AzTrials
AzTrials

Systemic SclerodermaPhase 2STATNCT01413100

Scleroderma Treatment With Autologous Transplant (STAT) Study

A Phase 2 study testing Anti-Thymocyte Globulin, Autologous Hematopoietic Stem Cell Transplantation and Cyclophosphamide in people with Systemic Scleroderma, run by Fred Hutchinson Cancer Center at 12 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial summary

This phase II trial studies how well giving cyclophosphamide and anti-thymocyte globulin together followed by peripheral blood stem cell transplant (PBSCT) and mycophenolate mofetil works in treating patients with systemic scleroderma (SSc). Read more about this study

Who is it for?
Ages Up to 70 years
Conditions the study is for
Systemic Scleroderma
What is being tested?
Anti-Thymocyte Globulin, Autologous Hematopoietic Stem Cell Transplantation and Cyclophosphamide
Could I receive a placebo?
No, everyone receives an active treatment
How many people
21 participants
Where
12 sitesUnited States
Study dates
15 Sep 2011to 11 Sep 2024
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

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 Up to 70 years
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • Patients with SSc as defined by the American College of Rheumatology with diffuse cutaneous disease (except Group 5) at risk of disease progression
  • Patients must have failed a prior >= 4-mponth course of either MMF/Myfortic or cyclophosphamide before being eligible for the study (determined at >= 1 week before start of mobilization); "failure" is defined as evidence of disease progression or absence of improvement; the response prior to MMF of cyclophosphamide will be assessed by the participating site study rheumatologist
  • Patients must meet eligibility in at least 1 of the following 6 groups:
  • GROUP 1:
  • Patients must have 1) both a and b below; and 2) either c, or d
  • a) Diffuse cutaneous scleroderma as defined by skin thickening proximal to the elbows and knees and/or involving the torso in addition to distal extremity involvement; a skin score will be obtained but not used to determine eligibility
Show all 39Show fewer
  • b) Duration of systemic sclerosis =< 7 years from the onset of first non-Raynaud's symptom; for those patients with disease activity between 5-7 years from the onset of first non-Raynaud's symptom, recent progression or activity of disease must be documented
  • c) Presence of SSc-related pulmonary disease with forced vital capacity (FVC) < 80% or hemoglobin-adjusted diffusing capacity for carbon monoxide (DLCO) < 70% of predicted AND evidence of alveolitis or SSc-related interstitial lung disease by high-resolution chest computed tomography (CT) scan and/or by bronchoalveolar lavage (BAL) (interstitial lung disease may be nonspecific interstitial pneumonia [NSIP] or usual interstitial pneumonia [UIP]; a bronchoalveolar lavage [BAL] should be done to confirm the findings of alveolitis only if the high resolution CT scan [HRCT] fails to show findings typically associated with systemic sclerosis changes [ground glass NSIP, UIP, SSc related interstitial lung disease]); alveolitis by BAL cell count will be defined based on a BAL cell differential count (> 3% neutrophils and/or > 2% eosinophils) from any lavaged lobe
  • d) History of SSc-related renal disease that may not be active at the time of screening; stable serum creatinine must be documented for a minimum of 3 months post-renal crisis at the time of the baseline visit; history of scleroderma hypertensive renal crisis is included in this criterion and is defined as follows:
  • History of new-onset hypertension based on any of the following (measurements must be repeated and confirmed at least 2 hours apart within 3 days of first event-associated observation, with a change from baseline):
  • Systolic blood pressure (SBP) >= 140 mmHg
  • Diastolic blood pressure (DBP) >= 90 mmHg
  • Rise in SBP >= 30 mmHg compared to baseline
  • Rise in DBP >= 20 mmHg compared to baseline
  • AND one of the following 5 laboratory criteria:
  • Increase of >= 50 % above baseline in serum creatinine
  • Proteinuria: >= 2+ by dipstick confirmed by protein:creatinine ratio > 2.5
  • Hematuria: >= 2+ by dipstick or > 10 red blood cell (RBC)s/hematopoietic-promoting factor (HPF) (without menstruation)
  • Thrombocytopenia: < 100,000 platelets/mm^3
  • Hemolysis: by blood smear or increased reticulocyte count
  • The above definition of SSc hypertensive renal crisis is independent of whether concomitant anti-hypertensive medications are used
  • Subjects who present with solely skin and renal disease in the absence of other organ involvement, except classic SSc renal crisis as described above and including non-hypertensive renal crisis, must see a nephrologist to confirm that their renal disease is secondary to only SSc
  • Note: Subjects may be re-screened if they fail to meet inclusion criteria on initial evaluation
  • GROUP 2:
  • Progressive pulmonary disease as defined by a decrease in the FVC or DLCO-adjusted by 10 or 15 percent or greater, respectively, from a prior FVC or DLCO-adjusted in the previous 18-month period
  • Patients will have diffuse cutaneous disease and may have both FVC and DLCOcorr >= 70% at screening for the study
  • Patients must also have evidence of alveolitis as defined by abnormal chest computed tomography (CT) or bronchoalveolar lavage (BAL)
  • GROUP 3: Diffuse scleroderma with disease duration =< 2 years since development of first sign of skin thickening plus modified Rodnan skin score >= 25 plus either
  • Erythrocyte sedimentation rate (ESR) > 25 mm/1st hour and/or hemoglobin (Hb) < 11 g/dL, not explained by causes other than active scleroderma
  • Lung involvement (either FVC or DLCO < 80% and evidence of interstitial lung disease by CT scan or alveolitis by BAL)
  • GROUP 4: Diffuse scleroderma with disease duration =< 2 years and skin score >= 30
  • GROUP 5:
  • Limited cutaneous scleroderma and SSc-related pulmonary disease with FVC < 80% or hemoglobin-adjusted DLCO < 70% of predicted
  • AND evidence of alveolitis/interstitial lung disease by high-resolution chest CT scan and/or by BAL (interstitial lung disease may be nonspecific interstitial pneumonia [NSIP] or usual interstitial pneumonia [UIP]; A bronchoalveolar lavage [BAL] should be done to confirm the findings of alveolitis only if the high resolution CT scan [HRCT] fails to show findings typically associated with systemic sclerosis changes [ground glass, NSIP, UIP, SSc related interstitial lung disease])
  • Alveolitis by BAL cell count will be defined based on a BAL cell differential count (> 3% neutrophils and/or > 2% eosinophils) from any lavaged lobe
  • GROUP 6: Progressive gastrointestinal disease as defined by all of the following items:
  • Disease duration of scleroderma =< 2 years.
  • Documented severe malabsorption syndrome requiring nutritional support; severe malabsorption syndrome is > 10% weight loss and on total parenteral nutrition (TPN) or enteral feedings
  • High score on distention/ bloating scale (>= 1.60 out of 3.00) on gastrointestinal (GI) questionnaire

Who cannot join

  • Subjects with pulmonary, cardiac, hepatic, or renal impairment that would limit their ability to receive cytoreductive therapy and compromise their survival; this includes, but is not restricted to, subjects with any of the following:
  • Pulmonary dysfunction defined as:
  • Severe pulmonary dysfunction with (1) a hemoglobin corrected DLCO < 40% of predicted at the Baseline Screening visit, or (3) FVC < 45% of predicted Baseline Screening visit, or
  • Partial pressure (pO2) < 70 mmHg or pCO2 >= 45 mmHg without supplemental oxygen, or
  • O2 saturation < 92% at rest without supplemental oxygen as measured by forehead pulse oximeter
  • Significant pulmonary artery hypertension (PAH) defined as:
Show all 37Show fewer
  • Peak systolic pulmonary artery pressure > 50 mmHg by resting echocardiogram will require right heart catheterization; if pulmonary artery pressure (PAP) is not evaluable on echocardiogram due to lack of a Tricuspid regurgitant jet, then normal anatomy and function as evidenced by normal right atrium and ventricle size, shape and wall thickness and septum shape must be documented to rule-out PAH; otherwise, right heart catheterization is indicated; prior history of PAH but controlled with medications will not exclude patients from the protocol; PAH is considered controlled with medications if peak systolic pulmonary artery pressure is < 45 mmHg or mean pulmonary artery pressure by right heart catheterization is < 30 mmHg at rest
  • Mean pulmonary artery pressure by right heart catheterization exceeding 30 mmHg at rest; if mean PAP is elevated and pulmonary vascular resistance and transpulmonary gradient are normal then the patient is eligible for the protocol
  • New York Heart Association (NYHA)/World Health Organization Class III or IV
  • Cardiac: Uncontrolled clinically significant arrhythmias; clinical evidence of significant congestive heart failure (CHF) (NYHA Class III or IV); left ventricular ejection fraction (LVEF) < 50% by echocardiogram
  • History/presence of arrhythmia (even controlled) on chemical anti-arrhythmic(s) must have cardiac consult to ensure the subject could safely proceed with protocol requirements
  • Significant renal pathology defined as:
  • Estimated creatinine clearance (CrCl) < 40 mL/min (using Cockcroft-Gault formula based on actual body weight) and serum creatinine > 2.0 mg/dL; OR
  • Active, untreated SSc renal crisis at the time of enrollment; presence of nephrotic range proteinuria (defined as >= 3.5 gms/24 hours, or protein:creatinine ratio >= 3.5), active urinary sediment, urinary RBCs > 25 per HPF, or red cell casts require further investigation by a nephrologist to rule out glomerulonephritis, overlap syndromes, or other causes of renal disease in all subjects; subjects with glomerulonephritis or overlap syndromes will be excluded
  • Hepatic: Active hepatitis (alanine aminotransferase [ALT], aspartate aminotransferase [AST], or bilirubin > 2 times the upper limit of normal [ULN]) or evidence of moderate to severe periportal fibrosis by liver biopsy
  • Active or clinically significant Gastric Antral Vascular Ectasia (GAVE, "watermelon stomach")
  • Unwilling or unable to discontinue disallowed disease-modifying antirheumatic drugs (DMARDs) for treatment of SSc prior to mobilization
  • History or presence of a 2nd autoimmune disease requiring immunosuppressive therapy that has substantial risk of immunosuppressive treatment beyond transplant with the following exceptions:
  • History and/or presence of Sjogren's Syndrome is allowed
  • Stable myositis (A history of myositis that is clinically stable as defined by lack of progressive proximal muscle weakness and a stable or decreasing creatine phosphokinase [CPK] < 3 x ULN) is allowed
  • The presence of anti-double stranded (ds)-deoxyribonucleic acid (DNA) without clinical systemic lupus erythematosus in a patient with a diagnosis of otherwise "pure" SSc is allowed
  • Concomitant rheumatoid arthritis without extra-articular disease characteristic of rheumatoid arthritis is allowed
  • Active uncontrolled infection that would be a contraindication to safe use of high-dose therapy
  • Positive study for Hepatitis B surface antigen or Hepatitis B or C confirmed by polymerase chain reaction (PCR)
  • Positive serology for human immunodeficiency virus (HIV)
  • Absolute neutrophil count (ANC) < 1500 cells/uL
  • Platelets < 100,000 cells/uL
  • Hematocrit < 27%
  • Hemoglobin < 9.0 g/dL
  • Malignancy within the 2 years prior to entry in study, excluding adequately treated squamous cell skin cancer, basal cell carcinoma, and carcinoma in situ; treatment must have been completed (with the exception of hormonal therapy for breast cancer) with cure/remission status verified for at least 2 years prior to entry in this study
  • Presence of other comorbid illnesses with an estimated median life expectancy < 5 years
  • Evidence of myelodysplasia (MDS); subjects with history of receiving any prior chemotherapy and/or radiotherapy for the treatment of malignant disease, history of greater than 2 months total prior cyclophosphamide for any condition (regardless of dose and route) and/or subjects presenting with abnormal peripheral blood counts require unilateral bone marrow aspiration for pathology, flow cytometry, cytogenetics, and fluorescence in situ hybridization (FISH) MDS panel (per institutional profile) to rule out MDS
  • Pregnancy
  • Inability to give voluntary informed consent
  • Unwilling to use contraceptive methods for at least 15 months after starting treatment
  • History of smoking tobacco (or other related/herbal products) in the prior 3 months
  • History of prior autologous hematopoietic cell transplantation

Trial locations and contacts

12 sites in 1 country.

12 sites in 1 country

United States12 sites

  • University of Michigan Comprehensive Cancer Center

    Ann Arbor, Michigan, 48109

  • Boston Medical Center

    Boston, Massachusetts, 02118

  • Boston University School of Medicine

    Boston, Massachusetts, 02118

  • University of Colorado

    Denver, Colorado, 80217-3364

  • Colorado Blood Cancer Institute

    Denver, Colorado, 80218

  • City of Hope Comprehensive Cancer Center

    Duarte, California, 91010

  • Duke University Medical Center

    Durham, North Carolina, 27710

  • M D Anderson Cancer Center

    Houston, Texas, 77030

  • The University of Texas Health Science Center, Houston

    Houston, Texas, 77030

  • UCLA / Jonsson Comprehensive Cancer Center

    Los Angeles, California, 90095

  • Seattle Children's Hospital

    Seattle, Washington, 98105

  • Fred Hutch/University of Washington Cancer Consortium

    Seattle, Washington, 98109

Other trials in United States

About this study

Official title: A Phase II Multi-center Study of High-Dose Cyclophosphamide and Antithymocyte Globulin Followed by Autologous Hematopoietic Cell Transplantation With Post Transplant Maintenance for the Treatment of Systemic Sclerosis

This phase II trial studies how well giving cyclophosphamide and anti-thymocyte globulin together followed by peripheral blood stem cell transplant (PBSCT) and mycophenolate mofetil works in treating patients with systemic scleroderma (SSc). Stem cells are collected from the patient's blood and stored prior to treatment. To store the stem cells patients are given colony-stimulating factors, such as filgrastim (G-CSF) or chemotherapy (cyclophosphamide) to help stem cells move from the bone marrow to the blood so they can be collected and stored. After storage, patients are then given high-dose chemotherapy, cyclophosphamide, and immunosuppression with anti-thymocyte globulin to suppress the immune system to prepare for the transplant. The stem cells are then returned to the patient to replace the blood-forming cells that were destroyed by the chemotherapy and immunosuppression. After the stem cells have "engrafted" and have matured enough to support the immune system at approximately 2-3 months, patients are given a medication called mycophenolate mofetil (MMF) or Myfortic. This medication is given to prevent worsening or reactivation of SSc and is referred to as maintenance therapy.

Read the detailed description

OUTLINE:

STEM CELL MOBILIZATION AND PREPARATION: Patients receive filgrastim subcutaneously (SC) on mobilization days 1-4 followed by apheresis until a target dose of CD34+ cells >= 2.5 x 10^6/kg are collected. Patients difficult to mobilize with filgrastim alone receive cyclophosphamide intravenously (IV) or *plerixafor subcutaneously (SC) on mobilization days 1-2 and filgrastim SC on mobilization days 5-7.

HDIT CONDITIONING: Patients receive high-dose cyclophosphamide IV over 1-2 hours on days -5 to -2 and anti-thymocyte globulin IV on days -5, -3, -1, 1, 3, and 5.

TRANSPLANTATION: Patients undergo autologous PBSCT on day 0.

MAINTENANCE THERAPY: Beginning 2-3 months after transplant, patients receive mycophenolate mofetil orally (PO) twice daily (BID) for 2 years.

NOTE: *Plerixafor is an alternative to the cyclophosphamide based mobilization.

After completion of study treatment, patients are followed at 1 month, weeks 12 and 26, and then annually for 5 years.

Being tested
Anti-Thymocyte Globulin, Autologous Hematopoietic Stem Cell Transplantation, Cyclophosphamide, Filgrastim, Laboratory Biomarker Analysis
Medical subject headings
Scleroderma, Systemic

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Anti-Thymocyte GlobulinBiological

    Also called Antithymocyte Globulin, Antithymocyte Serum, ATG, ATGAM

    Given IV

  • Autologous Hematopoietic Stem Cell TransplantationProcedure or surgery

    Also called Autologous Stem Cell Transplantation

    Undergo autologous PBSCT

  • CyclophosphamideDrug

    Also called (-)-Cyclophosphamide, 2H-1,3,2-Oxazaphosphorine, 2-[bis(2-chloroethyl)amino]tetrahydro-, 2-oxide, monohydrate, Carloxan, Ciclofosfamida

    Given IV

  • FilgrastimBiological

    Also called Filgrastim XM02, G-CSF, Neupogen, r-metHuG-CSF

    Given SC

  • Laboratory Biomarker AnalysisOther

    Correlative studies

  • Mycophenolate MofetilDrug

    Also called Cellcept, MMF

    Given PO

  • Peripheral Blood Stem Cell TransplantationProcedure or surgery

    Also called PBPC transplantation, Peripheral Blood Progenitor Cell Transplantation, Peripheral Stem Cell Support, Peripheral Stem Cell Transplantation

    Undergo autologous PBSCT

  • PlerixaforDrug

    Also called AMD 3100, JM-3100, Mozobil, SDZ SID 791

    Given SC

  • Quality-of-Life AssessmentOther

    Also called Quality of Life Assessment

    Ancillary studies

  • Questionnaire AdministrationOther

    Ancillary studies

Study groups in NCT01413100
Study groupRoleWhat they receive
Treatment (HDIT autologous PBSCT)ExperimentalSTEM CELL MOBILIZATION AND PREPARATION: Patients receive filgrastim SC on mobilization days 1-4 followed by apheresis until a target dose of CD34+ cells >= 2.5 x 10^6/kg are collected. Patients difficult to mobilize with filgrastim alone receive cyclophosphamide IV or *plerixafor SC on mobilization days 1-2 and filgrastim SC on mobilization days 5-7. HDIT CONDITIONING: Patients receive high-dose cyclophosphamide IV over 1-2 hours on days -5 to -2 and anti-thymocyte globulin IV on days -5, -3, -1, 1, 3, and 5. TRANSPLANTATION: Patients undergo autologous PBSCT on day 0. MAINTENANCE THERAPY: Beginning 2-3 months after transplant, patients receive mycophenolate mofetil PO BID for 2 years.
Receives: Biological: Anti-Thymocyte Globulin; Procedure: Autologous Hematopoietic Stem Cell Transplantation; Drug: Cyclophosphamide; Biological: Filgrastim; Other: Laboratory Biomarker Analysis; Drug: Mycophenolate Mofetil; Procedure: Peripheral Blood Stem Cell Transplantation; Drug: Plerixafor; Other: Quality-of-Life Assessment; Other: Questionnaire Administration

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. EFS of Patients Undergoing Transplant

    Event Free Survival (EFS) was defined as survival without meeting the protocol defined endpoint of organ injury (kidney injury requiring renal replacement dialysis for >6 months, sustained LVEF <30%, or sustained decline of FVC >20%).

    Measured: At 5 years

Secondary outcomes (15)

  1. All-cause Mortality

    Defined as any death.

    Measured: At 5 years

  2. The Number of Participants With Stable or Improved LVEF

    By echocardiogram, left ventricle ejection fraction (LVEF) greater than or equal to 30% for greater than or equal to 3 months

    Measured: From baseline to year 5

  3. Number of Participants Requiring Dialysis

    Measured by requiring chronic dialysis greater than or equal to 6 months after transplant or required kidney transplant.

    Measured: From baseline to year 5

  4. The Number of Participants With Disease Progression

    Chronic renal dysfunction requiring dialysis greater than or equal to 6 months post transplant or requiring kidney transplant, sustained by echo LVEJF <30% for at least 3 months post transplant, or sustained greater than or equal to 3 months decrease of FVC on pulmonary function test (PFT) > 20% post transplant.

    Measured: baseline to year 5

  5. The Number of Participants Who Completed ALL Health Care Utilization as Assessed by UCSD Healthcare Utilization Surveys

    UCSD healthcare utilization is a self-report instrument that asks the patient about outpatient and inpatient visits, prescription and non-prescription medications, any surgeries, and major medical expenses during the last 3 months.

    Measured: From baseline to 5 years

  6. The Median SHAQ Score

    Scleroderma Health Assessment Questionnaire (SHAQ) is a self reported questionnaire with 8 domains including the following scales: pain, patient global assessment, vascular digital ulcers, lung involvement, and gastrointestinal involvement. The SHAQ is a quality of life measure. Each question is scored from 0 (defined as without difficulty), to 3 (defined as unable to do). Some domains are visual analog scales that are measured first and then changed to the 0-3 scale. The individual scores are combined and divided by 8. A higher score indicates worse functionality and changes in the SHAQ is measured as medium change from baseline. The reported medium change can range from -3 to 3. A negative medium change indicates a better outcome.

    Measured: Baseline to year 4

  7. The Number of Participants With Improvement in Pulmonary Function

    Outcome measure was assessed as the number of participants who had greater than or equal to 3 months a > 10% improvement in predicted FVC or a > 15% improvement in DLCO.

    Measured: From baseline to year 5

  8. The Number of Participants With Significant Infectious Complications

    Infections of grade 3 or above

    Measured: Mobilization to Day + 100 post transplant

  9. The Number of Participants With Non-progression Mortality

    deaths without relapse of disease

    Measured: Baseline to year 5

  10. The Number of Participants Who Survived

    Measured: Baseline to year 5

  11. The Number of Participants Who Had Regimen-related Toxicities

    Defined as adverse events (AEs) >= Grade 3 and assessed by the investigator as 1 of the following: unrelated, unlikely, or possibly related to treatment; probably related to treatment; definitely related to treatment.

    The number of participants that experienced Grade 3 or higher adverse events is reported.

    Measured: Baseline to 1 year post-transplant, grade 3 or higher adverse events

  12. The Median Time of Initiation of Disease-modifying Antirheumatic Drugs (DMARDS) for Relapse After Transplant

    The time in months from transplant to starting new therapy not MMF maintenance for relapse disease.

    Measured: Transplant to year 5

  13. Median Time to Treatment Failure

    Time to treatment failure is defined as death or initiation of disease modifying antirheumatic drug (DMARD).

    Measured: From transplant (day 0) to death or disease progression to year 5

  14. The Number of Participants With Treatment-related Mortality

    Defined by death occurring at any time after start of mobilization procedure to day +90 after autologous HCT and definitely or probably resulting from treatment given in the study.

    Measured: Baseline to Day 90

  15. The Number of Participants Who Completed ALL Work Productivity Survey (WPS)

    The first question assesses employment status, type of job for the employed (non-manual, manual or mixed manual/non-manual) and the status of those unemployed (homemaker, retired, student, unable to work due to SSc, unable to work due to non-SSc health problems, or other, i.e. volunteer). The next 3 questions apply only to employed patients and assess absenteeism (full days of work missed due to SSc), presenteeism (days with work productivity reduced by greater than or equal to 50%), and how much SSc interfered with work productivity on a scale of 0-10.

    Measured: Baseline to year 5

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted10 Aug 2011 (estimated)
  2. Study start15 Sep 2011
  3. Primary completion15 Sep 2023
  4. Study completion11 Sep 2024

Resources

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
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
21 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01413100
Sponsor's protocol ID
2533.00
Other IDs
NCI-2011-01190, 2533, 2533.00, P30CA015704, RG1711052
Acronym
STAT
Status last verified
Dec 2024
First submitted
8 Aug 2011
Last updated
7 Jan 2025
Data monitoring committee
Yes

Publications

  • Georges GE, Khanna D, Wener MH, Mei MG, Mayes MD, Simms RW, Sanchorawala V, Hosing C, Kafaja S, Pawarode A, Holmberg LA, Kolfenbach J, Furst DE, Sullivan KM, Huang S, Gooley T, Nash RA. Autologous Nonmyeloablative Hematopoietic Stem Cell Transplantation for Diffuse Cutaneous Systemic Sclerosis: Identifying Disease Risk Factors for Toxicity and Long-Term Outcomes in a Prospective, Single-Arm Trial. Arthritis Rheumatol. 2025 May;77(5):571-581. doi: 10.1002/art.43072. Epub 2025 Jan 14.

    PubMed 39624016

  • Zielonka J, Higuero Sevilla JP. Autologous hematopoietic stem cell transplant for systemic sclerosis associated interstitial lung disease. Curr Opin Rheumatol. 2024 Nov 1;36(6):410-419. doi: 10.1097/BOR.0000000000001050. Epub 2024 Sep 27.

    PubMed 39348419

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Anti-Thymocyte Globulin, Autologous Hematopoietic Stem Cell Transplantation and Cyclophosphamide in people with Systemic Scleroderma, run by Fred Hutchinson Cancer Center at 12 sites 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 39 inclusion and 37 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: finished, as of the sponsor's last update on 7 Jan 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 12 sites 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?

Yes. Results are posted in the registry (first posted 7 Jan 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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?

EFS of Patients Undergoing Transplant (time frame: At 5 years). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01413100, which also lists 2 linked publications.

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 7 Jan 2025.

AzTrials uses cookies for analytics and advertising. You can accept or reject them; the site works either way. Privacy Policy.

Free account

Keep searching with a free account

You’ve used your 3 free searches for this session. Log in or create a free AzTrials account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in