Clinical trial NCT02091752: 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
Overall status: TERMINATED
Phase: PHASE2
Conditions: Primary Myelofibrosis
Keywords: Primary Myelofibrosis
Hematologic Diseases
Myeloproliferative Disorders
INC424
Ruxolitinib
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2014-03-19
Enrollment: 3
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


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

Primary outcomes:
measure: Proportion of Patients Achieving ≥20% Reduction From Baseline in Spleen Volume
timeFrame: Week 24
Secondary outcomes:
measure: Proportion of Patients Achieving ≥35% Reduction From Baseline in Spleen Volume
timeFrame: Week 24
measure: Proportion of Patients Achieving ≥25% and ≥50% Reduction, Respectively From Baseline, in Spleen Length
timeFrame: Week 24
measure: Change From Baseline in Spleen Length and Spleen Volume
timeFrame: Baseline, Week 24
measure: Proportion of Patients Achieving ≥25% and ≥50% Reduction, Respectively, From Baseline in Total Symptom Score (MPN-SAF TSS)
timeFrame: Week 24
measure: Change From Baseline in MPN-SAF TSS Score
timeFrame: Baseline, Week 24
measure: Patient Global Impression of Change (PGIC) Score
timeFrame: Week 1, Week 24
measure: Change From Baseline in European Organisation for Research and Treatment of Cancer (EORTC) QLQ-C30 and EuroQol (EQ)-5D-5L Scores
timeFrame: Baseline, Day 1, Week 8, Week 12, Week 16, Week 24
Other outcomes:
Locations:
city: Leipzig
country: Germany
facility: Novartis Investigative Site
city: Firenze
country: Italy
facility: Novartis Investigative Site
state: FI
city: Madrid
country: Spain
facility: Novartis Investigative Site
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: