NCT04707235

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®.

Trial Health

62
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
2,093

participants targeted

Target at P75+ for all trials

Timeline
Completed

Started Aug 2020

Longer than P75 for all trials

Geographic Reach
7 countries

19 active sites

Status
active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

August 21, 2020

Completed
4 months until next milestone

First Submitted

Initial submission to the registry

December 22, 2020

Completed
22 days until next milestone

First Posted

Study publicly available on registry

January 13, 2021

Completed
4.6 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 21, 2025

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

August 21, 2025

Completed
Last Updated

August 27, 2024

Status Verified

August 1, 2024

Enrollment Period

5 years

First QC Date

December 22, 2020

Last Update Submit

August 26, 2024

Conditions

Outcome Measures

Primary Outcomes (4)

  • 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 Outcomes (8)

  • Mean age of puberty

    5 years

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

    5 years

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

    5 years

  • Incidence of semen analysis performed

    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

    5 years

  • +3 more secondary outcomes

Study Arms (1)

Sickle cell disease patients treated with Siklos

Drug: Hydroxycarbamide

Interventions

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

Also known as: Siklos, Hydroxyurea
Sickle cell disease patients treated with Siklos

Eligibility Criteria

Age2 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-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.

You may qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (87)

Amiens - Picardie Hospital Adults

Amiens, 80054, France

Location

Amiens Picardie Hospital Children

Amiens, 80054, France

Location

Clinique de l'Europe Amiens

Amiens, France

Location

Angers Hospital Adults

Angers, 49933, France

Location

Angers Hospital Center Children

Angers, 49933, France

Location

Robert Ballanger Hospital Adults

Aulnay-sous-Bois, 93602, France

Location

Robert Ballanger Hospital Children

Aulnay-sous-Bois, 93602, France

Location

Avicenne Hospital Adults

Bobigny, 93009, France

Location

Jean Verdier Hospital Children

Bondy, 93143, France

Location

Bordeaux Hospital Adults

Bordeaux, 33000, France

Location

Bordeaux Hospital Children

Bordeaux, 33000, France

Location

Ambroise Paré Hospital Children

Boulogne-Billancourt, 92100, France

Location

Tours Regional University Hospital Center Adults

Chambray-lès-Tours, 37170, France

Location

Estaing Hospital Children

Clermont-Ferrand, 63003, France

Location

Louis Mourier Hospital Adults

Colombes, 92700, France

Location

Louis Mourier Hospital Children

Colombes, 92700, France

Location

Sud Francilien Hospital Center Adults

Corbeil-Essonnes, 91100, France

Location

Centre Hospitalier Sud Francilien

Corbeil-Essonnes, France

Location

Sud Oise Public Hospital Group Adults

Creil, 60100, France

Location

Henri Mondor Hospital Adults

Créteil, 94010, France

Location

Intercommunal Hospital Center of Créteil Adults and Children

Créteil, 94010, France

Location

Dreux Hospital Center Children

Dreux, 28102, France

Location

Gonesse Hospital Children

Gonesse, 95500, France

Location

Marne-La-Vallée Hospital Center Children

Jossigny, 77600, France

Location

Grenoble Hospital Adults

La Tronche, 38700, France

Location

Grenoble Hospital Children

La Tronche, 38700, France

Location

Bicêtre Hospital Adults

Le Kremlin-Bicêtre, 94270, France

Location

Hôpital Bicêtre

Le Kremlin-Bicêtre, France

Location

CH du Mans

Le Mans, France

Location

Jeanne De Flandre Hospital Children

Lille, 59037, France

Location

Hôpital Saint-Vincent de Paul

Lille, France

Location

Hôpital Mère-Enfant - CHU de Limoges

Limoges, France

Location

Edouard Herriot Hospital Adults

Lyon, 69003, France

Location

Hematology and oncology institute Children

Lyon, 69373, France

Location

Timone Hospital Adults

Marseille, 13005, France

Location

Timone Hospital Children

Marseille, France

Location

Meaux Hospital Center Adults

Meaux, 77100, France

Location

Meaux Hospital Center Children

Meaux, 77100, France

Location

St Eloi Hospital Adults

Montpellier, 34070, France

Location

Montpellier Hospital Children

Montpellier, 34295, France

Location

Emile Muller Hospital Adults

Mulhouse, 68100, France

Location

Nantes Hospital Children

Nantes, 44000, France

Location

Nantes Hospital Adults

Nantes, 44093, France

Location

Nice Hospital Children

Nice, 06200, France

Location

Orléans Hospital Adults

Orléans, 45066, France

Location

Armand Trousseau Hospital Children

Paris, 75012, France

Location

Saint-Antoine Hospital Adults

Paris, 75012, France

Location

Georges Pompidou European Hospital Adults

Paris, 75015, France

Location

Necker Hospital Adults

Paris, 75015, France

Location

Necker Hospital Children

Paris, 75015, France

Location

Robert Debré Hospital Children

Paris, 75019, France

Location

Centre hospitalier Perpignan

Perpignan, France

Location

Poitiers Hospital Adults

Poitiers, 86020, France

Location

American Hospital Children

Reims, 51100, France

Location

Reims Hospital Adults

Reims, 51100, France

Location

Pontchaillou Hospital Adults

Rennes, 35033, France

Location

Charles Nicolle Hospital Adults

Rouen, 76000, France

Location

Rouen Hospital Children

Rouen, 76000, France

Location

Yves Le Foll. Hospital Center Children

Saint-Brieuc, 22000, France

Location

Delafontaine Hospital Center Adults

Saint-Denis, 93200, France

Location

Delafontaine Hospital Center Children

Saint-Denis, 93200, France

Location

Saint-Quentin Hospital Center Adults

Saint-Quentin, 02321, France

Location

Hautepierre Hospital Children

Strasbourg, 67098, France

Location

Children Hospital

Toulouse, 31059, France

Location

Toulouse University Institute of cancer Adults

Toulouse, 31059, France

Location

Clocheville Regional University Hospital Center Children

Tours, 37000, France

Location

Nancy Hospital Children

Vandœuvre-lès-Nancy, 54500, France

Location

Versailles Hospital Center Children

Versailles, 78157, France

Location

CH de Cayenne

Cayenne, 97300, French Guiana

Location

Centre hospitalier de Kourou Children

Kourou, 97387, French Guiana

Location

Centre Hospitalier de l'Ouest Guyanais Franck Joly Adults

Saint-Laurent-du-Maroni, 97393, French Guiana

Location

Centre Hospitalier de l'Ouest Guyanais Franck Joly Children

Saint-Laurent-du-Maroni, 97393, French Guiana

Location

Charité Hospital Children

Berlin, Germany

Location

Düsseldorf Hospital Children

Düsseldorf, Germany

Location

Freiburg Hospital Children

Freiburg im Breisgau, Germany

Location

Hamburg Hospital Children

Hamburg, Germany

Location

Koblenz Hospital Children

Koblenz, Germany

Location

Laiko General Hospital Adults

Athens, Greece

Location

Hôpital RICOU - CHU Pointe-à-Pitre Abymes Children

Pointe-à-Pitre, 97159, Guadeloupe

Location

Hôpital RICOU - CHU Pointe-à-Pitre Abymes

Pointe-à-Pitre, 97159, Guadeloupe

Location

AUO Policlinico di Modena

Modena, Italy

Location

Napoli Hospital Children

Napoli, Italy

Location

Padova Hospital Children

Padua, Italy

Location

Verona Hospital Children

Verona, Italy

Location

CHU Fort de France Children

Fort-de-France, 97261, Martinique

Location

CHU Martinique Adults

Le Lamentin, 97232, Martinique

Location

CHU Sud Réunion

Saint-Pierre, Reunion

Location

MeSH Terms

Conditions

Anemia, Sickle Cell

Interventions

Hydroxyurea

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, CongenitalAnemia, HemolyticAnemiaHematologic DiseasesHemic and Lymphatic DiseasesHemoglobinopathiesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

UreaAmidesOrganic Chemicals

Study Officials

  • Mariane de Montalembert, MD, PhD

    Hospital Necker enfants malades

    STUDY CHAIR
  • Frédéric Galactéros, MD, PhD

    Henri Mondor University Hospital

    STUDY CHAIR

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

December 22, 2020

First Posted

January 13, 2021

Study Start

August 21, 2020

Primary Completion

August 21, 2025

Study Completion

August 21, 2025

Last Updated

August 27, 2024

Record last verified: 2024-08

Data Sharing

IPD Sharing
Will not share

Locations