Study of Dose Confirmation and Safety of Crizanlizumab in Pediatric Sickle Cell Disease Patients

Phase 1

• Conditions: Sickle Cell Disease with Vaso-Occlusive Crisis; MedDRA version: 20.0Level: LLTClassification code 10040644Term: Sickle cell diseaseSystem Organ Class: 100000004850; MedDRA version: 20.1Level: LLTClassification code 10002077Term: Anaemia sickle cellSystem Organ Class: 100000004850; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2017-001747-12-ES
• Lead Sponsor: ovartis Farmacéutica, S.A.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2019-01-23
• Last Update Posted: 16 May 2022

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 100

Inclusion Criteria

•Male or female patients aged 2 to <18 years (Group 3 will be expanded to allow enrolment of patients aged 6 to <24 months (and at least 6 kg) in Part B once the appropriate dose is confirmed in 2 to <6 year old patients)
•Confirmed diagnosis of sickle cell disease (SCD) (e.g. any genotype including HbSS, HbSC, HbSß0-thalassemia, HbSß+-thalassemia, and others) by hemoglobin electrophoresis or high performance liquid chromatography (HPLC) performed locally.
•Experienced at least 1 VOC within the preceding 12 months, as determined by medical history. Prior VOC must have resolved at least 7 days prior to the first dose in the study and should include all the following:
1.the occurrence of appropriate symptoms (see VOC definition in protocol Section 7.2.1.1)
2.either a visit to a medical facility or healthcare professional,
3.receipt of oral/parenteral opioid or other non-opioid parenteral analgesia.
•If receiving HU/HC or erythropoietin stimulating agent, must have been receiving the drug for at least 6 months prior to Screening and plan to continue taking at the same dose and schedule during the trial. Dose alterations of HU/HC during Part A are not allowed, and if this occurs, the patient will enter directly to the Part B.
•Received standard age-appropriate care for SCD, including penicillin prophylaxis, pneumococcal immunization, and parental education
•Transcranial Doppler (TCD) considered low risk within the past 6 months (for 2 to 16 years).

Other inclusion criteria as per protocol may apply
Are the trial subjects under 18? yes
Number of subjects for this age range: 100
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range 0
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range 0

Exclusion Criteria

•History of stem cell transplant.
•Received any blood products within 30 days of Day 1 dosing.
•Participating and maintaining in a chronic transfusion program (preplanned series of transfusions for prophylactic purposes).
•Patients with bleeding disorders
•Planning on undergoing an exchange transfusion during the duration of the study. Patients requiring episodic transfusion in response to worsened anemia or VOC are permitted.
•Contraindication or hypersensitivity to any drug from similar class as study drug or to any excipients of the study drug formulation.
•Planning to initiate or terminate HU/HC while on study, other than for safety reasons
•Significant active infection or immune deficiency (including chronic use of immunosuppressive drugs) in the opinion of the investigator.

Other exclusion criteria as per protocol may apply

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified