Anemia, Sickle CellNCT00448370
Role of Placenta Growth Factor in Sickle Acute Chest Syndrome
An observational study of Anemia, Sickle Cell, run by Children's Hospital Medical Center, Cincinnati at 1 site in United States.
Trial summary
The purpose of this research study is to find out whether Placenta Growth Factor (PlGF) and related tests can predict the development of acute chest syndrome (ACS) in patients with sickle cell disease (SCD) during a period where patients are well and during admission to the hospital for an acute sickle event to see if… Read more about this study
- Who is it for?
- Ages 66 months to 30 years
- Conditions the study is for
- Anemia, Sickle Cell
- How many people
- 136 participants
- Where
- 1 siteUnited States
- Study dates
- Mar 2007to Dec 2012
- Phase
- Observational
- Are there results?
- None posted in the registry
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 66 months to 30 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: Participants will have sickle cell disease (HbSS, HbSC, or Hb Sbeta-thalassemia) and will be equal to or greater than 5.5 years old.
Who may be able to join
- Patients must have a diagnosis of SCD (HbSS, HbSC or HbS beta/thal) by hemoglobin electrophoresis or gene mapping. Very few patients with HbSC or HbS beta/thal would go onto develop ACS, but they will serve as important controls.
- Patients must be ≥5.5 years of age and ≤30 years of age.
- Patients must be able to comply with pulmonary function tests.
Who cannot join
- Patients taking anti-leukotriene medications such as montelukast (Singulair®) or zileuton (Zyflo®) 30 days prior to enrollment will be ineligible, although patients with SCD and asthma who are not on leukotriene inhibitors will be eligible.
- Patients with known congenital heart disease or with congenital lung abnormality/disease that will affect the pulmonary function testing and methacholine challenge testing will be ineligible (examples include patients with cyanotic heart disease, immotile cilia syndrome, cystic fibrosis, diaphragmatic hernia).
- Any patient with neurologic abnormalities, stroke, developmental delay, or other medical condition that would preclude cooperation with pulmonary function testing or compliance with protocol procedures will be ineligible.
- Patients over 30 years of age will be ineligible.
- Patients who are pregnant or nursing.
- Patients on chronic transfusions will be ineligible.
- If patients come off transfusion therapy, they will be eligible 3 months following the last transfusion.
- Patients receiving simple transfusions will be eligible, but baseline measurements will be made 3 months following the simple transfusion.
Trial locations and contacts
1 site in 1 country.
United States1 site
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
About this study
The purpose of this research study is to find out whether Placenta Growth Factor (PlGF) and related tests can predict the development of acute chest syndrome (ACS) in patients with sickle cell disease (SCD) during a period where patients are well and during admission to the hospital for an acute sickle event to see if these measures can predict the development of ACS. Understanding events precipitating ACS may lead to preventative and interventional therapies which will improve patient outcomes and quality of life.
Read the detailed description
The proposed research is a clinical study designed to test the hypothesis that PlGF levels in blood at baseline in patients with SCD will correlate with leukotriene (LT) levels and will be reflective of the degree of airway obstruction; and that acute elevations in PlGF levels will occur during an acute sickle event and precede clinical and radiological recognition of ACS. This is a biological study that does not fall into the criteria of a phase I-IV trial.
Measurements will be done at two stages. First, patients who are admitted to the hospital with an acute sickle event will have a daily evaluation for the first 4 days of the admission or up until the patient has an ACS event. Measurements of inpatient spirometry (at primary site only) and impulse oscillometry will be performed daily. Measurements on 35 ACS events with ACS developing on the 3rd/4th day of admission will be collected. Measurements on patients developing ACS will be compared to those who do not develop ACS will allow for earlier prediction of ACS. Finally, patients who have an ACS or admission for an acute sickling event will be evaluated again after 3-4 weeks in order to get a baseline measurement. One hundred baseline measurements will be made. Twenty of these baseline patients will also have one or two additional baseline evaluations at subsequent clinic visits to evaluate the overall non-event baseline distribution and intra-subject baseline variability. Patients will have measurements repeated if admitted for an acute event greater than one year since the last baseline measurement . These measurements will be used to see if there is any prognostic information for a subsequent acute or ACS event. Some patients may only have baseline data collected.
- Conditions
- Anemia, Sickle Cell
- Keywords
- Anemia, sickle cell
- Medical subject headings
- Anemia, Sickle Cell
What is being measured
The primary outcome is the main question the study is designed to answer.
Changes in plasma PlGF levels and urine LT levels in SCD patients
Measured: Baseline and serially through the development of an Acute Sickle Event
Secondary outcomes (1)
Relationship of clinical parameters of severity of SCD and circulating levels of PlGF, leukotrienes, proinflammatory cytochemokines and pulmonary function tests
Measured: Baseline and serially through the development of an Acute Sickle Event
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Mar 2007 (estimated)
- Study startMar 2007
- Primary completionDec 2012
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Anemia, Sickle CellStudies of the same condition, open and under way.
- More trials run by Children's Hospital Medical Center, CincinnatiThe 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 136 participants (actual)
Sponsor and investigators
- Lead sponsor
- Children's Hospital Medical Center, Cincinnati
- Sponsor type
- University, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI)
- Responsible party
- Sponsor
- Investigators
- Karen Kalinyak, MD, Principal investigator, Children's Hospital Medical Center, CincinnatiPunam Malik, MD, Study chair, Children's Hospital Medical Center, Cincinnati
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00448370
- Sponsor's protocol ID
- CCHMC 06-09-57
- Other IDs
- R01
- Status last verified
- Dec 2017
- First submitted
- 14 Mar 2007
- Last updated
- 21 Dec 2017
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Dec 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Role of Placenta Growth Factor in Sickle Acute Chest Syndrome
- ClinicalTrials.gov ID
- NCT00448370
- Acronym / sponsor ID
- CCHMC 06-09-57
- Conditions
- Anemia, Sickle Cell
- Keywords
- Anemia, sickle cell
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 136 participants (actual)
- Lead sponsor
- Children's Hospital Medical Center, CincinnatiUniversity, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI)
- Study start
- Mar 2007
- Primary completion
- Dec 2012 (actual)
- First posted
- 16 Mar 2007 (estimated)
- Results
- Not posted in the source record
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 research study is to find out whether Placenta Growth Factor (PlGF) and related tests can predict the development of acute chest syndrome (ACS) in patients with sickle cell disease (SCD) during a period where patients are well and during admission to the hospital for an acute sickle event to see if these measures can predict the development of ACS. Understanding events precipitating ACS may lead to preventative and interventional therapies which will improve patient outcomes and quality of life.
Detailed description
The proposed research is a clinical study designed to test the hypothesis that PlGF levels in blood at baseline in patients with SCD will correlate with leukotriene (LT) levels and will be reflective of the degree of airway obstruction; and that acute elevations in PlGF levels will occur during an acute sickle event and precede clinical and radiological recognition of ACS. This is a biological study that does not fall into the criteria of a phase I-IV trial.
Measurements will be done at two stages. First, patients who are admitted to the hospital with an acute sickle event will have a daily evaluation for the first 4 days of the admission or up until the patient has an ACS event. Measurements of inpatient spirometry (at primary site only) and impulse oscillometry will be performed daily. Measurements on 35 ACS events with ACS developing on the 3rd/4th day of admission will be collected. Measurements on patients developing ACS will be compared to those who do not develop ACS will allow for earlier prediction of ACS. Finally, patients who have an ACS or admission for an acute sickling event will be evaluated again after 3-4 weeks in order to get a baseline measurement. One hundred baseline measurements will be made. Twenty of these baseline patients will also have one or two additional baseline evaluations at subsequent clinic visits to evaluate the overall non-event baseline distribution and intra-subject baseline variability. Patients will have measurements repeated if admitted for an acute event greater than one year since the last baseline measurement . These measurements will be used to see if there is any prognostic information for a subsequent acute or ACS event. Some patients may only have baseline data collected.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 66 months to 30 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Participants will have sickle cell disease (HbSS, HbSC, or Hb Sbeta-thalassemia) and will be equal to or greater than 5.5 years old.
Key inclusion criteria (3 listed)
- Patients must have a diagnosis of SCD (HbSS, HbSC or HbS beta/thal) by hemoglobin electrophoresis or gene mapping. Very few patients with HbSC or HbS beta/thal would go onto develop ACS, but they will serve as important controls.
- Patients must be ≥5.5 years of age and ≤30 years of age.
- Patients must be able to comply with pulmonary function tests.
Key exclusion criteria (8 listed)
- Patients taking anti-leukotriene medications such as montelukast (Singulair®) or zileuton (Zyflo®) 30 days prior to enrollment will be ineligible, although patients with SCD and asthma who are not on leukotriene inhibitors will be eligible.
- Patients with known congenital heart disease or with congenital lung abnormality/disease that will affect the pulmonary function testing and methacholine challenge testing will be ineligible (examples include patients with cyanotic heart disease, immotile cilia syndrome, cystic fibrosis, diaphragmatic hernia).
- Any patient with neurologic abnormalities, stroke, developmental delay, or other medical condition that would preclude cooperation with pulmonary function testing or compliance with protocol procedures will be ineligible.
- Patients over 30 years of age will be ineligible.
- Patients who are pregnant or nursing.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Changes in plasma PlGF levels and urine LT levels in SCD patients
Time frame: Baseline and serially through the development of an Acute Sickle Event
1 secondary outcome
Relationship of clinical parameters of severity of SCD and circulating levels of PlGF, leukotrienes, proinflammatory cytochemokines and pulmonary function tests
Time frame: Baseline and serially through the development of an Acute Sickle Event
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Dec 2017.
- Cincinnati Children's Hospital Medical CenterLink provided by the sponsor.
- Cincinnati Comprehensive Sickle Cell CenterLink provided by the sponsor.
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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.
Karen Kalinyak, MD principal investigator
Children's Hospital Medical Center, Cincinnati
Punam Malik, MD study chair
Children's Hospital Medical Center, Cincinnati
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Anemia, Sickle Cell, run by Children's Hospital Medical Center, Cincinnati 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 3 inclusion and 8 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: finished, as of the sponsor's last update on 21 Dec 2017. 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.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Changes in plasma PlGF levels and urine LT levels in SCD patients (time frame: Baseline and serially through the development of an Acute Sickle Event). 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 21 Dec 2017.