Pulmonary HypertensionPhase 2NCT00583115
Safety Study of Gleevec® in Children With Pulmonary Hypertension
A Phase 2 study testing Gleevec in people with Pulmonary Hypertension, run by Indiana University at 1 site in United States.
Trial summary
The purpose of this study is to find out if the drug, Gleevec, is safe and effective in treating children with Pulmonary Hypertension. Read more about this study
- Who is it for?
- Ages 8 to 18 years
- Conditions the study is for
- Pulmonary Hypertension
- What is being tested?
- Gleevec
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 1 participants
- Where
- 1 siteUnited States
- Study dates
- Oct 2007to Dec 2013
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 8 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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.
Who cannot join
- 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).
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- 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.
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Trial locations and contacts
1 site in 1 country.
United States1 site
Indiana University School of Medicine; Riley Hospital for Children
Indianapolis, Indiana, 46202
About this study
Official title: A Phase II Study of Gleevec® (Imatinib Mesylate, NSC 716051 Formerly ST1571) in Children With Pulmonary Hypertension
The purpose of this study is to find out if the drug, Gleevec, is safe and effective in treating children with Pulmonary Hypertension.
Read the detailed description
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 nationally, for a 6 month 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.
- Conditions
- Pulmonary Hypertension
- Being tested
- Gleevec
- Medical subject headings
- Hypertension, Pulmonary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GleevecDrug
Also called Imatinib Mesylate
260 mg/M2/day, given once daily by mouth
| Study group | Role | What they receive |
|---|---|---|
| Gleevec | Experimental | Drug taken orally 260mg/M2/day once per day Receives: Drug: Gleevec |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Dec 2007 (estimated)
- Study startOct 2007
- Primary completionAug 2013
- Study completionDec 2013
Why it stopped: Unable to enroll subjects
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Pulmonary HypertensionStudies of the same condition, open and under way.
- More trials run by Indiana UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 1 participants (actual)
Sponsor and investigators
- Lead sponsor
- Indiana University
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- R. Mark Payne, MD, Principal investigator, Indiana University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00583115
- Sponsor's protocol ID
- 0702-21
- Other IDs
- Internally funded
- Status last verified
- Sep 2015
- First submitted
- 20 Dec 2007
- Last updated
- 29 Oct 2015
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 29 Oct 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Study of Gleevec® (Imatinib Mesylate, NSC 716051 Formerly ST1571) in Children With Pulmonary Hypertension
- ClinicalTrials.gov ID
- NCT00583115
- Acronym / sponsor ID
- 0702-21
- Conditions
- Pulmonary Hypertension
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Unable to enroll subjects
- Enrolment
- 1 participants (actual)
- Lead sponsor
- Indiana UniversityUniversity, hospital or other
- Study start
- Oct 2007
- Primary completion
- Aug 2013 (actual)
- Study completion
- Dec 2013 (actual)
- First posted
- 31 Dec 2007 (estimated)
- Results
- Posted 29 Oct 2015
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to find out if the drug, Gleevec, is safe and effective in treating children with Pulmonary Hypertension.
Detailed description
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 nationally, for a 6 month 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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Gleevec | Experimental | Drug: GleevecDrug taken orally 260mg/M2/day once per day |
Who may qualify
- Ages
- 8 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (8 listed)
- 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.
Key exclusion criteria (12 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| GleevecAlso: Imatinib Mesylate260 mg/M2/day, given once daily by mouth | Drug | Gleevec |
Primary outcome
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.
Time frame: 6 months
1 secondary outcome
1) Decrease in Pulmonary Artery Pressures and Vascular Resistance as Determined by Cardiac Catheterization, 2) Time to Clinical Worsening,3) Survival.
Time frame: 6 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Oct 2015.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
R. Mark Payne, MD principal investigator
Indiana University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Gleevec in people with Pulmonary Hypertension, run by Indiana University at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 29 Oct 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 29 Oct 2015). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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. (time frame: 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Oct 2015.