18 and older, any sex, with Thalassemia Major. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Change of Hematocrit Adjusted Volume of Red Blood Cells (RBC)Primary· week 6 to week 30 interval
Change of RBC transfusion requirement measured as percent change of the hematocrit-adjusted volume of transfused RBC and observed during within on-treatment interval (any time-points of RBC transfusion between week 6 and week 30 driven by the individual patient's need) compared to baseline (defined by pre-treatment interval between Week - 24 to start of treatment).
Change of spleen volume from baseline at week 12 and week 30 as measured by magnetic imaging resonance (MRI) or computed tomography (CT).
% change from baseline at Week 12
Group
Value
95% CI
INC424 (Ruxolitinib) - Study Treatment
-19.733
± 16.0539
% change from baseline at Week 30
Group
Value
95% CI
INC424 (Ruxolitinib) - Study Treatment
-26.829
± 16.6936
Percentage Change in Mean Pre-transfusion Hemoglobin by 6 Week Time IntervalsSecondary· baseline, weeks 0 - 30
Change from baseline in pre-transfusion hemoglobin levels
Weeks 0 - 6 )
Group
Value
95% CI
INC424 (Ruxolitinib) - Study Treatment
0.43
± 10.135
Weeks 6 - 12
Group
Value
95% CI
INC424 (Ruxolitinib) - Study Treatment
2.87
± 10.555
Weeks 12 - 18
Group
Value
95% CI
INC424 (Ruxolitinib) - Study Treatment
2.78
± 11.081
Weeks 18 - 24
Group
Value
95% CI
INC424 (Ruxolitinib) - Study Treatment
-0.56
± 9.760
Weeks 24 - 30
Group
Value
95% CI
INC424 (Ruxolitinib) - Study Treatment
0.06
± 14.321
Percentage Change in Spleen Length (cm) Below the Left Coastal MarginSecondary· baseline, weeks 1,2,3,4,6,12,18,24,30
Change of spleen length from baseline over time measured by palpitation by time
Week 1
Group
Value
95% CI
INC424 (Ruxolitinib) - Study Treatment
-11.19
± 15.376
Week 2
Group
Value
95% CI
INC424 (Ruxolitinib) - Study Treatment
-22.11
± 23.604
Week 3
Group
Value
95% CI
INC424 (Ruxolitinib) - Study Treatment
-25.01
± 24.178
Week 4
Group
Value
95% CI
INC424 (Ruxolitinib) - Study Treatment
-26.94
± 25.343
Week 6
Group
Value
95% CI
INC424 (Ruxolitinib) - Study Treatment
-33.85
± 25.251
Week 12
Group
Value
95% CI
INC424 (Ruxolitinib) - Study Treatment
-49.29
± 26.792
Week 18
Group
Value
95% CI
INC424 (Ruxolitinib) - Study Treatment
-56.32
± 29.994
Week 24
Group
Value
95% CI
INC424 (Ruxolitinib) - Study Treatment
-56.93
± 29.552
Pharmacokinetics (PK) Parameter of CminSecondary· week 2, week 12
C min of INC424 by actual dose administered from 10mg bid to 20mg bid. Plasma PK samples were collected at Day 15 (Week 2), and Day 85 (Week 12). Cmin was collected immediately prior to dosing. n= number of patients with valid PK samples as per definition of the PK analysis set.
Week 2 (Day 15)
Group
Value
95% CI
10 mg Bid
7.5800
± 7.57959
15mg Bid
NA
± NA
20mg Bid
NA
± NA
Week 12 (Day 85)
Group
Value
95% CI
10 mg Bid
9.1300
± 7.61039
15mg Bid
18.5400
± 23.99940
20mg Bid
20.2300
± 25.98617
Pharmacokinetics (PK) Parameter of CmaxSecondary· Day 1, Week 2 (Day 15), Week 12 (Day 85)
Cmax (1h) of INC424 by actual dose administered from 10mg bid to 20mg bid. Plasma PK samples were collected at Day 1, Week 2, and Week 12. Cmax was collected within a +/- 1 hour post dose.
n= number of patients with valid PK samples as per definition of the PK analysis set.
Day 1
Group
Value
95% CI
5mg Bid
58.2000
± 0.00
10mg Bid
126.8000
± 58.70337
15mg Bid
0.00
± 0.00
20mg Bid
0.00
± 0.00
Week 2
Group
Value
95% CI
5mg Bid
56.7000
± 0.00
10mg Bid
125.400
± 40.61805
15mg Bid
0.00
± 0.00
20mg Bid
0.00
± 0.00
Week 12
Group
Value
95% CI
5mg Bid
0.00
± 0.00
10mg Bid
107.2100
± 50.07525
15mg Bid
245.6900
± 50.00362
20mg Bid
185.0000
± 97.58074
Adverse events — posted to ClinicalTrials.gov
Time frame: Adverse Events are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV). All Adverse Events reported in this record are from date of First Patient First Treatment until Last Patient Last Visit..
Reporting threshold: 5%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
Patients with severe thalassemia (thalassemia major) present with severe anemia that required life-long transfusion therapy, spleen enlargement that led to increased transfusion requirement, and other serious complications as early death, growth retardation, bone deformations and iron overload due to blood transfusions. Splenectomy can significantly reduce transfusion requirement in thalassemia patients, but it is associated with an increased risk of serious complications such as sepsis and thrombosis. Preliminary preclinical and clinical data suggested that JAK2 inhibition, by reducing spleen size, could improve hemoglobin levels, thereby eliminating the need for splenectomy and reducing transfusion requirement and related iron overload.
Publications & conference data
8 peer-reviewed publications reference this trial (live from Europe PMC):
NCT05621733 — A PMS of Jakavi® in Patients With Steroid-Refractory Graft-versus-Host Disease (SR-GvHD) in Korea
· recruiting
NCT04551053 — To Evaluate Efficacy and Safety of Parsaclisib and Ruxolitinib in Participants With Myelofibrosis Who Have Suboptimal Re
· Phase 3
· terminated
NCT04551066 — To Evaluate the Efficacy and Safety of Parsaclisib and Ruxolitinib in Participants With Myelofibrosis (LIMBER-313)
· Phase 3
· terminated
NCT04455841 — INCB000928 Administered as a Monotherapy or in Combination With Ruxolitinib in Participants With Anemia Due to Myeloprol
· Phase 1, PHASE2
· active not recruiting
NCT04530344 — Assess the Long Term Efficacy and Safety of Ruxolitinib Cream in Participants With Vitiligo
· Phase 3
· completed
Other recruiting trials for Thalassemia Major
Currently open trials in the same condition.
NCT07112703 — Effect of Incentive Respiratory Training on Pulmonary Functions and Functional Capacity in Children With B-thalassemia M
· NA
· recruiting
NCT06931912 — Growth Evaluation, Health Promotion, and Clinical Management in Children and Adolescents With Thalassemia
· NA
· recruiting
NCT06490627 — Unraveling the Impact of Thalidomide at Diverse Doses in Transfusion Dependent Beta Thalassemia
· Phase 2
· recruiting
NCT06041620 — Safety and Efficacy Evaluation of Autologous CRISPR-Cas12b Edited Hematopoietic Stem Cells
· NA
· recruiting
NCT06913634 — Study to Evaluate the Long- Term Safety and Efficacy of Luspatercept in Subjects Who Received at Least One Dose of Luspa
· active not recruiting
Other Novartis Pharmaceuticals trials
Trials by the same sponsor.
NCT07498335 — Study to Assess the Efficacy, Pharmacokinetics, Safety and Tolerability of Atrasentan in Pediatric Patients With Primary
· Phase 3
· not yet recruiting
NCT07489573 — Study of Efficacy and Safety of Secukinumab in Chinese Adult Patients With Moderate to Severe Hidradenitis Suppurativa
· Phase 4
· not yet recruiting
NCT07484269 — PULSE Registry: for Patients Receiving Lutetium (177Lu) Vipivotide Tetraxetan
· not yet recruiting
NCT07416162 — A Study of Iptacopan in Korean Patients With Paroxysmal Nocturnal Hemoglobinuria or C3 Glomerulopathy
· not yet recruiting
NCT07387926 — Safety and Efficacy of Asciminib in Pediatrics and Young Adults With Relapse/Refractory (r/r) Philadelphia Positive (Ph+
· Phase 1, PHASE2
· not yet recruiting
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Novartis Pharmaceuticals
Last refreshed: 17 July 2017
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT02049450.