CINC424A2X01B Rollover Protocol

Open Label, Multi-center, Phase IV Study of Ruxolitinib or Ruxolitinib and Panobinostat Combination, for Patients Who Have Completed Prior Global Novartis or Incyte Sponsored Studies

This is a long term safety study for patients that have been treated with either ruxolitinib or a combination of ruxolitinib with panobinostat, on a Novartis or Incyte sponsored study, who have been judged by the study Investigator to benefit from ongoing treatment.

Study Overview

• Status: Recruiting
• Conditions: Acute Myeloid Leukemia; Primary Myelofibrosis; Polycythemia Vera; Thalassemia; Graft Versus Host Disease
• Intervention / Treatment: Drug: ruxolitinib; Drug: panobinostat

Detailed Description

This roll-over protocol allows patients from multiple protocols, who are still receiving clinical benefit, to continue their treatment in one study that covers multiple indications. The population for the roll-over study should be consistent with the population defined in the parent studies. The primary eligibility criteria for a patient to enter the roll-over protocol is the participation and completion of a Novartis or Incyte study with ruxolitinib monotherapy or combination of ruxolitinib and panobinostat. Efficacy parameters will not be measured; however safety data and an evaluation of clinical benefit will be collected.

• Study Type: Interventional
• Enrollment (Estimated): 356
• Phase: Phase 4

Contacts and Locations

• Study Contact: Name: Novartis Pharmaceuticals; Phone Number: +41613241111
• Study Contact Backup: Name: Novartis Pharmaceuticals; Phone Number: +81337978748

Study Locations

• Australia
  • New South Wales
    • Darlinghurst, New South Wales, Australia, 2010
      • Completed
      • Novartis Investigative Site
    • St Leonards, New South Wales, Australia, 2065
      • Completed
      • Novartis Investigative Site
  • Queensland
    • Herston, Queensland, Australia, 4029
      • Completed
      • Novartis Investigative Site
    • Wooloongabba, Queensland, Australia, 4102
      • Completed
      • Novartis Investigative Site
  • South Australia
    • Bedford Park, South Australia, Australia, 5041
      • Completed
      • Novartis Investigative Site
  • Victoria
    • Box Hill, Victoria, Australia, 3128
      • Completed
      • Novartis Investigative Site
    • Clayton, Victoria, Australia, 3168
      • Completed
      • Novartis Investigative Site
    • Franston, Victoria, Australia, 3199
      • Completed
      • Novartis Investigative Site
  • Western Australia
    • Perth, Western Australia, Australia, 6000
      • Completed
      • Novartis Investigative Site
• Belgium
  • Antwerpen, Belgium, 2020
    • Completed
    • Novartis Investigative Site
  • Brugge, Belgium, 8000
    • Completed
    • Novartis Investigative Site
  • Leuven, Belgium, 3000
    • Completed
    • Novartis Investigative Site
  • Liege, Belgium, 4000
    • Completed
    • Novartis Investigative Site
• Bulgaria
  • Pleven, Bulgaria, 5800
    • Completed
    • Novartis Investigative Site
  • Plovdiv, Bulgaria, 4002
    • Completed
    • Novartis Investigative Site
  • Sofia, Bulgaria, 1413
    • Completed
    • Novartis Investigative Site
  • Sofia, Bulgaria, 1431
    • Completed
    • Novartis Investigative Site
• Chile
  • Santiago, Chile, 8420383
    • Completed
    • Novartis Investigative Site
  • RM
    • Santiago, RM, Chile, 7500922
      • Completed
      • Novartis Investigative Site
  • Valparaiso
    • Vina del Mar, Valparaiso, Chile, 2540364
      • Completed
      • Novartis Investigative Site
• China
  • Beijing, China, 100730
    • Active, not recruiting
    • Novartis Investigative Site
  • Zhejiang
    • Hangzhou, Zhejiang, China, 310003
      • Completed
      • Novartis Investigative Site
• Denmark
  • Copenhagen, Denmark, DK-2100
    • Withdrawn
    • Novartis Investigative Site
• France
  • Paris 10, France, 75475
    • Completed
    • Novartis Investigative Site
• Germany
  • Aachen, Germany, 52074
    • Active, not recruiting
    • Novartis Investigative Site
  • Dresden, Germany, 01307
    • Completed
    • Novartis Investigative Site
  • Hamburg, Germany, 20246
    • Completed
    • Novartis Investigative Site
  • Magdeburg, Germany, 39120
    • Active, not recruiting
    • Novartis Investigative Site
  • Mainz, Germany, 55131
    • Active, not recruiting
    • Novartis Investigative Site
  • Ulm, Germany, 89081
    • Completed
    • Novartis Investigative Site
• Greece
  • Athens, Greece, 115 27
    • Completed
    • Novartis Investigative Site
  • Patras, Greece, 265 00
    • Completed
    • Novartis Investigative Site
• Hungary
  • Budapest, Hungary, 1097
    • Completed
    • Novartis Investigative Site
  • Debrecen, Hungary, 4032
    • Completed
    • Novartis Investigative Site
  • Kaposvar, Hungary, 7400
    • Completed
    • Novartis Investigative Site
  • Kecskemet, Hungary, 6001
    • Completed
    • Novartis Investigative Site
  • Szeged, Hungary, H 6725
    • Completed
    • Novartis Investigative Site
  • Szombathely, Hungary, 9700
    • Completed
    • Novartis Investigative Site
• India
  • Maharashtra
    • Pune, Maharashtra, India, 411004
      • Active, not recruiting
      • Novartis Investigative Site
• Israel
  • Afula, Israel, 1834111
    • Completed
    • Novartis Investigative Site
  • Jerusalem, Israel, 9112001
    • Completed
    • Novartis Investigative Site
• Italy
  • Napoli, Italy, 80131
    • Completed
    • Novartis Investigative Site
  • AN
    • Ancona, AN, Italy, 60126
      • Completed
      • Novartis Investigative Site
  • BA
    • Bari, BA, Italy, 70124
      • Completed
      • Novartis Investigative Site
  • BO
    • Bologna, BO, Italy, 40138
      • Completed
      • Novartis Investigative Site
  • BS
    • Brescia, BS, Italy, 25123
      • Active, not recruiting
      • Novartis Investigative Site
  • FG
    • San Giovanni Rotondo, FG, Italy, 71013
      • Active, not recruiting
      • Novartis Investigative Site
  • FI
    • Firenze, FI, Italy, 50134
      • Active, not recruiting
      • Novartis Investigative Site
  • MI
    • Milano, MI, Italy, 20162
      • Completed
      • Novartis Investigative Site
    • Milano, MI, Italy, 20122
      • Completed
      • Novartis Investigative Site
    • Milano, MI, Italy, 20132
      • Active, not recruiting
      • Novartis Investigative Site
    • Rozzano, MI, Italy, 20089
      • Completed
      • Novartis Investigative Site
  • PA
    • Palermo, PA, Italy, 90146
      • Completed
      • Novartis Investigative Site
  • PV
    • Pavia, PV, Italy, 27100
      • Active, not recruiting
      • Novartis Investigative Site
  • RC
    • Reggio Calabria, RC, Italy, 89124
      • Active, not recruiting
      • Novartis Investigative Site
  • RM
    • Roma, RM, Italy, 00165
      • Recruiting
      • Novartis Investigative Site
    • Roma, RM, Italy, 00161
      • Completed
      • Novartis Investigative Site
  • TO
    • Orbassano, TO, Italy, 10043
      • Completed
      • Novartis Investigative Site
  • VA
    • Varese, VA, Italy, 21100
      • Active, not recruiting
      • Novartis Investigative Site
• Japan
  • Osaka, Japan, 545-8586
    • Completed
    • Novartis Investigative Site
  • Aichi
    • Nagoya, Aichi, Japan, 466 8560
      • Recruiting
      • Novartis Investigative Site
    • Nagoya, Aichi, Japan, 453-8511
      • Completed
      • Novartis Investigative Site
  • Fukuoka
    • Fukuoka city, Fukuoka, Japan, 812-8582
      • Completed
      • Novartis Investigative Site
  • Hokkaido
    • Sapporo city, Hokkaido, Japan, 060 8648
      • Completed
      • Novartis Investigative Site
  • Kanagawa
    • Isehara, Kanagawa, Japan, 259-1193
      • Completed
      • Novartis Investigative Site
  • Osaka
    • Suita, Osaka, Japan, 565 0871
      • Completed
      • Novartis Investigative Site
  • Tokyo
    • Bunkyo ku, Tokyo, Japan, 113-8677
      • Completed
      • Novartis Investigative Site
• Korea, Republic of
  • Seoul, Korea, Republic of, 03080
    • Recruiting
    • Novartis Investigative Site
  • Seoul, Korea, Republic of, 03080
    • Active, not recruiting
    • Novartis Investigative Site
  • Seoul, Korea, Republic of, 03722
    • Active, not recruiting
    • Novartis Investigative Site
  • Seocho Gu
    • Seoul, Seocho Gu, Korea, Republic of, 06591
      • Active, not recruiting
      • Novartis Investigative Site
• Lebanon
  • Beirut, Lebanon, 1107 2020
    • Completed
    • Novartis Investigative Site
• Mexico
  • Nuevo Leon
    • Monterrey, Nuevo Leon, Mexico, 64718
      • Completed
      • Novartis Investigative Site
• Poland
  • Wroclaw, Poland, 50 367
    • Active, not recruiting
    • Novartis Investigative Site
  • Slaskie
    • Gliwice, Slaskie, Poland, 44-101
      • Active, not recruiting
      • Novartis Investigative Site
• Portugal
  • Lisboa, Portugal, 1099 023
    • Completed
    • Novartis Investigative Site
  • Lisboa, Portugal, 1649-035
    • Completed
    • Novartis Investigative Site
• Russian Federation
  • Moscow, Russian Federation, 125167
    • Completed
    • Novartis Investigative Site
  • Moscow, Russian Federation, 129110
    • Completed
    • Novartis Investigative Site
  • Petrozavodsk, Russian Federation, 185019
    • Completed
    • Novartis Investigative Site
  • Saint Petersburg, Russian Federation, 197022
    • Active, not recruiting
    • Novartis Investigative Site
• Slovakia
  • Bratislava, Slovakia, 833 40
    • Withdrawn
    • Novartis Investigative Site
• South Africa
  • Pretoria, South Africa, 0027
    • Completed
    • Novartis Investigative Site
  • Pretoria, South Africa, 0044
    • Active, not recruiting
    • Novartis Investigative Site
  • Gauteng
    • Soweto, Gauteng, South Africa, 2013
      • Completed
      • Novartis Investigative Site
  • Western Province
    • Cape Town, Western Province, South Africa, 7800
      • Active, not recruiting
      • Novartis Investigative Site
• Spain
  • Castilla Y Leon
    • Salamanca, Castilla Y Leon, Spain, 37007
      • Completed
      • Novartis Investigative Site
  • Catalunya
    • Barcelona, Catalunya, Spain, 08003
      • Completed
      • Novartis Investigative Site
  • Navarra
    • Pamplona, Navarra, Spain, 31008
      • Completed
      • Novartis Investigative Site
• Sweden
  • Huddinge, Sweden, SE-14186
    • Active, not recruiting
    • Novartis Investigative Site
  • Lulea, Sweden, SE 971 80
    • Active, not recruiting
    • Novartis Investigative Site
  • Lund, Sweden, SE-221 85
    • Active, not recruiting
    • Novartis Investigative Site
  • Uddevalla, Sweden, 451 80
    • Active, not recruiting
    • Novartis Investigative Site
• Thailand
  • Bangkok, Thailand, 10700
    • Completed
    • Novartis Investigative Site
• Turkey
  • Istanbul, Turkey, 34093
    • Completed
    • Novartis Investigative Site
  • Izmir, Turkey, 35040
    • Completed
    • Novartis Investigative Site
  • Talas / Kayseri, Turkey, 38039
    • Completed
    • Novartis Investigative Site
  • Sihhiye
    • Ankara, Sihhiye, Turkey, 06100
      • Completed
      • Novartis Investigative Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 month and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Key Inclusion criteria:

1. Patient is currently enrolled in a Novartis GDD or GMA-sponsored or Incyte-sponsored clinical study, are receiving either ruxolitinib or combination of ruxolitinib and panobinostat, and fulfilled all of the requirements of the parent protocol.
2. Patient is currently benefiting from the treatment with ruxolitinib monotherapy or combination of ruxolitinib and panobinostat, as determined by the investigator
3. Patient has demonstrated compliance, as assessed by the investigator, with the parent study protocol requirements
4. Patient currently has no evidence of progressive disease, as determined by the investigator, following previous treatment with ruxolitinib or combination of ruxolitinib and panobinostat

Key Exclusion criteria:

1. Patient has been permanently discontinued from study treatment in the parent study due to any reason.
2. Patient's indication is currently approved and reimbursed in the corresponding country for ruxolitinib monotherapy or combination of ruxolitinib and panobinostat.
3. Pregnant or nursing (lactating) women.
4. Female patients of childbearing potential (e.g. are menstruating) who do not agree to abstinence or, if sexually active, do not agree to the use of highly effective contraception, throughout the study and for up to 30 days after stopping study treatment.

Other protocol-defined Inclusion / Exclusion criteria may apply.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Ruxolitinib monotherapy or ruxolitinib plus panobinostat in combination; All participants will receive either ruxolitinib monotherapy or ruxolitinib in combination with panobinostat, at the same dose/schedule that they were taking in the parent study.	Drug: ruxolitinib; ruxolitinib tablets or pediatric oral solution - participants continue ruxolitinib as they received in parent study; Other Names: Jakavi; INC424; Drug: panobinostat; panobinostat capsules - participants to continue receiving panobinostat in combination with ruxolitinib as per the parent study; Other Names: Farydak; LBH589

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence and severity of AEs and SAEs	The incidence of treatment-emergent AEs and SAEs (new or worsening from baseline) will be summarized by system organ class and/or preferred term, severity (based on CTCAE grades).	12 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Proportion of participants with clinical benefit as assessed by the investigator at scheduled visits.	The analysis of clinical benefit will be descriptive only and cannot be compared to efficacy results of other studies. In addition, as clinical benefit was only added in protocol amendment 1, it may not be available for all participants.	12 years
Incidence and severity of AEs and SAEs by treatment group	AEs and SAEs for participants in the Safety group will be listed and summarized by treatment group (ruxolitinib monotherapy / ruxolitinib + panobinostat combination)	12 years

Collaborators and Investigators

• Sponsor: Novartis Pharmaceuticals
• Investigators: Study Director: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Study record dates

Study Major Dates

• Study Start (Actual): March 5, 2015
• Primary Completion (Estimated): September 16, 2027
• Study Completion (Estimated): September 16, 2027

Study Registration Dates

• First Submitted: March 6, 2015
• First Submitted That Met QC Criteria: March 6, 2015
• First Posted (Estimated): March 12, 2015

Study Record Updates

• Last Update Posted (Actual): March 25, 2024
• Last Update Submitted That Met QC Criteria: March 22, 2024
• Last Verified: March 1, 2024

More Information

Terms related to this study

• Keywords: Acute Myeloid Leukemia; Ruxolitinib; AML; Myelofibrosis; Thalassemia; INC424; MF; Panobinostat; LBH589; Polycythemia vera; PV; Chronic Graft versus Host Disease; Acute Graft versus Host Disease; aGvHD; cGvHD
• Additional Relevant MeSH Terms: Immune System Diseases; Neoplasms by Histologic Type; Neoplasms; Neoplasms by Site; Bone Marrow Diseases; Hematologic Diseases; Genetic Diseases, Inborn; Myeloproliferative Disorders; Anemia; Leukemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Hemoglobinopathies; Bone Marrow Neoplasms; Hematologic Neoplasms; Primary Myelofibrosis; Leukemia, Myeloid; Leukemia, Myeloid, Acute; Thalassemia; Graft vs Host Disease; Polycythemia Vera; Polycythemia; Molecular Mechanisms of Pharmacological Action; Enzyme Inhibitors; Antineoplastic Agents; Histone Deacetylase Inhibitors; Panobinostat
• Other Study ID Numbers: CINC424A2X01B; 2014-003527-22 (EudraCT Number)

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No