Rollover Study for Patients With Sickle Cell Disease Who Have Completed a Prior Novartis-Sponsored Crizanlizumab Study

An Open-label, Multi-center, Phase IV, Rollover Study for Patients With Sickle Cell Disease Who Have Completed a Prior Novartis-Sponsored Crizanlizumab Study

This is a multi-center multi-national rollover study to allow continued access to crizanlizumab for patients with sickle cell disease (SCD) who are on crizanlizumab treatment in a Novartis-sponsored study (parent study) and are benefiting from the treatment as judged by the investigator.

Study Overview

• Status: Recruiting
• Conditions: Sickle Cell Disease
• Intervention / Treatment: Drug: Crizanlizumab

Detailed Description

There will be no screening period for this study as patients will transfer directly from parent studies. After providing informed consent, all eligible participants should start Crizanlizumab treatment at the earliest convenience following the treatment schedule of 28 days of the last dose in the parent study. Crizanlizumab will be administered at the same dose/schedule as in the parent study.

Study participants will have a safety follow up visit conducted 105 days after last administration of study treatment. The safety follow up at 105 days is not applicable for those participants who continue to receive Crizanlizumab after end of treatment visit either commercially or through PSDS.

The study is expected to remain open for 10 years from the first Patient's first visit (FPFV) in this clinical study or until study treatment becomes commercially available and is reimbursed in the respective indication or until such time that all enrolled patients no longer need treatment with Crizanlizumab, or a PSDS treatment plan is allowed and approved as per local laws and regulations, whichever comes first

• Study Type: Interventional
• Enrollment (Estimated): 130
• Phase: Phase 4

Contacts and Locations

• Study Contact: Name: Novartis Pharmaceuticals; Phone Number: 1-888-669-6682; Email: novartis.email@novartis.com
• Study Contact Backup: Name: Novartis Pharmaceuticals; Phone Number: +41613241111

Study Locations

• Belgium
  • Brussel, Belgium, 1000
    • Recruiting
    • Novartis Investigative Site
  • Laeken, Belgium, 1020
    • Recruiting
    • Novartis Investigative Site
  • Liege, Belgium, 4000
    • Recruiting
    • Novartis Investigative Site
• Brazil
  • BA
    • Salvador, BA, Brazil, 41253-190
      • Recruiting
      • Novartis Investigative Site
  • SP
    • Ribeirao Preto, SP, Brazil, 14051-140
      • Recruiting
      • Novartis Investigative Site
    • São Paulo, SP, Brazil, 01232-010
      • Recruiting
      • Novartis Investigative Site
• Colombia
  • Valle Del Cauca
    • Cali, Valle Del Cauca, Colombia, 760012
      • Recruiting
      • Novartis Investigative Site
• France
  • Creteil, France, 94010
    • Recruiting
    • Novartis Investigative Site
  • Paris cedex 15, France, 75015
    • Recruiting
    • Novartis Investigative Site
• Germany
  • Heidelberg, Germany, 69120
    • Recruiting
    • Novartis Investigative Site
• Italy
  • PD
    • Padova, PD, Italy, 35128
      • Recruiting
      • Novartis Investigative Site
  • TO
    • Orbassano, TO, Italy, 10043
      • Active, not recruiting
      • Novartis Investigative Site
• Lebanon
  • Beirut, Lebanon, 1107 2020
    • Recruiting
    • Novartis Investigative Site
  • Tripoli, Lebanon, 1434
    • Recruiting
    • Novartis Investigative Site
• Oman
  • Muscat, Oman, 123
    • Recruiting
    • Novartis Investigative Site
• Spain
  • Madrid, Spain, 28009
    • Recruiting
    • Novartis Investigative Site
• Turkey
  • Adana, Turkey, 01330
    • Recruiting
    • Novartis Investigative Site
  • Adana, Turkey, 01250
    • Recruiting
    • Novartis Investigative Site
  • Antakya / Hatay, Turkey, 31100
    • Recruiting
    • Novartis Investigative Site
• United States
  • Alabama
    • Birmingham, Alabama, United States, 35233
      • Recruiting
      • Novartis Investigative Site
      • Principal Investigator: Thomas Howard
      • Contact: Christy Patrick; Phone Number: 205-638-2792; Email: cpatrick@uabmc.edu
  • District of Columbia
    • Washington, District of Columbia, United States, 20010
      • Recruiting
      • Novartis Investigative Site
  • Georgia
    • Augusta, Georgia, United States, 30912
      • Recruiting
      • Novartis Investigative Site
  • North Carolina
    • Greenville, North Carolina, United States, 27834
      • Recruiting
      • Novartis Investigative Site
    • Greenville, North Carolina, United States, 27858
      • Recruiting
      • Novartis Investigative Site
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104-4399
      • Recruiting
      • Novartis Investigative Site
      • Principal Investigator: Helge D Hartung
      • Contact: Phone Number: 267-426-9338
  • Texas
    • Fort Worth, Texas, United States, 76104
      • Recruiting
      • Novartis Investigative Site
      • Contact: Patty Penn; Email: Patty.Penn2@cookchildrens.org
      • Principal Investigator: Clarissa Johnson

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 6 months and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Written informed consent/assent, according to local guidelines, signed by the adult patients. In the population under 18 years, it will be signed by the patient and/or by the parents or legal guardian prior to enrolling in the rollover study and receiving study medication
2. SCD patient currently enrolled in a Novartis-sponsored study receiving crizanlizumab and has fulfilled all the requirements in the parent study. Patient is currently benefiting from the treatment with crizanlizumab as determined by the investigator and has completed the treatment schedule as planned in the parent study
3. Patient has demonstrated compliance to the planned visit schedule in the parent study, and in the opinion of the investigator has shown willingness and ability to comply with future visit schedules

Exclusion Criteria:

1. Patient had permanently discontinued from crizanlizumab study treatment in the parent study before the parent study completion
2. Ongoing/unresolved treatment-related Grade 3 or higher AEs, and/or any ongoing AE requiring dose interruption. Patients meeting all other eligibility criteria may be enrolled once toxicities have resolved unless those toxicities were grade 4
3. Concurrent participation in any other investigational clinical trial other than the parent study or plan to participate in any other investigational clinical trial
4. Pregnant or nursing women
5. Women of childbearing potential who are unwilling to be on highly effective contraceptives during dosing and until 15 weeks after stopping treatment with crizanlizumab
6. SCD patients who do not meet parent study protocol criteria to continue with crizanlizumab

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Crizanlizumab; All participants will receive crizanlizumab (SEG101) at the same dose/schedule as in the parent study.	Drug: Crizanlizumab; Concentrate for solution for infusion for Intravenous use; Other Names: SEG101

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Not Applicable as this protocol is to provide an option for continued access to crizanlizumab for patients with Sickle Cell Disease who have completed a prior Novartis-sponsored Crizanlizumab study	Protocol to provide an option for continued access to crizanlizumab for patients with Sickle Cell Disease who have completed a prior Novartis-sponsored Crizanlizumab study, benefited from the treatment and do not have access to reimbursed, commercially available crizanlizumab.	Not Applicable - Study Completion

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of participants with treatment emergent adverse events	The number of participants with Frequency, severity and causality of treatment emergent adverse events will be collected.	from day of first dose of study medication to 105 days after last dose of study medication

Collaborators and Investigators

• Sponsor: Novartis Pharmaceuticals
• Investigators: Study Director: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Study record dates

Study Major Dates

• Study Start (Actual): June 10, 2021
• Primary Completion (Estimated): October 27, 2031
• Study Completion (Estimated): October 27, 2031

Study Registration Dates

• First Submitted: December 1, 2020
• First Submitted That Met QC Criteria: December 1, 2020
• First Posted (Actual): December 8, 2020

Study Record Updates

• Last Update Posted (Actual): July 27, 2023
• Last Update Submitted That Met QC Criteria: July 26, 2023
• Last Verified: July 1, 2023

More Information

Terms related to this study

• Keywords: SCD; crizanlizumab; P-selectin; SEG101; Sickle cell disease; VOC; Sickle cell disorder; Sickle cell anemia; Vaso-occlusive Crisis
• Additional Relevant MeSH Terms: Hematologic Diseases; Genetic Diseases, Inborn; Anemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Hemoglobinopathies; Anemia, Sickle Cell
• Other Study ID Numbers: CSEG101A2401B; 2020-004225-22 (EudraCT Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES

IPD Plan Description

Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent expert panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.

This trial data is currently available according to the process described on www.clinicalstudydatarequest.com.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No