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Penn Transplant Institute

Heart Transplant

Human heart tissue protocol

Clinical Trials in Organ Transplantation (CTOT) Studies

Prevention of Cardiac Allograft Vasculopathy Using Rituximab (Rituxan) Therapy in Cardiac Transplantation

  • Investigator: Lee Goldberg, MD
  • Sponsored by: National Institutes of Health (NIH)

A Prospective, Randomized, Multicenter, Two-Parallel Arm Study Evaluating the Overall Efficacy and Safety of Desensitization Therapy on Selected Patients Awaiting Heart Transplantation

  • Investigator: Lee Goldberg, MD
  • Sponsored by: National Institutes of Health (NIH)

Kidney Transplant

Clinical trials in organ transplantation (CTOT)

Eculizumab study (Eculizumab for Prevention of Delayed Graft Function in Deceased Donor Kidney Transplantation)

  • Name of PI and Coordinator: Roy Bloom, M.D., Robin Neubauer, R.N., Jennifer Trofe-Clark, PharmD
  • DGF is both an outcome following kidney transplantation and a predictor for long term graft function. In an era of a tremendous shortage of kidneys for transplantation, every effort should be made to improve the survival of the transplanted kidneys in the recipient. Therefore, it is imperative that we implement strategies to reduce the incidence of DGF in an effort to improve long-term graft survival. There are currently no accepted or FDA approved therapies for prevention or treatment of DGF following transplantation. The Sponsor Investigator of this proposed trial previously performed a pilot study for safety and preliminary efficacy on 8 recipients of first deceased donor kidney transplants at high risk for DGF, half of whom received eculizumab IV in the OR prior to reperfusion and the other half received saline as a control. All patients were treated with rabbit anti-thymocyte globulin induction. While 2 of 4 patients in the control group required dialysis for post-transplant DGF, none of the 4 given eculizumab required dialysis. Urine outputs trended to be greater and serum creatinine on Day 30 post-transplant trended lower in the patients given Eculizumab. There was no difference in adverse event rates between the 2 groups. Based on these promising results suggesting efficacy without toxicity we designed this expanded follow up study.
  • Contact: Robin Neubauer, R.N. 215-615-0773

RESTARRT study (Immunosuppression with Anti-thymocyte Globulin, Rituximab, Tacrolimus, Mycophenolate Mofetil and Sirolimus, Followed by Immunosuppression Withdrawal in Living-donor Renal Transplant Recipients)

Circulating donor-derived cell-free DNA in blood for diagnosing acute rejection in kidney transplant recipients (DART), Protocol SN-C-00006

  • Name of PI and Coordinator: Roy Bloom, M.D., Robin Neubauer, R.N., Jennifer Trofe-Clark, PharmD
  • The purpose of the study is to observe the measurement of DNA in your blood during your routine post-transplant follow-up visits for use as a marker for kidney tissue injury. Measuring the release of DNA from the transplanted kidney in the blood has been proposed to provide information on tissue injury in the kidney due to rejection.
  • Contact: Robin Neubauer, R.N. 215-615-0773

Liver Transplant

Effects of Rapamycin on Gene Expression and Biomarkers Associated with Aging

Researchers at the University of Pennsylvania would like to learn about the effect of Sirolimus (or Rapamune) medication on aging. Recent studies have shown that Rapamycin extends lifespan and prevents aging-related diseases in worms, flies, and mice. Since certain liver transplant patients are on Sirolimus medication while other transplant patients are on Tacrolimus (or Prograf) medication we would like to investigate the differences between these patients. A group of healthy volunteers will serve as a control for this study. Researchers will be analyzing blood samples for differences in gene expression. In addition, the study includes computerized cognitive assessments as well as photographs.

Research Coordinator: Mary Shaw, RN, BBA
Tel: 215-614-0528

Penn Transplant Biobank and Registry (BioTip)

The purpose of the study is to see if substances in the donor organ can predict how well the organ will function after transplant. Tests on blood, urine and biopsy tissue samples will be used in this study to see if such a link exists. Patients who are expected to receive other organs (kidney, lung and heart) will also be part of this study. The research team will be looking at any injuries in the transplanted organs to see if there are similarities among the different organ groups. In this way, they hope to develop a better understanding of organ injury after transplant. Study participation lasts for the lifetime of the participant.

Research Coordinator: Mary Shaw, RN, BBA
Tel: 215-614-0528

Predictors of Renal Dysfunction Following Liver Transplantation

10 to 20 percent of liver transplant recipients develop kidney problems after their liver transplant. This is a research study for individuals who are going to receive either a liver transplant or a simultaneous liver and kidney transplant. The purpose of this study is to help physicians understand which liver transplant recipients are likely to develop kidney problems after transplant. Tests of urine, blood, and tissue biopsy from the kidney will be used to determine who is at risk for kidney dysfunction after the liver transplant.

Research Coordinator: Mary Shaw, RN, BBA
Tel: 215-614-0528

A Phase II Randomized Multicenter Placebo-Controlled Blinded Study of Sorafenib Adjuvant Therapy in High Risk Orthotopic Liver Transplant (OLT) Recipients with Hepatocellular Carcinoma (HCC)

Hepatocellular carcinoma (HCC) (liver cancer) is a significant health problem, accounting for more than 1,000,000 new cases every year world-wide and is the third leading cause of cancer deaths in the world. The incidence of liver cancer is also increasing in the United States and causes about 13,000 deaths yearly. Outcomes for patients with HCC have been historically poor, regardless of treatment. Liver transplantation is a treatment option for liver cancer patients, but despite transplantation, the HCC (liver cancer) can recur in the new transplanted liver. Nexavar® (sorafenib) is approved by the Food and Drug Administration (FDA) for treatment of patients with advanced kidney cancer and advanced unresectable (untreatable with surgery) liver cancer (HCC). It is not known whether sorafenib, the study medication, is effective in preventing cancer recurrence in high risk patients following liver transplantation. The purpose of this study is to determine if sorafenib is a safe and effective treatment option for preventing liver cancer in high risk patients following liver transplantation.

Research Coordinator: Mary Shaw, RN, BBA
Tel: 215-614-0528

Models for Optimal Liver Transplant Outcomes Study

At the time of liver transplantation, some patients have normal kidney function and some patients have poor kidney function. In some patients, the kidney function may improve with time but at this time it is unknown which patients will have improved function and which patients will not. The purpose of this study is to assist physicians understand which liver transplant recipients are likely to develop kidney problems after transplant. Patients who are listed for a liver transplant are eligible to participate in this study. Tests of urine, blood and tissue biopsy from the kidney will be used to determine who is at risk for kidney dysfunction after the liver transplant.

Research Coordinator: Mary Shaw, RN, BBA
Tel: 215-614-0528

A 36 Month, Multi-Center, Open-Label, Randomized, Comparator Study To Evaluate The Efficacy and Safety of Everolimus Immunosuppression Treatment in Liver Transplantation for Hepatocellular Carcinoma Exceeding Milan Criteria

  • Investigator: Maarouf Hoteit, MD

Liver transplantation is an effective therapy approach in patients with hepatocellular carcinoma (HCC) and cirrhosis. Using the Milan tumor inclusion criteria, both patient and graft survival of patients with HCC are similar to those of patients without HCC. The same has been observed with more expanded tumor inclusion criteria. However, while imaging has improved, some of the patients have more tumor burden in the liver transplant than seen on imaging. Tumor size and number has correlated with worse outcome. The same applies to tumor differentiation and vascular invasion; both pathological parameters cannot be reliably predicted based on pre-transplant testing. While post-transplant tumor recurrence has decreased over the years, it is still of concern, as it typically leads to patient demise. Therefore, all attempts should be made to decrease the likelihood of tumor recurrence. This study is to evaluate use of everolimus and tacrolimus in a prospective manner to see if the use of these two medications results in lower tumor recurrence and better patient survival than tacrolimus and mycophenolic acid/mycophenolate mofetil after liver transplantation for HCC.

Research Coordinator: Mary Shaw, RN, BBA
Tel: 215-614-0528

Lung Transplant

Clinical Risk Factors of Primary Graft Failure, a Multicenter Study

Genetics of Primary Graft Failure, a Multicenter Study

Clinical Trials in Organ Transplantation

The association of anti-Col (V) antibodies with development of Primary Graft Dysfunction following lung transplantation

Regulatory T Cells and Primary Graft Failure

Innate Immunity and lung transplantation

Prospective Registry of Outcomes in Patients Electing Lung Transplantation (PROPEL): A correlative analysis using gene signature array

NOVEL Lung Trial: Normothermic Ex Vivo Lung Perfusion (EVLP) as an Assessment of Extended/Marginal Donor Lungs

A Multi-center, Multinational, Retrospective Review of Voriconazole Experience for the Prophylaxis against Invasive Aspergillosis in Lung Transplant Recipients

Advanced Lung Disease Research Studies


A Phase 3, Double-Blind, Multicenter, Randomized, Placebo-Controlled Trial Evaluating Repeated Courses of Aztreonam for Inhalation Solution in Subjects with non-CF Bronchiectasis and Gram-Negative Endobronchial Infection (AIR-BX1), Gilead

A Consolidated Database of Non-Cystic Fibrosis Bronchiectasis Patients from Major Clinical and Research Institutions (NON-TREATMENT)

Nontuberculous Mycobacterial Lung Disease (NTM): A randomized, double-blind, placebo-controlled study of Liposomal Amikacin for inhalation (Arikayce) in patients with recalcitrant nontuberculous mycobacterial lung disease

Genomic Research in Alpha 1 Anti-trypsin disease and Sarcoidosis


A prospective, longitudinal, non-pharmacologic, case-controlled study to evaluate longitudinal disease behavior and biomarker data over a 52 week period in idiopathic pulmonary fibrosis patients

A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study of the Efficacy and Safety of Pirfenidone in Patients with Idiopathic Pulmonary Fibrosis, InterMune (TREATMENT)

Pulmonary Hypertension

GRIPHON: Prostacyclin (PGI2) Receptor agonist In Pulmonary arterial Hypertension (TREATMENT): A multicenter, double-blind, placebo-controlled Phase 3 study to demonstrate the efficacy and safety of ACT-293987 in patients with pulmonary arterial hypertension

AIPH: Anastrozole in Patients with Pulmonary Arterial Hypertension (TREATMENT)

Cystic Fibrosis

A randomized, multi-center, open-label, cross over, phase IV study in cystic fibrosis (CF) patients to compare the tolerability, safety and efficacy of tobramycin inhalation powder (TIP) with tobramycin nebulizer solution (TOBI) as assessed by the switch-over of treatments

A prospective, 5-year Registry Study to Monitor the Susceptibility to Aztreonam of Pseudomonas aeruginosa (PA) Isolates from Patients with Cystic Fibrosis in the United States

Cystic Fibrosis Foundation Patient Registry

Continuous Alternating Therapy (CAT) Regimen of Inhaled Antibiotics for the Treatment of Chronic Pulmonary Pseudomonas aeruginosa infection

Cell Mediated Immunity Against the Cystic Fibrosis Transmembrane Conductance Regulator Protein in Patients with Cystic Fibrosis (3 blood draws)

Modulation of inflammation and oxidation biomarkers in cystic fibrosis by a botanical phenolic

  • Investigator: Jason Turowski, MD

Improving the Effectiveness of Pulmozyme at sites of infection by Combination with Multivalent Anions and Acting Depolymerizing Factors

  • Investigator: Paul Jamney, PhD

Pancreas Transplant

Islet transplantation in type 1 diabetes using immunosuppression and strategies to improve long term graft survival

Using anti-B lymphocyte agent rituximab (Rituxan) to improve long-term graft survival

Islet transplantation vs. intensive insulin therapy in patients with type 1 diabetes who have already received a kidney transplant

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