Clinical trial NCT04657822: 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
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE4
Conditions: Sickle Cell Disease
Keywords: SCD
Vaso-occlusive Crisis
crizanlizumab
SEG101
Sickle cell disease
Sickle cell disorder
VOC
P-selectin
Sickle cell anemia
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2020-12-08
Enrollment: 130
Sex: ALL
Minimum age (years): 6
Maximum age (years): 100
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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

Eligibility criteria: 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

Primary outcomes:
measure: 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
timeFrame: Not Applicable - Study Completion
Secondary outcomes:
measure: Number of participants with treatment emergent adverse events
timeFrame: from day of first dose of study medication to 105 days after last dose of study medication
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: University Of Alabama
state: Alabama
city: Washington
country: United States
facility: Childrens National Hospital
state: District of Columbia
city: Augusta
country: United States
facility: Augusta University Georgia
state: Georgia
city: Greenville
country: United States
facility: East Carolina University
state: North Carolina
city: Greenville
country: United States
facility: East Carolina University
state: North Carolina
city: Philadelphia
country: United States
facility: Childrens Hospital Of Philadelphia
state: Pennsylvania
city: Fort Worth
country: United States
facility: Cook Childrens Medical Center
state: Texas
city: Brussel
country: Belgium
facility: Novartis Investigative Site
city: Laeken
country: Belgium
facility: Novartis Investigative Site
city: Liege
country: Belgium
facility: Novartis Investigative Site
city: Salvador
country: Brazil
facility: Novartis Investigative Site
state: BA
city: Ribeirao Preto
country: Brazil
facility: Novartis Investigative Site
state: SP
city: Sao Paulo
country: Brazil
facility: Novartis Investigative Site
state: SP
city: Cali
country: Colombia
facility: Novartis Investigative Site
state: Valle Del Cauca
city: Monteria
country: Colombia
facility: Novartis Investigative Site
city: Creteil
country: France
facility: Novartis Investigative Site
city: Paris 15
country: France
facility: Novartis Investigative Site
city: Paris 15
country: France
facility: Novartis Investigative Site
city: Heidelberg
country: Germany
facility: Novartis Investigative Site
city: Padova
country: Italy
facility: Novartis Investigative Site
state: PD
city: Orbassano
country: Italy
facility: Novartis Investigative Site
state: TO
city: Beirut
country: Lebanon
facility: Novartis Investigative Site
city: Tripoli
country: Lebanon
facility: Novartis Investigative Site
city: Muscat
country: Oman
facility: Novartis Investigative Site
city: Barcelona
country: Spain
facility: Novartis Investigative Site
state: Catalunya
city: Madrid
country: Spain
facility: Novartis Investigative Site
city: Adana
country: Turkey
facility: Novartis Investigative Site
city: Adana
country: Turkey
facility: Novartis Investigative Site
city: Antakya Hatay
country: Turkey
facility: Novartis Investigative Site
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: