A study for patients who have completed a prior global Novartis or Incyte sponsored ruxolitinib (INC424) study or ruxolitinib and panobinostat (LBH589) combination study and are judged by the investigator to benefit from continued treatment

• Register: EUCTR
• Last refreshed on: 10 December 2019
• Main ID: EUCTR2014-003527-22-BE
• Date of registration: 24/03/2015
• Prospective Registration: Yes
• Primary sponsor: Novartis Pharma Services AG
• Public title: A study for patients who have completed a prior global Novartis or Incyte sponsored ruxolitinib (INC424) study or ruxolitinib and panobinostat (LBH589) combination study and are judged by the investigator to benefit from continued treatment
• Scientific title: An open label, multi-center, Phase IV roll-over protocol for patients who have completed a prior global Novartis or Incyte sponsored ruxolitinib (INC424) study or ruxolitinib and panobinostat (LBH589) combination study and are judged by the investigator to benefit from continued treatment
• Date of first enrolment: 21/04/2015
• Target sample size: 356
• Recruitment status: Authorised-recruitment may be ongoing or finished
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2014-003527-22
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no; Randomised: no; Open: yes; Single blind: no; Double blind: no; Parallel group: no; Cross over: no; Other: no; If controlled, specify comparator, Other Medicinial Product: no; Placebo: no; Other: no
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): yes

Countries of recruitment

Australia, Belgium, Bulgaria, Chile, China, Czech Republic, Denmark, France, Germany, Greece, Hungary, India, Israel, Italy, Japan, Jordan, Korea, Republic of, Lebanon, Mexico, Norway, Poland, Portugal, Romania, Russian Federation, Saudi Arabia, South Africa, Spain, Sweden, Thailand, Turkey, United Kingdom

Contacts

• Name: Clinical trial information desk
• Affiliation: Novartis Pharma AG

• Name: Clinical trial information desk
• Affiliation: Novartis Pharma AG

Key inclusion & exclusion criteria

Inclusion criteria:
- Patient is currently enrolled in a Novartis GDD or GMA-sponsored or Incyte-sponsored clinical study (where Incyte can delegate the sponsorship to a preferred CRO, if applicable) that is approved to enroll into this rollover study, and are receiving either ruxolitinib or combination of ruxolitinib and panobinostat and has fulfilled all of the requirements of the parent protocol.
- Patient is currently benefiting from the treatment with ruxolitinib monotherapy or combination of ruxolitinib and panobinostat, as determined by the investigator
- Patient has demonstrated compliance, as assessed by the investigator, with the parent study protocol requirements
- Willingness and ability to comply with scheduled visits, treatment plans and any other study procedures
- Patient currently has no evidence of progressive disease, as determined by the investigator, following previous treatment with ruxolitinib or combination of ruxolitinib and panobinostat
- Written informed consent obtained prior to enrolling in roll-over study and receiving study medication. If consent cannot be expressed in writing, it must be formally documented and witnessed, ideally via an independent trusted witness.
Note 1: If the patient is a minor, the parent who signs the informed
consent for the minor
must be a legally recognized parent or guardian. Where deemed
appropriate by the clinician, and the child's parent or guardian, the child
will also be included in the alldiscussions about the trials and the minor
aged 12 and above assent will be obtained. The parent or guardian will
sign on the designated line on the ICF attesting to the fact that the child
had given consent.
Note 2: if the minor is an adolescent female, she will be informed during
the assent process that for safety purpose, a pregnancy test is required.
She will also be told that if it is positive, she will be counseled and will
be assisted in telling her parents. If the minor does not want to proceed,
she will be advised not to sign consent and her enrollment in this
protocol will end.
Are the trial subjects under 18? yes
Number of subjects for this age range: 18
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 206
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 132

Exclusion criteria:
- Patient has been permanently discontinued from study treatment in the parent study due to any reason.
- Patient’s indication is currently approved and reimbursed in the local country
- Patient has participated in a combination trial other than the ruxolitinib and panobinostat combination trial (CLBH589X2106) where ruxolitinib was dispensed in combination with another study medication and the patient is still receiving combination therapy.
- Pregnant or nursing (lactating) women, where pregnancy is defined as the state of a female after conception and until the termination of gestation, confirmed by a positive hCG laboratory test.
- Female patients between = 12 and < 18 years of age and 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 as defined below, throughout the study and for up to 30 days after stopping treatment.
- Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using highly effective methods of contraception throughout the study duration inclusive of the 30-day safety follow up. Highly effective contraception methods include: see protocol.



Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Therapeutic area: Diseases [C] - Cancer [C04]

This roll-over study is designed to accept patients with varied disease origins. Please refer to the parent protocol for the disease background information and rationale for use of ruxolitinib in their individual indications.
MedDRA version: 20.1 Level: LLT Classification code 10054658 Term: Thalassemia System Organ Class: 100000004850
MedDRA version: 20.1 Level: PT Classification code 10066264 Term: Acute graft versus host disease in intestine System Organ Class: 10021428 - Immune system disorders
MedDRA version: 20.1 Level: PT Classification code 10066262 Term: Acute graft versus host disease in skin System Organ Class: 10021428 - Immune system disorders
MedDRA version: 20.1 Level: PT Classification code 10066263 Term: Acute graft versus host disease in liver System Organ Class: 10021428 - Immune system disorders
MedDRA version: 20.1 Level: PT Classification code 10066260 Term: Acute graft versus host disease System Organ Class: 10021428 - Immune system disorders
MedDRA version: 20.1 Level: PT Classification code 10066261 Term: Chronic graft versus host disease System Organ Class: 10021428 - Immune system disorders
MedDRA version: 20.1 Level: PT Classification code 10072160 Term: Chronic graft versus host disease in liver System Organ Class: 10021428 - Immune system disorders
MedDRA version: 20.1 Level: PT Classification code 10072158 Term: Chronic graft versus host disease in intestine System Organ Class: 10021428 - Immune system disorders
MedDRA version: 20.1 Level: PT Classification code 10072159 Term: Chronic graft versus host disease in skin System Organ Class: 10021428 - Immune system disorders
MedDRA version: 21.0 Level: LLT Classification code 10074689 Term: Post polycythemia vera myelofibrosis System Organ Class: 100000004864
MedDRA version: 21.0 Level: LLT Classification code 10074690 Term: Post essential thrombocythemia myelofibrosis System Organ Class: 100000004864
MedDRA version: 21.1 Level: L

Intervention(s)

Trade Name: Jakavi
Product Name: Ruxolitinib
Product Code: INC424
Pharmaceutical Form: Tablet
INN or Proposed INN: Ruxolitinib
CAS Number: 1092939-17-7
Current Sponsor code: INC424
Other descriptive name: RUXOLITINIB PHOSPHATE
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 5-

Trade Name: Jakavi
Product Name: Ruxolitinib
Product Code: INC424
Pharmaceutical Form: Tablet
INN or Proposed INN: Ruxolitinib
CAS Number: 1092939-17-7
Current Sponsor code: INC424
Other descriptive name: RUXOLITINIB PHOSPHATE
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 5-

Trade Name: Jakavi
Product Name: Ruxolitinib
Product Code: INC424
Pharmaceutical Form: Tablet
INN or Proposed INN: Ruxolitinib
CAS Number: 1092939-17-7
Current Sponsor code: INC424
Other descriptive name: RUXOLITINIB PHOSPHATE
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 10-

Trade Name: Jakavi
Product Name: Ruxolitinib
Product Code: INC424
Pharmaceutical Form: Tablet
INN or Proposed INN: Ruxolitinib
CAS Number: 1092939-17-7
Current Sponsor code: INC424
Other descriptive name: RUXOLITINIB PHOSPHATE
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 15-

Trade Name: Jakavi
Product Name: Ruxolitinib
Product Code: INC424
Pharmaceutical Form: Tablet
INN or Proposed INN: Ruxolitinib
CAS Number: 1092939-17-7
Current Sponsor code: INC424
Other descriptive name: RUXOLITINIB PHOSPHATE
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 20-

Primary Outcome(s)

Main Objective: To evaluate long term safety data i.e. SAEs and AEs.

Primary end point(s): Frequency and severity of SAEs/AEs

Secondary Objective: To evaluate clinical benefit as assessed by the investigator
Other secondary:
- To evaluate long term safety data by ruxolitinib in monotherapy or in
combination with panobinostat
- To evaluate clinical benefit by ruxolitinib in monotherapy or in combination with panobinostat

Timepoint(s) of evaluation of this end point: Though study completion estimated to be approximately 10 years.

Secondary Outcome(s)

Secondary end point(s): Proportion of patients with clinical benefit as assessed by the investigator at scheduled visits
Other secondary:
- frequency and severity of AEs/SAEs
- Proportion of patients with clinical benefit as assessed by the
investigator at scheduled visits

Timepoint(s) of evaluation of this end point: at every visit that occur every 12 weeks until EOT
other secondary:
- at every visit that occur every 12 weeks until End of Trial
- at every visit that occur every 12 weeks until End of Trial

Secondary ID(s)

2014-003527-22-ES

CINC424A2X01B

NCT02386800

Source(s) of Monetary Support

Novartis Pharma Services AG

Ethics review

Status: Approved
Approval date:
Contact: