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Sickle Cell AnemiaPhase 2NCT02961218

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

A Phase 2 study testing ACZ885 in people with Sickle Cell Anemia, run by Novartis Pharmaceuticals at 15 sites in 7 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

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

Who is it for?
Ages 8 to 20 years
Conditions the study is for
Sickle Cell Anemia
What is being tested?
ACZ885
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
49 participants
Where
15 sitesin 7 countries
Study dates
5 Apr 2017to 27 Apr 2020
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 8 to 20 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • 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.

Who cannot join

  • 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.

Trial locations and contacts

15 sites in 7 countries.

15 sites in 7 countries

United Kingdom5 sites
  • Novartis Investigative Site

    London, E1 1BB

  • Novartis Investigative Site

    London, NW1 2BU

  • Novartis Investigative Site

    London, SE1 7EH

  • Novartis Investigative Site

    London, SE5 9RS

  • Novartis Investigative Site

    Wolverhampton, Staffordshire, WS11 5XY

Other trials in United Kingdom

United States3 sites
  • Novartis Investigative Site

    Atlanta, Georgia, 30329

  • Novartis Investigative Site

    Augusta, Georgia, 30912

  • Novartis Investigative Site

    Greenville, North Carolina, 27834

Other trials in United States

Turkey (Türkiye)3 sites
  • Novartis Investigative Site

    Adana, 01330

  • Novartis Investigative Site

    Ankara, 06100

  • Novartis Investigative Site

    Mersin, 33343

Other trials in Turkey (Türkiye)

Canada1 site
  • Novartis Investigative Site

    Toronto, Ontario, M5G 1X8

Other trials in Canada

Germany1 site
  • Novartis Investigative Site

    Hamburg, 20246

Other trials in Germany

Israel1 site
  • Novartis Investigative Site

    Afula, 1834111

Other trials in Israel

South Africa1 site
  • Novartis Investigative Site

    Johannesburg, Guateng, 2193

Other trials in South Africa

About this study

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

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

Read the detailed description

This was an ambulatory-based 24-week study followed by an additional 24-week open label phase. It was a subject- and investigator-blinded, randomized, placebo-controlled, parallel group, non-confirmatory study to assess the clinical efficacy of ACZ885 administered s.c. in six injections given 28 days apart (in each phase of the study).

Pediatric and young adult subjects diagnosed with sickle cell anemia (SCA) were planned to be randomized to either ACZ885 treatment or placebo treatment in a 1:1 ratio,.

For each subject, there was a maximum 28-day screening period that included recording of daily pain frequency and intensity by e-diary for at least 1 week. Subjects who met the eligibility criteria at screening underwent evaluation of baseline clinical and biomarker assessments prior to first dose administration.

On Day 1, monthly s.c. dosing with ACZ885 started at 4 mg/kg for subjects weighing ≤40 kg and 300 mg for all other subjects. Subjects in the placebo treatment arm were injected with placebo in a like manner. All subjects returned to the study centers for safety checks on a monthly basis when they received treatment with either ACZ885 or placebo.

The final blinded dosing was given on Week 20, followed by blinded clinical assessments at Week 24. Subjects from both study arms were then offered optional, open label monthly dosing of ACZ885 for an additional 24 weeks (Weeks 24-48) with clinical outcome assessment.

Subjects returned for the end of study (EOS) visit at Week 56. For subjects who chose not to participate in the optional, open label portion of the study, or for those stopping treatment early for any other reason, an EOS visit occurred approximately 8 weeks after last dose received.

After enrollment of 49 subjects, Novartis decided to terminate the study early due to strategic reasons not related to safety and decided that no additional enrollment was needed in order to interpret the study objectives.

Being tested
ACZ885, Placebo
Keywords
Sickle cell disease, hemoglobinopathy, pediatric
Medical subject headings
Anemia, Sickle Cell, Hemoglobinopathies

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • ACZ885Drug

    Also called Canakinumab

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

  • PlaceboDrug

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

Study groups in NCT02961218
Study groupRoleWhat they receive
PlaceboPlacebo comparatorMonthly doses of 4 mg/kg for subjects weighing ≤40 kg and 300 mg for all other subjects
Receives: Drug: Placebo
ACZ885ExperimentalMonthly doses of 300 mg (4 mg/kg for patients ≤ 40 kg) canakinumab s.c.
Receives: Drug: ACZ885

What is being measured

The primary outcome is the main question the study is designed to answer.

  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.

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

Secondary outcomes (14)
  1. Change From Baseline of Average Daily Pain VAS Over 4 Weeks Intervals up to Week 24

    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). The average of 4 weeks interval up to week 24 was calculated.

    Measured: 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

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

    hs-CRP is a biomarker that represents the inflammation process.

    Measured: Baseline, Week 12

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

    WBC count was used as a laboratory marker to determine the effect of the drug

    Measured: Baseline, Week 12

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

    Absolute count of neutrophils was measured as a laboratory marker to determine the effect of the drug

    Measured: Baseline, Week 12

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

    Absolute count of blood monocytes was measured as a laboratory marker to determine the effect of the drug.

    Measured: Baseline, Week 12

  6. Change in the Concentration of Hemoglobin From Baseline to Week 12

    Hemoglobin was used as a hemolysis marker to determine the effect of the drug.

    Measured: Baseline, Week 12

  7. Change in the Reticulocyte Count From Baseline to Week 12

    Reticulocyte count was used as a hemolysis marker to determine the effect of the drug

    Measured: Baseline, Week 12

  8. Change in the Concentration of Bilirubin From Baseline to Week 12

    Bilirubin was used as a hemolysis marker to determine the effect of the drug

    Measured: Baseline, Week 12

  9. Change in the Concentration of Lactate Dehydrogenase (LDH) From Baseline to Week 12

    LDH was used as a hemolysis marker to determine the effect of the drug

    Measured: Baseline, Week 12

  10. Change in the Concentration of Haptoglobin From Baseline to Week 12

    Haptoglobin was used as a hemolysis marker to determine the effect of the drug

    Measured: Baseline, Week 12

  11. Change in the Concentration of Oxygen Percent Saturation (SAO2) From Baseline to Week 12

    SAO2 was used as a hemolysis marker to determine the effect of the drug

    Measured: Baseline, Week 12

  12. Number of Days Absent From School or Work Due to Pain as Recorded by E-diary

    The number of SCA-related days absent from school or work were derived from eDiary records.

    Measured: up to Week 24

  13. Number of Acute Blood Transfusions Per Patient by Study Period - Double-blind Period

    The occurrence of acute blood transfusions was summarized as the proportion of subjects who received at least one acute blood transfusion and the event rate of acute blood transfusions per subject, by study period, group and reason of transfusion.

    Measured: 12 weeks

  14. Mean Serum Concentration After Repeated Dosing of ACZ885

    PK samples were collected at Baseline, Week 4, 12, 20 and 24. Mean and standard deviation of the ACZ885 concentration was reported. Only those participants available at the specified time points were analyzed

    Measured: Baseline, Week 4, 12, 20 and 24

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted10 Nov 2016 (estimated)
  2. Study start5 Apr 2017
  3. Primary completion27 Jun 2019
  4. Study completion27 Apr 2020

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
49 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02961218
Sponsor's protocol ID
CACZ885X2206
Status last verified
Dec 2025
First submitted
20 Oct 2016
Last updated
13 Jan 2026
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com

Publications

  • 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.

    PubMed 35226723

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing ACZ885 in people with Sickle Cell Anemia, run by Novartis Pharmaceuticals at 15 sites in 7 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 4 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 13 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 15 sites in United Kingdom, United States, Turkey (Türkiye), Canada, Germany and 2 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 17 Dec 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Change From Baseline of 4- Week Average Daily Pain Measured by Visual Analog Score (VAS) Over the Period of Week 8 to 12 (time frame: Baseline (upto 28 days prior to start of treatment), Week 8 to 12). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02961218, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Jan 2026.

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