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

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ClinicalTrials.gov Identifier: NCT04707235
Recruitment Status : Recruiting
First Posted : January 13, 2021
Last Update Posted : January 13, 2021
Sponsor:
Collaborator:
International Clinical Trials Association
Information provided by (Responsible Party):
ADDMEDICA SASA

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®.

Condition or disease Intervention/treatment
Sickle Cell Disease Drug: Hydroxycarbamide

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Study Type : Observational
Estimated Enrollment : 2500 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: ESCORT-HU Extension: European Sickle Cell Disease Cohort - Hydroxyurea - Extension
Actual Study Start Date : August 21, 2020
Estimated Primary Completion Date : August 21, 2025
Estimated Study Completion Date : August 21, 2025

Resource links provided by the National Library of Medicine

Drug Information available for: Hydroxyurea

Group/Cohort Intervention/treatment
Sickle cell disease patients treated with Siklos Drug: Hydroxycarbamide
Patients, aged ≥ 2 years old, with symptomatic SCD, treated with Siklos®.
Other Names:
  • Siklos
  • Hydroxyurea




Primary Outcome Measures :
  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 Outcome Measures :
  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



Information from the National Library of Medicine

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Ages Eligible for Study:   2 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04707235


Contacts
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Contact: Corinne Duguet, MD + 33 1 72 69 01 86 corinne.duguet@addmedica.com
Contact: Laura Thomas-bourgneuf +33 1 72 69 01 86 laura.thomas-bourgneuf@addmedica.com

Locations
Show Show 79 study locations
Sponsors and Collaborators
ADDMEDICA SASA
International Clinical Trials Association
Investigators
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Study Chair: Mariane de Montalembert, MD, PhD Hospital Necker enfants malades
Study Chair: Frédéric Galactéros, MD, PhD Henri Mondor University Hospital
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Responsible Party: ADDMEDICA SASA
ClinicalTrials.gov Identifier: NCT04707235    
Other Study ID Numbers: ESCORT-HU Extension
First Posted: January 13, 2021    Key Record Dates
Last Update Posted: January 13, 2021
Last Verified: January 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn
Hydroxyurea
Antineoplastic Agents
Antisickling Agents
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Nucleic Acid Synthesis Inhibitors