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

• Sponsor: Novartis Pharmaceuticals (Industry)
• Overall Status: Recruiting
• CT.gov ID: NCT04657822
• Collaborator: (none)
• Enrollment: 130
• Locations: 20
• Arm: 1
• Anticipated Duration (Months): 91.2
• Patients Per Site: 6.5
• Patients Per Site Per Month: 0.1

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Sickle Cell Disease	Drug: Crizanlizumab	Phase 4

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 Design

• Study Type: Interventional
• Anticipated Enrollment: 130 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• 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
• Actual Study Start Date: Jun 10, 2021
• Anticipated Primary Completion Date: Jan 15, 2029
• Anticipated Study Completion Date: Jan 15, 2029

Arms and Interventions

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

Outcome Measures

Primary Outcome Measures

1. 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 [Not Applicable - Study Completion]

   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.

Secondary Outcome Measures

1. Number of participants with treatment emergent adverse events [from day of first dose of study medication to 105 days after last dose of study medication]

   The number of participants with Frequency, severity and causality of treatment emergent adverse events will be collected.

Eligibility Criteria

Criteria

• Ages Eligible for Study: 6 Months and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	University of Alabama 1600 7th ave	Birmingham	Alabama	United States	35233
2	Children s Hospital of Philadelphia Patient Treatment	Philadelphia	Pennsylvania	United States	19104-4399
3	Cook Childrens Medical Center	Fort Worth	Texas	United States	76104
4	Novartis Investigative Site	Brussel		Belgium	1000
5	Novartis Investigative Site	Liege		Belgium	4000
6	Novartis Investigative Site	Salvador	BA	Brazil	41253-190
7	Novartis Investigative Site	Ribeirao Preto	SP	Brazil	14051-140
8	Novartis Investigative Site	São Paulo	SP	Brazil	01232-010
9	Novartis Investigative Site	Cali	Valle Del Cauca	Colombia
10	Novartis Investigative Site	Paris		France	75015
11	Novartis Investigative Site	Heidelberg		Germany	69120
12	Novartis Investigative Site	Padova	PD	Italy	35128
13	Novartis Investigative Site	Orbassano	TO	Italy	10043
14	Novartis Investigative Site	Beirut		Lebanon	1107 2020
15	Novartis Investigative Site	Tripoli		Lebanon	1434
16	Novartis Investigative Site	Muscat		Oman	123
17	Novartis Investigative Site	Madrid		Spain	28009
18	Novartis Investigative Site	Adana		Turkey	01250
19	Novartis Investigative Site	Adana		Turkey	01330
20	Novartis Investigative Site	Antakya / Hatay		Turkey	31100

Sponsors and Collaborators

• Novartis Pharmaceuticals

Investigators

• Study Director: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Novartis Pharmaceuticals
• ClinicalTrials.gov Identifier: NCT04657822
• Other Study ID Numbers: CSEG101A2401B; 2020-004225-22
• First Posted: Dec 8, 2020
• Last Update Posted: Jun 27, 2022
• Last Verified: Jun 1, 2022

• Individual Participant Data (IPD) Sharing Statement: Yes
• Plan to Share IPD: Yes
• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No
• Keywords provided by Novartis Pharmaceuticals: SCD; Vaso-occlusive Crisis; crizanlizumab; SEG101; Sickle cell disease; Sickle cell disorder; VOC; P-selectin; Sickle cell anemia
• Additional relevant MeSH terms: Anemia, Sickle Cell

Study Results

No Results Posted as of Jun 27, 2022