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

Active, not recruiting

• Conditions: Sickle Cell Disease
• Interventions: Drug: Hydroxycarbamide

• Registration Number: NCT04707235
• Lead Sponsor: Theravia

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®.

Detailed Description

Not available

Study Timeline

• Study Start: 2020-08-21
• Study First Submitted: 2020-12-22
• Study First Submitted QC: 2021-01-12
• Study First Posted: 2021-01-13
• Status Verified: 2024-08
• Last Update Submitted: 2024-08-26
• Last Update Posted: 2024-08-27
• Primary Completion: 2025-08-21
• Study Completion: 2025-08-21

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 2093

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.

Exclusion Criteria

Not provided

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Sickle cell disease patients treated with Siklos	Hydroxycarbamide	-

Primary Outcome Measures

Name	Time	Method
Occurrence and incidence of the number of malignancies, leg ulcers, male fertility impairment and serious unexpected AEs causally related to Siklos®	5 years
Occurrence and incidence of the number of leg ulcers	5 years
Occurrence and incidence of male fertility impairment	5 years
Occurrence and incidence of serious unexpected AEs causally related to Siklos®	5 years

Secondary Outcome Measures

Name	Time	Method
Mean age of puberty	5 years	Document changes in fertility status in males and females
Mean number of patients with the use of a contraception, incidence of the use of semen cryopreservation and of semen analysis performed	5 years	Document changes in fertility status in males and females
Frequency and reason for temporary or permanent discontinuations of Siklos®	5 years	Potential barriers to the compliance to the prescription of Siklos
Number of switch and combination with alternative treatment	5 years
Measure of quality of life with SF12 questionnaire	5 years	Quality of life for adults SCD patients
Incidence of the use of semen cryopreservation and of semen analysis performed	5 years	Document changes in fertility status in males and females
Incidence of semen analysis performed	5 years	Document changes in fertility status in males and females
Management of Siklos® during pregnancy and outcome of pregnancy following exposure at the time of conception or at any time during the pregnancy	5 years	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

Trial Locations

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; 🇫🇷 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

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