Phase 3 N=161 Treatment

Expanded Treatment Protocol (ETP) of Ruxolitinib in Patients With Polycythemia Vera Who Were Hydroxyurea Resistant or Intolerant and for Whom no Treatment Alternatives Was Available.

Polycythemia Vera

Bottom Line

View on ClinicalTrials.gov: NCT02292446 ↗

Enrolled (actual)

161

Serious AEs

16.2%

Results posted

Apr 2019

Primary outcome: Primary: Number of Participants With Adverse Events - All Grades — 143; 26 Participants

Study Design & Population

Study type: Interventional
Phase: Phase 3
Interventions: Ruxolitinib (Drug)
Age: Adult, Older Adult · 18+ yrs
Sex: All
Sponsor: Novartis Pharmaceuticals
Primary completion: Dec 2017

Outcome Measures

Outcome	Result	p-value
PRIMARY Number of Participants With Adverse Events - All Grades	143; 26	—
SECONDARY Change From Baseline in Hematocrit Levels at All Visits	45.26; 43.70; -1.55; 45.21; 40.73; -4.48	—
SECONDARY Change From Baseline in Spleen Length	23.92; 16.83; -7.09; 22.66; 16.26; -6.40	—
SECONDARY Change From Baseline in Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MPN-SAF TSS)	23.92; 16.83; -7.09; 22.66; 16.26; -6.40	—

Summary

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

Eligibility Criteria

Inclusion Criteria

•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.

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Data sourced from ClinicalTrials.gov (NCT02292446). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.