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Polycythemia VeraPhase 3NCT02292446

Expanded Treatment Protocol (ETP) of Ruxolitinib in Patients With Polycythemia Vera Who Were Hydroxyurea Resistant or Intolerant and for Whom no Treatment Alternatives Was Available.

A Phase 3 study testing Ruxolitinib in people with Polycythemia Vera, run by Novartis Pharmaceuticals at 65 sites in 12 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial summary

The purpose of this open-label, single arm, multi-center Expanded Treatment Protocol (ETP) was to provide early access to ruxolitinib and evaluate safety information in patients with polycythemia vera (PV) who were hydroxyurea (HU) resistant or intolerant and who had no other standard treatment option, nor did they… Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Polycythemia Vera
What is being tested?
Ruxolitinib
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
161 participants
Where
65 sitesin 12 countries
Study dates
21 Nov 2014to 29 Dec 2017
Phase
Phase 3
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • •Confirmed diagnosis of PV according to the 2008 World Health Organization criteria, palpable spleen, Resistant to or intolerant of hydroxyurea, ECOG performance status of 0, 1 or 2; did not have access to a comparable or satisfactory alternative treatment

Who cannot join

  • •Inadequate liver or renal function, Significant bacterial, fungal, parasitic, or viral infection requiring treatment, Active malignancy within the past 5 years, except treated cervical intraepithelial neoplasia, basal cell carcinoma of the skin, or squamous cell carcinoma of the skin, with no evidence for recurrence in the past 3 years., Women who were pregnant or nursing.

Trial locations and contacts

65 sites in 12 countries.

65 sites in 12 countries

Germany19 sites

  • Novartis Investigative Site

    Aschaffenburg, 63739

  • Novartis Investigative Site

    Augsburg, 86150

  • Novartis Investigative Site

    Bad Soden, 65812

  • Novartis Investigative Site

    Berlin, 13357

  • Novartis Investigative Site

    Bottrop, 46236

  • Novartis Investigative Site

    Eisenach, 99817

  • Novartis Investigative Site

    Erlangen, 91054

  • Novartis Investigative Site

    Frankfurt, 60389

  • Novartis Investigative Site

    Frankfurt, 60596

  • Novartis Investigative Site

    Friedrichshafen, 88045

  • Novartis Investigative Site

    Hamburg, 22081

  • Novartis Investigative Site

    Hamm, 59063

  • Novartis Investigative Site

    Heidelberg, 69115

  • Novartis Investigative Site

    Heilbronn, 74072

  • Novartis Investigative Site

    Koblenz, 56068

  • Novartis Investigative Site

    Mannheim, Baden-Wurttemberg, 68305

  • Novartis Investigative Site

    Mutlangen, 73557

  • Novartis Investigative Site

    Stuttgart, 70376

  • Novartis Investigative Site

    Würzburg, 97080

Other trials in Germany

France17 sites

  • Novartis Investigative Site

    Angers, 49033

  • Novartis Investigative Site

    Avignon, 84902

  • Novartis Investigative Site

    Bayonne, Bayonne Cedex, 64109

  • Novartis Investigative Site

    Bordeaux, 33076

  • Novartis Investigative Site

    Chambéry, 73011

  • Novartis Investigative Site

    Le Mans, Cedex 09, 72037

  • Novartis Investigative Site

    Marseille, 13273

  • Novartis Investigative Site

    Meaux, 77104

  • Novartis Investigative Site

    Metz, 57000

  • Novartis Investigative Site

    Mulhouse, 68070

  • Novartis Investigative Site

    Nice, 06202

  • Novartis Investigative Site

    Paris, 75010

  • Novartis Investigative Site

    Perpignan, 66046

  • Novartis Investigative Site

    Pringy, 74374

  • Novartis Investigative Site

    Toulouse, 31059

  • Novartis Investigative Site

    Vandœuvre-lès-Nancy, 54511

  • Novartis Investigative Site

    Villejuif, 94805

Other trials in France

Belgium6 sites

  • Novartis Investigative Site

    Antwerp, 2060

  • Novartis Investigative Site

    Bruges, 8000

  • Novartis Investigative Site

    Brussels, 1070

  • Novartis Investigative Site

    Leuven, 3000

  • Novartis Investigative Site

    Liège, 4000

  • Novartis Investigative Site

    Yvoir, 5530

Other trials in Belgium

Bulgaria4 sites

  • Novartis Investigative Site

    Pleven, 5800

  • Novartis Investigative Site

    Plovdiv, 4002

  • Novartis Investigative Site

    Sofia, 1413

  • Novartis Investigative Site

    Sofia, 1431

Other trials in Bulgaria

Thailand4 sites

  • Novartis Investigative Site

    Bangkok, 10400

  • Novartis Investigative Site

    Bangkok, 10700

  • Novartis Investigative Site

    Chiang Mai, 50200

  • Novartis Investigative Site

    Khon Kaen, THA, 40002

Other trials in Thailand

Austria3 sites

  • Novartis Investigative Site

    Linz, A-4010

  • Novartis Investigative Site

    Salzburg, 5020

  • Novartis Investigative Site

    Wels, A 4600

Other trials in Austria

Chile3 sites

  • Novartis Investigative Site

    Santiago, 8420383

  • Novartis Investigative Site

    Santiago

  • Novartis Investigative Site

    Viña del Mar, Valparaiso, 2540364

Other trials in Chile

Canada2 sites

  • Novartis Investigative Site

    Hamilton, Ontario, L8V 5C2

  • Novartis Investigative Site

    Vancouver, British Columbia, V5Z 1M9

Other trials in Canada

Norway2 sites

  • Novartis Investigative Site

    Fredrikstad, NO-1603

  • Novartis Investigative Site

    Tromsø, 9038

Other trials in Norway

Portugal2 sites

  • Novartis Investigative Site

    Lisbon, 1099 023

  • Novartis Investigative Site

    Lisbon, 1749-035

Other trials in Portugal

Sweden2 sites

  • Novartis Investigative Site

    Luleå, SE 971 80

  • Novartis Investigative Site

    Uddevalla, 451 80

Other trials in Sweden

Mexico1 site

  • Novartis Investigative Site

    Monterrey, Nuevo León, 64000

Other trials in Mexico

About this study

Official title: An Open-label, Multi-center, Expanded Treatment Protocol (ETP) of Ruxolitinib in Patients With Polycythemia Vera Who Are Hydroxyurea Resistant or Intolerant and for Whom no Treatment Alternatives Are Available.

The purpose of this open-label, single arm, multi-center Expanded Treatment Protocol (ETP) was to provide early access to ruxolitinib and evaluate safety information in patients with polycythemia vera (PV) who were hydroxyurea (HU) resistant or intolerant and who had no other standard treatment option, nor did they qualify for another clinical study for PV

Being tested
Ruxolitinib
Keywords
Polycythemia Vera, Hematologic Diseases, Myeloproliferative Disorders, INC424, Ruxolitinib, hydroxyurea resistant, adult
Medical subject headings
Polycythemia Vera, Hematologic Diseases, Myeloproliferative Disorders

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • RuxolitinibDrug

    supplied as 5 mg, 10 mg and 20 mg tablets to be taken orally

Study groups in NCT02292446
Study groupRoleWhat they receive
All patientsExperimentalAll patients will receive ruxolitinib at a starting dose of 10 mg twice daily which could be titrated to most appropriate dose. Dose was not to exceed 25 mg bid nor be less than 5 mg once a day
Receives: Drug: Ruxolitinib

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Number of Participants With Adverse Events - All Grades

    Summary of adverse events (all grades).

    Measured: Baseline up to approximately 26 months

Secondary outcomes (3)

  1. Change From Baseline in Hematocrit Levels at All Visits

    Change in hematocrit levels from Baseline to each visit were measured

    Measured: Up to approximately 26 months

  2. Change From Baseline in Spleen Length

    Change in spleen length from Baseline to each visit

    Measured: Up to approximately 26 months

  3. Change From Baseline in Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MPN-SAF TSS)

    The MPN-SAF (Appendix 6) was a disease specific questionnaire comprised of 10 items that measures fatigue related to MPN disease and the severity of nine of the most prevalent associated symptoms including: early satiety, abdominal discomfort, inactivity, concentration, night sweats, itching, bone pain, fever and weight loss. There were three recall periods used in this questionnaire, which were 24 hours for fatigue, the past week for symptoms of early satiety, abdominal discomfort, inactivity, concentration, night sweats, itching, bone pain and fever, and the past 6 months for weight loss, Each item was scored on a scale ranging from 0 (no fatigue/absent) to 10 (As bad as you can imagine/worst imaginable). The MPN-SAF TSS was computed as the average of the observed items multiplied by 10 to achieve a 0-to-100 scale.

    The MPN-SAF TSS thus had a possible score range of 0 to 100.

    Measured: Up to approximately 26 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted17 Nov 2014 (estimated)
  2. Study start21 Nov 2014
  3. Primary completion29 Dec 2017
  4. Study completion29 Dec 2017

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
161 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02292446
Sponsor's protocol ID
CINC424B2001X
Status last verified
Jul 2019
First submitted
5 Nov 2014
Last updated
18 Jul 2019
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Ruxolitinib in people with Polycythemia Vera, run by Novartis Pharmaceuticals at 65 sites in 12 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 1 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 18 Jul 2019. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 65 sites in Germany, France, Belgium, Bulgaria, Thailand and 7 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 2 Apr 2019). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of Participants With Adverse Events - All Grades (time frame: Baseline up to approximately 26 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02292446.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Jul 2019.

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