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

Official title

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

Overview

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.

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

Interventions

• Drug Crizanlizumab
  Concentrate for solution for infusion for Intravenous use

Primary outcome measures

• 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 [Time frame: Not Applicable - Study Completion]

Secondary outcome measures (1)

• Number of participants with treatment emergent adverse events [Time frame: from day of first dose of study medication to 105 days after last dose of study medication]

Eligibility criteria

Inclusion criteria

• 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
• 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
• 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

• Patient had permanently discontinued from crizanlizumab study treatment in the parent study before the parent study completion
• 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
• Concurrent participation in any other investigational clinical trial other than the parent study or plan to participate in any other investigational clinical trial
• Pregnant or nursing women
• Women of childbearing potential who are unwilling to be on highly effective contraceptives during dosing and until 15 weeks after stopping treatment with crizanlizumab
• SCD patients who do not meet parent study protocol criteria to continue with crizanlizumab

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation

N/A

Model

Single group

Masking

Open label

Primary purpose

Treatment

Study locations

United States · 7 centers

• University Of Alabama — Birmingham
• Childrens National Hospital — Washington D.C.
• Augusta University Georgia — Augusta
• East Carolina University — Greenville
• East Carolina University — Greenville
• Childrens Hospital of Philadelphia — Philadelphia
• Cook Childrens Medical Center — Fort Worth

Colombia · 4 centers

• Novartis Investigative Site — Barranquilla
• Novartis Investigative Site — Valledupar
• Novartis Investigative Site — Cali
• Novartis Investigative Site — Montería

Belgium · 3 centers

• Novartis Investigative Site — Brussels
• Novartis Investigative Site — Laken
• Novartis Investigative Site — Liège

Brazil · 3 centers

• Novartis Investigative Site — Salvador
• Novartis Investigative Site — Ribeirão Preto
• Novartis Investigative Site — São Paulo

France · 3 centers

• Novartis Investigative Site — Créteil
• Novartis Investigative Site — Paris
• Novartis Investigative Site — Paris

Turkey (Türkiye) · 3 centers

• Novartis Investigative Site — Hatay
• Novartis Investigative Site — Adana
• Novartis Investigative Site — Adana

Italy · 2 centers

• Novartis Investigative Site — Padova
• Novartis Investigative Site — Orbassano

Lebanon · 2 centers

• Novartis Investigative Site — Beirut
• Novartis Investigative Site — Tripoli

Spain · 2 centers

• Novartis Investigative Site — Barcelona
• Novartis Investigative Site — Madrid

Germany · 1 center

• Novartis Investigative Site — Heidelberg

Oman · 1 center

• Novartis Investigative Site — Khoudh

Identifiers

NCT: NCT04657822 · CSEG101A2401B · 2020-004225-22

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗