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Trial details
Study of Dose Confirmation and Safety of Crizanlizumab in Pediatric Sickle Cell Disease Patients
Current status:
Approved
|
Date registered:
11/04/2022
Trial version(s)
History: 20/02/2019
History: 20/02/2019
History: 20/02/2019
History: 20/02/2019
History: 20/02/2019
History: 20/02/2019
History: 20/02/2019
History: 20/02/2019
History: 20/02/2019
History: 20/02/2019
History: 20/02/2019
History: 20/02/2019
History: 20/02/2019
History: 20/02/2019
History: 20/02/2019
History: 20/02/2019
History: 20/02/2019
History: 20/02/2019
History: 20/02/2019
History: 20/02/2019
History: 20/02/2019
History: 20/02/2019
History: 20/02/2019
History: 20/02/2019
History: 20/02/2019
History: 20/02/2019
History: 20/02/2019
History: 20/02/2019
History: 20/02/2019
History: 20/02/2019
History: 20/02/2019
Current: 20/02/2019
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Main Information
Primary registry identifying number
LBCTR2019020198
Protocol number
SEG101B2201
MOH registration number
Trial already registered with the MoPH
Study registered at the country of origin
Type of registration
Prospective
Date of registration in national regulatory agency
Primary sponsor
Novartis Pharma Services Inc.
Primary sponsor: Country of origin
Novartis Pharmaceuticals
Public title
Study of Dose Confirmation and Safety of Crizanlizumab in Pediatric Sickle Cell Disease Patients
Acronym
Scientific title
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
Acronym
Brief summary of the study: English
The purpose of the Phase 2 CSEG101B2201 study is to confirm and to establish appropriate dosing and to evaluate the safety in pediatric patients ages 6 months to <18 years with a history of VOC with or without HU/HC, receiving crizanlizumab for 2 years. The efficacy and safety of crizanlizumab was already demonstrated in adults with sickle cell disease. The approach is to extrapolate from the PK/pharmacodynamics (PD) already established in the adult population. The study is designed as a Phase II, multicenter, open-label study.
Brief summary of the study: Arabic
دراسة مفتوحة اللصاقة، متعددة المراكز، في المرحلة الثانية لتقييم الجرعات المناسبة وسلامة دواء كريزانليزوماب، مع أو بدون هيدروكسي يوريا / هيدروكسيكارباميد، لدى مرضى مصابين بداء الكريات المنجليّة لدى الأطفال مع نوبة انسداد وعائيّ من فئات عمريّة تسلسليّة وتنازليّة
Health conditions/problem studied: Specify
Sickle Cell Disease
Interventions: Specify
Drug: Crizanlizumab Crizanlizumab (SEG101) is a concentrate for solution for infusion, i.v. use. Supplied in single use 10 mL vials at a concentration of 10 mg/mL. One vial contains 100 mg of crizanlizumab. Other Name: SEG101
Key inclusion and exclusion criteria: Inclusion criteria
•Male or female patients aged 2 to <18 years (Group 3 will be expanded to allow enrolment of patients aged 6 to <24 months (and at least 6 kg) in Part B once the appropriate dose is confirmed in 2 to <6 year old patients) •Confirmed diagnosis of sickle cell disease (SCD) (e.g. any genotype including HbSS, HbSC, HbSβ0-thalassemia, HbSβ+-thalassemia, and others) by hemoglobin electrophoresis or high-performance liquid chromatography (HPLC) performed locally. •Experienced at least 1 VOC within the preceding 12 months, as determined by medical history. Prior VOC must have resolved at least 7 days prior to the first dose in the study and should include all the following: 1.the occurrence of appropriate symptoms (see VOC definition in protocol Section 7.2.1.1) 2.either a visit to a medical facility or healthcare professional, 3.receipt of oral/parenteral opioid or other non-opioid parenteral analgesia. •If receiving HU/HC or erythropoietin stimulating agent, must have been receiving the drug for at least 6 months prior to Screening and plan to continue taking at the same dose and schedule during the trial. Dose alterations of HU/HC during Part A are not allowed, and if this occurs, the patient will enter directly to the Part B. •Received standard age-appropriate care for SCD, including penicillin prophylaxis, pneumococcal immunization, and parental education •Transcranial Doppler (TCD) considered low risk within the past 6 months (for 2 to 16 years).
Key inclusion and exclusion criteria: Gender
Both
Key inclusion and exclusion criteria: Age minimum
1
Key inclusion and exclusion criteria: Age maximum
18
Key inclusion and exclusion criteria: Exclusion criteria
•History of stem cell transplant. •Received any blood products within 30 days of Day 1 dosing. •Participating 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. •Received a monoclonal antibody or immunoglobulin-based therapy within 6 months of Screening, or has documented immunogenicity to a prior monoclonal antibody. •Received active treatment on another investigational trial within 30 days (or 5 half lives of that agent, whichever is greater) prior to Screening or plans to participate in another investigational drug trial. •Pregnant females or females who have given birth within the past 90 days or who are breastfeeding. •Any documented history of a stroke or intracranial hemorrhage, or an uninvestigated neurologic finding within the past 12 months •Any conditional TCD within the past 12 months •Use of therapeutic anticoagulation (prophylactic doses permitted) or antiplatelet therapy (other than aspirin) within the 10 days prior to Week 1 Day 1 dosing •Hospitalized at Screening •Planning to undergo a major surgical procedure during the duration of the study •Planning to initiate or terminate HU/HC while on study, other than for safety reasons •Patient with active HIV infection (detectable viral load) •Patients with known active Hepatitis B infection. •Patients with known Hepatitis C history. •Significant active infection or immune deficiency (including chronic use of immunosuppressive drugs) in the opinion of the investigator. •Malignant disease. Exceptions to this exclusion include the following: malignancies that were treated curatively and have not recurred within 2 years prior to study treatment; any completely resected carcinoma in situ. •Has a serious mental or physical illness, which, in the opinion of the Investigator would compromise participation in the study. •Resting QTcF ≥450 msec at pretreatment (baseline) for patients under 12 years of age and ≥450 msec for males and ≥460 msec for female patients 12 years and older. •Cardiac or cardiac repolarization abnormality •Long QT syndrome, family history of idiopathic sudden death or congenital long QT syndrome •Sexually active females who are unwilling to comply with reliable method of birth control until 15 weeks following last dose of study drug. •Current drug or alcohol abuse: 1.Has a positive qualitative urine drug test at Screening for cocaine, phencyclidine (PCP), or amphetamines (opioids are permitted). 2.Consumes >12 (for males) or >8 (for females) standard alcoholic beverages per week. •Not able to understand and to comply with study instructions and requirements. •Subjects, who are an employee of the sponsor or investigator or otherwise dependent on them. •Subjects, who are committed to an institution by virtue of an order issued either by the judicial or the administrative authorities.
Type of Study
Type
Interventional
Type of intervention
Pharmaceutical
Trial scope
Other
Trial scope: Specify scope
Dose confirmation and Safety
Study design: Allocation
N/A: Single arm study
Study design: Masking
Open (masking not used)
Study design: Control
N/A
Study phase
2
Study design: Purpose
Prevention
Study design: Assignment
Single
IMP has market authorization
No
Name of IMP
SEG101 - Crizanlizumab
Type of IMP
Immunological
Pharmaceutical class
anti-human P-selectin antibody G1
Therapeutic indication
prevention of vaso-occlusive crises (VOCs) in patients of all genotypes with sickle cell disease (SCD)
Therapeutic benefit
1.Number of Vaso Occusive Crisis (VOC) events leading to healthcare visit in clinic/ER/hospital 2.Number of Vaso Occusive Crisis (VOC) events treated at home 3.Number of each subcategory of VOC event (uncomplicated pain crisis, acute chest syndrome, hepatic sequestration, splenic sequestration, priapism)
Biospecimen retention
Samples without DNA
Biospecimen description
All Blood samples and Urine Samples will be shipped to Covance Geneva Central Lab
Target sample size
8
Actual enrollment target size
8
Date of first enrollment: Type
Actual
Date of first enrollment: Date
13/12/2019
Date of study closure: Type
Actual
Date of study closure: Date
20/07/2022
Recruitment status
Suspended
Date of completion
30/06/2021
IPD sharing statement plan
No
IPD sharing statement 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.
Additional data URL
https://clinicaltrials.gov/ct2/show/NCT03474965?term=seg101&rank=2
Summary Results
Secondary Identifying Numbers
Full name of issuing authority
Secondary identifying number
Clinical Trials. gov
NCT03474965
Sources of Monetary or Material Support
Name
Novartis Pharma Services Inc.
Secondary Sponsors
Name
NA
Contact for Public/Scientific Queries
Contact type
First name
Last name
Address
Country
Telephone
Email
Affiliation
Public
Adlette
Inati
Tripoli
Lebanon
009613228 033
adlette.inati@lau.edu.lb
Nini Hospital
Scientific
Hind
Khairallah
Sin El Fil
Lebanon
+961 1 512002 Ext. 271
Hind.Khairallah@fattal.com.lb
Khalil Fattal et Fils s.a.l.
Public
Miguel
Abboud
Beirut
Lebanon
00961 (0) 3 534 213
ma56@aub.edu.lb
American University of Beirut Medical Center
Centers/Hospitals Involved in the Study
Center/Hospital name
Name of principles investigator
Principles investigator speciality
Ethical approval
Nini Hospital
Dr Adlette Inati
Hematology
Approved
American University of Beirut Medical Center
Dr Miguel Abboud
Hematology
Approved
Ethics Review
Ethics approval obtained
Approval date
Contact name
Contact email
Contact phone
Nini Hospital
17/12/2018
Nabil Kabbara
Nabil.kabbara@hopitalnini.com
+961 (0) 6 431 400 ext 1062
American University of Beirut Medical Center
16/10/2019
Fuad Ziyadeh
fz05@aub.edu.lb
+961 (0) 1 350 000 ext:5445
Countries of Recruitment
Name
Lebanon
Belgium
Germany
India
United States of America
Canada
Colombia
France
Italy
Oman
Spain
Switzerland
Turkey
United Kingdom
Health Conditions or Problems Studied
Condition
Code
Keyword
Sickle Cell Disease
Sickle-cell disorders (D57)
SCD
Interventions
Intervention
Description
Keyword
Please refer to table 7- 1 in the attached Protocol V00, page 51Consenting process, Physical Exam, Vital Signs, Medical History, Lab assessments, efficacy assessments and Biomarker assessments, pharmacokinetics and immunogenicity
Consenting process, Physical Exam, Vital Signs, Medical History, Lab assessments, efficacy assessments and Biomarker assessments, pharmacokinetics and immunogenicity
ICF, IMP, Lab
Primary Outcomes
Name
Time points
Measure
PK (AUCd15) after 1st dose, Confirm appropriate dosing of crizanlizumab in patients aged 2 to < 18 years
15 days
15 days
•PK (AUCtau) after 5th dose
week 15
week 15
•Frequency of any adverse events (AEs) as a measure of safety and tolerability
6 months , 2 years
6 months , 2 years
Key Secondary Outcomes
Name
Time points
Measure
•Number of Vaso Occusive Crisis (VOC) events leading to healthcare visit in clinic/ER/hospital
6 months, 2 years
6 months, 2 years
•Number of Vaso Occusive Crisis (VOC) events treated at home (based on documentation by health care provider following phone contact with the patient)
6 months, 2 years
6 months, 2 years
•Number of hospitalizations and ER visits (both overall and VOC-related)
6 months, 2 years
6 months, 2 years
•Absolute change from baseline in hemoglobin
Baseline, 6 months, 2 years
Baseline, 6 months, 2 years
Trial Results
Summary results in Lebanon
Study results globally
Date of posting of results summaries
Date of first journal publication of results
Results URL link
Baseline characteristics
Participant flow
Adverse events
Outcome measures
URL to protocol files
Link(s) to publications related to the study
Changes History
Change
Date
IRB Approval_PA04_AUBMC
05/04/2022
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