CClinicalTrials.gg
Sign In
CompletedNCT02961218Updated Jan 13, 2026Results posted

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

A Phase 2 interventional study of ACZ885 and Placebo in Sickle Cell Anemia, sponsored by Novartis Pharmaceuticals. Completed at 15 sites in 7 countries. Open to participants aged 8 Years to 20 Years. Per ClinicalTrials.gov, last updated 2026-01-13.

Sponsored by Novartis Pharmaceuticals · Phase 2, Interventional, and Treatment

Phase
Phase 2
Study type
Interventional
Enrollment
49
Allocation
Randomized
Ages
8 Years to 20 Years
Sex
All
01

Study summary

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.

02

Conditions studied

  • Sickle Cell Anemia

Keywords

  • Sickle cell disease
  • hemoglobinopathy
  • pediatric
03

In context

Anemia, Sickle Cell

1,103 studies on the registry are indexed under Anemia, Sickle Cell; 235 are open to participants now.

This study's enrollment of 49 is above the median of 40 across 750 interventional studies indexed under Anemia, Sickle Cell.

Browse Anemia, Sickle Cell studies →

Lead sponsor

Novartis Pharmaceuticals is the lead sponsor of 2,673 studies on the registry; 228 are open to participants now.

Of its 576 completed or terminated interventional studies of FDA-regulated products, 431 (75%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
8 Years to 20 Years
Sexes eligible
All
Accepts healthy volunteers
No

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

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.

05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Quadruple (Participant, Care provider, Investigator, Outcomes assessor)
Enrollment
49 participants (actual)

Study arms

  • Placebo comparator
    Placebo

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

    Drug: Placebo

  • Experimental
    ACZ885

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

    Drug: ACZ885

Interventions

  • DrugACZ885

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

    Also known as: Canakinumab

  • DrugPlacebo

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

06

What researchers measure

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.

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

Secondary outcomes

  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.

    Time frame: 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.

    Time frame: 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

    Time frame: 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

    Time frame: 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.

    Time frame: 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.

    Time frame: 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

    Time frame: 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

    Time frame: 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

    Time frame: 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

    Time frame: 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

    Time frame: 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.

    Time frame: 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.

    Time frame: 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

    Time frame: Baseline, Week 4, 12, 20 and 24

07

Results

Posted Dec 17, 2020

Participant flow

A total of 49 participants were randomized into the study from 15 centers in seven countries: Greater Britain (5), Israel (1), Germany (1), Turkey (3), South Africa (1), USA (3) and Canada (1).

Participant flow — Overall Study
MilestoneACZ885Placebo
Started2524
Safety analysis set2524
Pharmacokinetics (pk) analysis set250
Primary pd analysis set2524
Completed2219
Not completed35
Withdrew: Lost to follow-up02
Withdrew: Physician decision02
Withdrew: Subject/guardian decision31

Outcome measures

PrimaryChange 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.

Time frame:
Baseline (upto 28 days prior to start of treatment), Week 8 to 12
Reported as:
Mean · Score on a scale
Change From Baseline of 4- Week Average Daily Pain Measured by Visual Analog Score (VAS) Over the Period of Week 8 to 12
Score on a scaleACZ885Placebo
Change From Baseline of 4- Week Average Daily Pain Measured by Visual Analog Score (VAS) Over the Period of Week 8 to 12-0.45 ± 0.384-0.37 ± 0.402
Statistical analysis
  • ACZ885 vs Placebo · Bayesian model for repeated measures · p = 0.55 (probability reduction of average pain score in ACZ885 \> Placebo) · Mean difference (net): -0.07 · 90% CI -1.03 to 0.85Lower limit and upper limit represents the credibility interval from the Bayesian analysis.
SecondaryChange 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.

Time frame:
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
Reported as:
Mean · Score on a scale
Change From Baseline of Average Daily Pain VAS Over 4 Weeks Intervals up to Week 24
Score on a scaleACZ885Placebo
0-4 Weeks-0.337 ± 1.6290.007 ± 1.428
4-8 Weeks-0.173 ± 1.5150.158 ± 1.847
8-12 Weeks-0.444 ± 1.437-0.376 ± 2.104
12-16 Weeks-0.505 ± 1.7440.057 ± 2.371
16-20 Weeks-0.388 ± 1.7720.151 ± 1.811
20-24 Weeks-0.752 ± 1.6780.036 ± 2.131
SecondaryChange 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.

Time frame:
Baseline, Week 12
Reported as:
Geometric least squares mean · mg/L
Change in the Concentration of High Sensitivity C-Reactive Protein (hsCRP) From Baseline to Week 12
mg/LACZ885Placebo
Change in the Concentration of High Sensitivity C-Reactive Protein (hsCRP) From Baseline to Week 120.338 (0.237 to 0.483)0.830 (0.576 to 1.194)
Statistical analysis
  • ACZ885 vs Placebo · Mixed-effect Model for Repeated Measures · p = 0.002 · Ratio: 0.408 · 90% CI 0.253 to 0.658
SecondaryChange 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

Time frame:
Baseline, Week 12
Reported as:
Geometric least squares mean · 10^9 cells/liter
Change in the Concentration of White Blood Cell (WBC) Count From Baseline to Week 12
10^9 cells/literACZ885Placebo
Change in the Concentration of White Blood Cell (WBC) Count From Baseline to Week 120.813 (0.737 to 0.898)1.081 (0.973 to 1.201)
Statistical analysis
  • ACZ885 vs Placebo · Mixed-effect Model for Repeated Measures · p = <.001 · Ratio: 0.752 · 90% CI 0.657 to 0.862
SecondaryChange 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

Time frame:
Baseline, Week 12
Reported as:
Geometric least squares mean · 10^9 cells/liter
Change in the Concentration of Absolute Count of Neutrophils From Baseline to Week 12
10^9 cells/literACZ885Placebo
Change in the Concentration of Absolute Count of Neutrophils From Baseline to Week 120.717 (0.611 to 0.842)1.052 (0.888 to 1.246)
Statistical analysis
  • ACZ885 vs Placebo · Mixed-effect Model for Repeated Measures · p = 0.004 · Ratio: 0.682 · 90% CI 0.547 to 0.849
SecondaryChange 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.

Time frame:
Baseline, Week 12
Reported as:
Geometric least squares mean · 10^9 cells/liter
Change in the Concentration of Absolute Count of Blood Monocytes From Baseline to Week 12
10^9 cells/literACZ885Placebo
Change in the Concentration of Absolute Count of Blood Monocytes From Baseline to Week 120.712 (0.590 to 0.859)0.992 (0.813 to 1.209)
Statistical analysis
  • ACZ885 vs Placebo · Mixed-effect Model for Repeated Measures · p = 0.032 · Ratio: 0.718 · 90% CI 0.556 to 0.925
SecondaryChange in the Concentration of Hemoglobin From Baseline to Week 12

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

Time frame:
Baseline, Week 12
Reported as:
Mean · g/L
Change in the Concentration of Hemoglobin From Baseline to Week 12
g/LACZ885Placebo
Change in the Concentration of Hemoglobin From Baseline to Week 12-0.97 ± 4.7271.11 ± 7.975
SecondaryChange in the Reticulocyte Count From Baseline to Week 12

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

Time frame:
Baseline, Week 12
Reported as:
Mean · 10^9 cells/liter
Change in the Reticulocyte Count From Baseline to Week 12
10^9 cells/literACZ885Placebo
Change in the Reticulocyte Count From Baseline to Week 12-6.578 ± 64.113125.358 ± 47.6483
SecondaryChange in the Concentration of Bilirubin From Baseline to Week 12

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

Time frame:
Baseline, Week 12
Reported as:
Mean · umol/L
Change in the Concentration of Bilirubin From Baseline to Week 12
umol/LACZ885Placebo
Change in the Concentration of Bilirubin From Baseline to Week 125.05 ± 19.796-1.95 ± 11.591
SecondaryChange 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

Time frame:
Baseline, Week 12
Reported as:
Mean · Units per liter (U/L)
Change in the Concentration of Lactate Dehydrogenase (LDH) From Baseline to Week 12
Units per liter (U/L)ACZ885Placebo
Change in the Concentration of Lactate Dehydrogenase (LDH) From Baseline to Week 1219.06 ± 70.862-33.74 ± 209.259
SecondaryChange in the Concentration of Haptoglobin From Baseline to Week 12

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

Time frame:
Baseline, Week 12
Reported as:
Mean · g/L
Change in the Concentration of Haptoglobin From Baseline to Week 12
g/LACZ885Placebo
Change in the Concentration of Haptoglobin From Baseline to Week 12-0.0112 ± 0.04022-0.0213 ± 0.07923
SecondaryChange 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

Time frame:
Baseline, Week 12
Reported as:
Mean · Oxygen Saturation Percent
Change in the Concentration of Oxygen Percent Saturation (SAO2) From Baseline to Week 12
Oxygen Saturation PercentACZ885Placebo
Change in the Concentration of Oxygen Percent Saturation (SAO2) From Baseline to Week 12-0.5 ± 2.16-0.3 ± 1.82
SecondaryNumber 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.

Time frame:
up to Week 24
Reported as:
Mean · Days
Number of Days Absent From School or Work Due to Pain as Recorded by E-diary
DaysACZ885Placebo
Number of Days Absent From School or Work Due to Pain as Recorded by E-diary2.20 (1.69 to 2.70)1.86 (1.31 to 2.41)
Statistical analysis
  • ACZ885 vs Placebo · Generalized Linear Model (GLM) · p = 0.455 · Ratio: 1.40 · 90% CI 0.58 to 3.40
SecondaryNumber 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.

Time frame:
12 weeks
Reported as:
Count of participants · Participants
Number of Acute Blood Transfusions Per Patient by Study Period - Double-blind Period
ParticipantsACZ885Placebo
Overall Acute blood transfusion — 0 transfusions2018
Overall Acute blood transfusion — 1 transfusion44
Overall Acute blood transfusion — 2 transfusions11
Overall Acute blood transfusion — 3 transfusions01
Acute blood transfusion: Rescue — 0 transfusions2319
Acute blood transfusion: Rescue — 1 transfusion24
Acute blood transfusion: Rescue — 2 transfusions01
Acute blood transfusion: Rescue — 3 transfusions00
Acute blood transfusion: Prophylactic — 0 transfusions2222
Acute blood transfusion: Prophylactic — 1 transfusion21
Acute blood transfusion: Prophylactic — 2 transfusions11
Acute blood transfusion: Prophylactic — 3 transfusions00
SecondaryMean 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

Time frame:
Baseline, Week 4, 12, 20 and 24
Reported as:
Mean · ng/mL
Mean Serum Concentration After Repeated Dosing of ACZ885
ng/mLACZ885
Baseline0 ± 0
Week 413100 ± 5490
Week 1218700 ± 5860
Week 2019700 ± 5810
Week 2420600 ± 5930

Adverse events

Collected over Adverse events were collected from first dose of study treatment until end of study treatment (Week 48) plus 8 weeks post treatment, up to maximum duration of 56 weeks.. Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
ACZ8850/25 (0%)11/25 (44%)19/25 (76%)
Placebo0/24 (0%)15/24 (62.5%)20/24 (83.3%)
ACZ885 / ACZ8850/22 (0%)11/22 (50%)17/22 (77.3%)
Placebo / ACZ8850/20 (0%)11/20 (55%)18/20 (90%)
Most frequent serious events
Showing 10 of 24
Most frequent serious events
EventACZ885PlaceboACZ885 / ACZ885Placebo / ACZ885
Sickle cell anaemia with crisisBlood and lymphatic system disorders10/2511/249/2210/20
PneumoniaInfections and infestations0/251/242/221/20
Acute chest syndromeRespiratory, thoracic and mediastinal disorders0/252/240/220/20
AstheniaGeneral disorders0/250/240/221/20
PyrexiaGeneral disorders0/250/240/221/20
CholelithiasisHepatobiliary disorders0/251/240/221/20
AlloimmunisationImmune system disorders0/250/240/221/20
DizzinessNervous system disorders0/250/240/221/20
HeadacheNervous system disorders0/250/240/221/20
Pulmonary embolismRespiratory, thoracic and mediastinal disorders0/250/240/221/20
Most frequent other events
Showing 10 of 123
Most frequent other events
EventACZ885PlaceboACZ885 / ACZ885Placebo / ACZ885
PainGeneral disorders6/255/243/227/20
Upper respiratory tract infectionInfections and infestations1/255/242/226/20
Back painMusculoskeletal and connective tissue disorders2/252/242/225/20
HeadacheNervous system disorders5/253/242/224/20
Abdominal painGastrointestinal disorders0/250/240/223/20
Pain in extremityMusculoskeletal and connective tissue disorders3/253/240/223/20
Non-cardiac chest painGeneral disorders1/251/243/222/20
ConstipationGastrointestinal disorders0/250/241/222/20
NauseaGastrointestinal disorders1/252/240/222/20
InfluenzaInfections and infestations0/251/240/222/20

Baseline characteristics

Age, Continuous
Age, Continuous(Years)ACZ885PlaceboTotal
Mean15.8 ± 2.6915.6 ± 3.2815.7 ± 2.97
Sex: Female, Male
Sex: Female, Male(Participants)ACZ885PlaceboTotal
Female101121
Male151328
Race/Ethnicity, Customized
Race/Ethnicity, Customized(Participants)ACZ885PlaceboTotal
Black or African American121325
White121022
Other112
08

Study locations

15 sites
  • Novartis Investigative Site
    Atlanta, Georgia 30329, United States
  • Novartis Investigative Site
    Augusta, Georgia 30912, United States
  • Novartis Investigative Site
    Greenville, North Carolina 27834, United States
  • Novartis Investigative Site
    Toronto, Ontario M5G 1X8, Canada
  • Novartis Investigative Site
    Hamburg, 20246, Germany
  • Novartis Investigative Site
    Afula, 1834111, Israel
  • Novartis Investigative Site
    Johannesburg, Guateng 2193, South Africa
  • Novartis Investigative Site
    Adana, 01330, Turkey (Türkiye)
  • Novartis Investigative Site
    Ankara, 06100, Turkey (Türkiye)
  • Novartis Investigative Site
    Mersin, 33343, Turkey (Türkiye)
  • Novartis Investigative Site
    Wolverhampton, Staffordshire WS11 5XY, United Kingdom
  • Novartis Investigative Site
    London, E1 1BB, United Kingdom
  • Novartis Investigative Site
    London, NW1 2BU, United Kingdom
  • Novartis Investigative Site
    London, SE1 7EH, United Kingdom
  • Novartis Investigative Site
    London, SE5 9RS, United Kingdom
09

References and documents

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 ↗

Study documents

  • Study protocol · Mar 22, 2018
  • Statistical analysis plan · Jun 20, 2019

Documents are hosted by the registry — open the source record to download them.

Individual participant data

Plan to share: 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

10

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Jan 13, 2026, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT02961218
Lead sponsor
Novartis Pharmaceuticals
Responsible party
Sponsor
First posted
Nov 10, 2016
Start date
Apr 5, 2017
Primary completion
Jun 27, 2019
Completion
Apr 27, 2020
Results posted
Dec 17, 2020
Last update
Jan 13, 2026

Oversight

Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is completed, as verified in Dec 2025. You cannot join it, but the record below documents what was studied.

Follow this study

Get an email when the registry record changes — status, dates, results — or when someone posts here.

Sign in to follow

Discussion

Questions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.

Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.

Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.

Other studies in this condition

Anemia, Sickle Cell→

Other studies from this sponsor

Novartis Pharmaceuticals

Start the discussion