Cardiac Safe Transplants for Systemic Sclerosis

Stopped early · Phase 2/Phase 3

Conditions studied: Systemic Sclerosis, Scleroderma

In brief

This study is designed to treat systemic sclerosis (scleroderma) patients with an autologous stem cell transplant using a regimen of immune suppressant drugs and chemotherapy that is less toxic to your heart.

Key facts

Study ID

NCT03593902

Run by

Northwestern University

People needed

9

Starts

2018-05-17

Expected to finish

2019-10-09

Last updated by the study team

2020-08-12

Who can join

Age: 18 and older, up to 65. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Age 18 - 65 years old at the time of pre-transplant evaluation
• An established diagnosis of systemic sclerosis
• Diffuse cutaneous systemic sclerosis with involvement proximal to the elbow or knee and a modified Rodnan Skin Score of ≥ 14 (see Appendix A)
• AND
• Any one of the following:
• DLCO < 80% of predicted or decrease in lung function (DLCO, DLCO/VA or FVC) of 10% or more over 12 months.
• Pulmonary fibrosis or alveolitis on CT scan or chest x-ray (ground glass appearance of alveolitis).
• Abnormal EKG (non-specific ST-T wave abnormalities, low QRS voltage, or ventricular hypertrophy), or pericardial effusion or pericardial enhancement without constriction on MRI
• Gastrointestinal tract involvement confirmed on radiological study. Radiologic findings of scleroderma are small bowel radiographs showing thickened folds with dilated loops, segmentation, and flocculation +/- diverticula, or pseudodiverticula. A hide-bound appearance may be present (e.g. dilated and crowded circular folds). GI involvement may also be confirmed by D-xylose malabsorption, patulous esophagus on high-resolution computed tomography (HRCT), or esophageal manometry.
• OR
• Limited cutaneous systemic sclerosis (SSc) (modified Rodnan Skin Score <14) with lung involvement defined as active alveolitis on bronchoalveolar lavage (BAL), ground-glass opacity on CT scan, a DLCO < 80% predicted, or decrease in lung function (DLCO/VA, DLCO, FVC) of 10% or more in last 12 months.
• Other Inclusion Criteria for "CAST" Conditioning Regimen (presence of any of the following):
• Septal flattening or D-sign on MRI (without deep breathing)
• PASP >40 mm Hg or >45 mm Hg with fluid challenge*
• mPAP >25 mm Hg or >30 mm Hg with fluid challenge*
• Non-ischemia diffuse ventricular hypokinesis or non-ischemia wall hypokinesis
• Fluid challenge is 1000 ml normal saline over 10 minutes. Fluid challenge will not be done if right atrial pressure is >13 mm Hg at rest or pulmonary capillary wedge pressure is >20 mm Hg at rest.

You may not qualify if…

• Active ischemic heart disease or untreated coronary artery disease
• Untreated life-threatening cardiac arrhythmia on EKG or 24-hour holter
• Pericardial effusion > 1 cm on cardiac MRI unless successful pericardiocentesis has been performed
• LVEF <35%
• End-stage lung disease characterized by TLC<45% of predicted value, or DLCO hemoglobin corrected < 30 % predicted.
• Creatinine clearance <40 by 24-hour urine
• History of breast implants that have not been removed (unless they cannot be surgically removed due to risks of surgery)
• Liver cirrhosis, transaminases >2x of normal limits, or bilirubin > 2.0 unless due to Gilbert's disease
• Uncontrolled diabetes mellitus or any other illness that in the opinion of the investigators would jeopardize the ability of the patient to tolerate aggressive treatment
• Prior history of malignancy
• Positive pregnancy test, inability or unable to pursue effective means of birth control, or failure to willingly accept or comprehend irreversible sterility as a side effect of therapy
• Psychiatric illness or mental deficiency making compliance with treatment or informed consent impossible
• Major hematological abnormalities such as platelet count < 100,000/ul or absolute neutrophil count (ANC) < 1000/ul
• HIV positive
• Hepatitis B or C positive
• PASP >50 mmHg without fluid challenge
• mPAP >34 mmHg without fluid challenge
• Coronary artery disease not reversed by cardiology and interventional radiology

Where it is running

• Northwestern University, Feinberg School of Medicine — Chicago, Illinois, United States
• Northwestern University — Chicago, Illinois, United States

Full record on ClinicalTrials.gov

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