Safety Study of Gleevec® in Children With Pulmonary Hypertension

NCT00583115 | Terminated Phase 2 Results DMC

• 2 other identifiers: 0702-21Internally funded
• Study Type: interventional
• Target: 1
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to find out if the drug, Gleevec, is safe and effective in treating children with Pulmonary Hypertension.

Conditions

Pulmonary Hypertension

Outcome Measures

Primary Outcomes (1)

• 1) Safety and Tolerability as Determined by Laboratory Evaluation, Physical Examination, Echocardiographic Analysis, and Adverse Events, and 2) Efficacy as Determined by an Increase in the Non-encouraged 6 Minute Walk Test From Baseline.

  6 months

Secondary Outcomes (1)

• 1) Decrease in Pulmonary Artery Pressures and Vascular Resistance as Determined by Cardiac Catheterization, 2) Time to Clinical Worsening,3) Survival.

  6 months

Study Arms (1)

Gleevec

EXPERIMENTAL

Drug taken orally 260mg/M2/day once per day

Drug: Gleevec

Interventions

Gleevec DRUG

260 mg/M2/day, given once daily by mouth

Also known as: Imatinib Mesylate

Gleevec

Eligibility Criteria

• Age: 8 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Male and female patients between the ages of 8 -18 years of age.
• Diagnosis of idiopathic (or primary) pulmonary arterial hypertension according to the Venice Classification system (2003).54, 55
• Functional classification of WHO class III - IV.
• Pulmonary vascular resistance (PVR) \>300 dynes / sec / cm5.
• IPAH medications stable for at least 3 mo prior to baseline visit.
• Female patients of child bearing potential who are sexually active must have negative pregnancy test within 7 days prior to initiation of study drug and use a double-barrier local contraception, i.e., intra-uterine device plus condom, or spermicidal gel plus condom up to the Study Completion visit.
• Able to communicate well with the investigator, to understand and comply with the requirements of the study. Able to verbalize understanding and sign the written informed assent.
• Parents, or legal guardians, must be able to communicate well with the investigator, to understand and comply with the requirements of the study. They must verbalize understanding and sign the written informed consent statement.

You may not qualify if:

• Pre-existing lung disease including parasitic diseases affecting lungs, bronchial asthma, congenital abnormalities of the lungs, thorax and diaphragm.
• Congenital heart disease, left ventricular failure, or left-sided obstructive lesion (pulmonary venous hypertension with pulmonary capillary wedge pressure \> 12 mmHg) detected at right heart catheterization.
• Chronic thromboembolic pulmonary hypertension, congenital or acquired deficiencies of blood coagulation, deficient thrombocyte function, thrombocytopenia \< 40,000/μl, or Sickle Cell anemia.
• Pregnancy, breast feeding, or lack of safe contraception (hormonal contraception, IUD, bilateral tubal ligation, hysterectomy) in premenopausal women.
• Hepatic insufficiency with transaminase levels \>4-fold the upper limit of normal, or a bilirubin \> 2-fold the upper limit of normal.
• Renal insufficiency (serum creatinine \> 200 μmol/l).
• History of clinically significant drug allergy or history of atopic allergy (asthma, urticaria, eczematous dermatitis). A known hypersensitivity to the study drug, or drugs similar to the study drug.
• Previous therapeutic radiation of lungs or mediastinum.
• Participation in any treatment studies within 3 months prior to dosing, or longer if required by local regulations, and for any other limitation of participation based on local regulations.
• History of immunodeficiency diseases, including a positive HIV (ELISA and Western blot) test result, or a positive Hepatitis B surface antigen (HBsAg), or positive Hepatitis C test result.
• 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.

Sponsors & Collaborators

• Indiana University: lead

Study Sites (1)

Indiana University School of Medicine; Riley Hospital for Children

Indianapolis, Indiana, 46202, United States

Location

MeSH Terms

Conditions

Hypertension, Pulmonary

Interventions

Imatinib Mesylate

Condition Hierarchy (Ancestors)

Lung Diseases; Respiratory Tract Diseases; Hypertension; Vascular Diseases; Cardiovascular Diseases

Intervention Hierarchy (Ancestors)

Benzamides; Amides; Organic Chemicals; Benzoates; Acids, Carbocyclic; Carboxylic Acids; Benzene Derivatives; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons; Piperazines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Pyrimidines

Results Point of Contact

• Title: Ronald Mark Payne MD
• Organization: Indiana University

rpayne@iu.edu

317-278-6329

Study Officials

• R. Mark Payne, MD

  Indiana University School of Medicine

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 20, 2007
• First Posted: December 31, 2007
• Study Start: October 1, 2007
• Primary Completion: August 1, 2013
• Study Completion: December 1, 2013
• Last Updated: October 29, 2015
• Results First Posted: October 29, 2015

Record last verified: 2015-09

Locations

US (1)