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Study of two doses of crizanlizumab versus placebo in adolescent and adult sickle cell disease patients

Phase 1
Conditions
Sickle Cell Disease with vaso-occlusive crisis
MedDRA version: 21.0Level: PTClassification code 10040644Term: Sickle cell diseaseSystem Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.1Level: LLTClassification code 10002077Term: Anaemia sickle cellSystem Organ Class: 10010331 - Congenital, familial and genetic disorders
Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Registration Number
EUCTR2017-001746-10-IT
Lead Sponsor
OVARTIS PHARMA AG
Brief Summary

Not available

Detailed Description

Not available

Recruitment & Eligibility

Status
Authorised-recruitment may be ongoing or finished
Sex
All
Target Recruitment
240
Inclusion Criteria

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
4a. 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 resolve at least 7 days prior to Week 1 Day 1 and 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 antiinflammatory drug (NSAID) analgesics. Acute chest syndrome (ACS), priapism, and hepatic or splenic sequestration will be considered VOC in this study.
5. If receiving HU/HC or erythropoietin stimulating agent or L-glutamine (local HA approved medicinal product), must have been receiving the drug for at least 6 months and at a stable dose fro at least 3 months prior to Screening visit and plan to continue taking at the same dose and schedule until the participant 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:
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

Exclusion Criteria

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
NameTimeMethod
Secondary Outcome Measures
NameTimeMethod
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