Kinderonkologie und —hämatologie - CSEG101B2201 - Study of Dose Confirmation and Safety of Crizanlizumab in Pediatric Sickle Cell Disease Patients (Solace)

PrüfplancodeISRCTNEudraCTClinicaltrials.govDRKS
CSEG101B22012017-001747-12NCT03474965

A phase 2 Multicenter, Open-Label Study to Assess Appropriate Dosing and to Evaluate Safety of Crizanlizumab, with or without Hydroxyurea/Hydroxycarbamide, in Sequential, Descending Age Groups of Pediatric Sickle Cell Disease Patients with Vaso-Occlusive Crisis

Status: Aktiv (Rekrutierung geschlossen)

Studienziel / Fragestellung

Primäres Prüfziel

  • To confirm and establish appropriate dosing in participants from 6 months to less than 18 years of age (Parts A and B)
  • To evaluate the safety of crizanlizumab in participants from 6 months to less than 18 years of age (Parts A and B).

Sekundäre Prüfziele

  • To assess the long-term efficacy of crizanlizumab in participants aged 6 months to <18 year old, as measured by annualized rate of VOC events leading to healthcare visit in clinic/ER/hospital, annualized rate of VOC events treated at home, annualized rate of each subcategory of VOC event (uncomplicated pain crisis, acute chest syndrome, hepatic sequestration, splenic sequestration, priapism), annualized rate of Hospitalizations and ER visits (both overall and VOC-related), annualized rate of days of ER/hospitalization (both overall and VOC-related), annualized rate of Dactylitis events
  • To assess other safety measures in participants aged 6 months to < 18 year old in terms of frequency, seriousness, severity and causality of treatment emergent AEs, absolute change from baseline in hemoglobin, measurement of anti-drug antibodies (ADA) to crizanlizumab, ECG measures at the time of PK, growth and sexual maturation assessments.
  • Characterize the long-term PK and PD of crizanlizumab in participants aged 6 months to <18 years at the time of study entry as measured by pre-dose concentrations prior to each study drug dose and percentage of P-selectin inhibition prior to dosing.

Diagnose

  • Anämien
  • Kinderonkologie und -hämatologie
  • Sichelzellanämie

Confirmed diagnosis of  Sickle Cell Disease (all genotypes), with at least 1 VOC within the preceding 12 months

Patientenmerkmale

Einschlusskriterien

  • Male or female patients ages 2 to
  • Confirmed diagnosis of SCD (any genotype including HbSS, HbSC, HbSβ0-thalassemia, HbSβ+-thalassemia, and others) by hemoglobin electrophoresis and/or high-performance liquid chromatography (HPLC) performed locally. Confirmation of diagnosis by two accepted methods is recommended.
  • Experienced at least 1 VOC within the preceding 12 months prior to screening, as determined by medical history. Prior VOC must have resolved at least 7 days prior to the first dose in the study and must include all the following:
    • the occurrence of appropriate symptoms (see VOC definition in Section 7.2.1.1)
    • either a visit to a medical facility or healthcare professional
    • receipt of oral/parenteral opioid or parenteral non-steroidal anti-inflammatory drugs (NSAID).
  • If receiving HU/HC, L-glutamine or erythropoietin stimulating agent, must have been receiving the drug consistently for at least 6 months prior to Screening and plan to continue taking it at the same dose and schedule during the trial. Patients not receiving such drugs must have been off them for at least 6 months prior to screening.
  • Received standard age-appropriate care for SCD, including penicillin prophylaxis, pneumococcal immunization, and parental education.
  • Transcranial Doppler (TCD) for patients aged 2 to < 16 years at the time of screening, with HbSS, HbSβ0-thalassemia, and HbSD disease, indicating low risk for stroke (per investigator) as outlined in Section 7.2.2.6

(Protocol Version 04)

Ausschlusskriterien

  • History of stem cell transplant
  • Received any blood products within 30 days prior to Week 1 Day 1 dosing
  • Plan to participate in a chronic transfusion program (pre-planned series of transfusions for prophylactic purposes) or undergo exchange transfusions/plasmapheresis during the study. Patients requiring episodic transfusion (simple or exchange) in response to worsened anemia or VOC are permitted.
  • Patients with bleeding disorders
  • 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 or L-glutamine 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.
  • Patients under voxelotor within 30 days prior to screening or planning to initiate voxelotor while on study.

(Protocol Version 04)

Studiendesign

Phase II, Multizentrisch, Prospektiv, Einarmig, Open Label

Dokumente (passwortgeschützt)

Prüfzentren

Köln

Klinik und Poliklinik für Kinder- und Jugendmedizin

Studienbüro

    Early Clinical Trials

Status

Aktiv (Rekrutierung geschlossen)

Prüfer (Hauptprüfer im Zentrum)

Dr. med. Marc Hömberg

Stellvertretender Prüfer

  • Dr. med. Claudia Potthoff
  • Prof. Dr. med. Thorsten Simon

Studienkontakt im Prüfzentrum