Sickle Cell DiseasePhase 3NCT01794000
A Study of Prasugrel in Pediatric Participants With Sickle Cell Disease (SCD)
A Phase 3 study testing Prasugrel in people with Sickle Cell Disease, run by Eli Lilly and Company at 50 sites in 14 countries.
Trial summary
The main purpose of the study is to evaluate the efficacy and safety of the study drug known as prasugrel for the reduction of Vaso-Occlusive Crisis events in pediatric participants with sickle cell disease. The study will also investigate reduction in daily pain in children who have sickle cell disease. Read more about this study
- Who is it for?
- Ages 2 to 17 years
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- Prasugrel
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 341 participants
- Where
- 50 sitesin 14 countries
- Study dates
- Apr 2013to Dec 2015
- 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 2 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Have SCD [homozygous sickle cell (HbSS) or hemoglobin (HbS) Beta^0 thalassemia]
- Are participants with SCD who have had ≥2 episodes of vaso-occlusive crisis (VOC) in the past year
- Have a body weight ≥19 kilograms (kg) and are ≥2 and <18 years of age, inclusive at the time of screening
- If participants are ≥2 and ≤16 years of age, must have had a transcranial Doppler within the last year
Who cannot join
- History of: transient ischemic attack (TIA)/ ischemic or hemorrhagic stroke, severe head trauma, intracranial hemorrhage, intracranial neoplasm, arteriovenous malformation, or aneurysm
- History of abnormal or conditional [velocity in middle or anterior cerebral, or internal carotid artery ≥170 centimeter per second (cm/sec)] transcranial Doppler within the last year
- History of, or are undergoing treatment with, chronic red blood cell (RBC) transfusion therapy
- Are at an increased risk for bleeding complications
- Are receiving chronic treatment with nonsteroidal anti-inflammatory drug (NSAID)s and cannot be switched to another analgesic
Trial locations and contacts
50 sites in 14 countries.
50 sites in 14 countries
United States20 sites
Emory University
Atlanta, Georgia, 30322
Boston Children's Hospital
Boston, Massachusetts, 02115
University of NC at Chapel Hill School of Medicine
Chapel Hill, North Carolina, 27599
Medical University of South Carolina
Charleston, South Carolina, 29425
Ann & Robert H Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
Childrens Hospital Medical Center
Cincinnati, Ohio, 45229
Rainbow Babies and Children's Hospital
Cleveland, Ohio, 44106
Childrens Hospital of Michigan
Detroit, Michigan, 48201
Connecticut Children's Medical Center
Hartford, Connecticut, 06106
Children's Mercy Hospital
Kansas City, Missouri, 64108
St Jude Childrens Research Hospital
Memphis, Tennessee, 38105
Children's Hospital of Oakland
Oakland, California, 94609
Stanford Univ Medical Center
Palo Alto, California, 94304
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19134
St Christophers Hospital For Children
Philadelphia, Pennsylvania, 19134
Childrens Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224
Memorial Health University Medical Center
Savannah, Georgia, 31404
Mary Bridge Children's Hospital and Health Center
Tacoma, Washington, 98405
Albert Einstein College of Medicine
The Bronx, New York, 10467
Howard University Hospital
Washington D.C., District of Columbia, 20060
Egypt6 sites
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Al Fayyum, 63514
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Al Mansurah, 35516
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Alexandria, 21131
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Cairo, 11566
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Ismailia
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Zagazig, 44519
Italy5 sites
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Genova, 16128
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Modena, 40124
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Monza, 20900
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Padova, 35138
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Verona, 37126
Kenya4 sites
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Busia, 40100
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Kisumu
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Kombewa
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Nairobi
United Kingdom3 sites
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London, SE1 7EH
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Manchester, M13 9WL
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Tooting, London, SW17 0QT
Belgium2 sites
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Brussels, 1200
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Montegnée, 4420
Ghana2 sites
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Agogo
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Korle Bu
Turkey (Türkiye)2 sites
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Balcali Adana, 01330
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Mersin, 33079
Brazil1 site
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Rio de Janeiro, 20211-030
Canada1 site
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Montreal, Quebec, H3T 1C5
Lebanon1 site
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Beirut, 5244
Oman1 site
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Muscat, 123
Saudi Arabia1 site
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Jeddah, 21859
United Arab Emirates1 site
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Abu Dhabi
About this study
Official title: A Phase 3, Double-Blind, Randomized, Efficacy and Safety Comparison of Prasugrel and Placebo in Pediatric Patients With Sickle Cell Disease.
The main purpose of the study is to evaluate the efficacy and safety of the study drug known as prasugrel for the reduction of Vaso-Occlusive Crisis events in pediatric participants with sickle cell disease. The study will also investigate reduction in daily pain in children who have sickle cell disease.
Read the detailed description
The submission database was validated for data reported through the data cutoff date for the submission database lock (SDBL). The SDBL data cutoff was 17 July 2015 for all participants except for 2 in the youngest age group, for whom the SDBL data cutoff occurred on 08 August 2015. The data cutoff date for SDBL corresponds to the primary completion date for the study. The SDBL occurred on 31 August 2015.
The study was stopped following SDBL and review of the topline information indicated that the primary and secondary efficacy endpoints were not met. Subsequently, the Sponsor requested that participants discontinue study drug immediately and that discontinuation visits for all active study participants be conducted as soon as feasible.
After the data cutoff date for SDBL, the Sponsor continued to collect safety data through the final participants contact; some additional efficacy data were collected through the final visit. The last patient visit (LPV) occurred on 17 December 2015, which corresponds to the study completion date and led to the planned supplemental database lock (PSDBL) on 22 January 2016. This supplemental data base was originally designed to capture additional blinded and randomized information to enhance safety data for labeling should the study have been positive.
The safety information contained in this record reflects the entire safety information and reflects the information from the supplemental data base lock in January of 2016. The efficacy information contained in this record reflects the information collected through primary completion date in the submission database. Primary analyses of the major efficacy objectives were repeated using the entire double-blind period data from the PSDBL and did not change the original conclusions and were consistent with the results from the original efficacy analyses included in the SDBL.
- Conditions
- Sickle Cell Disease
- Being tested
- Prasugrel, 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.
PrasugrelDrug
Also called LY640315, Effient, Efient
Administered orally
PlaceboDrug
Administered orally
| Study group | Role | What they receive |
|---|---|---|
| Prasugrel | Experimental | Participants will be titrated from initial daily dose of 0.08 milligram per kilogram (mg/kg) of orally administered prasugrel monotherapy at randomization to a dose that will achieve a P2Y12 reaction units (PRU) level of 231 to 136, as measured by VerifyNow instrument. This corresponds to a range of platelet inhibition of approximately 30% to 60%. The maximum possible dose allowed is 0.12 mg/kg daily, not to exceed 10 mg daily. Receives: Drug: Prasugrel |
| Placebo | Placebo comparator | Participants in this treatment group will receive daily orally administered placebo and will follow visit schedule identical to that in the active treatment group. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Vaso-Occlusive Crisis (VOC) Events Per Participant Per Year (Rate of VOC)
The VOC is a composite endpoint of painful crisis or acute chest syndrome. Events that occurred within 7 days from the prior event onset date were not counted as a new episode. Data collected through the primary completion date reported below.
Measured: Randomization through 24 Months
Secondary outcomes (12)
Monthly Rate of Days With Pain
Monthly rate of days with pain was measured through participant diaries using a modified version of the Faces Pain Scale-Revised (FPS-R). Each day participants selected the face on the scale that reflected their worst pain related to sickle cell disease (SCD) on that day. This pain scale contains six faces corresponding to the pain intensity of 0, 2, 4, 6, 8 or 10, in which 0 denotes no pain and 10 denotes the worst pain possible. Any day the participant selected a face other than face 0 was considered a day with pain. Monthly rate of days with pain was calculated for each participant by summing the number of days reported with any pain divided by the number of non-missing diary entries completed in the month. A month was defined as 4 weeks (28 days).The monthly rate was set to missing if there were more than 14 missing entries for the FPS-R in a specific month. Data collected through the primary completion date are present below.
Measured: Randomization through 9 Months
Monthly Mean in Faces Pain Scale-Revised Score
Each day participants selected the face on the FPS-R scale that reflected their worst pain related to sickle cell disease (SCD) on that day. Monthly mean in FPS-R score was calculated for each participant by summing the FPS-R score divided by the number of non-missing diary entries completed in the month. This pain scale contains six faces corresponding to the pain intensity of 0, 2, 4, 6, 8 or 10, in which 0 denotes no pain and 10 denotes the worst pain possible. A month was defined as 4 weeks (28 days). The monthly mean in FPS-R score was set to missing if there were more than 14 missing entries for the FPS-R in a specific month. Data collected through the primary completion date are presented below.
Measured: Randomization through 9 Months
Number of Painful Crisis Events Per Participant Per Year (Rate of Painful Crisis)
A painful crisis is defined as an onset of moderate to severe pain that lasts at least 2 hours for which there is no explanation other than vaso-occlusion and which requires therapy with oral or parenteral opioids, ketorolac, or other analgesics prescribed by a health care provider (HCP) in a medical setting such as a hospital, clinic, emergency room visit, or telephone management. The painful crisis that occurred within 7 days from the prior event onset date was not counted as a new episode. Data collected through the primary completion date are presented below.
Measured: Randomization through 24 Months
Number of Hospitalizations for VOC Per Participant Per Year (Rate of Hospitalizations)
Hospitalization that occurred within 7 days of the prior event onset date were not counted as a new episode. Data collected through the primary completion date are presented below.
Measured: Randomization through 24 Months
Number of Acute Chest Syndrome Per Participant Per Year (Rate of Acute Chest Syndrome)
Acute chest syndrome was defined as an acute illness characterized by fever and/or respiratory symptoms, accompanied by a new pulmonary infiltrate on a chest X-ray. Acute chest syndrome that occurred within 7 days of the prior event onset date was not counted as a new episode. Data collected through the primary completion date are presented below.
Measured: Randomization through 24 Months
Number of Red Blood Cell (RBC) Transfusions Due to Sickle Cell Disease (SCD) Per Participant Per Year (Rate of RBC Transfusions)
RBC transfusions that occurred within 7 days of the prior event onset date were not counted as a new episode. Data collected through the primary completion date are presented below.
Measured: Randomization through 24 Months
Monthly Rate of Days of Analgesic Use
Monthly rate of days of analgesic use was measured through participant diaries and was calculated for each participant by summing the number of days they reported analgesic use divided by the number of diary entries completed in the month. A month was defined as 4 weeks (28 days). The monthly rate was set to missing if there were more than 14 missing entries for analgesic use in a specific month. Data collected through the primary completion date are presented below.
Measured: Randomization through 9 Months
Quarterly Rate of School Absence Due to Sickle Cell Pain
Quarterly rate of school absence due to sickle cell pain was measured through participant diaries and was calculated for each participant by summing the number of days with school absence due to sickle cell pain divided by the number of school dates in the quarter. A quarter was defined as 12 weeks. The quarterly rate was set to missing if there were more than 6 weeks of missing diary entries during a specific quarter. Data collected through the primary completion date are presented below.
Measured: Randomization through 9 Months
Time to First Transient Ischemic Attack (TIA)/Ischemic Stroke
Measured: Randomization through 24 Months
Number of Days Hospitalized for VOC
The total length of hospitalization in days for VOC was calculated for each participant. Data collected through the primary completion date are presented below.
Measured: Randomization through 24 Months
Time From Randomization to First and Second VOC
Data collected through the primary completion date are presented below.
Measured: Randomization to First VOC and Second VOC respectively (up to 24 Months)
Percentage of Participants With Hemorrhagic Events Requiring Medical Intervention
Medical intervention was defined as any medical evaluation resulting in therapy or further investigation, as determined by a trained medical professional. Data collected from the first dose of study medication through 10 days after last dose of study medication during the double blind study period are presented below.
Measured: First Dose through 24 Months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Feb 2013 (estimated)
- Study startApr 2013
- Primary completionJul 2015
- Study completionDec 2015
Why it stopped: The study is being terminated for lack of efficacy.
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 DiseaseStudies of the same condition, open and under way.
- More trials run by Eli Lilly and CompanyThe 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
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 341 participants (actual)
Sponsor and investigators
- Lead sponsor
- Eli Lilly and Company
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Daiichi Sankyo
- Responsible party
- Sponsor
- Investigators
- Call 1-877-CTLILLY (1-877-285-4559) or 1-317-615-4559 Mon-Fri 9 AM - 5 PM Eastern time (UTC/GMT - 5 hours, EST), Study director, Eli Lilly and Company
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01794000
- Sponsor's protocol ID
- 13038
- Other IDs
- H7T-MC-TADO, 2012-003837-41
- Status last verified
- Sep 2019
- First submitted
- 14 Feb 2013
- Last updated
- 25 Sep 2019
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- Individual data sharing
- Yes: Anonymized individual patient level data will be provided in a secure access environment upon approval of a research proposal and a signed data sharing agreement.
Publications
Heeney MM, Hoppe CC, Abboud MR, Inusa B, Kanter J, Ogutu B, Brown PB, Heath LE, Jakubowski JA, Zhou C, Zamoryakhin D, Agbenyega T, Colombatti R, Hassab HM, Nduba VN, Oyieko JN, Robitaille N, Segbefia CI, Rees DC; DOVE Investigators. A Multinational Trial of Prasugrel for Sickle Cell Vaso-Occlusive Events. N Engl J Med. 2016 Feb 18;374(7):625-35. doi: 10.1056/NEJMoa1512021. Epub 2015 Dec 8.
PubMed 26644172
Study at a glance
From the registry record, last updated by the sponsor on 25 Sep 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3, Double-Blind, Randomized, Efficacy and Safety Comparison of Prasugrel and Placebo in Pediatric Patients With Sickle Cell Disease.
- ClinicalTrials.gov ID
- NCT01794000
- Acronym / sponsor ID
- 13038
- Conditions
- Sickle Cell Disease
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: The study is being terminated for lack of efficacy.
- Enrolment
- 341 participants (actual)
- Lead sponsor
- Eli Lilly and CompanyIndustry (pharma, biotech, devices)
- Collaborators
- Daiichi Sankyo
- Study start
- Apr 2013
- Primary completion
- Jul 2015 (actual)
- Study completion
- Dec 2015 (actual)
- First posted
- 18 Feb 2013 (estimated)
- Results
- Posted 27 Jul 2016
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The main purpose of the study is to evaluate the efficacy and safety of the study drug known as prasugrel for the reduction of Vaso-Occlusive Crisis events in pediatric participants with sickle cell disease. The study will also investigate reduction in daily pain in children who have sickle cell disease.
Detailed description
The submission database was validated for data reported through the data cutoff date for the submission database lock (SDBL). The SDBL data cutoff was 17 July 2015 for all participants except for 2 in the youngest age group, for whom the SDBL data cutoff occurred on 08 August 2015. The data cutoff date for SDBL corresponds to the primary completion date for the study. The SDBL occurred on 31 August 2015.
The study was stopped following SDBL and review of the topline information indicated that the primary and secondary efficacy endpoints were not met. Subsequently, the Sponsor requested that participants discontinue study drug immediately and that discontinuation visits for all active study participants be conducted as soon as feasible.
After the data cutoff date for SDBL, the Sponsor continued to collect safety data through the final participants contact; some additional efficacy data were collected through the final visit. The last patient visit (LPV) occurred on 17 December 2015, which corresponds to the study completion date and led to the planned supplemental database lock (PSDBL) on 22 January 2016. This supplemental data base was originally designed to capture additional blinded and randomized information to enhance safety data for labeling should the study have been positive.
The safety information contained in this record reflects the entire safety information and reflects the information from the supplemental data base lock in January of 2016. The efficacy information contained in this record reflects the information collected through primary completion date in the submission database. Primary analyses of the major efficacy objectives were repeated using the entire double-blind period data from the PSDBL and did not change the original conclusions and were consistent with the results from the original efficacy analyses included in the SDBL.
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 |
|---|---|---|
| Prasugrel | Experimental | Drug: PrasugrelParticipants will be titrated from initial daily dose of 0.08 milligram per kilogram (mg/kg) of orally administered prasugrel monotherapy at randomization to a dose that will achieve a P2Y12 reaction units (PRU) level of 231 to 136, as measured by VerifyNow instrument. This corresponds to a range of platelet inhibition of approximately 30% to 60%. The maximum possible dose allowed is 0.12 mg/kg daily, not to exceed 10 mg daily. |
| Placebo | Placebo comparator | Drug: PlaceboParticipants in this treatment group will receive daily orally administered placebo and will follow visit schedule identical to that in the active treatment group. |
Who may qualify
- Ages
- 2 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- Have SCD [homozygous sickle cell (HbSS) or hemoglobin (HbS) Beta^0 thalassemia]
- Are participants with SCD who have had ≥2 episodes of vaso-occlusive crisis (VOC) in the past year
- Have a body weight ≥19 kilograms (kg) and are ≥2 and <18 years of age, inclusive at the time of screening
- If participants are ≥2 and ≤16 years of age, must have had a transcranial Doppler within the last year
Key exclusion criteria (5 listed)
- History of: transient ischemic attack (TIA)/ ischemic or hemorrhagic stroke, severe head trauma, intracranial hemorrhage, intracranial neoplasm, arteriovenous malformation, or aneurysm
- History of abnormal or conditional [velocity in middle or anterior cerebral, or internal carotid artery ≥170 centimeter per second (cm/sec)] transcranial Doppler within the last year
- History of, or are undergoing treatment with, chronic red blood cell (RBC) transfusion therapy
- Are at an increased risk for bleeding complications
- Are receiving chronic treatment with nonsteroidal anti-inflammatory drug (NSAID)s and cannot be switched to another analgesic
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 |
|---|---|---|
| PrasugrelAlso: LY640315, Effient, EfientAdministered orally | Drug | Prasugrel |
| PlaceboAdministered orally | Drug | Placebo |
Primary outcome
Number of Vaso-Occlusive Crisis (VOC) Events Per Participant Per Year (Rate of VOC)
Time frame: Randomization through 24 Months
The VOC is a composite endpoint of painful crisis or acute chest syndrome. Events that occurred within 7 days from the prior event onset date were not counted as a new episode. Data collected through the primary completion date reported below.
12 secondary outcomes
Monthly Rate of Days With Pain
Time frame: Randomization through 9 Months
Monthly Mean in Faces Pain Scale-Revised Score
Time frame: Randomization through 9 Months
Number of Painful Crisis Events Per Participant Per Year (Rate of Painful Crisis)
Time frame: Randomization through 24 Months
Number of Hospitalizations for VOC Per Participant Per Year (Rate of Hospitalizations)
Time frame: Randomization through 24 Months
Number of Acute Chest Syndrome Per Participant Per Year (Rate of Acute Chest Syndrome)
Time frame: Randomization through 24 Months
Number of Red Blood Cell (RBC) Transfusions Due to Sickle Cell Disease (SCD) Per Participant Per Year (Rate of RBC Transfusions)
Time frame: Randomization through 24 Months
Monthly Rate of Days of Analgesic Use
Time frame: Randomization through 9 Months
Quarterly Rate of School Absence Due to Sickle Cell Pain
Time frame: Randomization through 9 Months
Time to First Transient Ischemic Attack (TIA)/Ischemic Stroke
Time frame: Randomization through 24 Months
Number of Days Hospitalized for VOC
Time frame: Randomization through 24 Months
Time From Randomization to First and Second VOC
Time frame: Randomization to First VOC and Second VOC respectively (up to 24 Months)
Percentage of Participants With Hemorrhagic Events Requiring Medical Intervention
Time frame: First Dose through 24 Months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Sep 2019.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 26644172Heeney MM, Hoppe CC, Abboud MR, Inusa B, Kanter J, Ogutu B, Brown PB, Heath LE, Jakubowski JA, Zhou C, Zamoryakhin D, Agbenyega T, Colombatti R, Hassab HM, Nduba VN, Oyieko JN, Robitaille N, Segbefia CI, Rees DC; DOVE Investigators. A Multinational Trial of Prasugrel for Sickle Cell Vaso-Occlusive Events. N Engl J Med. 2016 Feb 18;374(7):625-35. doi: 10.1056/NEJMoa1512021. Epub 2015 Dec 8.
- Individual participant dataSharing planned: Yes. Anonymized individual patient level data will be provided in a secure access environment upon approval of a research proposal and a signed data sharing agreement.
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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.
Call 1-877-CTLILLY (1-877-285-4559) or 1-317-615-4559 Mon-Fri 9 AM - 5 PM Eastern time (UTC/GMT - 5 hours, EST) study director
Eli Lilly and Company
50 sites in 14 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Prasugrel in people with Sickle Cell Disease, run by Eli Lilly and Company at 50 sites in 14 countries.
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 4 inclusion and 5 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 25 Sep 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 50 sites in United States, Egypt, Italy, Kenya, United Kingdom and 9 more countries. 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 27 Jul 2016). 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 Vaso-Occlusive Crisis (VOC) Events Per Participant Per Year (Rate of VOC) (time frame: Randomization through 24 Months). 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 NCT01794000, 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 25 Sep 2019.