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

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

Not yet recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2017-001747-12-ES

Enrollment

100

Registered

2019-01-23

Start date

2018-12-28

Completion date

Unknown

Last updated

2022-05-16

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Sickle Cell Disease with Vaso-Occlusive Crisis MedDRA version: 20.0 Level: LLT Classification code 10040644 Term: Sickle cell disease System Organ Class: 100000004850 MedDRA version: 20.1 Level: LLT Classification code 10002077 Term: Anaemia sickle cell System Organ Class: 100000004850

Interventions

Product Name: crizanlizumab Product Code: SEG101 Pharmaceutical Form: Concentrate for solution for infusion INN or Proposed INN: CRIZANLIZUMAB Current Sponsor code: SEG101 Concentration unit: mg/ml mi

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: •Male or female patients aged 2 to =65 years) no F.1.3.1 Number of subjects for this age range 0

Exclusion criteria

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

Design outcomes

Primary

Measure	Time frame
Primary end point(s): 1.PK (AUCd15) after 1st dose Confirm appropriate dosing of crizanlizumab in patients aged 2 to < 18 years (Parts A) 2.PD (AUCd15) after 1st dose Confirm appropriate dosing of crizanlizumab in patients aged 2 to < 18 years (Parts A) 3.PK (AUCtau) after 5th dose Confirm appropriate dosing of Crizanlizumab in patients aged 2 to < 18 years old 4.PD (AUCtau) after 5th dose Confirm appropriate dosing of Crizanlizumab in patients aged 2 to < 18 years old 5.PK (Cmax) after 1st dose and 5th dose Confirm appropriate dosing of crizanlizumab in patients aged 2 to < 18 years (Parts A) 6.PK pre-dose concentrations Confirm appropriate dosing of crizanlizumab in patients aged 6 months to less than 24 months of age (Part B) 7.Frequency of any adverse events (AEs) as a measure of safety and tolerability Safety of crizanlizumab in patients aged 6 months to < 18 years (Parts A and B);Timepoint(s) of evaluation of this end point: 1. Day 15 2. Day 15 3. Week 15 4. Week 15 5. Week 1 and Week 15 6. Week 3 and Week 19 7. 6 months, 2 years;Main Objective: - To confirm and establish appropriate dosing of crizanlizumab in patients ages 6 months to <18 years at the time of study entry (Part A and B) - To evaluate the safety of crizanlizumab in patients ages 6 months to <18 years at the time of study entry (Parts A and B);Secondary Objective: - To assess the long-term efficacy of crizanlizumab in 6 months to < 18 year old patients at the time of study entry (Parts A and B) - To assess other safety measures in patients aged 6 months to < 18 years at the time of study entry - To characterize long-term PK and PD of crizanlizumab in patients ages 6 months to <18 years at the time of study entry	—

Measure

Time frame

Primary end point(s): 1.PK (AUCd15) after 1st dose Confirm appropriate dosing of crizanlizumab in patients aged 2 to < 18 years (Parts A) 2.PD (AUCd15) after 1st dose Confirm appropriate dosing of crizanlizumab in patients aged 2 to < 18 years (Parts A) 3.PK (AUCtau) after 5th dose Confirm appropriate dosing of Crizanlizumab in patients aged 2 to < 18 years old 4.PD (AUCtau) after 5th dose Confirm appropriate dosing of Crizanlizumab in patients aged 2 to < 18 years old 5.PK (Cmax) after 1st dose and 5th dose Confirm appropriate dosing of crizanlizumab in patients aged 2 to < 18 years (Parts A) 6.PK pre-dose concentrations Confirm appropriate dosing of crizanlizumab in patients aged 6 months to less than 24 months of age (Part B) 7.Frequency of any adverse events (AEs) as a measure of safety and tolerability Safety of crizanlizumab in patients aged 6 months to < 18 years (Parts A and B);Timepoint(s) of evaluation of this end point: 1. Day 15 2. Day 15 3. Week 15 4. Week 15 5. Week 1 and Week 15 6. Week 3 and Week 19 7. 6 months, 2 years;Main Objective: - To confirm and establish appropriate dosing of crizanlizumab in patients ages 6 months to <18 years at the time of study entry (Part A and B) - To evaluate the safety of crizanlizumab in patients ages 6 months to <18 years at the time of study entry (Parts A and B);Secondary Objective: - To assess the long-term efficacy of crizanlizumab in 6 months to < 18 year old patients at the time of study entry (Parts A and B) - To assess other safety measures in patients aged 6 months to < 18 years at the time of study entry - To characterize long-term PK and PD of crizanlizumab in patients ages 6 months to <18 years at the time of study entry

—

Secondary

Measure	Time frame
Secondary end point(s): 1.Number of Vaso Occusive Crisis (VOC) events leading to healthcare visit in clinic/ER/hospital To assess the long-term efficacy of crizanlizumab in 6 months to 18 years 13.PD pre-dose concentrations prior to each study drug dose. Characterize long-term PK and PD of crizanlizumab in patients aged 6 months to >18 years 14.Percentage P-selectin inhibition prior to dosing Characterize long-term PK and PD of crizanlizumab in patients aged 6 months to >18 years;Timepoint(s) of evaluation of this end point: 1. 6 months, 2 years 2. 6 months, 2 years 3. 6 months, 2 years 4. 6 months, 2 years 5. 6 months, 2 years 6. 6 months, 2 years 7. 6 months, 2 years 8. 6 months, 2 years 9. Week 1, Week 3, Week 15, Week 27 and End of Treatment (EOT) 10. Screening, Week 7, Week 11, week 15, week 27 and Week 51 11. Screening, Week 51 and End of Treatment (EOT) 12. Week 1, Week 3, Week 7, Week 15, Week 19, Week 23, Week 27, Week 31, Week 35, Week 39, Week 43, Week 47 and Week 51 13. Week 1, Week 3, Week 7, Week 15, Week 19, Week 23, Week 27, Week 31, Week 35, Week 39, Week 43, Week 47 and Week 51 14. Week 3, Week 15, Week 27 and Week 51	—

Measure

Time frame

Secondary end point(s): 1.Number of Vaso Occusive Crisis (VOC) events leading to healthcare visit in clinic/ER/hospital To assess the long-term efficacy of crizanlizumab in 6 months to 18 years 13.PD pre-dose concentrations prior to each study drug dose. Characterize long-term PK and PD of crizanlizumab in patients aged 6 months to >18 years 14.Percentage P-selectin inhibition prior to dosing Characterize long-term PK and PD of crizanlizumab in patients aged 6 months to >18 years;Timepoint(s) of evaluation of this end point: 1. 6 months, 2 years 2. 6 months, 2 years 3. 6 months, 2 years 4. 6 months, 2 years 5. 6 months, 2 years 6. 6 months, 2 years 7. 6 months, 2 years 8. 6 months, 2 years 9. Week 1, Week 3, Week 15, Week 27 and End of Treatment (EOT) 10. Screening, Week 7, Week 11, week 15, week 27 and Week 51 11. Screening, Week 51 and End of Treatment (EOT) 12. Week 1, Week 3, Week 7, Week 15, Week 19, Week 23, Week 27, Week 31, Week 35, Week 39, Week 43, Week 47 and Week 51 13. Week 1, Week 3, Week 7, Week 15, Week 19, Week 23, Week 27, Week 31, Week 35, Week 39, Week 43, Week 47 and Week 51 14. Week 3, Week 15, Week 27 and Week 51

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Countries

Belgium, Brazil, Canada, Colombia, France, Germany, India, Italy, Lebanon, Oman, Spain, Switzerland, Turkey, United Kingdom, United States

Contacts

Public ContactTrial Monitoring Organization (TMo)

Novartis Farmacéutica, S.A.

eecc.novartis@novartis.com+34 90 0353036

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 13, 2026