ESCORT-HU Extension: European Sickle Cell Disease Cohort - Hydroxyurea - Extension Study

N/AActive Not RecruitingNCT04707235

Theravia2,093 enrolled

Overview

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®.

Study Type

OBSERVATIONAL

Enrollment

2,093

Conditions

Sickle Cell Disease

Interventions

HydroxycarbamideDRUG

Patients, aged ≥ 2 years old, with symptomatic SCD, treated with Siklos®.

Eligibility

Sex: ALLMin age: 2 Years

Medical Language ↔ Plain English

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.

Locations (87)

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

Outcomes

Primary Outcomes

Occurrence and incidence of the number of malignancies, leg ulcers, male fertility impairment and serious unexpected AEs causally related to Siklos®

Time frame: 5 years

Occurrence and incidence of the number of leg ulcers

Time frame: 5 years

Occurrence and incidence of male fertility impairment

Time frame: 5 years

Occurrence and incidence of serious unexpected AEs causally related to Siklos®

Time frame: 5 years

Secondary Outcomes

Mean age of puberty

Document changes in fertility status in males and females

Time frame: 5 years

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

Time frame: 5 years

Incidence of the use of semen cryopreservation and of semen analysis performed

Document changes in fertility status in males and females

Time frame: 5 years

Incidence of semen analysis performed

Document changes in fertility status in males and females

Time frame: 5 years

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

Time frame: 5 years

Frequency and reason for temporary or permanent discontinuations of Siklos®

Potential barriers to the compliance to the prescription of Siklos

Time frame: 5 years

Number of switch and combination with alternative treatment

Time frame: 5 years

Measure of quality of life with SF12 questionnaire

Quality of life for adults SCD patients

Time frame: 5 years