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Active, Not Recruiting

NCT Number: NCT04707235

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

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®.

Active, Not Recruiting

This study is active but is not currently recruiting participants.

Key information

Conditions

Sickle Cell Disease Anemia Anemia, Hemolytic Anemia, Hemolytic, Congenital Anemia, Sickle Cell Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinopathies

Age range

2 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Amiens - Picardie Hospital Adults, Amiens, France

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

hydroxycarbamide

Drug

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

Other names: Siklos, Hydroxyurea

Primary outcomes

  1. 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

  2. Occurrence and incidence of the number of leg ulcers

    Time frame: 5 years

  3. Occurrence and incidence of male fertility impairment

    Time frame: 5 years

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

    Time frame: 5 years

Secondary outcomes

  1. Mean age of puberty

    Time frame: 5 years

    Document changes in fertility status in males and females

  2. Mean number of patients with the use of a contraception, incidence of the use of semen cryopreservation and of semen analysis performed

    Time frame: 5 years

    Document changes in fertility status in males and females

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

    Time frame: 5 years

    Document changes in fertility status in males and females

  4. Incidence of semen analysis performed

    Time frame: 5 years

    Document changes in fertility status in males and females

  5. Management of Siklos® during pregnancy and outcome of pregnancy following exposure at the time of conception or at any time during the pregnancy

    Time frame: 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

  6. Frequency and reason for temporary or permanent discontinuations of Siklos®

    Time frame: 5 years

    Potential barriers to the compliance to the prescription of Siklos

  7. Number of switch and combination with alternative treatment

    Time frame: 5 years

  8. Measure of quality of life with SF12 questionnaire

    Time frame: 5 years

    Quality of life for adults SCD patients

Sponsors and collaborators

Lead sponsor

Theravia

Industry

Collaborators

  • International Clinical Trials Association

Registry information

Official study title

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

Important dates

Study start
2020
Primary completion
2025
Study completion
2025
First posted
Jan 13, 2021
Registry last updated
Aug 27, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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