An observational study in Sickle Cell Disease, sponsored by Theravia. Completed at 60 sites in 7 countries. Open to participants aged 2 Years and older. Per ClinicalTrials.gov, last updated 2020-03-19.
In the context of the Risk Management Plan (RMP), as requested from Addmedica by the EMEA, to collect information about long-term safety of Siklos® (hydroxycarbamide) when used in patients with Sickle Cell Disease.
1,134 studies on the registry are indexed under Anemia, Sickle Cell; 238 are open to participants now.
This study's enrollment of 1,906 is above the median of 100 across 296 observational studies indexed under Anemia, Sickle Cell.
Browse Anemia, Sickle Cell studies →Theravia is the lead sponsor of 8 studies on the registry; 1 is open to participants now.
First submitted before Theravia became part of Norgine.
Counted across the registry records on this site, refreshed daily.
Patient with sickle-cell disease
Inclusion Criteria:
Also known as: hydrocarbamide
% of Patient-years With Malignancies
Time frame: During the follow-up of participant, up to 10 years
% of Patient-years With Skin Ulcerations
Patients with at least one skin ulceration
Time frame: During the follow-up of participant, up to 10 years
% of Patient-years With Myelosuppressions
Patients with at least one myelosuppression
Time frame: During the follow-up of participant, up to 10 years
| Milestone | Patients With Sickle Cell Disease Treated With Siklos® |
|---|---|
| Started | 1906 |
| Completed | 1906 |
| Not completed | 0 |
| % patient-years | Patients With Sickle Cell Disease Treated With Siklos® |
|---|---|
| % of Patient-years With Malignancies | 0.001 |
Patients with at least one skin ulceration
| % patient-years | Patients With Sickle Cell Disease Treated With Siklos® |
|---|---|
| % of Patient-years With Skin Ulcerations | 1.13 |
Patients with at least one myelosuppression
| % patient-years | Patients With Sickle Cell Disease Treated With Siklos® |
|---|---|
| % of Patient-years With Myelosuppressions | 5.99 |
Collected over Adverse events were collected during the follow-up of participant, up to 10 years. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Patients With Sickle Cell Disease Treated With Siklos® | 33/1,903 (1.7%) | 705/1,903 (37%) | 1,308/1,903 (68.7%) |
| Event | Patients With Sickle Cell Disease Treated With Siklos® |
|---|---|
| infections and infestationsInfections and infestations | 346/1903 |
| Blood and lymphatic system disordersBlood and lymphatic system disorders | 155/1903 |
| respiratory, thoracic and mediastinal disordersRespiratory, thoracic and mediastinal disorders | 151/1903 |
| general disorders and administration site conditionsGeneral disorders | 97/1903 |
| Event | Patients With Sickle Cell Disease Treated With Siklos® |
|---|---|
| infections and infestationsInfections and infestations | 645/1903 |
| General disorders and administration site conditionsGeneral disorders | 386/1903 |
| Skin and subcutaneous tissue disordersSkin and subcutaneous tissue disorders | 370/1903 |
1906 patients included but 1903 patient took at least one dose of Siklos and are included in the analysis
| Age, Continuous(years) | Patients With Sickle Cell Disease Treated With Siklos® |
|---|---|
| Median | 21.4 (0.8 to 70.4) |
| Sex: Female, Male(Participants) | Patients With Sickle Cell Disease Treated With Siklos® |
|---|---|
| Female | 1051 |
| Male | 852 |
| Race and Ethnicity Not Collected(Participants) | Patients With Sickle Cell Disease Treated With Siklos® |
|---|
| Region of Enrollment(participants) | Patients With Sickle Cell Disease Treated With Siklos® |
|---|---|
| Greece | 173 |
| Italy | 10 |
| France | 1575 |
| Germany | 145 |
PDFs hosted by the registry, ClinicalTrials.gov.
This study is completed, as verified in Mar 2020. You cannot join it, but the record below documents what was studied.
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