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Sponsored by: |
Indiana University |
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Information provided by: | Indiana University |
ClinicalTrials.gov Identifier: | NCT00583115 |
The purpose of this study is to find out if the drug, Gleevec, is safe and effective in treating children with Pulmonary Hypertension.
Condition | Intervention | Phase |
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Pulmonary Hypertension |
Drug: Imatinib Mesylate |
Phase II |
Study Type: | Interventional |
Study Design: | Treatment, Open Label, Active Control, Single Group Assignment, Safety/Efficacy Study |
Official Title: | A Phase II Study of Gleevec® (Imatinib Mesylate, NSC 716051 Formerly ST1571) in Children With Pulmonary Hypertension |
Estimated Enrollment: | 5 |
Study Start Date: | October 2007 |
Estimated Study Completion Date: | December 2009 |
Estimated Primary Completion Date: | December 2009 (Final data collection date for primary outcome measure) |
Arms | Assigned Interventions |
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Single Group: Experimental
Children with PAH
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Drug: Imatinib Mesylate
260 mg/M2/day, given once daily by mouth
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Idiopathic pulmonary artery hypertension (IPAH) is a rare but progressive disease in children and adults with a very high mortality from right heart failure. Platelet derived growth factor (PDGF) has been implicated in the pulmonary artery remodeling and vascular proliferation that is a pathologic hallmark of IPAH. Animal and clinical data support the hypothesis that activation of the PDGF receptor is critical in the development of IPAH, and inhibition of the PDGF signaling pathways may be a potential therapeutic target. Gleevec is a tyrosine kinase inhibitor developed for treatment of certain cancers and inhibits the PDGF receptor. Animal and preliminary clinical data suggest that Gleevec can reverse vascular remodeling and improve right heart failure. A small clinical trial for adults with IPAH is ongoing in Europe, but there are no trials in children where the disease has a worse prognosis. This project is a phase II, single dose pilot study to generate the hypothesis that activation of the PDGF receptor is critical in the development of IPAH, and inhibition of the PDGF signaling pathways is a potential therapeutic target. Five patients between the ages of 8 yr to 18 yr with severe IPAH will be recruited from 2 sites, Indianapolis and New York, for a 1 yr trial of Imatinib. The Specific Aim of this study is to collect preliminary data on the safety and efficacy of Gleevec in children with IPAH. Results from this study are needed to develop a larger, multi-center trial to determine the efficacy and therapeutic impact of Gleevec in children with IPAH. The long term goal of this work is to develop novel therapeutic strategies for treatment of IPAH in children. This translational study of the inhibition of the PDGF receptor in children with IPAH could provide insights into the etiology of IPAH and have a major impact on the development of new treatment strategies for both children and adults with pulmonary artery hypertension from multiple origins.
Ages Eligible for Study: | 8 Years to 18 Years |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
Any surgical or medical condition which might significantly alter the absorption, distribution, metabolism or excretion of drugs, such as a history of inflammatory bowel syndrome, gastritis, ulcers, gastrointestinal or rectal bleeding, or a history of major gastrointestinal tract surgery such as gastrectomy, gastroenterostomy, or bowel resection.
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Contact: R. Mark Payne, MD | 317-274-1526 | rpayne@iupui.edu |
United States, Indiana | |
Indiana University School of Medicine; Riley Hospital for Children | Recruiting |
Indianapolis, Indiana, United States, 46202 | |
Contact: R.Mark Payne, MD 317-274-1526 rpayne@iupui.edu | |
Contact: Lucy C Miller, BSN 317-278-7809 lucmille@iupui.edu | |
Principal Investigator: R. Mark Payne, MD |
Principal Investigator: | R. Mark Payne, MD | Indiana University School of Medicine |
Responsible Party: | Indiana University School of Medicine ( Ronald Mark Payne, MD, FAAP, FACC ) |
Study ID Numbers: | IUPedPDT-01, IUPedPDT-01-PAYN |
Study First Received: | December 20, 2007 |
Last Updated: | December 28, 2007 |
ClinicalTrials.gov Identifier: | NCT00583115 |
Health Authority: | United States: Food and Drug Administration |
Imatinib Respiratory Tract Diseases Hypertension, Pulmonary |
Lung Diseases Vascular Diseases Hypertension |
Molecular Mechanisms of Pharmacological Action Antineoplastic Agents Therapeutic Uses Enzyme Inhibitors |
Cardiovascular Diseases Protein Kinase Inhibitors Pharmacologic Actions |