Study to Determine the Safety and Efficacy of INCB018424 in Patients With Polycythemia Vera or Essential Thrombocythemia

NCT00726232 | Terminated Phase 2 Results

• 3 other identifiers: INCB 18424-256Ruxolitinib2008-001382-28
• Study Type: interventional
• Target: 73
• Locations: 2 countries
• Sites: 4

Brief Summary

To evaluate the safety and efficacy profile of different treatment regimens of Ruxolitinib (INCB018424) administered to two groups of patients; those with polycythemia vera (PV) and those with essential thrombocythemia (ET). Patients in each group were refractory to hydroxyurea or for whom hydroxyurea is contraindicated.

Conditions

Myeloproliferative Neoplasm (MPN)

Outcome Measures

Primary Outcomes (2)

• Percentage of Polycythemia Vera Participants With a Confirmed Clinical Partial Response (PR) or Complete Response (CR)

  For a confirmed response all criteria must have been sustained for at least 2 months. CR: * Hematocrit \< 45% in men and \< 42% in women * No phlebotomy for 1 month * No palpable splenomegaly * White blood cells \< 10 x 10\^9/L with normal differential and platelets \< 400 x 10\^9/L * No sustained leucopenia or thrombocytopenia (\>2 weeks) * No systemic PV symptoms (pruritus, night sweats, bone pain, fever, weight loss) PR: * Hematocrit \< 45% in men and \< 42% in women * 50% reduction in phlebotomy requirements from 6 months before treatment started * 50% reduction in palpable splenomegaly

  Assessed after 2 cycles (56 days) of treatment on Day 1 of Cycle 3

• Percentage of Essential Thrombocythemia (ET) Participants With a Confirmed Clinical Partial Response (PR) or Complete Response (CR)

  For a confirmed response all criteria must have been sustained for at least 2 months. Complete Clinical Response: * Platelet count \< 400 x 10\^9/L * White blood cell count \< 10 x 10\^9/L with normal differential and Hematocrit ≤ upper limit of normal * Absence of sustained (\> 2 weeks) anemia or leucopenia based on institutional normal ranges * Absence of systemic ET symptoms (pruritus, bone pain, weakness, night sweats, paresthesias) * Absence of palpable splenomegaly Partial Clinical Response: * Platelet count \< 400 x 10\^9/L * 50% reduction in palpable splenomegaly

  Assessed after 2 cycles (56 days) of treatment on Day 1 of Cycle 3.

Secondary Outcomes (9)

• Percentage of Polycythemia Vera Participants Who Achieved Individual Components of Clinical Response at 12 Weeks

  Baseline and Week 12 (Cycle 4, Day 1)

• Percentage of Polycythemia Vera Participants Who Achieved Individual Components of Clinical Response at 24 Weeks

  Baseline and Week 24 (Cycle 7, Day 1)

• Percentage of Polycythemia Vera Participants Who Achieved Individual Components of Clinical Response at 36 Weeks

  Baseline and Week 36 (Cycle 10, Day 1)

• Percentage of Essential Thrombocythemia Participants Who Achieved Individual Components of Clinical Response at 4 Weeks

  Baseline and 4 weeks (Cycle 2, Day 1)

• Percentage of Essential Thrombocythemia Participants Who Achieved Individual Components of Clinical Response at 24 Weeks

  Baseline and 24 weeks (Cycle 7, Day 1)

Study Arms (3)

Ruxolitinib 10 mg BID

EXPERIMENTAL

Participants received 10 mg Ruxolitinib orally twice a day (BID) for 56 days (two 28-day cycles) during the dose-ranging phase. After patients completed 2 cycles of treatment at the randomized dose, Investigators were permitted to adjust the dose/regimen on an individual basis to achieve an optimal balance of efficacy and safety. Treatment continued until a patient met a withdrawal criterion, had intolerable toxicity, progression of disease, or withdrew consent.

Drug: Ruxolitinib

Ruxolitinib 25 mg BID

EXPERIMENTAL

Participants received 25 mg Ruxolitinib orally twice a day (BID) for 56 days (two 28-day cycles) during the dose-ranging phase. After patients completed 2 cycles of treatment at the randomized dose, Investigators were permitted to adjust the dose/regimen on an individual basis to achieve an optimal balance of efficacy and safety. Treatment continued until a patient met a withdrawal criterion, had intolerable toxicity, progression of disease, or withdrew consent.

Drug: Ruxolitinib

Ruxolitinib 50 mg QD

EXPERIMENTAL

Participants received 50 mg Ruxolitinib orally once a day (QD) for 56 days (two 28-day cycles) during the dose-ranging phase. After patients completed 2 cycles of treatment at the randomized dose, Investigators were permitted to adjust the dose/regimen on an individual basis to achieve an optimal balance of efficacy and safety. Treatment continued until a patient met a withdrawal criterion, had intolerable toxicity, progression of disease, or withdrew consent.

Drug: Ruxolitinib

Interventions

Ruxolitinib DRUG

Ruxolitinib was administered orally and supplied as 5 mg and 25 mg tablets.

Also known as: INCB018424

Ruxolitinib 10 mg BID; Ruxolitinib 25 mg BID; Ruxolitinib 50 mg QD

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 polycythemia vera or essential thrombocythemia as determined by treating physician
• Disease refractory to hydroxyurea or for whom treatment with hydroxyurea is contraindicated or have refused further treatment with hydroxyurea due to side effects.
• Patient meets baseline clinical lab parameters

You may not qualify if:

• Treatment with interferon alpha or anagrelide within 7 days and hydroxyurea within 1 day of starting INCB018424.
• Patients diagnosed with another malignancy unless the malignancy was cervical intraepithelial neoplasia or basal or squamous cell skin cancer and the patient has been disease free for \> 3 years
• Patients receiving therapy with intermediate or high dose steroids greater than the equivalent of 10 mg prednisone per day
• Clinically significant cardiac disease (New York Heart Association (NYHA) Class III or IV)

Sponsors & Collaborators

• Incyte Corporation: lead

Study Sites (4)

Unknown Facility

Houston, Texas, 77030, United States

Location

Unknown Facility

Bergamo, Italy

Location

Unknown Facility

Florence, Italy

Location

Unknown Facility

Pavia, Italy

Location

Related Publications (1)

• Pieri L, Pancrazzi A, Pacilli A, Rabuzzi C, Rotunno G, Fanelli T, Guglielmelli P, Fjerza R, Paoli C, Verstovsek S, Vannucchi AM. JAK2V617F complete molecular remission in polycythemia vera/essential thrombocythemia patients treated with ruxolitinib. Blood. 2015 May 21;125(21):3352-3. doi: 10.1182/blood-2015-01-624536. No abstract available.

  PMID: 25999444 DERIVED

MeSH Terms

Conditions

Myeloproliferative Disorders

Interventions

ruxolitinib

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Results Point of Contact

• Title: Study Director
• Organization: Incyte Corporation

medinfo@incyte.com

1-855-463-3463

Study Officials

• Albert Assad, MD

  Incyte Corporation

  STUDY DIRECTOR

Publication Agreements

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

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR
• Expanded Access: Yes

Study Record Dates

• First Submitted: July 29, 2008
• First Posted: July 31, 2008
• Study Start: August 20, 2008
• Primary Completion: June 20, 2010
• Study Completion: August 20, 2018
• Last Updated: October 29, 2025
• Results First Posted: February 24, 2012

Record last verified: 2025-10

Locations

US (1); IT (3)