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Clinical Trials at UMass

 

Eye Diseases

Neovascular Age-related Macular degeneration 

A Phase 1, Open Label, Multi-Center, Dose Escalating, Safety and Tolerability Study of a Single Intravitreal Injection of AAV2-sFLT01 in Patients with Neovascular Age-Related Macular Degeneration  PI Terry Flotte MD | Registration:  NCT01024998

This study is now in Long Term Follow Up. The purpose of this research study was to examine the safety and ability of an experimental study drug rAAV2-sFLT01. The study drug used a virus to transfer a gene into the cells of the eye to produce the sFLT01 protein. sFLT01 stops VEGF another protein in the body from stimulating growth of abnormal blood vessels under the retina which cause leakage of blood and fluids leading to damage of cells in the retina and macula and thus loss of vision.

Leber's Congenital Amaurosis 2 (LCA-2 or RPE65 deficiency)

A Multiple-Site, Phase I & II Safety and Efficacy Trial of a Recombinant Adeno-associated Virus Vector Expressing RPE65 (rAAV2-CB-hRPE65) in Patients with Leber Congenital Amaurosis Type 2  PI: Terry Flotte MD  Sponsor: AGTC | Registration: NCT00749957

This study is now in Long-Term Follow up. The purpose of this research study was to examine whether a study drug, called rAAV-CB-hRPE65, is safe and can improve vision. A normal copy of the RPE65 gene which makes the RPE65 protein that is needed to make the retina work was transferred by injecting rAAV2-CB-hRPE65 in affected patients.


Pulmonary Diseases

Alpha-1 Antitrypsin Deficiency

Phase 2, Safety and Efficacy Trial of a Recombinant Adeno-associated Virus Vector Expressing Alpha 1 Antitrypsin (rAAV1-CB-hAAT) in Patients with Alpha 1 Antitrypsin Deficiency  PI: Terry Flotte MD  Sponsor: AGTC/NHLBI | Registration:  NCT01054339

This research study is in long term follow up. This study tested whether a study drug, called rAAV1-CB-hAAT, is safe and was designed to test what dose vector is needed to increase the amount of alpha-1 protein in the blood in affected individulas. The study drug contains a normal copy of the AAT gene and that is delivered via a rAAV1 through injections into the muscles. 


Urology Diseases

Prostate Cancer

A Randomized Controlled Trial of Prostatak™ As Adjuvant To Up-Front Radiation Therapy For Localized Prostate Cancer  PI Mitchell Sokoloff MD  Sponsor: Advantagene | Registration:  NCT01436968

Gene Vaccine Clinical Trial for Men with Newly Diagnosed Prostate Cancer Who Are Candidates for Radiation Therapy (pdf)

This is a multicenter, randomized, placebo-controlled study testing a research product called ProstAtak™. It is being given to patients newly diagnosed with prostate cancer undergoing radiation treatment. ProstAtak™ consists of a virus carrying a gene being injected into the prostate gland along with being given valacyclovir pills (normally given to treat herpes infection). This is being done to determine if when injected into the prostate it kills prostate cancer within the prostate and stimulates an immune response throughout the body to kill prostate cancer in the event that it spreads. Approximately two thirds of the patients will receive ProstAtak™ and the other one third of the patients will receive a placebo injection (a liquid similar to salt water) and the valacyclovir pills. The decision as to whether a patient receives drug or placebo will be made by chance. The study patients, medical provider and the sponsor will not know whether the patient is receiving the drug or placebo.

Gene Vaccine Clinical Trial for Men with Newly Diagnosed Prostate Cancer Who Are Candidates for Active Surveillance (pdf)

Next steps?

For more information on Clinical Trials at the University of Massachusetts Medical School and to learn more about our ongoing efforts to translate research bench work into clinical therapies visit our new Center for Clinical and Translational Research site (CCTS).

To find new and ongoing other clinical studies at the University of Massachusetts and to learn how to volunteer for a clinical tiral visit the Conquering Diseases Programs (CDP).

 

CCTS's website  CDP's website