Trial · NCT02049450
Study of Efficacy and Safety of INC424 in Regularly Transfused Patients With Thalassemia.
NCT02049450, Phase 2, Completed, Novartis Pharmaceuticals; results first posted 2017-06-15
3 sourcestrials-2026-11-12-0.5.0
Download JSON
Cite this record
ConductScience Library. Study of Efficacy and Safety of INC424 in Regularly Transfused Patients With Thalassemia. Trials, record NCT02049450. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02049450
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study of Efficacy and Safety of INC424 in Regularly Transfused Patients With Thalassemia.
- NCT number
- NCT02049450
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 30 participants, actual
- Lead sponsor
- Novartis Pharmaceuticals
- Conditions
- Thalassemia Major
- Interventions
- ruxolitinib
- First posted
- 2014-01-30
- Last updated
- 2017-07-17
- Other registries' IDs
- eudract:2013-002812-28
- Results first posted
- 2017-06-15
Source ClinicalTrials.gov
Registered outcomes6
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change of Hematocrit Adjusted Volume of Red Blood Cells (RBC) | 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). |
| Secondary | Percentage Change in Spleen Volume (cm3) | baseline, week 12, week 30 | Change of spleen volume from baseline at week 12 and week 30 as measured by magnetic imaging resonance (MRI) or computed tomography (CT). |
| Secondary | Percentage Change in Mean Pre-transfusion Hemoglobin by 6 Week Time Intervals | baseline, weeks 0 - 30 | Change from baseline in pre-transfusion hemoglobin levels |
| Secondary | Percentage Change in Spleen Length (cm) Below the Left Coastal Margin | baseline, weeks 1,2,3,4,6,12,18,24,30 | Change of spleen length from baseline over time measured by palpitation by time |
| Secondary | Pharmacokinetics (PK) Parameter of Cmin | 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. |
| Secondary | Pharmacokinetics (PK) Parameter of Cmax | 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. |
Source ClinicalTrials.gov
Outcomes6
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change of Hematocrit Adjusted Volume of Red Blood Cells (RBC) | week 6 to week 30 interval | Per-Protocol Set (PPS) consisted of a subset of patients in the Safety Set who were compliant with requirements of the Study Protocol. Patients were excluded from the PPS if: they had no or incomplete history of RBC transfusions within 24 weeks prior to the first dose of ruxolitinib or discontinued treatment with ruxolitinib prior to Week 18. | % change of hematocrit-adjusted volume | MEAN |
| SECONDARY | Percentage Change in Spleen Volume (cm3) | baseline, week 12, week 30 | The Safety Set consisted of all patients who received at least one dose of ruxolitinib. All safety data was analyzed using the Safety set. The FAS and Safety set are identical in this study. | percentage change | MEAN |
| SECONDARY | Percentage Change in Mean Pre-transfusion Hemoglobin by 6 Week Time Intervals | baseline, weeks 0 - 30 | The Safety Set consists of all patients who received at least one dose of ruxolitinib. All safety data was analyzed using the Safety set. The FAS and Safety set are identical in this study. | percentage change of hemoglobin levels | MEAN |
| SECONDARY | Percentage Change in Spleen Length (cm) Below the Left Coastal Margin | baseline, weeks 1,2,3,4,6,12,18,24,30 | The Safety Set consists of all patients who received at least one dose of ruxolitinib. All safety data was analyzed using the Safety set. The FAS and Safety set are identical in this study. | percentage change in spleen length | MEAN |
| SECONDARY | Pharmacokinetics (PK) Parameter of Cmin | week 2, week 12 | The PK analysis set includes all patients with at least one evaluable PK sample at any visit. | ng/mL | MEAN |
| SECONDARY | Pharmacokinetics (PK) Parameter of Cmax | Day 1, Week 2 (Day 15), Week 12 (Day 85) | The PK analysis set includes all patients with at least one evaluable PK sample at any visit. | ng/mL | MEAN |
Source ClinicalTrials.gov
Results by group35
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change of Hematocrit Adjusted Volume of Red Blood Cells (RBC) | INC424 (Ruxolitinib) - Study Treatment | -5.934 | Standard Deviation 22.1681 | % change of hematocrit-adjusted volume | |
| Percentage Change in Spleen Volume (cm3) | INC424 (Ruxolitinib) - Study Treatment | % change from baseline at Week 12 | -19.733 | Standard Deviation 16.0539 | percentage change |
| Percentage Change in Spleen Volume (cm3) | INC424 (Ruxolitinib) - Study Treatment | % change from baseline at Week 30 | -26.829 | Standard Deviation 16.6936 | percentage change |
| Percentage Change in Mean Pre-transfusion Hemoglobin by 6 Week Time Intervals | INC424 (Ruxolitinib) - Study Treatment | Weeks 0 - 6 ) | 0.43 | Standard Deviation 10.135 | percentage change of hemoglobin levels |
| Percentage Change in Mean Pre-transfusion Hemoglobin by 6 Week Time Intervals | INC424 (Ruxolitinib) - Study Treatment | Weeks 6 - 12 | 2.87 | Standard Deviation 10.555 | percentage change of hemoglobin levels |
| Percentage Change in Mean Pre-transfusion Hemoglobin by 6 Week Time Intervals | INC424 (Ruxolitinib) - Study Treatment | Weeks 12 - 18 | 2.78 | Standard Deviation 11.081 | percentage change of hemoglobin levels |
| Percentage Change in Mean Pre-transfusion Hemoglobin by 6 Week Time Intervals | INC424 (Ruxolitinib) - Study Treatment | Weeks 18 - 24 | -0.56 | Standard Deviation 9.760 | percentage change of hemoglobin levels |
| Percentage Change in Mean Pre-transfusion Hemoglobin by 6 Week Time Intervals | INC424 (Ruxolitinib) - Study Treatment | Weeks 24 - 30 | 0.06 | Standard Deviation 14.321 | percentage change of hemoglobin levels |
| Percentage Change in Spleen Length (cm) Below the Left Coastal Margin | INC424 (Ruxolitinib) - Study Treatment | Week 1 | -11.19 | Standard Deviation 15.376 | percentage change in spleen length |
| Percentage Change in Spleen Length (cm) Below the Left Coastal Margin | INC424 (Ruxolitinib) - Study Treatment | Week 2 | -22.11 | Standard Deviation 23.604 | percentage change in spleen length |
1 to 10 of 35Source ClinicalTrials.gov
See all 35 results by groupParticipant flow5
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | INC424 (Ruxolitinib) - Study Treatment | 30 | |
| Overall Study | Discontinued Treatment Prior to Week 30 | INC424 (Ruxolitinib) - Study Treatment | 4 | |
| Overall Study | Entered Extension Phase | INC424 (Ruxolitinib) - Study Treatment | 18 | |
| Overall Study | COMPLETED | INC424 (Ruxolitinib) - Study Treatment | 26 | |
| Overall Study | NOT COMPLETED | INC424 (Ruxolitinib) - Study Treatment | 4 |
Source ClinicalTrials.gov
Adverse events29
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Anaemia | Blood and lymphatic system disorders | INC424 (Ruxolitinib) - Study Treatment | 4 | 30 | |
| other | Abdominal pain | Gastrointestinal disorders | INC424 (Ruxolitinib) - Study Treatment | 2 | 30 | |
| other | Abdominal pain upper | Gastrointestinal disorders | INC424 (Ruxolitinib) - Study Treatment | 5 | 30 | |
| other | Constipation | Gastrointestinal disorders | INC424 (Ruxolitinib) - Study Treatment | 2 | 30 | |
| other | Diarrhoea | Gastrointestinal disorders | INC424 (Ruxolitinib) - Study Treatment | 5 | 30 | |
| other | Nausea | Gastrointestinal disorders | INC424 (Ruxolitinib) - Study Treatment | 5 | 30 | |
| other | Fatigue | General disorders | INC424 (Ruxolitinib) - Study Treatment | 3 | 30 | |
| other | Pyrexia | General disorders | INC424 (Ruxolitinib) - Study Treatment | 2 | 30 | |
| other | Gastroenteritis | Infections and infestations | INC424 (Ruxolitinib) - Study Treatment | 3 | 30 | |
| other | Nasopharyngitis | Infections and infestations | INC424 (Ruxolitinib) - Study Treatment | 3 | 30 |
1 to 10 of 29Source ClinicalTrials.gov
See all 29 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | INC424 (Ruxolitinib) - Study Treatment | ||
| Total, other adverse events | INC424 (Ruxolitinib) - Study Treatment | 23 | 30 |
| Total, serious adverse events | INC424 (Ruxolitinib) - Study Treatment | 6 | 30 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT02049450
GET https://conductscience.org/api/trials/v1/NCT02049450/results?page=2