A Phase II Study of Re-treatment of Myelofibrosis Patients With Ruxolitinib/Jakavi After Treatment Interruption Due to Loss of Response and/or Adverse Event (ReTreatment Trial)

Phase 2

Terminated

• Conditions: Primary Myelofibrosis
• Interventions: Drug: Ruxolitinib

• Registration Number: NCT02091752
• Lead Sponsor: Novartis Pharmaceuticals

Brief Summary

The aim of the study is to assess the efficacy and safety of restarting ruxolitinib after treatment interruption due to loss of response and/or adverse events.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2014-03-06
• Study First Submitted QC: 2014-03-17
• Study First Posted: 2014-03-19
• Study Start: 2014-09
• Primary Completion: 2015-02
• Study Completion: 2015-02
• Results First Submitted: 2016-01-05
• Results First Submitted QC: 2016-01-05
• Status Verified: 2016-02
• Results First Posted: 2016-02-10
• Last Update Submitted: 2016-02-24
• Last Update Posted: 2016-03-24

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 3

Inclusion Criteria

• Confirmed diagnosis of PMF, PPV MF or PET-MF, irrespective of JAK2 mutational status according to the 2008 revised International Standard Criteria
• Peripheral blast count < 10%
• Requires therapy for MF in the opinion of the investigator
• Received prior monotherapy treatment with ruxolitinib for at least 12 consecutive weeks and experienced treatment interruption because of lossof response or adverse event
• Patients adhering to the Screening phase assessments and undergoing a a ruxolitinib-free washout period of a minimum of 1 week and a maximum of 8 weeks
• ECOG performance status 0, 1, 2, or 3
• Adequate bone marrow function
• Written informed consent

Exclusion Criteria

• Patients not initially responding (primary resistance) to ruxolitinib therapy
• Patients who underwent a splenectomy or spleen radiation
• Patients currently scheduled for bone marrow transplant
• Patients who have discontinued ruxolitinib < 14 days prior to screening
• Patients who are not able to receive a starting dose of ruxolitinib of at least 15 mg total daily dose
• Leukemic transformation
• Inadequate renal function
• Presence of clinically meaningful active bacterial, fungal, parasitic or viral infection which requires therapy
• Previous history of Progressive Multifocal Leuko-encephalopathy (PML)
• Clinically significant cardiac disease or significant concurrent medical condition

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Ruxolitinib	Ruxolitinib	All participants received ruxolitinib.

Primary Outcome Measures

Name	Time	Method
Proportion of Patients Achieving ≥20% Reduction From Baseline in Spleen Volume	Week 24

Secondary Outcome Measures

Name	Time	Method
Change From Baseline in Spleen Length and Spleen Volume	Baseline, Week 24
Proportion of Patients Achieving ≥25% and ≥50% Reduction, Respectively From Baseline, in Spleen Length	Week 24
Patient Global Impression of Change (PGIC) Score	Week 1, Week 24
Change From Baseline in European Organisation for Research and Treatment of Cancer (EORTC) QLQ-C30 and EuroQol (EQ)-5D-5L Scores	Baseline, Day 1, Week 8, Week 12, Week 16, Week 24
Change From Baseline in MPN-SAF TSS Score	Baseline, Week 24
Proportion of Patients Achieving ≥35% Reduction From Baseline in Spleen Volume	Week 24
Proportion of Patients Achieving ≥25% and ≥50% Reduction, Respectively, From Baseline in Total Symptom Score (MPN-SAF TSS)	Week 24

Trial Locations

Locations (1)

Novartis Investigative Site; 🇪🇸 Madrid, Spain