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| Sponsored by: |
Children's Memorial Hospital |
|---|---|
| Information provided by: | Children's Memorial Hospital |
| ClinicalTrials.gov Identifier: | NCT00350844 |
Purpose
The goal of this study is to test the hypothesis that hydroxyurea is effective for the specific treatment of secondary pulmonary hypertension found on screening in children and young adults with sickle cell disease.
| Condition | Intervention | Phase |
|---|---|---|
|
Sickle Cell Disease Pulmonary Hypertension |
Drug: Hydroxyurea |
Phase I Phase II |
| Study Type: | Interventional |
| Study Design: | Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study |
| Official Title: | A Pilot Study of Hydroxyurea for the Treatment of Pulmonary Hypertension in Children and Young Adults With Sickle Cell Disease |
| Estimated Enrollment: | 18 |
| Study Start Date: | July 2006 |
| Estimated Study Completion Date: | June 2008 |
Increasing evidence suggests that pulmonary hypertension, defined by an elevated tricuspid regurgitant jet velocity (TRJV) on echocardiogram, is a major cause of morbidity and mortality in adults with sickle cell disease (SCD). However, both the prevalence and optimal treatment of pulmonary hypertension in children and young adults with SCD are unknown.
We hypothesize that short term therapy with hydroxyurea will decrease TRJV in children and young adults with pulmonary hypertension found on screening. Patients eligible for treatment will have had evidence of pulmonary hypertension on at least 2 screening echocardiograms. Baseline laboratory tests will be obtained and other causes of secondary pulmonary hypertension will be excluded prior to initiation of treatment. Patients will be treated with hydroxyurea according to a standard dose escalation schedule for a total of 12 months. A clinic visit will be required every 2 months and standard screening for toxicity will be performed monthly. There will be an interim analysis of the primary outcome at 6 months following therapy.
Eligibility| Ages Eligible for Study: | 10 Years to 25 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
Contacts and Locations| Contact: Robert I Liem, MD | 773-880-3977 | rliem@childrensmemorial.org |
| Contact: Diane M Calamaras, RN, CPNP | 773-880-8953 | dcalamaras@childrensmemorial.org |
| United States, Illinois | |
| Children's Memorial Hospital | Recruiting |
| Chicago, Illinois, United States, 60614-3394 | |
| Contact: Robert I Liem, MD 773-880-3977 rliem@childrensmemorial.org | |
| Principal Investigator: Robert I Liem, MD | |
| United States, Maryland | |
| Johns Hopkins University | Recruiting |
| Baltimore, Maryland, United States, 21205 | |
| Contact: John J. Strouse, MD | |
| Principal Investigator: John J. Strouse, MD | |
| Sub-Investigator: James Casella, MD | |
| United States, Missouri | |
| St. Louis Children's Hospital and Washington University | Recruiting |
| St. Louis, Missouri, United States, 63110 | |
| Contact: Michael R. DeBaun, MD, MPH DeBaun_M@kids.wustl.edu | |
| Principal Investigator: Michael R. DeBaun, MD, MPH | |
| Sub-Investigator: Joshua Field, MD | |
| United States, Wisconsin | |
| Children's Hospital Wisconsin and Medical College of Wisconsin | Recruiting |
| Milwaukee, Wisconsin, United States, 53236 | |
| Contact: Julie Panepinto, MD, MPH jpanepin@mcw.edu | |
| Principal Investigator: Julie Panepinto, MD, MPH | |
| Principal Investigator: | Robert I Liem, MD | Children's Memorial Hospital |
More Information
| Study ID Numbers: | 12735 |
| Study First Received: | July 10, 2006 |
| Last Updated: | December 5, 2007 |
| ClinicalTrials.gov Identifier: | NCT00350844 |
| Health Authority: | United States: Food and Drug Administration |
|
Sickle Cell Disease Pulmonary Hypertension |
|
Hydroxyurea Hematologic Diseases Anemia Vascular Diseases Anemia, Hemolytic Sickle cell anemia Anemia, Hemolytic, Congenital Genetic Diseases, Inborn |
Respiratory Tract Diseases Hypertension, Pulmonary Hemoglobinopathies Lung Diseases Hemoglobinopathy Anemia, Sickle Cell Hypertension |
|
Antisickling Agents Molecular Mechanisms of Pharmacological Action Antineoplastic Agents Therapeutic Uses Hematologic Agents |
Enzyme Inhibitors Cardiovascular Diseases Nucleic Acid Synthesis Inhibitors Pharmacologic Actions |
