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Study of Efficacy, Safety and Tolerability of ACZ885 (Canakinumab) in Pediatric and Young Adult Patients With Sickle Cell Anemia

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ClinicalTrials.gov Identifier: NCT02961218
Recruitment Status : Recruiting
First Posted : November 10, 2016
Last Update Posted : July 19, 2019
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

Brief Summary:
To determine the effect of canakinumab versus placebo on daily pain experienced by sickle cell anemia patients (Reduction of average daily pain VAS over the period of Week 8 to 12 as compared to baseline levels).

Condition or disease Intervention/treatment Phase
Sickle Cell Anemia Drug: Ilaris, Canakinumab Drug: Placebo Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 60 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Multiple-dose, Subject- and Investigator-blinded, Placebo-controlled, Parallel Design Study to Assess the Efficacy, Safety and Tolerability of ACZ885 (Canakinumab) in Pediatric and Young Adult Patients With Sickle Cell Anemia
Actual Study Start Date : April 5, 2017
Estimated Primary Completion Date : June 10, 2020
Estimated Study Completion Date : June 10, 2020

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Anemia
Drug Information available for: Canakinumab

Arm Intervention/treatment
Placebo Comparator: Placebo
Matching placebo administered subcutaneously
Drug: Placebo
Monthly doses of placebo to match the administered dose of canakinumab s.c.

Experimental: Ilaris, Canakinumab
Randomized drug administration for 24 weeks duration (6 drug administrations each 28 days apart) followed by an additional 24-week open label phase (6 drug administrations each 28 days apart).
Drug: Ilaris, Canakinumab
Monthly doses of 300 mg canakinumab subcutaneously (s.c)




Primary Outcome Measures :
  1. Reduction of average daily pain [ Time Frame: Week 8 to 12 ]
    Primary endpoint is the reduction of average daily pain measured with a visual analog scale over the period of Week 8 to 12 as compared to baseline levels


Secondary Outcome Measures :
  1. Reduction of average daily pain over 24 weeks [ Time Frame: Baseline, 24 weeks ]
    Reduction of average daily pain measured with a visual analog scale over 4-week intervals up to Week 24 as compared to baseline levels

  2. The effect of canakinumab versus placebo on sickle cell anemia related days missed from school or work [ Time Frame: 56 weeks ]
    The number of days absent from school or work due to pain as recorded daily in an e-diary

  3. Measurement of pharmacokinetics of canakinumab in sickle cell anemia patients, which will be presented as descriptive summary statistics of pharmacokinetic concentrations. [ Time Frame: 48 weeks ]
    serial serum PK determination, 9 samples over 48 weeks, in patients with sickle cell anemia

  4. Week 12 versus baseline of white blood cell count [ Time Frame: Baseline, 12 weeks ]
    The effect of canakinumab versus placebo measured with laboratory markers of inflammation

  5. Week 12 versus baseline of absolute counts of blood neutrophils [ Time Frame: Baseline, 12 weeks ]
    The effect of canakinumab versus placebo measured with laboratory markers of inflammation

  6. Week 12 versus baseline of absolute counts of blood monocytes [ Time Frame: Baseline, 12 weeks ]
    The effect of canakinumab versus placebo measured with laboratory markers of inflammation

  7. Week 12 versus baseline of serum hs-CRP [ Time Frame: Baseline, 12 weeks ]
    The effect of canakinumab versus placebo measured with laboratory markers of inflammation

  8. Week 12 versus baseline of Hemoglobin concentration [ Time Frame: Baseline, 12 weeks ]
    The effect of canakinumab versus placebo measured with laboratory and functional markers of hemolysis

  9. Week 12 versus baseline of reticulocyte count [ Time Frame: Baseline, 12 weeks ]
    The effect of canakinumab versus placebo measured with laboratory and functional markers of hemolysis

  10. Week 12 versus baseline of Haptoglobin [ Time Frame: Baseline, 12 weeks ]
    The effect of canakinumab versus placebo measured with laboratory and functional markers of hemolysis

  11. Week 12 versus baseline of LDH [ Time Frame: Baseline, 12 weeks ]
    The effect of canakinumab versus placebo measured with laboratory and functional markers of hemolysis

  12. Week 12 versus baseline of total bilirubin [ Time Frame: Baseline, 12 weeks ]
    The effect of canakinumab versus placebo measured with laboratory and functional markers of hemolysis

  13. Week 12 versus baseline of oxygen percent saturation (SAO2) [ Time Frame: Baseline, 12 weeks ]
    The effect of canakinumab versus placebo measured with laboratory and functional markers of hemolysis



Information from the National Library of Medicine

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Ages Eligible for Study:   8 Years to 20 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Male and female subjects ages 8-20 years of age (both inclusive) diagnosed with sickle cell anemia (HbSS) or sickle beta0 thalassemia (documented by family studies, or analysis of either hemoglobin or DNA).
  • Patient's written informed consent from those ≥18 years of age must be obtained before any assessment is performed. Parent or legal guardian's written informed consent and child's assent, if appropriate, are required before any assessment is performed for patients < 18 years of age.
  • Detectable baseline of background or episodic pain measured by daily e-diary over 1 to 2 weeks during screening period as defined below: Average daily pain score ≥ 1 cm without analgesic use over a period of at least 7 days and/or, At least one episode of pain requiring analgesic use during a period of up to 14 days.
  • History of ≥2 vaso-occlusive pain episodes in the past year, as defined as pain with no other, non-sickle cell identifiable cause that requires analgesia and interferes with the patient's normal daily routine.

Exclusion Criteria:

  • History of known hypersensitivity to canakinumab.
  • Ongoing or treatment with the past 3 months with red blood cell transfusion therapy, or have evidence of iron overload requiring chelation therapy.
  • Transcranial Doppler ultrasound in the past year or at screening in patients with an accessible transtemporal window, demonstrating velocity in middle or anterior cerebral or internal carotid artery ≥200 cm/sec.
  • Administration of any other blood products within 3 weeks of screening visit.

Other protocol-defined inclusion/exclusion criteria may apply.


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02961218


Contacts
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Contact: Novartis Pharmaceuticals 1-888-669-6682 novartis.email@novartis.co
Contact: Novartis Pharmaceuticals +41613241111

Locations
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United States, Florida
Novartis Investigative Site Completed
Miami, Florida, United States, 33136
United States, Georgia
Novartis Investigative Site Active, not recruiting
Atlanta, Georgia, United States, 30329
Novartis Investigative Site Active, not recruiting
Augusta, Georgia, United States, 30912
United States, New York
Novartis Investigative Site Withdrawn
New York, New York, United States, 10029
United States, North Carolina
Novartis Investigative Site Active, not recruiting
Greenville, North Carolina, United States, 27834
United States, Pennsylvania
Novartis Investigative Site Terminated
Philadelphia, Pennsylvania, United States, 19104-4399
Canada, Ontario
Novartis Investigative Site Active, not recruiting
Toronto, Ontario, Canada, M5G 1X8
Germany
Novartis Investigative Site Completed
Hamburg, Germany, 20246
Israel
Novartis Investigative Site Completed
Afula, Israel, 1834111
South Africa
Novartis Investigative Site Recruiting
Johannesburg, Guateng, South Africa, 2193
Turkey
Novartis Investigative Site Active, not recruiting
Adana, Turkey, 01330
Novartis Investigative Site Active, not recruiting
Ankara, Turkey, 06100
Novartis Investigative Site Active, not recruiting
Mersin, Turkey, 33343
United Kingdom
Novartis Investigative Site Active, not recruiting
London, United Kingdom, E1 1BB
Novartis Investigative Site Active, not recruiting
London, United Kingdom, NW1 2BU
Novartis Investigative Site Active, not recruiting
London, United Kingdom, SE1 7EH
Novartis Investigative Site Active, not recruiting
London, United Kingdom, SE5 9RS
Novartis Investigative Site Active, not recruiting
Staffordshire, United Kingdom, WS11 5XY
Sponsors and Collaborators
Novartis Pharmaceuticals

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Responsible Party: Novartis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02961218     History of Changes
Other Study ID Numbers: CACZ885X2206
First Posted: November 10, 2016    Key Record Dates
Last Update Posted: July 19, 2019
Last Verified: July 2019

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Novartis ( Novartis Pharmaceuticals ):
Sickle cell disease
hemoglobinopathy
pediatric
Additional relevant MeSH terms:
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Anemia
Anemia, Sickle Cell
Hematologic Diseases
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Hemoglobinopathies
Genetic Diseases, Inborn
Antibodies, Monoclonal
Immunologic Factors
Physiological Effects of Drugs