Back to resultsA 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)
Primary Purpose
Primary Myelofibrosis
Status
Terminated
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
Ruxolitinib
Sponsored by
About this trial
This is an interventional treatment trial for Primary Myelofibrosis focused on measuring Primary Myelofibrosis, Hematologic Diseases, Myeloproliferative Disorders, INC424, Ruxolitinib
Eligibility Criteria
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
Sites / Locations
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Ruxolitinib
Arm Description
All participants received ruxolitinib.
Outcomes
Primary Outcome Measures
Proportion of Patients Achieving ≥20% Reduction From Baseline in Spleen Volume
Secondary Outcome Measures
Proportion of Patients Achieving ≥35% Reduction From Baseline in Spleen Volume
Proportion of Patients Achieving ≥25% and ≥50% Reduction, Respectively From Baseline, in Spleen Length
Change From Baseline in Spleen Length and Spleen Volume
Proportion of Patients Achieving ≥25% and ≥50% Reduction, Respectively, From Baseline in Total Symptom Score (MPN-SAF TSS)
Change From Baseline in MPN-SAF TSS Score
Patient Global Impression of Change (PGIC) Score
Change From Baseline in European Organisation for Research and Treatment of Cancer (EORTC) QLQ-C30 and EuroQol (EQ)-5D-5L Scores
Full Information
NCT ID
NCT02091752
First Posted
March 6, 2014
Last Updated
February 24, 2016
Sponsor
Novartis Pharmaceuticals
1. Study Identification
Unique Protocol Identification Number
NCT02091752
Brief Title
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)
Official Title
The ReTreatment Trial: A Phase II, Open-label, Single-arm Study of Re-treating Myelofibrosis Patients With Ruxolitinib/Jakavi After Treatment Interruption Due to Loss of Response and/or Adverse Event.
Study Type
Interventional
2. Study Status
Record Verification Date
February 2016
Overall Recruitment Status
Terminated
Why Stopped
The study was terminated due to low enrollment.
Study Start Date
September 2014 (undefined)
Primary Completion Date
February 2015 (Actual)
Study Completion Date
February 2015 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Novartis Pharmaceuticals
4. Oversight
Data Monitoring Committee
No
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Primary Myelofibrosis
Keywords
Primary Myelofibrosis, Hematologic Diseases, Myeloproliferative Disorders, INC424, Ruxolitinib
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
3 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Ruxolitinib
Arm Type
Experimental
Arm Description
All participants received ruxolitinib.
Intervention Type
Drug
Intervention Name(s)
Ruxolitinib
Intervention Description
Starting dose was based on reason for previous discontinuation of ruxolitinib (i.e. loss of response or AE) and baseline platelet count. For participants who previously discontinued ruxolitinib due to loss of response, the starting dose was determined based on baseline platelet counts as follows: participants with a baseline platelet count of ≥ 200 x 109/L began dosing at 20 mg po bid; participants with a baseline platelet count of 100 x 109/L to <200 x 109/L began dosing at 15 mg po bid. Participants who previously discontinued ruxolitinib due to an AE initiated therapy at a total daily dose 5 mg lower than the total daily dose prior to discontinuation.
Primary Outcome Measure Information:
Title
Proportion of Patients Achieving ≥20% Reduction From Baseline in Spleen Volume
Time Frame
Week 24
Secondary Outcome Measure Information:
Title
Proportion of Patients Achieving ≥35% Reduction From Baseline in Spleen Volume
Time Frame
Week 24
Title
Proportion of Patients Achieving ≥25% and ≥50% Reduction, Respectively From Baseline, in Spleen Length
Time Frame
Week 24
Title
Change From Baseline in Spleen Length and Spleen Volume
Time Frame
Baseline, Week 24
Title
Proportion of Patients Achieving ≥25% and ≥50% Reduction, Respectively, From Baseline in Total Symptom Score (MPN-SAF TSS)
Time Frame
Week 24
Title
Change From Baseline in MPN-SAF TSS Score
Time Frame
Baseline, Week 24
Title
Patient Global Impression of Change (PGIC) Score
Time Frame
Week 1, Week 24
Title
Change From Baseline in European Organisation for Research and Treatment of Cancer (EORTC) QLQ-C30 and EuroQol (EQ)-5D-5L Scores
Time Frame
Baseline, Day 1, Week 8, Week 12, Week 16, Week 24
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Novartis Pharmaceuticals
Organizational Affiliation
Novartis Pharmaceuticals
Official's Role
Study Director
Facility Information:
Facility Name
Novartis Investigative Site
City
Leipzig
ZIP/Postal Code
04103
Country
Germany
Facility Name
Novartis Investigative Site
City
Firenze
State/Province
FI
ZIP/Postal Code
50134
Country
Italy
Facility Name
Novartis Investigative Site
City
Madrid
ZIP/Postal Code
28034
Country
Spain
12. IPD Sharing Statement
Learn more about this trial
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)
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