Sickle Cell DiseaseNCT04707235
ESCORT-HU Extension: European Sickle Cell Disease Cohort - Hydroxyurea - Extension Study
An observational study of Sickle Cell Disease, run by Theravia at 87 sites in 8 countries.
Trial 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… Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- Hydroxycarbamide
- How many people
- 2,093 participants
- Where
- 87 sitesin 8 countries
- Study dates
- 21 Aug 2020to 21 Aug 2025
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
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.
Who may be able to join
- 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,
Show all 12Show fewer
- 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.
Who cannot join
None listed.
Trial locations and contacts
87 sites in 8 countries.
87 sites in 8 countries
France68 sites
Amiens - Picardie Hospital Adults
Amiens, 80054
Amiens Picardie Hospital Children
Amiens, 80054
Clinique de l'Europe Amiens
Amiens
Angers Hospital Adults
Angers, 49933
Angers Hospital Center Children
Angers, 49933
Robert Ballanger Hospital Adults
Aulnay-sous-Bois, 93602
Robert Ballanger Hospital Children
Aulnay-sous-Bois, 93602
Avicenne Hospital Adults
Bobigny, 93009
Jean Verdier Hospital Children
Bondy, 93143
Bordeaux Hospital Adults
Bordeaux, 33000
Bordeaux Hospital Children
Bordeaux, 33000
Ambroise Paré Hospital Children
Boulogne-Billancourt, 92100
Tours Regional University Hospital Center Adults
Chambray-lès-Tours, 37170
Estaing Hospital Children
Clermont-Ferrand, 63003
Louis Mourier Hospital Adults
Colombes, 92700
Louis Mourier Hospital Children
Colombes, 92700
Sud Francilien Hospital Center Adults
Corbeil-Essonnes, 91100
Centre Hospitalier Sud Francilien
Corbeil-Essonnes
Sud Oise Public Hospital Group Adults
Creil, 60100
Henri Mondor Hospital Adults
Créteil, 94010
Intercommunal Hospital Center of Créteil Adults and Children
Créteil, 94010
Dreux Hospital Center Children
Dreux, 28102
Gonesse Hospital Children
Gonesse, 95500
Marne-La-Vallée Hospital Center Children
Jossigny, 77600
Grenoble Hospital Adults
La Tronche, 38700
Grenoble Hospital Children
La Tronche, 38700
Bicêtre Hospital Adults
Le Kremlin-Bicêtre, 94270
Hôpital Bicêtre
Le Kremlin-Bicêtre
CH du Mans
Le Mans
Jeanne De Flandre Hospital Children
Lille, 59037
Hôpital Saint-Vincent de Paul
Lille
Hôpital Mère-Enfant - CHU de Limoges
Limoges
Edouard Herriot Hospital Adults
Lyon, 69003
Hematology and oncology institute Children
Lyon, 69373
Timone Hospital Adults
Marseille, 13005
Timone Hospital Children
Marseille
Meaux Hospital Center Adults
Meaux, 77100
Meaux Hospital Center Children
Meaux, 77100
St Eloi Hospital Adults
Montpellier, 34070
Montpellier Hospital Children
Montpellier, 34295
Emile Muller Hospital Adults
Mulhouse, 68100
Nantes Hospital Children
Nantes, 44000
Nantes Hospital Adults
Nantes, 44093
Nice Hospital Children
Nice, 06200
Orléans Hospital Adults
Orléans, 45066
Armand Trousseau Hospital Children
Paris, 75012
Saint-Antoine Hospital Adults
Paris, 75012
Georges Pompidou European Hospital Adults
Paris, 75015
Necker Hospital Adults
Paris, 75015
Necker Hospital Children
Paris, 75015
Robert Debré Hospital Children
Paris, 75019
Centre hospitalier Perpignan
Perpignan
Poitiers Hospital Adults
Poitiers, 86020
American Hospital Children
Reims, 51100
Reims Hospital Adults
Reims, 51100
Pontchaillou Hospital Adults
Rennes, 35033
Charles Nicolle Hospital Adults
Rouen, 76000
Rouen Hospital Children
Rouen, 76000
Yves Le Foll. Hospital Center Children
Saint-Brieuc, 22000
Delafontaine Hospital Center Adults
Saint-Denis, 93200
Delafontaine Hospital Center Children
Saint-Denis, 93200
Saint-Quentin Hospital Center Adults
Saint-Quentin, 02321
Hautepierre Hospital Children
Strasbourg, 67098
Children Hospital
Toulouse, 31059
Toulouse University Institute of cancer Adults
Toulouse, 31059
Clocheville Regional University Hospital Center Children
Tours, 37000
Nancy Hospital Children
Vandœuvre-lès-Nancy, 54500
Versailles Hospital Center Children
Versailles, 78157
Germany5 sites
Charité Hospital Children
Berlin
Düsseldorf Hospital Children
Düsseldorf
Freiburg Hospital Children
Freiburg im Breisgau
Hamburg Hospital Children
Hamburg
Koblenz Hospital Children
Koblenz
French Guiana4 sites
CH de Cayenne
Cayenne, 97300
Centre hospitalier de Kourou Children
Kourou, 97387
Centre Hospitalier de l'Ouest Guyanais Franck Joly Adults
Saint-Laurent-du-Maroni, 97393
Centre Hospitalier de l'Ouest Guyanais Franck Joly Children
Saint-Laurent-du-Maroni, 97393
Italy4 sites
AUO Policlinico di Modena
Modena
Napoli Hospital Children
Naples
Padova Hospital Children
Padova
Verona Hospital Children
Verona
Guadeloupe2 sites
Hôpital RICOU - CHU Pointe-à-Pitre Abymes Children
Pointe-à-Pitre, 97159
Hôpital RICOU - CHU Pointe-à-Pitre Abymes
Pointe-à-Pitre, 97159
Martinique2 sites
CHU Fort de France Children
Fort-de-France, 97261
CHU Martinique Adults
Le Lamentin, 97232
About this study
Official title: 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®.
- Conditions
- Sickle Cell Disease
- Being tested
- Hydroxycarbamide
- Medical subject headings
- Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
HydroxycarbamideDrug
Also called Siklos, Hydroxyurea
Patients, aged ≥ 2 years old, with symptomatic SCD, treated with Siklos®.
| Study group | Role | What they receive |
|---|---|---|
| Sickle cell disease patients treated with Siklos | Not described Receives: Drug: Hydroxycarbamide |
What is being measured
The primary outcome is the main question the study is designed to answer.
Occurrence and incidence of the number of malignancies, leg ulcers, male fertility impairment and serious unexpected AEs causally related to Siklos®
Measured: 5 years
Occurrence and incidence of the number of leg ulcers
Measured: 5 years
Occurrence and incidence of male fertility impairment
Measured: 5 years
Occurrence and incidence of serious unexpected AEs causally related to Siklos®
Measured: 5 years
Secondary outcomes (8)
Mean age of puberty
Document changes in fertility status in males and females
Measured: 5 years
Mean number of patients with the use of a contraception, incidence of the use of semen cryopreservation and of semen analysis performed
Document changes in fertility status in males and females
Measured: 5 years
Incidence of the use of semen cryopreservation and of semen analysis performed
Document changes in fertility status in males and females
Measured: 5 years
Incidence of semen analysis performed
Document changes in fertility status in males and females
Measured: 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
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
Measured: 5 years
Frequency and reason for temporary or permanent discontinuations of Siklos®
Potential barriers to the compliance to the prescription of Siklos
Measured: 5 years
Number of switch and combination with alternative treatment
Measured: 5 years
Measure of quality of life with SF12 questionnaire
Quality of life for adults SCD patients
Measured: 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Jan 2021
- Study start21 Aug 2020
- Primary completion21 Aug 2025 (estimated)
- Study completion21 Aug 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 2,093 participants (actual)
Sponsor and investigators
- Lead sponsor
- Theravia
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- International Clinical Trials Association
- Responsible party
- Sponsor
- Investigators
- Mariane de Montalembert, MD, PhD, Study chair, Hospital Necker enfants maladesFrédéric Galactéros, MD, PhD, Study chair, Henri Mondor University Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04707235
- Sponsor's protocol ID
- ESCORT-HU Extension
- Status last verified
- Aug 2024
- First submitted
- 22 Dec 2020
- Last updated
- 27 Aug 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Aug 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- ESCORT-HU Extension: European Sickle Cell Disease Cohort - Hydroxyurea - Extension
- ClinicalTrials.gov ID
- NCT04707235
- Acronym / sponsor ID
- ESCORT-HU Extension
- Conditions
- Sickle Cell Disease
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 2,093 participants (actual)
- Lead sponsor
- TheraviaIndustry (pharma, biotech, devices)
- Collaborators
- International Clinical Trials Association
- Study start
- 21 Aug 2020 (actual)
- Primary completion
- 21 Aug 2025 (estimated)
- Study completion
- 21 Aug 2025 (estimated)
- First posted
- 13 Jan 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Sickle cell disease patients treated with Siklos | – | Drug: Hydroxycarbamide |
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- 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.
Key inclusion criteria (12 listed)
- 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:
Key exclusion criteria (0 listed)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| HydroxycarbamideAlso: Siklos, HydroxyureaPatients, aged ≥ 2 years old, with symptomatic SCD, treated with Siklos®. | Drug | Sickle cell disease patients treated with Siklos |
Primary outcome
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
Occurrence and incidence of the number of leg ulcers
Time frame: 5 years
Occurrence and incidence of male fertility impairment
Time frame: 5 years
Occurrence and incidence of serious unexpected AEs causally related to Siklos®
Time frame: 5 years
8 secondary outcomes
Mean age of puberty
Time frame: 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
Incidence of the use of semen cryopreservation and of semen analysis performed
Time frame: 5 years
Incidence of semen analysis performed
Time frame: 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
Frequency and reason for temporary or permanent discontinuations of Siklos®
Time frame: 5 years
Number of switch and combination with alternative treatment
Time frame: 5 years
Measure of quality of life with SF12 questionnaire
Time frame: 5 years
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Aug 2024.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Mariane de Montalembert, MD, PhD study chair
Hospital Necker enfants malades
Frédéric Galactéros, MD, PhD study chair
Henri Mondor University Hospital
87 sites in 8 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Sickle Cell Disease, run by Theravia at 87 sites in 8 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 12 inclusion and 0 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 27 Aug 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 87 sites in France, Germany, French Guiana, Italy, Guadeloupe and 3 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Aug 2024.