft-vs-host disease (GVHD), infectious complications, treatment-related mortality (TRM), and overall survival.
II. To evaluate treatment effects on disease activation/progression, as indicated by measures of cardiac, pulmonary, gastrointestinal and renal function, as well as skin thickness, overall functional assessment (SHAQ), use of concomitant disease-modifying antirheumatic drugs (DMARDs), and occurrence of myositis.
III. To evaluate disease responses after nonmyeloablative conditioning and allogeneic HCT or immunomodulatory therapy, assessing skin disease by modified Rodnan Skin Score (mRSS), pulmonary function as measured by diffusing capacity of the lung for carbon monoxide (DLCO) and forced vital capacity (FVC), Modified Scleroderma Health Assessment Questionnaire (SHAQ), and Quality of Life using Short Form 36 (SF36).
IV. To evaluate, by mechanistic studies, of the effect of allogeneic HCT on dermal fibrosis and vasculopathy.
- To evaluate the late complications of allogeneic HCT after nonmyeloablative conditioning including the incidence and prevalence of chronic GVHD and time to discontinue immunosuppression, infectious complications, TRM and overall survival.
VI. To evaluate the treatment effects on disease activation/ progression, as indicated by measures of cardiac, pulmonary, gastrointestinal and renal function, as well as skin thickness, overall functional assessment (SHAQ), use of concomitant DMARDs, and occurrence of myositis.
VII. To evaluate disease responses after nonmyeloablative conditioning and allogeneic HCT or immunomodulatory therapy, assessing skin disease by modified Rodnan Skin Score (mRSS), pulmonary function as measured by DLCO and FVC, Modified Scleroderma Health Assessment Questionnaire (SHAQ), and Quality of Life using Short Form 36 (SF36).
OUTLINE: Patients are assigned to 1 of 2 treatment arms and receive nonmyeloablative conditioning followed by an allogeneic peripheral blood stem cell transplantation. Treatment in both arms continues in the absence of disease progression of unacceptable toxicity.
ARM I (transplant): Patients receive fludarabine intravenously (IV) on days -4 to -2. Patients undergo total-body irradiation on day 0. Patients then undergo peripheral blood stem cell transplantation on day 0. Patients receive GVHD prophylaxis comprising tacrolimus orally (PO) twice daily on days -3 to 180 and taper and mycophenolate mofetil PO three times daily on days 0-28 and then twice daily until day 180 and taper.
ARM II (nontransplant): Patients will receive immunosuppressive therapy based on their history. The 3 options that they may receive include 1.)mycophenolate mofetil PO twice daily for 16 months, 2.) Rituximab IV on days 1 and 15 and then repeated at 6 months, and 3.) Cyclophosphamide IV at 28-32 day intervals or orally once daily for 16 months.
After completion of study treatment, patients are followed up periodically for 5 years.
- Conditions
- Systemic Scleroderma
- Being tested
- fludarabine phosphate, total-body irradiation, tacrolimus, mycophenolate mofetil, rituximab
- Keywords
- Scleroderma, systemic sclerosis, allogeneic hematopoietic stem cell transplantation
- Medical subject headings
- Scleroderma, Systemic, Scleroderma, Diffuse
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Also called 2-F-ara-AMP, Beneflur, Fludara
Given IV
total-body irradiationRadiation
Also called TBI
Undergo total-body irradiation
tacrolimusDrug
Also called FK 506, Prograf
Given orally
Also called Cellcept, MMF
Given orally
rituximabBiological
Also called IDEC-C2B8, IDEC-C2B8 monoclonal antibody, Mabthera, MOAB IDEC-C2B8
Given IV
cyclophosphamideDrug
Also called CPM, CTX, Cytoxan, Endoxan
Given orally
nonmyeloablative allogeneic hematopoietic stem cell transplantationProcedure or surgery
Undergo transplantation
peripheral blood stem cell transplantationProcedure or surgery
Also called PBPC transplantation, PBSC transplantation, peripheral blood progenitor cell transplantation, transplantation, peripheral blood stem cell
Undergo transplantation
| Study group | Role | What they receive |
|---|---|---|
| Arm I (transplant) | Experimental | Patients receive fludarabine IV on days -4 to -2. Patients undergo total-body irradiation on day 0. Patients then undergo peripheral blood stem cell transplantation on day 0. Patients receive GVHD prophylaxis comprising tacrolimus PO twice daily on days -3 to 180 and taper and mycophenolate mofetil PO three times daily on days 0-28 and then twice daily until day 180 and taper. Receives: Drug: fludarabine phosphate; Radiation: total-body irradiation; Drug: tacrolimus; Drug: mycophenolate mofetil; Procedure: nonmyeloablative allogeneic hematopoietic stem cell transplantation; Procedure: peripheral blood stem cell transplantation |
| Arm II (nontransplant) | Active comparator | Patients receive mycophenolate mofetil PO twice daily for 16 months, rituximab IV on days 1 and 15 and then repeated at 6 months, and cyclophosphamide IV at 28-32 day intervals or orally once daily for 16 months. Receives: Drug: mycophenolate mofetil; Biological: rituximab; Drug: cyclophosphamide |
What is being measured
The primary outcome is the main question the study is designed to answer.
Event-free survival
Measured: 2 years
Secondary outcomes (11)
Event-free survival
Measured: 5 years
Overall survival
Measured: 2 years
Overall survival
Measured: 5 years
Time to progression
Measured: 2 years
Time to progression
Measured: 5 years
Incidence of initiation of any SSC disease-modifying therapy other than that specified in the protocol treatment arms
Measured: Up to 5 years
Incidence of definite and probable viral, fungal, and bacterial infections
Measured: Up to 5 years
Median time to engraftment as defined as having greater than or equal to 5% donor T cells in the peripheral blood (donor T cell chimerism)
Measured: From date of transplant to time of engraftment
Rate of primary graft failure
Measured: Day 56
Rate of secondary graft failure
Measured: 1 year
Probability of acute and chronic GVHD
Measured: Up to 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Jan 2010 (estimated)
- Primary completionSep 2017 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Systemic SclerodermaStudies of the same condition, open and under way.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Fred Hutchinson Cancer Research Center/University of Washington Cancer Consortium
- Sponsor type
- University, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Investigators
- George Georges, Principal investigator, Fred Hutchinson Cancer Research Center/University of Washington Cancer Consortium
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01047072
- Sponsor's protocol ID
- 2363.00
- Other IDs
- NCI-2012-00139
- Status last verified
- Oct 2012
- First submitted
- 11 Jan 2010
- Last updated
- 31 Oct 2012
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 31 Oct 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase II Clinical Trial of Allogeneic Hematopoietic Cell Transplantation After Nonmyeloablative Conditioning for Patients With Severe Systemic Sclerosis
- ClinicalTrials.gov ID
- NCT01047072
- Acronym / sponsor ID
- 2363.00
- Conditions
- Systemic Scleroderma
- Keywords
- Scleroderma, systemic sclerosis, allogeneic hematopoietic stem cell transplantation
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Withdrawn
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Fred Hutchinson Cancer Research Center/University of Washington Cancer ConsortiumUniversity, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Primary completion
- Sep 2017 (estimated)
- First posted
- 12 Jan 2010 (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.
The purpose of the study is to see how well reduced intensity conditioning followed by a stem cell transplant from a donor (allogeneic) works in treating patients with severe systemic sclerosis. In an allogeneic stem cell transplant procedure, stem cells are taken from a healthy donor and transplanted into the patient. Stem cells can be donated by a family member or an unrelated donor who is a complete tissue type match.
Detailed description
PRIMARY OBJECTIVES:
- To evaluate the overall effects of allogeneic hematopoietic cell transplantation (HCT) in systemic sclerosis (SSc).
II. To assess the potential efficacy, in terms of event-free survival (EFS) at 2 (part 1) and 5 (part 2) years, of allogeneic HCT as treatment for patients with severe SSc.
SECONDARY OBJECTIVES:
- To evaluate the rate of complications of allogeneic HCT after nonmyeloablative conditioning including the incidence of graft rejection, regimen-related toxicities, severe acute and chronic graft-vs-host disease (GVHD), infectious complications, treatment-related mortality (TRM), and overall survival.
II. To evaluate treatment effects on disease activation/progression, as indicated by measures of cardiac, pulmonary, gastrointestinal and renal function, as well as skin thickness, overall functional assessment (SHAQ), use of concomitant disease-modifying antirheumatic drugs (DMARDs), and occurrence of myositis.
III. To evaluate disease responses after nonmyeloablative conditioning and allogeneic HCT or immunomodulatory therapy, assessing skin disease by modified Rodnan Skin Score (mRSS), pulmonary function as measured by diffusing capacity of the lung for carbon monoxide (DLCO) and forced vital capacity (FVC), Modified Scleroderma Health Assessment Questionnaire (SHAQ), and Quality of Life using Short Form 36 (SF36).
IV. To evaluate, by mechanistic studies, of the effect of allogeneic HCT on dermal fibrosis and vasculopathy.
- To evaluate the late complications of allogeneic HCT after nonmyeloablative conditioning including the incidence and prevalence of chronic GVHD and time to discontinue immunosuppression, infectious complications, TRM and overall survival.
VI. To evaluate the treatment effects on disease activation/ progression, as indicated by measures of cardiac, pulmonary, gastrointestinal and renal function, as well as skin thickness, overall functional assessment (SHAQ), use of concomitant DMARDs, and occurrence of myositis.
VII. To evaluate disease responses after nonmyeloablative conditioning and allogeneic HCT or immunomodulatory therapy, assessing skin disease by modified Rodnan Skin Score (mRSS), pulmonary function as measured by DLCO and FVC, Modified Scleroderma Health Assessment Questionnaire (SHAQ), and Quality of Life using Short Form 36 (SF36).
OUTLINE: Patients are assigned to 1 of 2 treatment arms and receive nonmyeloablative conditioning followed by an allogeneic peripheral blood stem cell transplantation. Treatment in both arms continues in the absence of disease progression of unacceptable toxicity.
ARM I (transplant): Patients receive fludarabine intravenously (IV) on days -4 to -2. Patients undergo total-body irradiation on day 0. Patients then undergo peripheral blood stem cell transplantation on day 0. Patients receive GVHD prophylaxis comprising tacrolimus orally (PO) twice daily on days -3 to 180 and taper and mycophenolate mofetil PO three times daily on days 0-28 and then twice daily until day 180 and taper.
ARM II (nontransplant): Patients will receive immunosuppressive therapy based on their history. The 3 options that they may receive include 1.)mycophenolate mofetil PO twice daily for 16 months, 2.) Rituximab IV on days 1 and 15 and then repeated at 6 months, and 3.) Cyclophosphamide IV at 28-32 day intervals or orally once daily for 16 months.
After completion of study treatment, patients are followed up periodically for 5 years.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Arm I (transplant) | Experimental | Drug: fludarabine phosphate, Radiation: total-body irradiation, Drug: tacrolimus, Drug: mycophenolate mofetil, Procedure: nonmyeloablative allogeneic hematopoietic stem cell transplantation, Procedure: peripheral blood stem cell transplantationPatients receive fludarabine IV on days -4 to -2. Patients undergo total-body irradiation on day 0. Patients then undergo peripheral blood stem cell transplantation on day 0. Patients receive GVHD prophylaxis comprising tacrolimus PO twice daily on days -3 to 180 and taper and mycophenolate mofetil PO three times daily on days 0-28 and then twice daily until day 180 and taper. |
| Arm II (nontransplant) | Active comparator | Drug: mycophenolate mofetil, Biological: rituximab, Drug: cyclophosphamidePatients receive mycophenolate mofetil PO twice daily for 16 months, rituximab IV on days 1 and 15 and then repeated at 6 months, and cyclophosphamide IV at 28-32 day intervals or orally once daily for 16 months. |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (22 listed)
- Patients with severe SSc as defined by the American College of Rheumatology and at high-risk for a fatal outcome based on the following prognostic factors from groups 1-5:
- Unless patients have a DLCOcorr less than 45%, patients must have failed either oral or intravenous cyclophosphamide regimen defined as:
- Patient must have a sibling who is a) HLA-identical and b) could serve as a donor of a peripheral blood stem cell graft to be placed on the transplant arm or an unrelated donor matched at HLA-A, B, C, DRB1 and DQB1. Patients without an HLA-identical sibling or an HLA-matched unrelated donor that meet the above criteria will be placed on the non-transplant arm
- The donor must be an HLA-identical sibling of the patient or an HLA-matched unrelated donor.
- If the donor has reached the age of assent, then they must have completed the local institutional review board (IRB) assent process.
Key exclusion criteria (28 listed)
- Eligible for the NIH-sponsored randomized clinical trial (SCOT)
- Fertile men or women unwilling to use contraceptive techniques during and for 12 months or until immunosuppression is discontinued following transplantation
- Evidence of ongoing active infection
- Pregnancy
- Subjects with pulmonary, cardiac, hepatic, or renal impairment that would limit their ability to receive therapy and compromise their survival. This includes but is not restricted to, subjects with any of the following:
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 |
|---|---|---|
| fludarabine phosphateAlso: 2-F-ara-AMP, Beneflur, FludaraGiven IV | Drug | Arm I (transplant) |
| total-body irradiationAlso: TBIUndergo total-body irradiation | Radiation | Arm I (transplant) |
| tacrolimusAlso: FK 506, PrografGiven orally | Drug | Arm I (transplant) |
| mycophenolate mofetilAlso: Cellcept, MMFGiven orally | Drug | Arm I (transplant), Arm II (nontransplant) |
| rituximabAlso: IDEC-C2B8, IDEC-C2B8 monoclonal antibody, Mabthera, MOAB IDEC-C2B8Given IV | Biological | Arm II (nontransplant) |
| cyclophosphamideAlso: CPM, CTX, Cytoxan, EndoxanGiven orally | Drug | Arm II (nontransplant) |
| nonmyeloablative allogeneic hematopoietic stem cell transplantationUndergo transplantation | Procedure or surgery | Arm I (transplant) |
| peripheral blood stem cell transplantationAlso: PBPC transplantation, PBSC transplantation, peripheral blood progenitor cell transplantation, transplantation, peripheral blood stem cellUndergo transplantation | Procedure or surgery | Arm I (transplant) |
Primary outcome
Event-free survival
Time frame: 2 years
11 secondary outcomes
Event-free survival
Time frame: 5 years
Overall survival
Time frame: 2 years
Overall survival
Time frame: 5 years
Time to progression
Time frame: 2 years
Time to progression
Time frame: 5 years
Incidence of initiation of any SSC disease-modifying therapy other than that specified in the protocol treatment arms
Time frame: Up to 5 years
Incidence of definite and probable viral, fungal, and bacterial infections
Time frame: Up to 5 years
Median time to engraftment as defined as having greater than or equal to 5% donor T cells in the peripheral blood (donor T cell chimerism)
Time frame: From date of transplant to time of engraftment
Rate of primary graft failure
Time frame: Day 56
Rate of secondary graft failure
Time frame: 1 year
Probability of acute and chronic GVHD
Time frame: Up to 5 years
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Oct 2012.
Related trials for Systemic Scleroderma
Not yet recruitingPhase 2
Hyaluronidase Injections for Sclerodactyly in Patients With Scleroderma
Scleroderma, Sclerodactyly
RecruitingPhase 1
A Trial in Healthy Adult Participants and Adults With Autoimmune Diseases to Test How HBM7020 is Tolerated and How it Behaves in the Body
Rheumatoid Arthritis, Systemic Lupus Erythematosus
Active, not recruitingPhase 2
A Study Evaluating the Safety and Efficacy of Inhaled AP01 in Participants With Progressive Pulmonary Fibrosis
Pulmonary Fibrosis, Progressive Pulmonary Fibrosis
RecruitingObservational study
Quantification of Calcinosis Cutis Disease Burden Using Computed Tomography Images
Systemic Sclerosis (SSc), Calcinosis Cutis
RecruitingPhase 1/2
Phase I/II Study of AD-PluReceptor Plus Tafasitamab-cxix and Lymphodepleting Chemotherapy in Patients With Autoimmune Disorders
Autoimmune Disorders, Systemic Sclerosis
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.
George Georges principal investigator
Fred Hutchinson Cancer Research Center/University of Washington Cancer Consortium
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing fludarabine phosphate, total-body irradiation and tacrolimus in people with Systemic Scleroderma, run by Fred Hutchinson Cancer Research Center/University of Washington Cancer Consortium 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?
The sponsor lists 22 inclusion and 28 exclusion criteria. 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: withdrawn, as of the sponsor's last update on 31 Oct 2012. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 was withdrawn before enrolling participants, so there are no results.
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?
Event-free survival (time frame: 2 years). 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 31 Oct 2012.