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

• ClinicalTrials.gov Identifier: NCT04707235
• Recruitment Status: Recruiting
• First Posted: January 13, 2021
• Last Update Posted: March 25, 2022
• Sponsor: ADDMEDICA SASA
• Collaborator: International Clinical Trials Association
• Information provided by (Responsible Party): ADDMEDICA SASA

Tracking Information

• First Submitted Date: December 22, 2020
• First Posted Date: January 13, 2021
• Last Update Posted Date: March 25, 2022
• Actual Study Start Date: August 21, 2020
• Estimated Primary Completion Date: August 21, 2025 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: January 12, 2021)

• 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 ]

• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: January 12, 2021)

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

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: ESCORT-HU Extension: European Sickle Cell Disease Cohort - Hydroxyurea - Extension Study
• Official Title: ESCORT-HU Extension: European Sickle Cell Disease Cohort - Hydroxyurea - Extension
• 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®.
• Detailed Description: Not Provided
• Study Type: Observational
• Study Design: Observational Model: Cohort; Time Perspective: Prospective
• Target Follow-Up Duration: Not Provided
• Biospecimen: Not Provided
• 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.

• Condition: Sickle Cell Disease

Intervention

Drug: Hydroxycarbamide

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

Other Names:

• Siklos
• Hydroxyurea

Study Groups/Cohorts

Sickle cell disease patients treated with Siklos

Intervention: Drug: Hydroxycarbamide

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Recruiting
• Estimated Enrollment (submitted: January 12, 2021): 2500
• Original Estimated Enrollment: Same as current
• Estimated Study Completion Date: August 21, 2025
• Estimated Primary Completion Date: August 21, 2025 (Final data collection date for primary outcome measure)

Eligibility 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.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 2 Years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Contacts

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

• Listed Location Countries: France, French Guiana, Germany, Greece, Guadeloupe, Italy, Martinique

Administrative Information

• NCT Number: NCT04707235
• Other Study ID Numbers: ESCORT-HU Extension
• Has Data Monitoring Committee: No

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: No

• Current Responsible Party: ADDMEDICA SASA
• Original Responsible Party: Same as current
• Current Study Sponsor: ADDMEDICA SASA
• Original Study Sponsor: Same as current
• Collaborators: International Clinical Trials Association

Investigators

• Study Chair: Mariane de Montalembert, MD, PhD; Hospital Necker enfants malades
• Study Chair: Frédéric Galactéros, MD, PhD; Henri Mondor University Hospital

• PRS Account: ADDMEDICA SASA
• Verification Date: March 2022