Study of Two Doses of Crizanlizumab Versus Placebo in Adolescent and Adult Sickle Cell Disease Patients

A Phase III, Multicenter, Randomized, Double-blind Study to Assess Efficacy and Safety of Two Doses of Crizanlizumab Versus Placebo, With or Without Hydroxyurea/ Hydroxycarbamide Therapy, in Adolescent and Adult Sickle Cell Disease Patients With Vaso-Occlusive Crises (STAND)

ClinicalTrials.gov Identifier: NCT03814746

Novartis Reference Number: CSEG101A2301

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All compounds are either investigational or being studied for (a) new use(s). Efficacy and safety have not been established. There is no guarantee that they will become commercially available for the use(s) under investigation. 

Study Description

The purpose of this study is to compare the efficacy and safety of 2 doses of crizanlizumab (5.0 mg/kg and 7.5 mg/kg) versus placebo in adolescent and adult sickle cell disease (SCD) patients with history of vaso-occlusive crisis (VOC) leading to healthcare visit.

Condition 
Sickle Cell Disease (SCD)
Phase 
Phase 3
Overall status 
Recruiting
Enrollment count 
240 participants
Start date 
Jul 26, 2019
Completion date 
Dec 02, 2027
Gender 
All
Age(s)
12 Years and older (Child, Adult, Older Adult)

Interventions

Drug
Crizanlizumab (SEG101)
Crizanlizumab will be supplied in single use 10 mL glass vials at a concentration of 10 mg/mL. One vial contains 100 mg of crizanlizumab. This is a concentrate for solution for infusion IV.
Drug
Placebo
Placebo will be supplied in single use 10 mL glass vials at a concentration of 10 mg/mL. One vial contains 100 mg of placebo. This is a concentrate for solution for infusion IV.

Eligibility Criteria

Inclusion Criteria:

Written informed consent must be obtained prior to any screening procedures
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
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

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:

Pain crisis defined as an acute onset of pain for which there is no other medically determined explanation other than vaso- occlusion -
which requires a visit to a medical facility and/or healthcare professional,
and receipt of oral/parenteral opioids or parenteral nonsteroidal anti-inflammatory drug (NSAID) analgesia Acute chest syndrome (ACS), priapism and hepatic or splenic sequestration will be considered VOC in this study
If receiving HU/HC or L-glutamine (local HA approved medicinal product), must have been receiving the drug for at least 6 months and at a stable dose for at least 3 months prior to Screening visit and plan to continue taking it at the same dose and schedule until the subject has reached one year of study treatment. Patients who have not been receiving such drug must not have received it for at least 6 months prior to Screening visit to be included. Patients must have evidence of insufficient control of acute pain, such as at least one VOC leading to healthcare visit while on HU/HC or L-Glutamine treatment. If receiving erythropoietin stimulating agent, must have been receiving the drug for at least 6 months prior to Screening visit and plan to continue taking the treatment to maintain stable Hb levels at least until the subject has reached one year of study treatment

Patients must meet the following central laboratory values prior to Week 1 Day 1:

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
ECOG performance status ≤2.0 for adults and Karnofsky ≥ 50% for adolescents

Exclusion Criteria:

History of stem cell transplant.
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.
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.
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.
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.
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.

History or current diagnosis of ECG abnormalities indicating significant risk of safety such as:

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
Not able to understand and to comply with study instructions and requirements.
Received prior treatment with crizanlizumab or other selectin targeting agent

Other protocol-defined Inclusion/Exclusion may apply.

Study Locations

United States
Memorial Cancer Institute Regulatory Contact
Recruiting
Hollywood, 33021
Florida
United States
Childrens Healthcare of Atlanta
Recruiting
Atlanta, 30342
Georgia
United States
Boston Medical Center
Recruiting
Boston, 02118
Massachusetts
United States
Jacobi Medical Center
Recruiting
Bronx, 10461
New York
United States
Levine Cancer Insitute Carolinas Healthcare System
Recruiting
Charlotte, 28204
North Carolina
United States
Thomas Jefferson University Hospital
Recruiting
Philadelphia, 19107
Pennsylvania
United States
Univ of Tenn Health Sciences Ctr
Recruiting
Memphis, 38163
Tennessee
United States
Cook Childrens Medical Center
Withdrawn
Fort Worth, 76104
Texas
United States
University of Texas Health Science Center at Houston
Recruiting
Houston, 77030
Texas
United States
Belgium
Novartis Investigative Site
Recruiting
Edegem, 2650
Antwerpen
Belgium
Novartis Investigative Site
Recruiting
Brussel, 1000
-
Belgium
Novartis Investigative Site
Recruiting
Bruxelles, 1070
-
Belgium
Novartis Investigative Site
Recruiting
Laeken, 1020
-
Belgium
Novartis Investigative Site
Recruiting
Liege, 4000
-
Belgium
Brazil
Novartis Investigative Site
Recruiting
Salvador, 41253-190
Bahia
Brazil
Novartis Investigative Site
Recruiting
Recife, 50070-170
Pernambuco
Brazil
Novartis Investigative Site
Recruiting
Rio de Janeiro, 20.211-030
RJ
Brazil
Novartis Investigative Site
Recruiting
Ribeirao Preto, 14048-900
SP
Brazil
Novartis Investigative Site
Recruiting
Sao Paulo, 05403 000
SP
Brazil
Novartis Investigative Site
Recruiting
Sao Paulo, 08270-070
SP
Brazil
Novartis Investigative Site
Recruiting
São Paulo, 01232-010
SP
Brazil
Novartis Investigative Site
Recruiting
Porto Alegre, 90035-003
-
Brazil
Canada
Novartis Investigative Site
Recruiting
Montreal, H2X 1R9
Quebec
Canada
Colombia
Novartis Investigative Site
Recruiting
Monteria,
-
Colombia
Finland
Novartis Investigative Site
Recruiting
Helsinki, FIN 00290
-
Finland
France
Novartis Investigative Site
Recruiting
Creteil, 94000
-
France
Novartis Investigative Site
Recruiting
Marseille cedex 05, 13385
-
France
Novartis Investigative Site
Recruiting
Paris, 75015
-
France
Germany
Novartis Investigative Site
Recruiting
Berlin, 13353
-
Germany
Novartis Investigative Site
Recruiting
Essen, 45147
-
Germany
Novartis Investigative Site
Recruiting
Hamburg, 20246
-
Germany
Novartis Investigative Site
Recruiting
Koeln, 50937
-
Germany
Novartis Investigative Site
Recruiting
Stuttgart, 70376
-
Germany
Greece
Novartis Investigative Site
Recruiting
Athens, 115 27
-
Greece
Novartis Investigative Site
Recruiting
Patras, 265 00
-
Greece
Novartis Investigative Site
Recruiting
Thessaloniki, GR 54636
-
Greece
India
Novartis Investigative Site
Recruiting
Bhubaneswar, 751003
Odisha
India
Novartis Investigative Site
Recruiting
Hyderabad, 500082
Telangana
India
Novartis Investigative Site
Recruiting
Vellore,
-
India
Italy
Novartis Investigative Site
Recruiting
Genova, 16128
GE
Italy
Novartis Investigative Site
Recruiting
Palermo, 90146
PA
Italy
Novartis Investigative Site
Recruiting
Verona, 37126
VR
Italy
Novartis Investigative Site
Recruiting
Napoli, 80138
-
Italy
Jordan
Novartis Investigative Site
Recruiting
Irbid, 22110
-
Jordan
Lebanon
Novartis Investigative Site
Recruiting
Beirut, 1107 2020
-
Lebanon
Novartis Investigative Site
Recruiting
Tripoli, 1434
-
Lebanon
Netherlands
Novartis Investigative Site
Recruiting
Amsterdam, 1105 AZ
-
Netherlands
Novartis Investigative Site
Recruiting
Den Haag, 2545 CH
-
Netherlands
Novartis Investigative Site
Recruiting
Rotterdam, 3015 GD
-
Netherlands
Oman
Novartis Investigative Site
Recruiting
Muscat, 123
-
Oman
Panama
Novartis Investigative Site
Recruiting
Panama City, 0801
-
Panama
South Africa
Novartis Investigative Site
Recruiting
Soweto, 2013
Gauteng
South Africa
Spain
Novartis Investigative Site
Recruiting
Barcelona, 08035
Catalunya
Spain
Novartis Investigative Site
Recruiting
Salt, 17190
Cataluña
Spain
Novartis Investigative Site
Recruiting
Madrid, 28034
-
Spain
Novartis Investigative Site
Recruiting
Madrid, 28046
-
Spain
Turkey
Novartis Investigative Site
Recruiting
Adana, 01330
-
Turkey
Novartis Investigative Site
Recruiting
Antakya / Hatay, 31100
-
Turkey
United Kingdom
Novartis Investigative Site
Recruiting
Sheffield, S10 2JF
South Yorkshire
United Kingdom
Novartis Investigative Site
Recruiting
Cambridge, CB2 0QQ
-
United Kingdom
Novartis Investigative Site
Recruiting
London, E1 1BB
-
United Kingdom
Novartis Investigative Site
Recruiting
London, SE1 9RT
-
United Kingdom
Novartis Investigative Site
Recruiting
London, SE5 9RS
-
United Kingdom
Novartis Investigative Site
Recruiting
Sheffield, S10 2TH
-
United Kingdom

Contacts

Name: 
Novartis Pharmaceuticals
Phone: 
1-888-669-6682
Name: 
Novartis Pharmaceuticals
Phone: 
+41613241111
Email: 

Have a question?

Call 1-999-669-6682 or email [email protected]