Study of two doses of crizanlizumab versus placebo in adolescent and adult sickle cell disease patients
- Conditions
- Sickle Cell Disease with vaso-occlusive crisisMedDRA version: 20.0 Level: LLT Classification code 10040644 Term: Sickle cell disease System Organ Class: 100000004850MedDRA version: 20.1 Level: LLT Classification code 10002077 Term: Anaemia sickle cell System Organ Class: 100000004850Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
- Registration Number
- EUCTR2017-001746-10-ES
- Lead Sponsor
- ovartis Farmacéutica, S.A.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Authorised-recruitment may be ongoing or finished
- Sex
- Not specified
- Target Recruitment
- 240
1. Written informed consent must be obtained prior
to any screening procedures
2. Male or female patients aged 12 years and older
on the day of signing informed consent. Adolescent
include patients aged 12 to 17 years old and adults =
18 years and older
3. Confirmed diagnosis of SCD by hemoglobin
electrophoresis or high performance liquid
chromatography (HPLC) [performed locally]. All SCD
genotypes are eligible, genotyping is not required for study entry
4. Experienced at least 2 VOCs leading to healthcare visit within the 12 months prior to screening visit as determined by medical history. Prior VOC leading to healthcare visit must include:
a. Pain crisis defined as an acute onset of pain for
which there is no other medically determined
explanation other than vaso- occlusion -
b. a visit to a medical facility and/or healthcare
professional,
c. and receipt of oral/parenteral opioids or parenteral nonsteroidal anti-inflammatory drug (NSAID) analgesia
As well as other complicated crises, such as acute
chest syndrome, priapism, and hepatic or splenic
sequestration
5. If receiving HU/HC or erythropoietin stimulating
agent or L-glutamine, must have been receiving the
drug for at least 6 months prior to Screening visit and plan to continue taking at the same dose and schedule until the subject has reached one year of study treatment
6. Patients must meet the following central
laboratory values at the screening visit:
• Absolute Neutrophil Count =1.0 x 109/L
• Platelet count =75 x 109/L
• Hemoglobin: for adults (Hb) =4.0 g/dL and for
adolescents (Hb) =5.5 g/dL
• Glomerular filtration rate = 45 mL/min/1.73 m2
using CKD-EPI formula in adults, and Shwartz formula in adolescents
• Direct (conjugated) bilirubin < 2.0 x ULN
• Alanine transaminase (ALT) < 3.0 x ULN
7. ECOG performance status =2.0 for adults and
Karnofsky = 50% for adolescents
Are the trial subjects under 18? yes
Number of subjects for this age range: 49
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 190
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 1
1. History of stem cell transplant.
2. Participating in a chronic transfusion program
(pre-planned series of transfusions for prophylactic
purposes) and/or planning on undergoing an
exchange transfusion during the duration of the study; episodic transfusion in response to worsened anemia or VOC is permitted.
3. Contraindication or hypersensitivity to any drug or
metabolites from similar class as study drug or to any excipients of the study drug formulation. History of severe hypersensitivity reaction to other monoclonal antibodies, which in the opinion of the investigator may pose an increased risk of serious infusion reaction.
4. Received active treatment on another
investigational trial within 30 days (or 5 half-lives of
that agent, whichever is greater) prior to Screening
visit or plans to participate in another investigational
drug trial.
5. Women of child-bearing potential, defined as all
women physiologically capable of becoming pregnant unless they are using highly effective methods of contraception during dosing and for 15 weeks after stopping treatment.
6. Concurrent severe and/or uncontrolled medical
conditions which, in the opinion of the Investigator,
could cause unacceptable safety risks or compromise participation in the study.
7. History or current diagnosis of ECG abnormalities
indicating significant risk of safety such as:
• Resting QTcF =470 msec at pretreatment
(baseline) for both male and female or inestability to
determine QTc
• Concomitant clinically significant cardiac
arrhythmias (e.g ventricular tachycardia), and clinically significant second or third degree AV block without a pacemaker
• History of familial long QT syndrome or know
family history of Torsades de Pointes
8. Not able to understand and to comply with study
intructions and requirements.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method