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

August 26, 2024 updated by: Theravia

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

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 Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

Study Type

Observational

Enrollment (Actual)

2093

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • France
      • Amiens, France, 80054
        • Amiens - Picardie Hospital Adults
      • Amiens, France, 80054
        • Amiens Picardie Hospital Children
      • Amiens, France
        • Clinique de l'Europe Amiens
      • Angers, France, 49933
        • Angers Hospital Adults
      • Angers, France, 49933
        • Angers Hospital Center Children
      • Aulnay-sous-Bois, France, 93602
        • Robert Ballanger Hospital Adults
      • Aulnay-sous-Bois, France, 93602
        • Robert Ballanger Hospital Children
      • Bobigny, France, 93009
        • Avicenne Hospital Adults
      • Bondy, France, 93143
        • Jean Verdier Hospital Children
      • Bordeaux, France, 33000
        • Bordeaux Hospital Adults
      • Bordeaux, France, 33000
        • Bordeaux Hospital Children
      • Boulogne-Billancourt, France, 92100
        • Ambroise Paré Hospital Children
      • Chambray-lès-Tours, France, 37170
        • Tours Regional University Hospital Center Adults
      • Clermont-Ferrand, France, 63003
        • Estaing Hospital Children
      • Colombes, France, 92700
        • Louis Mourier Hospital Adults
      • Colombes, France, 92700
        • Louis Mourier Hospital Children
      • Corbeil-Essonnes, France
        • Centre hospitalier Sud Francilien
      • Corbeil-Essonnes, France, 91100
        • Sud Francilien Hospital Center Adults
      • Creil, France, 60100
        • Sud Oise Public Hospital Group Adults
      • Créteil, France, 94010
        • Henri Mondor Hospital Adults
      • Créteil, France, 94010
        • Intercommunal Hospital Center of Créteil Adults and Children
      • Dreux, France, 28102
        • Dreux Hospital Center Children
      • Gonesse, France, 95500
        • Gonesse Hospital Children
      • Jossigny, France, 77600
        • Marne-La-Vallée Hospital Center Children
      • La Tronche, France, 38700
        • Grenoble Hospital Adults
      • La Tronche, France, 38700
        • Grenoble Hospital Children
      • Le Kremlin-Bicêtre, France
        • Hôpital Bicêtre
      • Le Kremlin-Bicêtre, France, 94270
        • Bicêtre Hospital Adults
      • Le Mans, France
        • CH du Mans
      • Lille, France, 59037
        • Jeanne De Flandre Hospital Children
      • Lille, France
        • Hôpital Saint-Vincent de Paul
      • Limoges, France
        • Hôpital Mère-Enfant - CHU de Limoges
      • Lyon, France, 69003
        • Edouard Herriot Hospital Adults
      • Lyon, France, 69373
        • Hematology and oncology institute Children
      • Marseille, France, 13005
        • Timone Hospital Adults
      • Marseille, France
        • Timone Hospital Children
      • Meaux, France, 77100
        • Meaux Hospital Center Adults
      • Meaux, France, 77100
        • Meaux Hospital Center Children
      • Montpellier, France, 34070
        • St Eloi Hospital Adults
      • Montpellier, France, 34295
        • Montpellier Hospital Children
      • Mulhouse, France, 68100
        • Emile Muller Hospital Adults
      • Nantes, France, 44000
        • Nantes Hospital Children
      • Nantes, France, 44093
        • Nantes Hospital Adults
      • Nice, France, 06200
        • Nice Hospital Children
      • Orléans, France, 45066
        • Orléans Hospital Adults
      • Paris, France, 75012
        • Armand Trousseau Hospital Children
      • Paris, France, 75012
        • Saint-Antoine Hospital Adults
      • Paris, France, 75015
        • Georges Pompidou European Hospital Adults
      • Paris, France, 75015
        • Necker Hospital Adults
      • Paris, France, 75015
        • Necker Hospital Children
      • Paris, France, 75019
        • Robert Debré Hospital Children
      • Perpignan, France
        • Centre Hospitalier Perpignan
      • Poitiers, France, 86020
        • Poitiers Hospital Adults
      • Reims, France, 51100
        • American Hospital Children
      • Reims, France, 51100
        • Reims Hospital Adults
      • Rennes, France, 35033
        • Pontchaillou Hospital Adults
      • Rouen, France, 76000
        • Charles Nicolle Hospital Adults
      • Rouen, France, 76000
        • Rouen Hospital Children
      • Saint-Brieuc, France, 22000
        • Yves Le Foll. Hospital Center Children
      • Saint-Denis, France, 93200
        • Delafontaine Hospital Center Adults
      • Saint-Denis, France, 93200
        • Delafontaine Hospital Center Children
      • Saint-Quentin, France, 02321
        • Saint-Quentin Hospital Center Adults
      • Strasbourg, France, 67098
        • Hautepierre Hospital Children
      • Toulouse, France, 31059
        • Children Hospital
      • Toulouse, France, 31059
        • Toulouse University Institute of cancer Adults
      • Tours, France, 37000
        • Clocheville Regional University Hospital Center Children
      • Vandoeuvre les nancy, France, 54500
        • Nancy Hospital Children
      • Versailles, France, 78157
        • Versailles Hospital Center Children
  • French Guiana
      • Cayenne, French Guiana, 97300
        • CH de Cayenne
      • Kourou, French Guiana, 97387
        • Centre hospitalier de Kourou Children
      • Saint-Laurent-du-Maroni, French Guiana, 97393
        • Centre Hospitalier de l'Ouest Guyanais Franck Joly Adults
      • Saint-Laurent-du-Maroni, French Guiana, 97393
        • Centre Hospitalier de l'Ouest Guyanais Franck Joly Children
  • Germany
      • Berlin, Germany
        • Charité Hospital Children
      • Düsseldorf, Germany
        • Düsseldorf Hospital Children
      • Freiburg, Germany
        • Freiburg Hospital Children
      • Hamburg, Germany
        • Hamburg Hospital Children
      • Koblenz, Germany
        • Koblenz Hospital Children
  • Greece
      • Athens, Greece
        • Laiko General Hospital Adults
  • Guadeloupe
      • Pointe-à-Pitre, Guadeloupe, 97159
        • Hôpital RICOU - CHU Pointe-à-Pitre Abymes Children
      • Pointe-à-Pitre, Guadeloupe, 97159
        • Hôpital RICOU - CHU Pointe-à-Pitre Abymes
  • Italy
      • Modena, Italy
        • AUO Policlinico di Modena
      • Napoli, Italy
        • Napoli Hospital Children
      • Padova, Italy
        • Padova Hospital Children
      • Verona, Italy
        • Verona Hospital Children
  • Martinique
      • Fort-de-France, Martinique, 97261
        • CHU Fort de France Children
      • Le Lamentin, Martinique, 97232
        • CHU Martinique Adults
  • Réunion
      • Saint-Pierre, Réunion
        • CHU Sud Réunion

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

1 year and older (Child, Adult, Older Adult)

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.

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Prospective

Cohorts and Interventions

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:
  • Hydroxyurea
  • Siklos

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
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
5 years
Occurrence and incidence of the number of leg ulcers
Time Frame: 5 years
5 years
Occurrence and incidence of male fertility impairment
Time Frame: 5 years
5 years
Occurrence and incidence of serious unexpected AEs causally related to Siklos®
Time Frame: 5 years
5 years

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Mean age of puberty
Time Frame: 5 years
Document changes in fertility status in males and females
5 years
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
5 years
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
5 years
Incidence of semen analysis performed
Time Frame: 5 years
Document changes in fertility status in males and females
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
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
5 years
Frequency and reason for temporary or permanent discontinuations of Siklos®
Time Frame: 5 years
Potential barriers to the compliance to the prescription of Siklos
5 years
Number of switch and combination with alternative treatment
Time Frame: 5 years
5 years
Measure of quality of life with SF12 questionnaire
Time Frame: 5 years
Quality of life for adults SCD patients
5 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Theravia

Collaborators

International Clinical Trials Association

Investigators

  • Study Chair: Mariane de Montalembert, MD, PhD, Hôspital Necker Enfants Malades
  • Study Chair: Frédéric Galactéros, MD, PhD, Henri Mondor University Hospital

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

August 21, 2020

Primary Completion (Estimated)

August 21, 2025

Study Completion (Estimated)

August 21, 2025

Study Registration Dates

First Submitted

December 22, 2020

First Submitted That Met QC Criteria

January 12, 2021

First Posted (Actual)

January 13, 2021

Study Record Updates

Last Update Posted (Actual)

August 27, 2024

Last Update Submitted That Met QC Criteria

August 26, 2024

Last Verified

August 1, 2024

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ESCORT-HU Extension

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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