Clinical trial NCT04707235: ESCORT-HU Extension: European Sickle Cell Disease Cohort - Hydroxyurea - Extension Study
Official title: ESCORT-HU Extension: European Sickle Cell Disease Cohort - Hydroxyurea - Extension
Study type: OBSERVATIONAL
Overall status: ACTIVE_NOT_RECRUITING
Conditions: Sickle Cell Disease
Lead sponsor: Theravia
Sponsor class: INDUSTRY
Collaborators: name: International Clinical Trials Association
First posted: 2021-01-13
Enrollment: 2093
Sex: ALL
Minimum age (years): 2
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Investigators will be recruited among physicians experienced in SCD management who are the only ones authorised to initiate Siklos® treatment hence to enrol and to follow up the participants. Investigators who are already participating in ESCORT-HU study will be asked to participate in the extension study.

Patient selection will be based on a systematic sampling technique: during the recruitment period, patients fulfilling the inclusion criteria will be informed by their initiating physician about the ESCORT-HU Extension study and enrolled in case they agree to participate.

Brief summary: 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®.


Eligibility criteria: 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.

Primary outcomes:
measure: Occurrence and incidence of the number of malignancies, leg ulcers, male fertility impairment and serious unexpected AEs causally related to Siklos®
timeFrame: 5 years
measure: Occurrence and incidence of the number of leg ulcers
timeFrame: 5 years
measure: Occurrence and incidence of male fertility impairment
timeFrame: 5 years
measure: Occurrence and incidence of serious unexpected AEs causally related to Siklos®
timeFrame: 5 years
Secondary outcomes:
measure: Mean age of puberty
timeFrame: 5 years
measure: Mean number of patients with the use of a contraception, incidence of the use of semen cryopreservation and of semen analysis performed
timeFrame: 5 years
measure: Incidence of the use of semen cryopreservation and of semen analysis performed
timeFrame: 5 years
measure: Incidence of semen analysis performed
timeFrame: 5 years
measure: Management of Siklos® during pregnancy and outcome of pregnancy following exposure at the time of conception or at any time during the pregnancy
timeFrame: 5 years
measure: Frequency and reason for temporary or permanent discontinuations of Siklos®
timeFrame: 5 years
measure: Number of switch and combination with alternative treatment
timeFrame: 5 years
measure: Measure of quality of life with SF12 questionnaire
timeFrame: 5 years
Other outcomes:
Locations:
city: Amiens
country: France
facility: Amiens - Picardie Hospital Adults
city: Amiens
country: France
facility: Amiens Picardie Hospital Children
city: Amiens
country: France
facility: Clinique de l'Europe Amiens
city: Angers
country: France
facility: Angers Hospital Adults
city: Angers
country: France
facility: Angers Hospital Center Children
city: Aulnay-sous-Bois
country: France
facility: Robert Ballanger Hospital Adults
city: Aulnay-sous-Bois
country: France
facility: Robert Ballanger Hospital Children
city: Bobigny
country: France
facility: Avicenne Hospital Adults
city: Bondy
country: France
facility: Jean Verdier Hospital Children
city: Bordeaux
country: France
facility: Bordeaux Hospital Adults
city: Bordeaux
country: France
facility: Bordeaux Hospital Children
city: Boulogne-Billancourt
country: France
facility: Ambroise Paré Hospital Children
city: Chambray-lès-Tours
country: France
facility: Tours Regional University Hospital Center Adults
city: Clermont-Ferrand
country: France
facility: Estaing Hospital Children
city: Colombes
country: France
facility: Louis Mourier Hospital Adults
city: Colombes
country: France
facility: Louis Mourier Hospital Children
city: Corbeil-Essonnes
country: France
facility: Sud Francilien Hospital Center Adults
city: Corbeil-Essonnes
country: France
facility: Centre Hospitalier Sud Francilien
city: Creil
country: France
facility: Sud Oise Public Hospital Group Adults
city: Créteil
country: France
facility: Henri Mondor Hospital Adults
city: Créteil
country: France
facility: Intercommunal Hospital Center of Créteil Adults and Children
city: Dreux
country: France
facility: Dreux Hospital Center Children
city: Gonesse
country: France
facility: Gonesse Hospital Children
city: Jossigny
country: France
facility: Marne-La-Vallée Hospital Center Children
city: La Tronche
country: France
facility: Grenoble Hospital Adults
city: La Tronche
country: France
facility: Grenoble Hospital Children
city: Le Kremlin-Bicêtre
country: France
facility: Bicêtre Hospital Adults
city: Le Kremlin-Bicêtre
country: France
facility: Hôpital Bicêtre
city: Le Mans
country: France
facility: CH du Mans
city: Lille
country: France
facility: Jeanne De Flandre Hospital Children
city: Lille
country: France
facility: Hôpital Saint-Vincent de Paul
city: Limoges
country: France
facility: Hôpital Mère-Enfant - CHU de Limoges
city: Lyon
country: France
facility: Edouard Herriot Hospital Adults
city: Lyon
country: France
facility: Hematology and oncology institute Children
city: Marseille
country: France
facility: Timone Hospital Adults
city: Marseille
country: France
facility: Timone Hospital Children
city: Meaux
country: France
facility: Meaux Hospital Center Adults
city: Meaux
country: France
facility: Meaux Hospital Center Children
city: Montpellier
country: France
facility: St Eloi Hospital Adults
city: Montpellier
country: France
facility: Montpellier Hospital Children
city: Mulhouse
country: France
facility: Emile Muller Hospital Adults
city: Nantes
country: France
facility: Nantes Hospital Children
city: Nantes
country: France
facility: Nantes Hospital Adults
city: Nice
country: France
facility: Nice Hospital Children
city: Orléans
country: France
facility: Orléans Hospital Adults
city: Paris
country: France
facility: Armand Trousseau Hospital Children
city: Paris
country: France
facility: Saint-Antoine Hospital Adults
city: Paris
country: France
facility: Georges Pompidou European Hospital Adults
city: Paris
country: France
facility: Necker Hospital Adults
city: Paris
country: France
facility: Necker Hospital Children
city: Paris
country: France
facility: Robert Debré Hospital Children
city: Perpignan
country: France
facility: Centre hospitalier Perpignan
city: Poitiers
country: France
facility: Poitiers Hospital Adults
city: Reims
country: France
facility: American Hospital Children
city: Reims
country: France
facility: Reims Hospital Adults
city: Rennes
country: France
facility: Pontchaillou Hospital Adults
city: Rouen
country: France
facility: Charles Nicolle Hospital Adults
city: Rouen
country: France
facility: Rouen Hospital Children
city: Saint-Brieuc
country: France
facility: Yves Le Foll. Hospital Center Children
city: Saint-Denis
country: France
facility: Delafontaine Hospital Center Adults
city: Saint-Denis
country: France
facility: Delafontaine Hospital Center Children
city: Saint-Quentin
country: France
facility: Saint-Quentin Hospital Center Adults
city: Strasbourg
country: France
facility: Hautepierre Hospital Children
city: Toulouse
country: France
facility: Children Hospital
city: Toulouse
country: France
facility: Toulouse University Institute of cancer Adults
city: Tours
country: France
facility: Clocheville Regional University Hospital Center Children
city: Vandoeuvre les nancy
country: France
facility: Nancy Hospital Children
city: Versailles
country: France
facility: Versailles Hospital Center Children
city: Cayenne
country: French Guiana
facility: CH de Cayenne
city: Kourou
country: French Guiana
facility: Centre hospitalier de Kourou Children
city: Saint-Laurent-du-Maroni
country: French Guiana
facility: Centre Hospitalier de l'Ouest Guyanais Franck Joly Adults
city: Saint-Laurent-du-Maroni
country: French Guiana
facility: Centre Hospitalier de l'Ouest Guyanais Franck Joly Children
city: Berlin
country: Germany
facility: Charité Hospital Children
city: Düsseldorf
country: Germany
facility: Düsseldorf Hospital Children
city: Freiburg
country: Germany
facility: Freiburg Hospital Children
city: Hamburg
country: Germany
facility: Hamburg Hospital Children
city: Koblenz
country: Germany
facility: Koblenz Hospital Children
city: Athens
country: Greece
facility: Laiko General Hospital Adults
city: Pointe-à-Pitre
country: Guadeloupe
facility: Hôpital RICOU - CHU Pointe-à-Pitre Abymes Children
city: Pointe-à-Pitre
country: Guadeloupe
facility: Hôpital RICOU - CHU Pointe-à-Pitre Abymes
city: Modena
country: Italy
facility: AUO Policlinico di Modena
city: Napoli
country: Italy
facility: Napoli Hospital Children
city: Padova
country: Italy
facility: Padova Hospital Children
city: Verona
country: Italy
facility: Verona Hospital Children
city: Fort-de-France
country: Martinique
facility: CHU Fort de France Children
city: Le Lamentin
country: Martinique
facility: CHU Martinique Adults
city: Saint-Pierre
country: Réunion
facility: CHU Sud Réunion
Study officials:
affiliation: Hospital Necker enfants malades
name: Mariane de Montalembert, MD, PhD
role: STUDY_CHAIR
affiliation: Henri Mondor University Hospital
name: Frédéric Galactéros, MD, PhD
role: STUDY_CHAIR
References: