Sickle Cell AnemiaPhase 2NCT01788631
A Phase II Trial of Regadenoson in Sickle Cell Anemia
A Phase 2 study testing Regadenoson in people with Sickle Cell Anemia, run by Dana-Farber Cancer Institute at 12 sites in United States.
Trial summary
This research study is a Phase II clinical trial, which tests the safety and effectiveness of an investigational drug called Regadenoson (or Lexiscan) to learn whether the drug works in treating a specific disease, in this case Sickle Cell Disease (SCD). "Investigational" means that the drug is being studied. Read more about this study
- Who is it for?
- Ages 10 to 70 years
- Conditions the study is for
- Sickle Cell Anemia
- What is being tested?
- Regadenoson
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 100 participants
- Where
- 12 sitesUnited States
- Study dates
- Jul 2013to 12 Dec 2016
- 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 10 to 70 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Must have sickle cell anemia confirmed by hemoglobin analysis
- Must be admitted to hospital for pain or ACS
- Reliable IV access as determined by the study physician
- Participants must have the laboratory indices as defined below:
- Hemoglobin ≥ 5 g/dL
- Platelets > 100,000/mcL
Show all 9Show fewer
- ALT (SGPT) < 3 X institutional upper limit of normal
- Serum creatinine ≤ 1.5 mg/dL
- INR ≤2.0, PTT ≤ 48 seconds
Who cannot join
- Pregnant or breastfeeding
- Current physician diagnosis of asthma defined by treatment with systemic corticosteroids within the last 12 months or predicted/current use of asthma controller medications
- 10 or more hospitalizations for pain in the last 12 months
- Receiving regularly scheduled transfusions
- Severe ACS
- Second or third degree AV block or sinus node dysfunction
Show all 17Show fewer
- History of a bleeding diathesis
- History of clinically overt stroke within 3 years
- History of severe hypertension not adequately controlled with anti-hypertensive medications
- Receiving chronic anti-coagulation or anti-platelet therapy
- History of metastatic cancer
- Receiving any other study agents or have received a study agent in the past 30 days
- Uncontrolled intercurrent illness
- Known HIV
- Have previously enrolled and received the investigational agent as part of this study
- Taking medications that may interact with the investigational agent
- Have previously undergone a hematopoietic stem cell transplant or solid organ transplant
Trial locations and contacts
12 sites in 1 country.
12 sites in 1 country
United States12 sites
Johns Hopkins University
Baltimore, Maryland, 21205
Boston Children's Hospital
Boston, Massachusetts, 02215
Brigham and Women's Hospital
Boston, Massachusetts, 02215
Dana-Farber Cancer Institute
Boston, Massachusetts, 02215
University of Illinois at Chicago
Chicago, Illinois, 60612
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Wayne State University/Karmanos Cancer Institute
Detroit, Michigan, 48201
Duke University
Durham, North Carolina, 27705
Baylor College of Medicine
Houston, Texas, 77030
Medical College of Wisconsin
Milwaukee, Wisconsin, 53226
Children's Hospital and Research Center at Oakland
Oakland, California, 94609
Washington University in St. Louis
St Louis, Missouri, 63110
About this study
Official title: A Phase II, Randomized, Placebo-Controlled Trial of Regadenoson in Sickle Cell Anemia
This research study is a Phase II clinical trial, which tests the safety and effectiveness of an investigational drug called Regadenoson (or Lexiscan) to learn whether the drug works in treating a specific disease, in this case Sickle Cell Disease (SCD). "Investigational" means that the drug is being studied. It also means that the FDA has not yet approved the drug for your type of disease.
SCD is an inherited blood disorder that causes the red blood cells to change their shape from a round shape to a half-moon/crescent or sickled shape. People who have SCD have a different type of protein that carries oxygen in their blood (hemoglobin) than people without SCD. This different type of hemoglobin makes the red blood cells change into crescent shape under certain conditions. Sickle-shaped cells are a problem because they often get stuck in the blood vessels blocking the flow of blood, and cause inflammation and injury to important areas in the body.
Read more
Regadenoson (trade name Lexiscan) is a drug that may prevent this inflammation and injury caused by the sickle shaped cells. This drug is approved by the FDA to be used as a fast infusion during a heart stress test in people who are unable to exercise enough to put stress on their heart by making the heart beat faster. Regadenoson has been studied as a long infusion at this dose in adults, and no safety issues have been identified (ClinicalTrials.gov Identifier: NCT01085201). This is the first study to look at patient benefit with the long infusion of the drug. This drug has been used in laboratory experiments and information from those other research studies suggests that this drug may help to protect the body from damage caused by sickle-shaped cells in this research study.
In this research study, the investigators are specifically looking to see if Regadenoson is an effective treatment for pain crises and acute chest syndrome in SCD.
Read the detailed description
If you are willing to participate in this research study you will be asked to undergo some screening tests and procedures to confirm your eligibility. Many of these tests and procedures are likely to be part of regular sickle cell anemia care and may be done even if it turns out that you do not take part in the research study. If you have had some of these tests and procedures recently, they may or may not have to be repeated. The tests and procedures include: a medical history, physical examination, blood tests, blood or urine pregnancy test (if applicable) and an electrocardiogram. If these tests show that you are eligible to participate in the research study, you will begin the study treatment. If you do not meet the eligibility criteria, you will not be able to participate in the research study. At the time of screening we will also ask you about your pain level.
Because no one knows which of the study options is best, you will be "randomized" into one of the study groups: the "study drug" group, which will receive Regadenoson, or the "control" group, which will receive placebo. Randomization means that you are put into a group by chance. It is like flipping a coin. Neither you nor the research doctor will choose what group you will be in. You will have an equal (50/50) chance of being placed in either group. Neither you nor the research doctor will know what group you are in.
You will be given a study medication and it will contain either Regadenoson or placebo (fluids with no medicine).
You will be given one infusion of the study drug while you are admitted to the hospital for a pain crisis. The study drug will be infused with fluids. You will stay in the hospital for at least 3 days and 2 nights. Your infusion will be 48 hours long, followed by a 6-hour observation period. During your infusion, you will receive standard treatment for your pain crisis. The study drug will be given through a separate part of your body from the infusions that are part of your standard treatment. The study drug will not be available after your participation in the study ends.
Before the infusion: We will place a small tube in your vein called an IV, which will be used only to infuse the study drug. It will not be used for infusions that are part of standard treatment for your pain crisis. During the study drug infusion, standard treatment will be given through a separate IV. We can use your standard treatment IV or a needle to draw blood for the required blood test. If it is hard to draw blood from your veins, we may ask you if you would like to use a peripherally inserted central catheter (PICC line) for your blood draws. A PICC line is a small tube that is placed in a vein in your arm and goes through to a vein in your chest. A chest x-ray is usually done to make sure it is in the right veins. It is your choice to decide whether you would like to use a PICC line. We will record your blood pressure and heart rate every 5-10 minutes, until they have stabilized. We will also ask you about your pain level at the time of your blood test.
During the 48 hour infusion: Your heart rate and the amount of oxygen in your blood will be monitored continuously using a device that fits over your finger. We will take about 2-3 teaspoons of blood at 24 and 48 hours after the beginning of your infusion for tests to try to understand how the drug affects your body. We will ask you about your pain level at the time of each blood test. We will take your blood pressure every 30 minutes for the first 2 hours, then every hour for the next two hours, then every 2 hours for the remainder of the infusion.
A six hour observation period will take place immediately after the infusion. At this time you will undergo the following: your heart rate and the amount of oxygen in your blood will be monitored continuously with a device that fits over your finger. We will take about 5 teaspoons of blood at the end of the period to try to understand how the study drug affects your body. We will ask you about your pain level at the time of your blood test. We will take your blood pressure every 2 hours for the full duration of the observation period.
You may not eat or drink anything that contains caffeine, such as coffee, tea, chocolate or sodas during the infusion and observation periods.
We would like to keep track of your medical condition for 30 days after you receive the study drug. We would like to do this by contacting you on the telephone weekly during the 30 days after your participation to see how you are doing.
- Conditions
- Sickle Cell Anemia
- Being tested
- Regadenoson, Placebo
- Medical subject headings
- Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RegadenosonDrug
Also called Lexiscan
Regadenoson is an A2AR agonist that is a coronary vasodilator. It is chemically described as adenosine, 2-[4-[(methylamino)carbonyl]-1H-pyrazol-1-yl]-, monohydrate. Its molecular formula is C15H18N8O5. Regadenoson has an FDA indication for use in radionuclide myocardial perfusion imaging in patients unable to undergo adequate exercise stress. It has lower affinity for non-A2A adenosine receptor subtypes thought to be associated with some of the adverse effects associated with non-selective adenosine receptor agonists, which increase extracellular adenosine by blocking its uptake into cells. The maximal plasma concentration of regadenoson is achieved within 1 to 4 minutes after injection and parallels the onset of the pharmacodynamic response. Its half-life is approximately 2 to 4 minutes.
PlaceboDrug
Also called Regadenoson Placebo, Lexiscan Placebo, 0.9% Normal Saline
This study uses 0.9% Normal Saline (NS) as placebo. This is a sterile sodium chloride solution usually used to replenish fluids and electrolytes. It contains no additives, and is a standard solution used as placebo in clinical trials where the study drug is administration intravenously. NS will be prepared by investigational pharmacy.
| Study group | Role | What they receive |
|---|---|---|
| Regadenoson Arm | Active comparator | 1.44 mcg/kg/hour infused over 48 hours Receives: Drug: Regadenoson |
| Placebo Arm | Placebo comparator | Placebo infused over 48 hours Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With a Reduction in Invariant Natural-Killer T-Cell (iNKT Cell) Activation by 70% or More
To determine if infusional Regadenoson reduced iNKT cell activation among individuals with sickle cell anemia (SCA) and pain or acute chest syndrome (ACS) compared to placebo by 70% or greater.
Measured: Baseline-End of study infusion over 48 hours
Secondary outcomes (6)
Length of Hospital Stay
To determine if regadenoson reduces length of hospital stay among individuals admitted with SCA and pain or ACS compared to placebo
Measured: Hospital Presentation- Hospital Discharge, assessed up to 1 month
Number of Participants With an Improvement in Respiratory Symptoms
To determine if regadenoson improved respiratory symptoms among individuals with sickle cell anemia (SCA) and pain or acute chest syndrome (ACS) compared to placebo. Patients were classified as having an improvement in respiratory symptoms if they experienced any of the following outcomes:(1) respiratory rate decreased by 25% from baseline or normalized (≤20 bpm) or (2) degree of hypoxia (SpO2) on room air increased by 10% from baseline or normalized (≥92%) or (3) thoracic pain improved by 3 points from baseline on a 10-point visual analog scale.
Measured: Baseline-End of study infusion over 48 hours
Opioid Use
To determine if regadenoson reduces opioid use among individuals with SCA and pain or ACS compared to placebo.
Measured: Baseline-End of study infusion over 48 hours
Level of Inflammatory Markers (A2A)
To determine if regadenoson reduces levels of inflammatory markers among individuals with SCA and pain or ACS compared to placebo.
Measured: Baseline-End of study infusion over 48 hours
Level of Inflammatory Markers (IL-4)
To determine if regadenoson reduces levels of inflammatory markers among individuals with SCA and pain or ACS compared to placebo.
Measured: Baseline-End of study infusion over 48 hours
Level of Inflammatory Markers (IFN-gamma)
To determine if regadenoson reduces levels of inflammatory markers among individuals with SCA and pain or ACS compared to placebo.
Measured: Baseline-End of study infusion over 48 hours
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Feb 2013 (estimated)
- Study startJul 2013
- Primary completion10 Nov 2016
- Study completion12 Dec 2016
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 Sickle Cell AnemiaStudies of the same condition, open and under way.
- More trials run by Dana-Farber Cancer InstituteThe 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 100 participants (actual)
Sponsor and investigators
- Lead sponsor
- Dana-Farber Cancer Institute
- Sponsor type
- University, hospital or other
- Collaborators
- Brigham and Women's Hospital, Boston Children's Hospital, La Jolla Institute for Allergy & Immunology, National Heart, Lung, and Blood Institute (NHLBI), Washington University School of Medicine, Children's Hospital Medical Center, Cincinnati, University of Illinois Chicago, Medical College of Wisconsin, Duke University, Johns Hopkins University, Wayne State University, Baylor College of Medicine, UCSF Benioff Children's Hospital Oakland
- Responsible party
- Principal investigator, David G. Nathan, MD, Dana-Farber Cancer Institute
- Investigators
- David Nathan, MD, Principal investigator, Dana-Farber Cancer Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01788631
- Sponsor's protocol ID
- 13-005
- Other IDs
- 1P50HL110790-01
- Status last verified
- Jan 2018
- First submitted
- 7 Feb 2013
- Last updated
- 7 Feb 2018
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Field JJ, Majerus E, Gordeuk VR, Gowhari M, Hoppe C, Heeney MM, Achebe M, George A, Chu H, Sheehan B, Puligandla M, Neuberg D, Lin G, Linden J, Nathan DG. Randomized phase 2 trial of regadenoson for treatment of acute vaso-occlusive crises in sickle cell disease. Blood Adv. 2017 Aug 28;1(20):1645-1649. doi: 10.1182/bloodadvances.2017009613. eCollection 2017 Sep 12.
PubMed 29296811
Study at a glance
From the registry record, last updated by the sponsor on 7 Feb 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II, Randomized, Placebo-Controlled Trial of Regadenoson in Sickle Cell Anemia
- ClinicalTrials.gov ID
- NCT01788631
- Acronym / sponsor ID
- 13-005
- Conditions
- Sickle Cell Anemia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 100 participants (actual)
- Lead sponsor
- Dana-Farber Cancer InstituteUniversity, hospital or other
- Collaborators
- Brigham and Women's Hospital, Boston Children's Hospital, La Jolla Institute for Allergy & Immunology, National Heart, Lung, and Blood Institute (NHLBI), Washington University School of Medicine, Children's Hospital Medical Center, Cincinnati, University of Illinois Chicago, Medical College of Wisconsin, Duke University, Johns Hopkins University, Wayne State University, Baylor College of Medicine, UCSF Benioff Children's Hospital Oakland
- Study start
- Jul 2013
- Primary completion
- 10 Nov 2016 (actual)
- Study completion
- 12 Dec 2016 (actual)
- First posted
- 11 Feb 2013 (estimated)
- Results
- Posted 7 Feb 2018
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This research study is a Phase II clinical trial, which tests the safety and effectiveness of an investigational drug called Regadenoson (or Lexiscan) to learn whether the drug works in treating a specific disease, in this case Sickle Cell Disease (SCD). "Investigational" means that the drug is being studied. It also means that the FDA has not yet approved the drug for your type of disease.
SCD is an inherited blood disorder that causes the red blood cells to change their shape from a round shape to a half-moon/crescent or sickled shape. People who have SCD have a different type of protein that carries oxygen in their blood (hemoglobin) than people without SCD. This different type of hemoglobin makes the red blood cells change into crescent shape under certain conditions. Sickle-shaped cells are a problem because they often get stuck in the blood vessels blocking the flow of blood, and cause inflammation and injury to important areas in the body.
Regadenoson (trade name Lexiscan) is a drug that may prevent this inflammation and injury caused by the sickle shaped cells. This drug is approved by the FDA to be used as a fast infusion during a heart stress test in people who are unable to exercise enough to put stress on their heart by making the heart beat faster. Regadenoson has been studied as a long infusion at this dose in adults, and no safety issues have been identified (ClinicalTrials.gov Identifier: NCT01085201). This is the first study to look at patient benefit with the long infusion of the drug. This drug has been used in laboratory experiments and information from those other research studies suggests that this drug may help to protect the body from damage caused by sickle-shaped cells in this research study.
In this research study, the investigators are specifically looking to see if Regadenoson is an effective treatment for pain crises and acute chest syndrome in SCD.
Detailed description
If you are willing to participate in this research study you will be asked to undergo some screening tests and procedures to confirm your eligibility. Many of these tests and procedures are likely to be part of regular sickle cell anemia care and may be done even if it turns out that you do not take part in the research study. If you have had some of these tests and procedures recently, they may or may not have to be repeated. The tests and procedures include: a medical history, physical examination, blood tests, blood or urine pregnancy test (if applicable) and an electrocardiogram. If these tests show that you are eligible to participate in the research study, you will begin the study treatment. If you do not meet the eligibility criteria, you will not be able to participate in the research study. At the time of screening we will also ask you about your pain level.
Because no one knows which of the study options is best, you will be "randomized" into one of the study groups: the "study drug" group, which will receive Regadenoson, or the "control" group, which will receive placebo. Randomization means that you are put into a group by chance. It is like flipping a coin. Neither you nor the research doctor will choose what group you will be in. You will have an equal (50/50) chance of being placed in either group. Neither you nor the research doctor will know what group you are in.
You will be given a study medication and it will contain either Regadenoson or placebo (fluids with no medicine).
You will be given one infusion of the study drug while you are admitted to the hospital for a pain crisis. The study drug will be infused with fluids. You will stay in the hospital for at least 3 days and 2 nights. Your infusion will be 48 hours long, followed by a 6-hour observation period. During your infusion, you will receive standard treatment for your pain crisis. The study drug will be given through a separate part of your body from the infusions that are part of your standard treatment. The study drug will not be available after your participation in the study ends.
Before the infusion: We will place a small tube in your vein called an IV, which will be used only to infuse the study drug. It will not be used for infusions that are part of standard treatment for your pain crisis. During the study drug infusion, standard treatment will be given through a separate IV. We can use your standard treatment IV or a needle to draw blood for the required blood test. If it is hard to draw blood from your veins, we may ask you if you would like to use a peripherally inserted central catheter (PICC line) for your blood draws. A PICC line is a small tube that is placed in a vein in your arm and goes through to a vein in your chest. A chest x-ray is usually done to make sure it is in the right veins. It is your choice to decide whether you would like to use a PICC line. We will record your blood pressure and heart rate every 5-10 minutes, until they have stabilized. We will also ask you about your pain level at the time of your blood test.
During the 48 hour infusion: Your heart rate and the amount of oxygen in your blood will be monitored continuously using a device that fits over your finger. We will take about 2-3 teaspoons of blood at 24 and 48 hours after the beginning of your infusion for tests to try to understand how the drug affects your body. We will ask you about your pain level at the time of each blood test. We will take your blood pressure every 30 minutes for the first 2 hours, then every hour for the next two hours, then every 2 hours for the remainder of the infusion.
A six hour observation period will take place immediately after the infusion. At this time you will undergo the following: your heart rate and the amount of oxygen in your blood will be monitored continuously with a device that fits over your finger. We will take about 5 teaspoons of blood at the end of the period to try to understand how the study drug affects your body. We will ask you about your pain level at the time of your blood test. We will take your blood pressure every 2 hours for the full duration of the observation period.
You may not eat or drink anything that contains caffeine, such as coffee, tea, chocolate or sodas during the infusion and observation periods.
We would like to keep track of your medical condition for 30 days after you receive the study drug. We would like to do this by contacting you on the telephone weekly during the 30 days after your participation to see how you are doing.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Regadenoson Arm | Active comparator | Drug: Regadenoson1.44 mcg/kg/hour infused over 48 hours |
| Placebo Arm | Placebo comparator | Drug: PlaceboPlacebo infused over 48 hours |
Who may qualify
- Ages
- 10 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (9 listed)
- Must have sickle cell anemia confirmed by hemoglobin analysis
- Must be admitted to hospital for pain or ACS
- Reliable IV access as determined by the study physician
- Participants must have the laboratory indices as defined below:
Key exclusion criteria (17 listed)
- Pregnant or breastfeeding
- Current physician diagnosis of asthma defined by treatment with systemic corticosteroids within the last 12 months or predicted/current use of asthma controller medications
- 10 or more hospitalizations for pain in the last 12 months
- Receiving regularly scheduled transfusions
- Severe ACS
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 |
|---|---|---|
| RegadenosonAlso: LexiscanRegadenoson is an A2AR agonist that is a coronary vasodilator. It is chemically described as adenosine, 2-[4-[(methylamino)carbonyl]-1H-pyrazol-1-yl]-, monohydrate. Its molecular formula is C15H18N8O5. Regadenoson has an FDA indication for use in radionuclide myocardial perfusion imaging in patients unable to undergo adequate exercise stress. It has lower affinity for non-A2A adenosine receptor subtypes thought to be associated with some of the adverse effects associated with non-selective adenosine receptor agonists, which increase extracellular adenosine by blocking its uptake into cells. The maximal plasma concentration of regadenoson is achieved within 1 to 4 minutes after injection and parallels the onset of the pharmacodynamic response. Its half-life is approximately 2 to 4 minutes. | Drug | Regadenoson Arm |
| PlaceboAlso: Regadenoson Placebo, Lexiscan Placebo, 0.9% Normal SalineThis study uses 0.9% Normal Saline (NS) as placebo. This is a sterile sodium chloride solution usually used to replenish fluids and electrolytes. It contains no additives, and is a standard solution used as placebo in clinical trials where the study drug is administration intravenously. NS will be prepared by investigational pharmacy. | Drug | Placebo Arm |
Primary outcome
Number of Participants With a Reduction in Invariant Natural-Killer T-Cell (iNKT Cell) Activation by 70% or More
Time frame: Baseline-End of study infusion over 48 hours
To determine if infusional Regadenoson reduced iNKT cell activation among individuals with sickle cell anemia (SCA) and pain or acute chest syndrome (ACS) compared to placebo by 70% or greater.
6 secondary outcomes
Length of Hospital Stay
Time frame: Hospital Presentation- Hospital Discharge, assessed up to 1 month
Number of Participants With an Improvement in Respiratory Symptoms
Time frame: Baseline-End of study infusion over 48 hours
Opioid Use
Time frame: Baseline-End of study infusion over 48 hours
Level of Inflammatory Markers (A2A)
Time frame: Baseline-End of study infusion over 48 hours
Level of Inflammatory Markers (IL-4)
Time frame: Baseline-End of study infusion over 48 hours
Level of Inflammatory Markers (IFN-gamma)
Time frame: Baseline-End of study infusion over 48 hours
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Feb 2018.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 29296811Field JJ, Majerus E, Gordeuk VR, Gowhari M, Hoppe C, Heeney MM, Achebe M, George A, Chu H, Sheehan B, Puligandla M, Neuberg D, Lin G, Linden J, Nathan DG. Randomized phase 2 trial of regadenoson for treatment of acute vaso-occlusive crises in sickle cell disease. Blood Adv. 2017 Aug 28;1(20):1645-1649. doi: 10.1182/bloodadvances.2017009613. eCollection 2017 Sep 12.
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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.
David Nathan, MD principal investigator
Dana-Farber Cancer Institute
12 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Regadenoson in people with Sickle Cell Anemia, run by Dana-Farber Cancer Institute at 12 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 17 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 7 Feb 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 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 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 7 Feb 2018). 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?
Number of Participants With a Reduction in Invariant Natural-Killer T-Cell (iNKT Cell) Activation by 70% or More (time frame: Baseline-End of study infusion over 48 hours). 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 NCT01788631, 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 7 Feb 2018.