A Phase 3 interventional study of Ruxolitinib in Polycythemia Vera, sponsored by Novartis Pharmaceuticals. Completed at 65 sites in 12 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2019-07-18.
Sponsored by Novartis Pharmaceuticals · Phase 3, Interventional, and Treatment
The purpose of this open-label, single arm, multi-center Expanded Treatment Protocol (ETP) was to provide early access to ruxolitinib and evaluate safety information in patients with polycythemia vera (PV) who were hydroxyurea (HU) resistant or intolerant and who had no other standard treatment option, nor did they qualify for another clinical study for PV
229 studies on the registry are indexed under Polycythemia Vera; 54 are open to participants now.
This study's enrollment of 161 is above the median of 55 across 174 interventional studies indexed under Polycythemia Vera.
Browse Polycythemia Vera studies →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.
•Confirmed diagnosis of PV according to the 2008 World Health Organization criteria, palpable spleen, Resistant to or intolerant of hydroxyurea, ECOG performance status of 0, 1 or 2; did not have access to a comparable or satisfactory alternative treatment
Exclusion Criteria:
•Inadequate liver or renal function, Significant bacterial, fungal, parasitic, or viral infection requiring treatment, Active malignancy within the past 5 years, except treated cervical intraepithelial neoplasia, basal cell carcinoma of the skin, or squamous cell carcinoma of the skin, with no evidence for recurrence in the past 3 years., Women who were pregnant or nursing.
All patients will receive ruxolitinib at a starting dose of 10 mg twice daily which could be titrated to most appropriate dose. Dose was not to exceed 25 mg bid nor be less than 5 mg once a day
Drug: Ruxolitinib
supplied as 5 mg, 10 mg and 20 mg tablets to be taken orally
Number of Participants With Adverse Events - All Grades
Summary of adverse events (all grades).
Time frame: Baseline up to approximately 26 months
Change From Baseline in Hematocrit Levels at All Visits
Change in hematocrit levels from Baseline to each visit were measured
Time frame: Up to approximately 26 months
Change From Baseline in Spleen Length
Change in spleen length from Baseline to each visit
Time frame: Up to approximately 26 months
Change From Baseline in Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MPN-SAF TSS)
The MPN-SAF (Appendix 6) was a disease specific questionnaire comprised of 10 items that measures fatigue related to MPN disease and the severity of nine of the most prevalent associated symptoms including: early satiety, abdominal discomfort, inactivity, concentration, night sweats, itching, bone pain, fever and weight loss. There were three recall periods used in this questionnaire, which were 24 hours for fatigue, the past week for symptoms of early satiety, abdominal discomfort, inactivity, concentration, night sweats, itching, bone pain and fever, and the past 6 months for weight loss, Each item was scored on a scale ranging from 0 (no fatigue/absent) to 10 (As bad as you can imagine/worst imaginable). The MPN-SAF TSS was computed as the average of the observed items multiplied by 10 to achieve a 0-to-100 scale. The MPN-SAF TSS thus had a possible score range of 0 to 100.
Time frame: Up to approximately 26 months
| Milestone | All Patients |
|---|---|
| Started | 161 |
| Completed | 141 |
| Not completed | 20 |
| Withdrew: Adverse event | 12 |
| Withdrew: Death | 1 |
| Withdrew: Disease progression | 2 |
| Withdrew: Subject/guardian decision | 2 |
| Withdrew: Withdrawal by subject | 3 |
Summary of adverse events (all grades).
| Participants | All Patients |
|---|---|
| Adverse events | 143 |
| Serious adverse events | 26 |
Change in hematocrit levels from Baseline to each visit were measured
| percentage | Baseline | Post-baseline | Change |
|---|---|---|---|
| Week 4 | 45.26 ± 5.246 | 43.70 ± 5.220 | -1.55 ± 3.652 |
| Week 8 | 45.21 ± 5.136 | 40.73 ± 4.967 | -4.48 ± 5.055 |
| Week 12 | 45.44 ± 5.324 | 39.97 ± 5.419 | -5.47 ± 6.522 |
| Week 16 | 45.64 ± 5.395 | 39.32 ± 5.454 | -6.32 ± 6.928 |
| Week 20 | 45.96 ± 5.353 | 40.47 ± 5.323 | -5.49 ± 6.840 |
| Week 24 | 45.93 ± 5.109 | 40.32 ± 4.960 | -5.62 ± 6.073 |
| Week 36 | 45.30 ± 4.602 | 40.43 ± 4.959 | -4.86 ± 6.184 |
| Week 48 | 45.41 ± 4.714 | 39.82 ± 4.959 | -5.59 ± 6.318 |
| Week 60 | 46.56 ± 5.299 | 40.60 ± 5.105 | 40.60 ± 5.929 |
| Week 72 | 47.66 ± 5.377 | 40.59 ± 4.056 | -7.07 ± 6.388 |
| Week 84 | 48.15 ± 5.758 | 41.93 ± 5.097 | -6.23 ± 6.492 |
| Week 96 | 50.03 ± 4.702 | 40.82 ± 4.496 | -9.21 ± 6.861 |
| EOT | 45.38 ± 5.202 | 39.93 ± 5.730 | -5.45 ± 5.847 |
Change in spleen length from Baseline to each visit
| cm | Baseline | Post-baseline | Change |
|---|---|---|---|
| Week 12 | 23.92 ± 14.618 | 16.83 ± 12.931 | -7.09 ± 12.770 |
| Week 24 | 22.66 ± 14.319 | 16.26 ± 13.896 | -6.40 ± 14.101 |
| Week 36 | 21.87 ± 13.228 | 16.70 ± 14.419 | -5.18 ± 10.929 |
| Week 48 | 22.14 ± 13.994 | 18.76 ± 16.880 | -3.38 ± 14.438 |
| Week 60 | 20.00 ± 13.007 | 17.35 ± 12.357 | -2.65 ± 13.753 |
| Week 72 | 18.41 ± 13.148 | 13.47 ± 11.441 | -4.94 ± 13.840 |
| Week 84 | 19.57 ± 14.233 | 13.36 ± 9.443 | -6.21 ± 12.135 |
| Week 96 | 19.92 ± 14.494 | 12.33 ± 8.026 | -7.58 ± 11.237 |
| End of treatment | 3.03 ± 3.419 | 0.54 ± 1.661 | -2.49 ± 3.025 |
The MPN-SAF (Appendix 6) was a disease specific questionnaire comprised of 10 items that measures fatigue related to MPN disease and the severity of nine of the most prevalent associated symptoms including: early satiety, abdominal discomfort, inactivity, concentration, night sweats, itching, bone pain, fever and weight loss. There were three recall periods used in this questionnaire, which were 24 hours for fatigue, the past week for symptoms of early satiety, abdominal discomfort, inactivity, concentration, night sweats, itching, bone pain and fever, and the past 6 months for weight loss, Each item was scored on a scale ranging from 0 (no fatigue/absent) to 10 (As bad as you can imagine/worst imaginable). The MPN-SAF TSS was computed as the average of the observed items multiplied by 10 to achieve a 0-to-100 scale. The MPN-SAF TSS thus had a possible score range of 0 to 100.
| scores | Baseline | Post-baseline | Change |
|---|---|---|---|
| Week 12 | 23.92 ± 14.618 | 16.83 ± 12.931 | -7.09 ± 12.770 |
| Week 24 | 22.66 ± 14.319 | 16.26 ± 13.896 | -6.40 ± 14.101 |
| Week 36 | 21.87 ± 13.228 | 16.70 ± 14.419 | -5.18 ± 10.929 |
| Week 48 | 22.14 ± 13.994 | 18.76 ± 16.880 | -3.38 ± 14.438 |
| Week 60 | 20.00 ± 13.007 | 17.35 ± 12.357 | -2.65 ± 13.753 |
| Week 72 | 18.41 ± 13.148 | 13.47 ± 11.441 | -4.94 ± 13.840 |
| Week 84 | 19.57 ± 14.233 | 13.36 ± 9.443 | -6.21 ± 12.135 |
| Week 96 | 19.92 ± 14.494 | 12.33 ± 8.026 | -7.58 ± 11.237 |
| End of treatment | 22.86 ± 14.225 | 18.12 ± 15.130 | -4.74 ± 13.954 |
Collected over Adverse Events (AEs) were collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV). All AEs reported in this record are from date of First Patient First Treatment until Last Patient Last Visit) up to approximately 26 months. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| All Patients | 1/161 (0.6%) | 26/161 (16.1%) | 107/161 (66.5%) |
| Event | All Patients |
|---|---|
| AnaemiaBlood and lymphatic system disorders | 2/161 |
| PneumoniaInfections and infestations | 2/161 |
| Squamous cell carcinomaNeoplasms benign, malignant and unspecified (incl cysts and polyps) | 2/161 |
| Bundle branch block bilateralCardiac disorders | 1/161 |
| PericarditisCardiac disorders | 1/161 |
| Ventricular extrasystolesCardiac disorders | 1/161 |
| CataractEye disorders | 1/161 |
| AscitesGastrointestinal disorders | 1/161 |
| DiarrhoeaGastrointestinal disorders | 1/161 |
| Retroperitoneal haematomaGastrointestinal disorders | 1/161 |
| Event | All Patients |
|---|---|
| AnaemiaBlood and lymphatic system disorders | 35/161 |
| HeadacheNervous system disorders | 27/161 |
| DiarrhoeaGastrointestinal disorders | 15/161 |
| ConstipationGastrointestinal disorders | 14/161 |
| FatigueGeneral disorders | 14/161 |
| Weight increasedInvestigations | 13/161 |
| PruritusSkin and subcutaneous tissue disorders | 13/161 |
| AstheniaGeneral disorders | 12/161 |
| DizzinessNervous system disorders | 12/161 |
| ThrombocytosisBlood and lymphatic system disorders | 11/161 |
| Age, Customized(participants) | All Patients |
|---|---|
| <= 60 | 52 |
| >60 | 109 |
| Sex: Female, Male(Participants) | All Patients |
|---|---|
| Female | 65 |
| Male | 96 |
| Race/Ethnicity, Customized(participants) | All Patients |
|---|---|
| Caucasian | 128 |
| Asian | 3 |
| Other | 30 |
| Number of participants resistant or intolerant to hydroxyurea (HU)(participants) | All Patients |
|---|---|
| Resistant to hydroxyurea | 60 |
| Intolerant to hydroxyurea | 101 |
| Number of participants' frequency of phlebotomy in 52 weeks prior to screening(participants) | All Patients |
|---|---|
| 1 phlebotomy | 21 |
| 2 phlebotomies | 19 |
| >=3 phlebotomies | 74 |
| Missing | 47 |
| Number of participants' use of prior antineoplastic therapy(participants) | All Patients |
|---|---|
| Prior Hydroxyurea use | 160 |
| Prior other anti-neoplastic medications use | 59 |
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Novartis Pharmaceuticals