Sickle Cell DiseaseNCT02709681
Hydroxyurea in the Treatment of Sickle Cell Disease
An observational study of Sickle Cell Disease, run by Società Italiana Talassemie ed Emoglobinopatie at 30 sites in Italy.
Trial summary
This is a retrospective cohort study of Sickle Cell Disease (SCD) patients attending 32 treatment centers across Italy. The aim of this study will be to report the Italian experience with the use of hydroxyurea in a large cohort of SCD patients and to evaluate the benefits and safety of this intervention for the… Read more about this study
- Who is it for?
- Ages 1 year and older
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- Physician standard-of-care in SCD patients
- How many people
- 628 participants
- Where
- 30 sitesItaly
- Study dates
- Nov 2015to Jul 2017
- 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 1 year and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: A population of SCD (Homozigous HbS and Beta Thal /HbS) patients followed in 32 Italian Centers was included in this analysis.
Who may be able to join
- Sickle Cell Disease affected patients
- 2-3 vaso-occlusive crisis and/or hospitalizations in the last year
Who cannot join
- none
Trial locations and contacts
30 sites in 1 country.
30 sites in 1 country
Italy30 sites
Azienda Ospedaliero-Universitaria
Bari
U.O. Oncoematologia Pediatrica Ospedali Civili
Brescia, 25100
Ospedale "A. Perrino"
Brindisi, 72100
Azienda Ospedaliera Universitaria Di Cagliari
Cagliari, 09121
Ospedale Vittorio Emanuele
Catania, 95123
ARNAS "Garibaldi"
Catania, 95124
University of Catania
Catania, 95124
A.O. "Pugliese-Ciaccio"
Catanzaro, 88100
Osp.San Giovanni Di Dio
Crotone
Azienda Ospedaliero Universitaria Meyer
Florence, 50139
Ospedale Vittorio Emanuele III
Gela, Caltanisetta, 93012
E.O. Ospedali Galliera
Genova, 16128
Università degli Studi di Milano
Milan, 20122
Azienda Ospedaliero-Universitaria di Modena - Policlinico
Modena, 41124
Clinica pediatrica Monza S. Gerardo
Monza, 20052
Azienda Ospedaliera San Gerardo di Monza
Monza, 20900
Aorn A. Cardarelli
Naples
Clinica di Onco-Ematologia Pediatrica, Università di Padova
Padova, 35128
A.R.N.A.S. "Civico"
Palermo, 90100
Azienda Ospedaliera Ospedali Riuniti Villa Sofia Cervello
Palermo, 90146
Azienda Ospedaliero-Universitaria di Parma
Parma, 43126
Policlinico San Matteo
Pavia, 27100
Centro Emofilia e Medicina Trasfusionale - Pres. Ospedaliero
Ravenna, 48121
A. O. Bianchi Melacrino Morelli
Reggio Calabria, 89100
Ospedale S. Eugenio - FF UOSD DH Talassemici
Roma, 00144
Università Cattolica del Sacro Cuore - Policlinico A.Gemelli
Roma, 00168
Ospedali Civili Riuniti di Sciacca
Sciacca, Agrigento, 92019
U.O.S. Talassemia P.O. Umberto I°
Syracuse, 96100
Università degli Suidi di Torino
Torino, 90146
Policlinico G.B. Rossi
Verona, 37122
About this study
Official title: Hydroxyurea in Sickle Cell Disease: a Large Nation-wide Cohort Study From Italy
This is a retrospective cohort study of Sickle Cell Disease (SCD) patients attending 32 treatment centers across Italy. The aim of this study will be to report the Italian experience with the use of hydroxyurea in a large cohort of SCD patients and to evaluate the benefits and safety of this intervention for the prevention and management of a wide range of clinical morbidities
Read the detailed description
The indication for hydroxyurea initiation was 2-3 vaso-occlusive crisis and/or hospitalizations in the last year.
The study will analyze demographics (age and gender), origin, genotype, clinical phenotype (vaso-occlusive or hemolytic), transfusion history (including exchange), and folic acid use, average laboratory values up to three years pre-hydroxyurea and for the period post-hydroxyurea therapy including total hemoglobin level, fetal hemoglobin level, hemoglobin S level, white blood count, platelet count, lactate dehydrogenase level, total and direct bilirubin levels, aspartate and alanine aminotransferase levels, and serum creatinine level.
The incidence of complications pre- and post-hydroxyurea therapy will be also analyzed including: stroke, silent cerebral infraction, acute chest syndrome, vaso-occlusive crisis, hospitalization, pulmonary hypertension, leg ulcers, bone necrosis, and kidney injury. Safety data included adverse events as reported by the treating physician and the incidence of malignancy or death as well as pregnancy incidents and their outcomes will be also pointed out.
- Conditions
- Sickle Cell Disease
- Being tested
- Physician standard-of-care in SCD patients
- Keywords
- Sickle Cell Disease, Hydroxyurea
- 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.
Physician standard-of-care in SCD patientsOther
| Study group | Role | What they receive |
|---|---|---|
| SCD patients | Patients followed in 32 Italian Centers. Receives: Other: Physician standard-of-care in SCD patients |
What is being measured
The primary outcome is the main question the study is designed to answer.
Changes in laboratory parameters is being assessed
Increases or decreases in percentage of total hemoglobin, fetal hemoglobin and hemoglobin S level will be assessed. Changing of white blood cells and platelets counts, lactate dehydrogenase, bilirubin, aspartate aminotransferase and serum creatinine level will be also evaluated.
Measured: An average of 3 years before and an average of 3 years after initiation of hydroxyurea therapy
Changes in complication rates is being assessed
Changing in the incidence of stroke, silent cerebral infraction, acute chest syndrome, vaso-occlusive crisis, pulmonary hypertension, leg ulcers, bone necrosis and kidney injury will be evaluated.
Measured: An average of 3 years before and an average of 3 years after initiation of hydroxyurea therapy
Rate of hospitalizations
Changing in rate of hospitalizations before and after start hydroxyurea therapy
Measured: An average of 3 years before and an average of 3 years after initiation of hydroxyurea therapy
Secondary outcomes (1)
Changing in the incidence of complications according to specific subgroups
We also stratified the analysis according to age (≥18 years), origin (Italian and African), genotype (βS/β0, βS/β+ and βS/βS) duration of hydroxyurea treatment (≥10 years) and hydroxyurea dose(≥15 mg/kg/day).
Measured: An average of 3 years before and an average of 3 years after initiation of hydroxyurea therapy
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Mar 2016 (estimated)
- Study startNov 2015
- Primary completionNov 2016
- Study completionJul 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
- 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
- Retrospective
- Enrolment
- 628 participants (actual)
Sponsor and investigators
- Lead sponsor
- Società Italiana Talassemie ed Emoglobinopatie
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Paolo Rigano, MD, Principal investigator, Servico Integrado de Tecnicas Endovasculares
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02709681
- Sponsor's protocol ID
- SocietaITE
- Status last verified
- Mar 2016
- First submitted
- 1 Mar 2016
- Last updated
- 6 Oct 2017
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Publications
Rigano P, De Franceschi L, Sainati L, Piga A, Piel FB, Cappellini MD, Fidone C, Masera N, Palazzi G, Gianesin B, Forni GL; Italian Multicenter Study of Hydroxyurea in Sickle Cell Anemia Investigators. Real-life experience with hydroxyurea in sickle cell disease: A multicenter study in a cohort of patients with heterogeneous descent. Blood Cells Mol Dis. 2018 Mar;69:82-89. doi: 10.1016/j.bcmd.2017.08.017. Epub 2017 Oct 9.
PubMed 29107441
Study at a glance
From the registry record, last updated by the sponsor on 6 Oct 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Hydroxyurea in Sickle Cell Disease: a Large Nation-wide Cohort Study From Italy
- ClinicalTrials.gov ID
- NCT02709681
- Acronym / sponsor ID
- SocietaITE
- Conditions
- Sickle Cell Disease
- Keywords
- Sickle Cell Disease, Hydroxyurea
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 628 participants (actual)
- Lead sponsor
- Società Italiana Talassemie ed EmoglobinopatieUniversity, hospital or other
- Study start
- Nov 2015
- Primary completion
- Nov 2016 (actual)
- Study completion
- Jul 2017 (actual)
- First posted
- 16 Mar 2016 (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.
This is a retrospective cohort study of Sickle Cell Disease (SCD) patients attending 32 treatment centers across Italy. The aim of this study will be to report the Italian experience with the use of hydroxyurea in a large cohort of SCD patients and to evaluate the benefits and safety of this intervention for the prevention and management of a wide range of clinical morbidities
Detailed description
The indication for hydroxyurea initiation was 2-3 vaso-occlusive crisis and/or hospitalizations in the last year.
The study will analyze demographics (age and gender), origin, genotype, clinical phenotype (vaso-occlusive or hemolytic), transfusion history (including exchange), and folic acid use, average laboratory values up to three years pre-hydroxyurea and for the period post-hydroxyurea therapy including total hemoglobin level, fetal hemoglobin level, hemoglobin S level, white blood count, platelet count, lactate dehydrogenase level, total and direct bilirubin levels, aspartate and alanine aminotransferase levels, and serum creatinine level.
The incidence of complications pre- and post-hydroxyurea therapy will be also analyzed including: stroke, silent cerebral infraction, acute chest syndrome, vaso-occlusive crisis, hospitalization, pulmonary hypertension, leg ulcers, bone necrosis, and kidney injury. Safety data included adverse events as reported by the treating physician and the incidence of malignancy or death as well as pregnancy incidents and their outcomes will be also pointed out.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| SCD patients | – | Other: Physician standard-of-care in SCD patientsPatients followed in 32 Italian Centers. |
Who may qualify
- Ages
- 1 year and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- A population of SCD (Homozigous HbS and Beta Thal /HbS) patients followed in 32 Italian Centers was included in this analysis.
Key inclusion criteria (2 listed)
- Sickle Cell Disease affected patients
- 2-3 vaso-occlusive crisis and/or hospitalizations in the last year
Key exclusion criteria (1 listed)
- none
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 |
|---|---|---|
| Physician standard-of-care in SCD patients | Other | SCD patients |
Primary outcome
Changes in laboratory parameters is being assessed
Time frame: An average of 3 years before and an average of 3 years after initiation of hydroxyurea therapy
Increases or decreases in percentage of total hemoglobin, fetal hemoglobin and hemoglobin S level will be assessed. Changing of white blood cells and platelets counts, lactate dehydrogenase, bilirubin, aspartate aminotransferase and serum creatinine level will be also evaluated.
Changes in complication rates is being assessed
Time frame: An average of 3 years before and an average of 3 years after initiation of hydroxyurea therapy
Changing in the incidence of stroke, silent cerebral infraction, acute chest syndrome, vaso-occlusive crisis, pulmonary hypertension, leg ulcers, bone necrosis and kidney injury will be evaluated.
Rate of hospitalizations
Time frame: An average of 3 years before and an average of 3 years after initiation of hydroxyurea therapy
Changing in rate of hospitalizations before and after start hydroxyurea therapy
1 secondary outcome
Changing in the incidence of complications according to specific subgroups
Time frame: An average of 3 years before and an average of 3 years after initiation of hydroxyurea therapy
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 6 Oct 2017.
- Derived from this study: PubMed 29107441Rigano P, De Franceschi L, Sainati L, Piga A, Piel FB, Cappellini MD, Fidone C, Masera N, Palazzi G, Gianesin B, Forni GL; Italian Multicenter Study of Hydroxyurea in Sickle Cell Anemia Investigators. Real-life experience with hydroxyurea in sickle cell disease: A multicenter study in a cohort of patients with heterogeneous descent. Blood Cells Mol Dis. 2018 Mar;69:82-89. doi: 10.1016/j.bcmd.2017.08.017. Epub 2017 Oct 9.
- Individual participant dataSharing planned: No
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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.
Paolo Rigano, MD principal investigator
Servico Integrado de Tecnicas Endovasculares
30 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Sickle Cell Disease, run by Società Italiana Talassemie ed Emoglobinopatie at 30 sites in Italy.
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 2 inclusion and 1 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 6 Oct 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 30 sites in Italy. 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 laboratory parameters is being assessed (time frame: An average of 3 years before and an average of 3 years after initiation of hydroxyurea therapy). The study lists 3 primary outcomes in all. 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 NCT02709681, 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 6 Oct 2017.