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)

NCT02091752 | Terminated Phase 2 Results

• 1 other identifier: CINC424A2407
• Study Type: interventional
• Target: 3
• Locations: 3 countries
• Sites: 3

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.

Conditions

Primary Myelofibrosis

Keywords

Primary Myelofibrosis; Hematologic Diseases; Myeloproliferative Disorders; INC424; Ruxolitinib

Outcome Measures

Primary Outcomes (1)

• Proportion of Patients Achieving ≥20% Reduction From Baseline in Spleen Volume

  Week 24

Secondary Outcomes (7)

• 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 Spleen Length

  Week 24

• Change From Baseline in Spleen Length and Spleen Volume

  Baseline, Week 24

• Proportion of Patients Achieving ≥25% and ≥50% Reduction, Respectively, From Baseline in Total Symptom Score (MPN-SAF TSS)

  Week 24

• Change From Baseline in MPN-SAF TSS Score

  Baseline, Week 24

Study Arms (1)

Ruxolitinib

EXPERIMENTAL

All participants received ruxolitinib.

Drug: Ruxolitinib

Interventions

Ruxolitinib DRUG

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.

Ruxolitinib

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (3)

Novartis Investigative Site

Leipzig, 04103, Germany

Location

Novartis Investigative Site

Florence, FI, 50134, Italy

Location

Novartis Investigative Site

Madrid, Madrid, 28034, Spain

Location

MeSH Terms

Conditions

Primary Myelofibrosis; Hematologic Diseases; Myeloproliferative Disorders

Interventions

ruxolitinib

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hemic and Lymphatic Diseases

Results Point of Contact

• Title: Study Director
• Organization: Novartis

862-778-8300

Study Officials

• Novartis Pharmaceuticals

  Novartis Pharmaceuticals

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 6, 2014
• First Posted: March 19, 2014
• Study Start: September 1, 2014
• Primary Completion: February 1, 2015
• Study Completion: February 1, 2015
• Last Updated: March 24, 2016
• Results First Posted: February 10, 2016

Record last verified: 2016-02

Locations

ES (1); IT (1); DE (1)