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

NCT02961218 | Completed Phase 2 Results FDA Drug

• 1 other identifier: CACZ885X2206
• Study Type: interventional
• Target: 49
• Locations: 7 countries
• Sites: 15

Brief Summary

The study assesses the efficacy, safety and tolerability of ACZ885 (canakinumab) in pediatric and young adult patients with sickle cell anemia (SCA).

Conditions

Sickle Cell Anemia

Keywords

Sickle cell disease; hemoglobinopathy; pediatric

Outcome Measures

Primary Outcomes (1)

• Change From Baseline of 4- Week Average Daily Pain Measured by Visual Analog Score (VAS) Over the Period of Week 8 to 12

  Visual analog scale (VAS) was used to record severity. Pediatric and young adult participants rated their daily sickle cell associated pain intensity once each day in the evening using an 11-point numerical rating scale from 0 to 10 with higher ratings associated with more intense pain (0 = no pain, 10 = worst pain). For each subject, there was a maximum 28-day screening period that included recording of daily pain intensity by e-diary for at least 1 week. The average daily pain results in the screening period were used to derive the baseline value. The average over week 8 to 12 was calculated and the change from baseline in the average daily pain VAS was analyzed using a Bayesian model for repeated measures.

  Baseline (upto 28 days prior to start of treatment), Week 8 to 12

Secondary Outcomes (14)

• Change From Baseline of Average Daily Pain VAS Over 4 Weeks Intervals up to Week 24

  Baseline (upto 28 days prior to start of treatment), Week 0 to 4, Week 4 to 8, Week 8 to 12, Week 12 to 16, Week 16 to 20 and Week 20 to 24

• Change in the Concentration of High Sensitivity C-Reactive Protein (hsCRP) From Baseline to Week 12

  Baseline, Week 12

• Change in the Concentration of White Blood Cell (WBC) Count From Baseline to Week 12

  Baseline, Week 12

• Change in the Concentration of Absolute Count of Neutrophils From Baseline to Week 12

  Baseline, Week 12

• Change in the Concentration of Absolute Count of Blood Monocytes From Baseline to Week 12

  Baseline, Week 12

Study Arms (2)

Placebo

PLACEBO COMPARATOR

Monthly doses of 4 mg/kg for subjects weighing ≤40 kg and 300 mg for all other subjects

Drug: Placebo

ACZ885

EXPERIMENTAL

Monthly doses of 300 mg (4 mg/kg for patients ≤ 40 kg) canakinumab s.c.

Drug: ACZ885

Interventions

ACZ885 DRUG

Monthly doses of 4 mg/kg for subjects weighing ≤40 kg and 300 mg for all other subjects

Also known as: Canakinumab

ACZ885

Placebo DRUG

Monthly doses of placebo to match the administered dose of canakinumab s.c.

Placebo

Eligibility Criteria

• Age: 8 Years - 20 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (15)

Novartis Investigative Site

Atlanta, Georgia, 30329, United States

Location

Novartis Investigative Site

Augusta, Georgia, 30912, United States

Location

Novartis Investigative Site

Greenville, North Carolina, 27834, United States

Location

Novartis Investigative Site

Toronto, Ontario, M5G 1X8, Canada

Location

Novartis Investigative Site

Hamburg, 20246, Germany

Location

Novartis Investigative Site

Afula, 1834111, Israel

Location

Novartis Investigative Site

Johannesburg, Guateng, 2193, South Africa

Location

Novartis Investigative Site

Adana, 01330, Turkey (Türkiye)

Location

Novartis Investigative Site

Ankara, 06100, Turkey (Türkiye)

Location

Novartis Investigative Site

Mersin, 33343, Turkey (Türkiye)

Location

Novartis Investigative Site

Wolverhampton, Staffordshire, WS11 5XY, United Kingdom

Location

Novartis Investigative Site

London, E1 1BB, United Kingdom

Location

Novartis Investigative Site

London, NW1 2BU, United Kingdom

Location

Novartis Investigative Site

London, SE1 7EH, United Kingdom

Location

Novartis Investigative Site

London, SE5 9RS, United Kingdom

Location

Related Publications (1)

• Rees DC, Kilinc Y, Unal S, Dampier C, Pace BS, Kaya B, Trompeter S, Odame I, Mahlangu J, Unal S, Brent J, Grosse R, Fuh BR, Inusa BPD, Koren A, Leblebisatan G, Levin C, McNamara E, Meiser K, Hom D, Oliver SJ. A randomized, placebo-controlled, double-blind trial of canakinumab in children and young adults with sickle cell anemia. Blood. 2022 Apr 28;139(17):2642-2652. doi: 10.1182/blood.2021013674.

  PMID: 35226723 DERIVED

Related Links

• A Plain Language Trial Summary is available on novartisclinicaltrials.com
• A Pediatric Plain Language Trial Summary is available on novartisclinicaltrials.com

MeSH Terms

Conditions

Anemia, Sickle Cell; Hemoglobinopathies

Interventions

canakinumab

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Results Point of Contact

• Title: Study Director
• Organization: Novartis Pharmaceuticals

Novartis.email@novartis.com

862-778-8300

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 20, 2016
• First Posted: November 10, 2016
• Study Start: April 5, 2017
• Primary Completion: June 27, 2019
• Study Completion: April 27, 2020
• Last Updated: January 13, 2026
• Results First Posted: December 17, 2020

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will share

Locations

US (3); CA (1); TU (3); IL (1); DE (1); ZA (1); GB (5)