Sickle Cell Disease Clinical Trial
Official title:
ESCORT-HU Extension: European Sickle Cell Disease Cohort - Hydroxyurea - Extension
As safety information pertaining to the long-term use of HU remains incomplete in spite of the first safety study (ESCORT-HU), an extension of the latter is proposed. ESCORT-HU Extension study aims at evaluating the long-term safety of Siklos® focusing on some questions regarding its safety when used in current practice in adults and paediatric patients treated with Siklos® and followed for up to 5 years. The study will focus on the following concerns : occurrence and incidence of malignancies, leg ulcers, male fertility impairment and serious unexpected AEs causally related to Siklos®.
| Status | Recruiting |
| Enrollment | 2000 |
| Est. completion date | August 21, 2025 |
| Est. primary completion date | August 21, 2025 |
| Accepts healthy volunteers | No |
| Gender | All |
| Age group | 2 Years and older |
| Eligibility | Inclusion Criteria: - Male or female patients with symptomatic SCD, - = 2 years old, - Treated with Siklos®, - Having been informed of the study by the investigator and consenting to participate, or whose parents or legal guardians are consenting for their child to participate. To allow risk evaluation, participants must belong at least to one of the subpopulations defined below: - Participants previously enrolled in ESCORT-HU who agree to participate in ESCORT-HU Extension study, - New participants with any of the following criteria: - history of HU treatment for more than 5 years or - prepubescent over 10 years of age for girls and 13 years of age for boys at enrolment, or - with history of leg ulcer, or - pregnant women without interruption of Siklos® 3 months before the beginning of the pregnancy or, - males treated with Siklos® whose partner is pregnant and without discontinuation of Siklos® 3 months before the beginning of the pregnancy. |
| Country | Name | City | State |
|---|---|---|---|
| France | Amiens - Picardie Hospital Adults | Amiens | |
| France | Amiens Picardie Hospital Children | Amiens | |
| France | Clinique de l'Europe Amiens | Amiens | |
| France | Angers Hospital Adults | Angers | |
| France | Angers Hospital Center Children | Angers | |
| France | Robert Ballanger Hospital Adults | Aulnay-sous-Bois | |
| France | Robert Ballanger Hospital Children | Aulnay-sous-Bois | |
| France | Avicenne Hospital Adults | Bobigny | |
| France | Jean Verdier Hospital Children | Bondy | |
| France | Bordeaux Hospital Adults | Bordeaux | |
| France | Bordeaux Hospital Children | Bordeaux | |
| France | Ambroise Paré Hospital Children | Boulogne-Billancourt | |
| France | Tours Regional University Hospital Center Adults | Chambray-lès-Tours | |
| France | Estaing Hospital Children | Clermont-Ferrand | |
| France | Louis Mourier Hospital Adults | Colombes | |
| France | Louis Mourier Hospital Children | Colombes | |
| France | Centre Hospitalier Sud Francilien | Corbeil-Essonnes | |
| France | Sud Francilien Hospital Center Adults | Corbeil-Essonnes | |
| France | Sud Oise Public Hospital Group Adults | Creil | |
| France | Henri Mondor Hospital Adults | Créteil | |
| France | Intercommunal Hospital Center of Créteil Adults and Children | Créteil | |
| France | Dreux Hospital Center Children | Dreux | |
| France | Gonesse Hospital Children | Gonesse | |
| France | Marne-La-Vallée Hospital Center Children | Jossigny | |
| France | Grenoble Hospital Adults | La Tronche | |
| France | Grenoble Hospital Children | La Tronche | |
| France | Bicêtre Hospital Adults | Le Kremlin-Bicêtre | |
| France | Hôpital Bicêtre | Le Kremlin-Bicêtre | |
| France | CH du Mans | Le Mans | |
| France | Hôpital Saint-Vincent de Paul | Lille | |
| France | Jeanne De Flandre Hospital Children | Lille | |
| France | Hôpital Mère-Enfant - CHU de Limoges | Limoges | |
| France | Edouard Herriot Hospital Adults | Lyon | |
| France | Hematology and oncology institute Children | Lyon | |
| France | Timone Hospital Adults | Marseille | |
| France | Timone Hospital Children | Marseille | |
| France | Meaux Hospital Center Adults | Meaux | |
| France | Meaux Hospital Center Children | Meaux | |
| France | Montpellier Hospital Children | Montpellier | |
| France | St Eloi Hospital Adults | Montpellier | |
| France | Emile Muller Hospital Adults | Mulhouse | |
| France | Nantes Hospital Adults | Nantes | |
| France | Nantes Hospital Children | Nantes | |
| France | Nice Hospital Children | Nice | |
| France | Orléans Hospital Adults | Orléans | |
| France | Armand Trousseau Hospital Children | Paris | |
| France | Georges Pompidou European Hospital Adults | Paris | |
| France | Necker Hospital Adults | Paris | |
| France | Necker Hospital Children | Paris | |
| France | Robert Debré Hospital Children | Paris | |
| France | Saint-Antoine Hospital Adults | Paris | |
| France | Centre hospitalier Perpignan | Perpignan | |
| France | Poitiers Hospital Adults | Poitiers | |
| France | American Hospital Children | Reims | |
| France | Reims Hospital Adults | Reims | |
| France | Pontchaillou Hospital Adults | Rennes | |
| France | Charles Nicolle Hospital Adults | Rouen | |
| France | Rouen Hospital Children | Rouen | |
| France | Yves Le Foll. Hospital Center Children | Saint-Brieuc | |
| France | Delafontaine Hospital Center Adults | Saint-Denis | |
| France | Delafontaine Hospital Center Children | Saint-Denis | |
| France | Saint-Quentin Hospital Center Adults | Saint-Quentin | |
| France | Hautepierre Hospital Children | Strasbourg | |
| France | Children Hospital | Toulouse | |
| France | Toulouse University Institute of cancer Adults | Toulouse | |
| France | Clocheville Regional University Hospital Center Children | Tours | |
| France | Nancy Hospital Children | Vandoeuvre les nancy | |
| France | Versailles Hospital Center Children | Versailles | |
| French Guiana | CH de Cayenne | Cayenne | |
| French Guiana | Centre hospitalier de Kourou Children | Kourou | |
| French Guiana | Centre Hospitalier de l'Ouest Guyanais Franck Joly Adults | Saint-Laurent-du-Maroni | |
| French Guiana | Centre Hospitalier de l'Ouest Guyanais Franck Joly Children | Saint-Laurent-du-Maroni | |
| Germany | Charité Hospital Children | Berlin | |
| Germany | Düsseldorf Hospital Children | Düsseldorf | |
| Germany | Freiburg Hospital Children | Freiburg | |
| Germany | Hamburg Hospital Children | Hamburg | |
| Germany | Koblenz Hospital Children | Koblenz | |
| Greece | Laiko General Hospital Adults | Athens | |
| Guadeloupe | Hôpital RICOU - CHU Pointe-à-Pitre Abymes | Pointe-à-Pitre | |
| Guadeloupe | Hôpital RICOU - CHU Pointe-à-Pitre Abymes Children | Pointe-à-Pitre | |
| Italy | AUO Policlinico di Modena | Modena | |
| Italy | Napoli Hospital Children | Napoli | |
| Italy | Padova Hospital Children | Padova | |
| Italy | Verona Hospital Children | Verona | |
| Martinique | CHU Fort de France Children | Fort-de-France | |
| Martinique | CHU Martinique Adults | Le Lamentin | |
| Réunion | CHU Sud Réunion | Saint-Pierre |
| Lead Sponsor | Collaborator |
|---|---|
| ADDMEDICA SASA | International Clinical Trials Association |
France, French Guiana, Germany, Greece, Guadeloupe, Italy, Martinique, Réunion,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | Occurrence and incidence of the number of malignancies, leg ulcers, male fertility impairment and serious unexpected AEs causally related to Siklos® | 5 years | ||
| Primary | Occurrence and incidence of the number of leg ulcers | 5 years | ||
| Primary | Occurrence and incidence of male fertility impairment | 5 years | ||
| Primary | Occurrence and incidence of serious unexpected AEs causally related to Siklos® | 5 years | ||
| Secondary | Mean age of puberty | Document changes in fertility status in males and females | 5 years | |
| Secondary | Mean number of patients with the use of a contraception, incidence of the use of semen cryopreservation and of semen analysis performed | Document changes in fertility status in males and females | 5 years | |
| Secondary | Incidence of the use of semen cryopreservation and of semen analysis performed | Document changes in fertility status in males and females | 5 years | |
| Secondary | Incidence of semen analysis performed | Document changes in fertility status in males and females | 5 years | |
| Secondary | Management of Siklos® during pregnancy and outcome of pregnancy following exposure at the time of conception or at any time during the pregnancy | Outcome of pregnancies Following exposure to Siklos® at the time of conception (in participant and participant's partner) or at any time during the pregnancy | 5 years | |
| Secondary | Frequency and reason for temporary or permanent discontinuations of Siklos® | Potential barriers to the compliance to the prescription of Siklos | 5 years | |
| Secondary | Number of switch and combination with alternative treatment | 5 years | ||
| Secondary | Measure of quality of life with SF12 questionnaire | Quality of life for adults SCD patients | 5 years |
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