Anemia, Sickle CellPhase 3NCT00530270
Dexamethasone to Treat Acute Chest Syndrome in People With Sickle Cell Disease
A Phase 3 study testing Dexamethasone in people with Anemia, Sickle Cell, run by Children's Hospital Medical Center, Cincinnati at 6 sites in United States.
Trial summary
People with sickle cell disease (SCD) may develop acute chest syndrome (ACS), which is a common and serious lung condition that usually requires hospitalization. Dexamethasone is a medication that may decrease hospitalization time for people with ACS, but it may also bring about new sickle cell pain. Read more about this study
- Who is it for?
- Ages 5 years and older
- Conditions the study is for
- Anemia, Sickle Cell
- What is being tested?
- Dexamethasone
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 12 participants
- Where
- 6 sitesUnited States
- Study dates
- Dec 2006to Nov 2008
- Phase
- Phase 3
- 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 5 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Diagnosis of sickle cell anemia (Hgb SS) or sickle-β0-thalassemia (Hgb Sβ0)
- Current episode of ACS, defined as a new lobar or segmental pulmonary infiltrate seen on a chest radiograph and two or more of the following findings:
- Temperature of 38.5°C or higher
- Tachypnea (i.e., rapid breathing)
- Dyspnea or increased work of breathing
- Chest wall pain
Show all 9Show fewer
- Oxygen saturation of less than 90% in room air by pulse oximetry
- Current episode of ACS diagnosed in the 24 hours prior to study entry
- Ability to take medication in capsule form
Who cannot join
- Prior participation in this study
- Diagnosed with any medical condition that will likely be worsened by corticosteroid therapy, including any of the following conditions:
- Diabetes mellitus
- High blood pressure
- Esophageal or gastrointestinal ulceration or bleeding
- Known avascular necrosis
Show all 15Show fewer
- Diagnosis of ACS in the 6 months prior to study entry
- Treatment with oral or parenteral corticosteroid therapy for any reason in the 14 days prior to study entry
- Use of inhaled corticosteroids or systemic corticosteroids for respiratory illness in the 3 months prior to study entry
- Long-term lung condition that requires treatment with corticosteroids
- Participation in a program of chronic transfusions that ended fewer than 4 months ago. A program of chronic transfusions includes a regimen of serial simple or exchange transfusions given at least every 6 weeks for at least three consecutive transfusions for the prevention of SCD-related complications.
- Pregnant
- Treatment with any investigational drug in the 90 days prior to study entry
- History of either tuberculosis or a positive skin test for tuberculosis
- Known HIV infection or a current systemic fungal infection
Trial locations and contacts
6 sites in 1 country.
6 sites in 1 country
United States6 sites
Children's Hospital Boston
Boston, Massachusetts, 02115
University of North Carolina
Chapel Hill, North Carolina, 27599
Children's Medical Center of Dallas
Dallas, Texas, 75235
Kosair Children's Hospital
Louisville, Kentucky, 40202
St. Christopher's Hospital
Philadelphia, Pennsylvania, 19134
University of California - Davis
Sacramento, California, 95817
About this study
Official title: Randomized Trial of Oral Dexamethasone for Acute Chest Syndrome
People with sickle cell disease (SCD) may develop acute chest syndrome (ACS), which is a common and serious lung condition that usually requires hospitalization. Dexamethasone is a medication that may decrease hospitalization time for people with ACS, but it may also bring about new sickle cell pain. This study will evaluate the effectiveness of a dexamethasone regimen that includes a gradual dose reduction at decreasing hospitalization and recovery time in people with SCD and ACS.
Read the detailed description
SCD is an inherited blood disorder. Symptoms include anemia, infections, organ damage, and intense episodes of pain, which are called "sickle cell crises." ACS is a life-threatening, lung-related complication of SCD that can lower the level of oxygen in the blood. Repeat occurrences of ACS can cause lung damage. It is the second most common cause of hospitalizations among people with SCD and accounts for more than 25% of premature deaths in people with SCD. Symptoms of ACS include fever, chest pain, cough, and breathing difficulties. ACS can appear suddenly and often requires immediate hospitalization and treatment, including antibiotics, supplemental oxygen, and blood transfusions. Previous studies have shown that dexamethasone, a type of steroid medication that blocks inflammation, can decrease hospitalization time for people with ACS; however, some participants in these earlier studies were re-hospitalized due to new sickle cell pain. Slowly decreasing the dosage of dexamethasone over a period of time may decrease the chance that new sickle cell pain will occur. The purpose of this study is to evaluate the effectiveness of a dexamethasone regimen that includes a gradual dose reduction at decreasing hospitalization and recovery time in people with SCD and ACS.
This study will enroll people with SCD who are hospitalized and have been diagnosed with ACS within the past 24 hours. Participants will be randomly assigned to receive either dexamethasone or placebo on a daily basis for 8 days. Every 2 days the medication dose will be gradually reduced. While in the hospital, participants will receive usual care for ACS, including antibiotics, pain control medication, intravenous fluids, and other needed treatments. Each day, participants will undergo a physical exam, a pain assessment score, a test to measure the oxygen level in the body, blood collection, and, if needed, a chest x-ray. Vital signs and blood pressure measurements will be taken every 4 hours. Study staff will document the amount of pain medication, blood transfusions, oxygen, and breathing treatments participants receive.
Upon leaving the hospital, follow-up visits will occur 1 week after participants were originally admitted to the hospital (participants who are still hospitalized at this time will not attend this visit) and 1 month after hospital discharge. At both visits, information on hospital visits for pain treatment and blood transfusions will be collected, and evaluations performed earlier in the study will be repeated. The second visit will also include lung function tests.
- Conditions
- Anemia, Sickle Cell
- Being tested
- Dexamethasone, Placebo
- Keywords
- Sickle Cell Disease, ACS, Acute Chest Syndrome, Hgb SS, Hgb Sβ0, Dexamethasone
- Medical subject headings
- Anemia, Sickle Cell, Acute Chest Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DexamethasoneDrug
Individuals meeting entry criteria will be randomized to receive dexamethasone 0.3 mg/kg (12 mg maximum single dose). The study drug will be given by mouth every 12 hours until discharge from the hospital or for a maximum of 4 doses (2 days), whichever occurs first. Thereafter, study drug will be tapered over 6 days for a total duration of therapy not to exceed 8 days.
PlaceboDrug
Individuals meeting entry criteria will be randomized to receive 0.3 mg/kg (12 mg maximum single dose) of placebo. The study drug will be given by mouth every 12 hours until discharge from the hospital or for a maximum of 4 doses (2 days), whichever occurs first. Thereafter, study drug will be tapered over 6 days for a total duration of therapy not to exceed 8 days.
| Study group | Role | What they receive |
|---|---|---|
| Dexamethasone | Active comparator | Not described Receives: Drug: Dexamethasone |
| Placebo | Placebo comparator | Not described Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Log (Natural) of Duration of Signs and Symptoms of Acute Chest Syndrome (ACS) or Duration of Hospitalization, Whichever is Less
Resolution of symptoms of ACS includes respiratory rate <= upper limit of normal +2, no work of breathing (retractions, nasal flaring, and use of accessory muscles), thoracic pain <= 4, no use of supplemental oxygen, no use of ventilary support, and saturation of peripheral oxygen (Sp02) >= steady state value -2. Symptoms were measured every 4 hours from the first dose of study drug to resolution of symptoms or hospital discharge.
Measured: Measured from first dose to end of the hospital stay, no maximum number of days
Secondary outcomes (4)
Rating of Pain
Change from baseline rating of pain from randomization (baseline) to discharge from the hospital, evaluated every 4 hours. Pain was rated on the Oucher Scale for the pediatric population or numeric rating scale for the adult population, both 0 to 10 with 0 indicating no pain and 10 indicating severe pain.
Measured: Measured at the end of the hospital stay
Duration of Hospitalization
Duration in hours from treatment start time to hospital discharge.
Measured: Measured at the end of hospital stay, no maximum number of days
Duration of Supplemental Oxygen
Time period between the supplemental oxygen start date/time and first dose date/time, whichever is later, and the supplemental oxygen stop date/time
Measured: Measured at the end of hospital stay
Duration of Hypoxemia (Low Blood Oxygen)
Sum of time periods when subject was hypoxemic (Sp02 value less than 92%) since the first dose date/time
Measured: Measured at the end of hospital stay
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Sep 2007 (estimated)
- Study startDec 2006
- Primary completionJun 2008
- Study completionNov 2008
Why it stopped: Study was closed June 23, 2008 due to low enrollment.
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.
- Publications (1)Papers the sponsor has linked to this study.
- 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
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 12 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
- Principal investigator, Dr. Charles Quinn, Children's Hospital Medical Center, Cincinnati
- Investigators
- Charles Quinn, MD, Principal investigator, University of Texas, Southwestern Medical Center at Dallas
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00530270
- Sponsor's protocol ID
- 516
- Other IDs
- U54HL070587, U54HL070587-07
- Status last verified
- Mar 2013
- First submitted
- 14 Sep 2007
- Last updated
- 9 Apr 2013
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Quinn CT, Stuart MJ, Kesler K, Ataga KI, Wang WC, Styles L, Smith-Whitley K, Wun T, Raj A, Hsu LL, Krishnan S, Kuypers FA, Setty Y, Rhee S, Key NS, Buchanan GR; Investigators of the Comprehensive Sickle Cell Centers. Tapered oral dexamethasone for the acute chest syndrome of sickle cell disease. Br J Haematol. 2011 Oct;155(2):263-7. doi: 10.1111/j.1365-2141.2011.08827.x. Epub 2011 Aug 16.
PubMed 21848879
Study at a glance
From the registry record, last updated by the sponsor on 9 Apr 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Randomized Trial of Oral Dexamethasone for Acute Chest Syndrome
- ClinicalTrials.gov ID
- NCT00530270
- Acronym / sponsor ID
- 516
- Conditions
- Anemia, Sickle Cell
- Keywords
- Sickle Cell Disease, ACS, Acute Chest Syndrome, Hgb SS, Hgb Sβ0, Dexamethasone
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Study was closed June 23, 2008 due to low enrollment.
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Children's Hospital Medical Center, CincinnatiUniversity, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI)
- Study start
- Dec 2006
- Primary completion
- Jun 2008 (actual)
- Study completion
- Nov 2008 (actual)
- First posted
- 17 Sep 2007 (estimated)
- Results
- Posted 31 Dec 2009
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
People with sickle cell disease (SCD) may develop acute chest syndrome (ACS), which is a common and serious lung condition that usually requires hospitalization. Dexamethasone is a medication that may decrease hospitalization time for people with ACS, but it may also bring about new sickle cell pain. This study will evaluate the effectiveness of a dexamethasone regimen that includes a gradual dose reduction at decreasing hospitalization and recovery time in people with SCD and ACS.
Detailed description
SCD is an inherited blood disorder. Symptoms include anemia, infections, organ damage, and intense episodes of pain, which are called "sickle cell crises." ACS is a life-threatening, lung-related complication of SCD that can lower the level of oxygen in the blood. Repeat occurrences of ACS can cause lung damage. It is the second most common cause of hospitalizations among people with SCD and accounts for more than 25% of premature deaths in people with SCD. Symptoms of ACS include fever, chest pain, cough, and breathing difficulties. ACS can appear suddenly and often requires immediate hospitalization and treatment, including antibiotics, supplemental oxygen, and blood transfusions. Previous studies have shown that dexamethasone, a type of steroid medication that blocks inflammation, can decrease hospitalization time for people with ACS; however, some participants in these earlier studies were re-hospitalized due to new sickle cell pain. Slowly decreasing the dosage of dexamethasone over a period of time may decrease the chance that new sickle cell pain will occur. The purpose of this study is to evaluate the effectiveness of a dexamethasone regimen that includes a gradual dose reduction at decreasing hospitalization and recovery time in people with SCD and ACS.
This study will enroll people with SCD who are hospitalized and have been diagnosed with ACS within the past 24 hours. Participants will be randomly assigned to receive either dexamethasone or placebo on a daily basis for 8 days. Every 2 days the medication dose will be gradually reduced. While in the hospital, participants will receive usual care for ACS, including antibiotics, pain control medication, intravenous fluids, and other needed treatments. Each day, participants will undergo a physical exam, a pain assessment score, a test to measure the oxygen level in the body, blood collection, and, if needed, a chest x-ray. Vital signs and blood pressure measurements will be taken every 4 hours. Study staff will document the amount of pain medication, blood transfusions, oxygen, and breathing treatments participants receive.
Upon leaving the hospital, follow-up visits will occur 1 week after participants were originally admitted to the hospital (participants who are still hospitalized at this time will not attend this visit) and 1 month after hospital discharge. At both visits, information on hospital visits for pain treatment and blood transfusions will be collected, and evaluations performed earlier in the study will be repeated. The second visit will also include lung function tests.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Dexamethasone | Active comparator | Drug: Dexamethasone |
| Placebo | Placebo comparator | Drug: Placebo |
Who may qualify
- Ages
- 5 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (9 listed)
- Diagnosis of sickle cell anemia (Hgb SS) or sickle-β0-thalassemia (Hgb Sβ0)
- Current episode of ACS, defined as a new lobar or segmental pulmonary infiltrate seen on a chest radiograph and two or more of the following findings:
- Current episode of ACS diagnosed in the 24 hours prior to study entry
- Ability to take medication in capsule form
Key exclusion criteria (15 listed)
- Prior participation in this study
- Diagnosed with any medical condition that will likely be worsened by corticosteroid therapy, including any of the following conditions:
- Diagnosis of ACS in the 6 months prior to study entry
- Treatment with oral or parenteral corticosteroid therapy for any reason in the 14 days prior to study entry
- Use of inhaled corticosteroids or systemic corticosteroids for respiratory illness in the 3 months prior to study entry
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 |
|---|---|---|
| DexamethasoneIndividuals meeting entry criteria will be randomized to receive dexamethasone 0.3 mg/kg (12 mg maximum single dose). The study drug will be given by mouth every 12 hours until discharge from the hospital or for a maximum of 4 doses (2 days), whichever occurs first. Thereafter, study drug will be tapered over 6 days for a total duration of therapy not to exceed 8 days. | Drug | Dexamethasone |
| PlaceboIndividuals meeting entry criteria will be randomized to receive 0.3 mg/kg (12 mg maximum single dose) of placebo. The study drug will be given by mouth every 12 hours until discharge from the hospital or for a maximum of 4 doses (2 days), whichever occurs first. Thereafter, study drug will be tapered over 6 days for a total duration of therapy not to exceed 8 days. | Drug | Placebo |
Primary outcome
Log (Natural) of Duration of Signs and Symptoms of Acute Chest Syndrome (ACS) or Duration of Hospitalization, Whichever is Less
Time frame: Measured from first dose to end of the hospital stay, no maximum number of days
Resolution of symptoms of ACS includes respiratory rate <= upper limit of normal +2, no work of breathing (retractions, nasal flaring, and use of accessory muscles), thoracic pain <= 4, no use of supplemental oxygen, no use of ventilary support, and saturation of peripheral oxygen (Sp02) >= steady state value -2. Symptoms were measured every 4 hours from the first dose of study drug to resolution of symptoms or hospital discharge.
4 secondary outcomes
Rating of Pain
Time frame: Measured at the end of the hospital stay
Duration of Hospitalization
Time frame: Measured at the end of hospital stay, no maximum number of days
Duration of Supplemental Oxygen
Time frame: Measured at the end of hospital stay
Duration of Hypoxemia (Low Blood Oxygen)
Time frame: Measured at the end of hospital stay
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Apr 2013.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 21848879Quinn CT, Stuart MJ, Kesler K, Ataga KI, Wang WC, Styles L, Smith-Whitley K, Wun T, Raj A, Hsu LL, Krishnan S, Kuypers FA, Setty Y, Rhee S, Key NS, Buchanan GR; Investigators of the Comprehensive Sickle Cell Centers. Tapered oral dexamethasone for the acute chest syndrome of sickle cell disease. Br J Haematol. 2011 Oct;155(2):263-7. doi: 10.1111/j.1365-2141.2011.08827.x. Epub 2011 Aug 16.
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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.
Charles Quinn, MD principal investigator
University of Texas, Southwestern Medical Center at Dallas
6 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Dexamethasone in people with Anemia, Sickle Cell, run by Children's Hospital Medical Center, Cincinnati at 6 sites 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 9 inclusion and 15 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 9 Apr 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites 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. Some participants in this study may receive a placebo. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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 31 Dec 2009). 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?
Log (Natural) of Duration of Signs and Symptoms of Acute Chest Syndrome (ACS) or Duration of Hospitalization, Whichever is Less (time frame: Measured from first dose to end of the hospital stay, no maximum number of days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00530270, which also lists 1 linked publication.
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 9 Apr 2013.