Phase III Study of SAR302503 in Intermediate-2 and High Risk Patients With Myelofibrosis
- Conditions
- Hematopoietic Neoplasm
- Interventions
- Drug: PlaceboDrug: SAR302503
- Registration Number
- NCT01437787
- Lead Sponsor
- Sanofi
- Brief Summary
Primary Objective:
* To evaluate the efficacy of daily oral doses of 400 mg or 500 mg of SAR302503 (Investigational Medicinal Product, IMP) compared to placebo in the reduction of spleen volume as determined by magnetic resonance imaging (MRI) (or computed tomography scan in patients with contraindications for MRI).
Secondary Objectives:
* To evaluate the effect on Myelofibrosis (MF)-associated symptoms (key MF symptoms) as measured by the modified Myelofibrosis Symptom Assessment Form (MFSAF) diary.
* To evaluate the Overall Survival of patients treated with either 400 mg/day or 500 mg/day of IMP as compared to placebo.
* To evaluate the Progression Free Survival of patients treated with either 400 mg/day or 500 mg/day of IMP as compared to placebo.
* To evaluate the durability of splenic response.
* To evaluate the safety of IMP.
- Detailed Description
The expected duration of a patient's treatment in this study is approximately 8 months, based on a maximum 28-day screening period, followed by a โฅ6-month (6-cycle) treatment period, and an End Of Treatment (EOT) visit, which should be performed at least 30 days following the last administration of IMP or placebo.
Patients who continue to benefit clinically will be allowed to remain on IMP or placebo beyond the 6-month treatment period until the occurrence of disease progression or unacceptable toxicity.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 289
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- CROSSOVER
- Arm && Interventions
Group Intervention Description Placebo comparator Placebo once daily X 28 days, orally, empty stomach, approximately same time each day SAR302503 500 mg SAR302503 once daily X 28 days, orally, empty stomach, approximately same time each day SAR302503 400 mg SAR302503 once daily X 28 days, orally, empty stomach, approximately same time each day
- Primary Outcome Measures
Name Time Method Response Rate (RR), defined as the proportion of patients who have a โฅ35% reduction in volume of spleen size at the end of Cycle 6, and confirmed 4 weeks thereafter 6 months
- Secondary Outcome Measures
Name Time Method Proportion of patients who have โฅ25% reduction in volume of spleen size at end of Cycle 6, and confirmed 4 weeks thereafter. 6 months Symptom Response Rate (SRR): Proportion of patients with โฅ50% reduction from baseline to the end of Cycle 6 in the total symptom score. 6 months This assessment will be conducted through the modified MFSAF diary, which will be completed during the week prior to Day 1 of each treatment cycle up to Cycle 6, and during the week prior to the end of Cycle 6.
OS (overall survival) of either 400 mg/day or 500 mg/day of IMP as compared to placebo. approximately 5 years PFS (progression free survival) of either 400 mg/day or 500 mg/day of IMP as compared to placebo. approximately 5 years Duration of spleen response, measured by MRI (or CT scan in patients with contraindications for MRI. 2 years Clinical and laboratory events graded by the NCI CTCAE v4.03. approximately 5 years
Trial Locations
- Locations (101)
Investigational Site Number 840013
๐บ๐ธBaton Rouge, Louisiana, United States
Investigational Site Number 840014
๐บ๐ธScottsdale, Arizona, United States
Investigational Site Number 376002
๐ฎ๐ฑTel Hashomer, Israel
Investigational Site Number 440001
๐ฑ๐นKaunas, Lithuania
Investigational Site Number 620003
๐ต๐นPorto, Portugal
Investigational Site Number 643001
๐ท๐บMoscow, Russian Federation
Investigational Site Number 642002
๐ท๐ดBucuresti, Romania
Investigational Site Number 643007
๐ท๐บVolgograd, Russian Federation
Investigational Site Number 840004
๐บ๐ธHouston, Texas, United States
Investigational Site Number 276007
๐ฉ๐ชBonn, Germany
Investigational Site Number 348002
๐ญ๐บBudapest, Hungary
Investigational Site Number 840001
๐บ๐ธLa Jolla, California, United States
Investigational Site Number 840006
๐บ๐ธLos Angeles, California, United States
Investigational Site Number 840012
๐บ๐ธLa Jolla, California, United States
Investigational Site Number 840009
๐บ๐ธNewark, New Jersey, United States
Investigational Site Number 840008
๐บ๐ธRochester, Minnesota, United States
Investigational Site Number 840002
๐บ๐ธCanton, Ohio, United States
Investigational Site Number 036004
๐ฆ๐บTweed Heads, Australia
Investigational Site Number 036005
๐ฆ๐บHerston, Australia
Investigational Site Number 036003
๐ฆ๐บRandwick, Australia
Investigational Site Number 040001
๐ฆ๐นWien, Austria
Investigational Site Number 036002
๐ฆ๐บWodonga, Australia
Investigational Site Number 056003
๐ง๐ชAntwerpen, Belgium
Investigational Site Number 076004
๐ง๐ทPorto Alegre, Brazil
Investigational Site Number 056001
๐ง๐ชLeuven, Belgium
Investigational Site Number 124001
๐จ๐ฆMontreal, Canada
Investigational Site Number 076002
๐ง๐ทJau, Brazil
Investigational Site Number 076001
๐ง๐ทRio De Janeiro, Brazil
Investigational Site Number 124003
๐จ๐ฆMontreal, Canada
Investigational Site Number 250006
๐ซ๐ทMarseille, France
Investigational Site Number 124002
๐จ๐ฆSaint John, Canada
Investigational Site Number 250005
๐ซ๐ทNantes Cedex 01, France
Investigational Site Number 250004
๐ซ๐ทNimes, France
Investigational Site Number 276006
๐ฉ๐ชAachen, Germany
Investigational Site Number 250002
๐ซ๐ทPierre Benite Cedex, France
Investigational Site Number 250007
๐ซ๐ทPoitiers, France
Investigational Site Number 250003
๐ซ๐ทToulouse, France
Investigational Site Number 276001
๐ฉ๐ชMannheim, Germany
Investigational Site Number 276008
๐ฉ๐ชDresden, Germany
Investigational Site Number 348006
๐ญ๐บKecskemรฉt, Hungary
Investigational Site Number 348007
๐ญ๐บGyรถr, Hungary
Investigational Site Number 348001
๐ญ๐บDebrecen, Hungary
Investigational Site Number 348003
๐ญ๐บMiskolc, Hungary
Investigational Site Number 372002
๐ฎ๐ชDublin, Ireland
Investigational Site Number 376003
๐ฎ๐ฑHaifa, Israel
Investigational Site Number 372001
๐ฎ๐ชGalway, Ireland
Investigational Site Number 380002
๐ฎ๐นBergamo, Italy
Investigational Site Number 380004
๐ฎ๐นFirenze, Italy
Investigational Site Number 410002
๐ฐ๐ทBundang-Gu, Korea, Republic of
Investigational Site Number 410003
๐ฐ๐ทSeoul, Korea, Republic of
Investigational Site Number 410005
๐ฐ๐ทSeoul, Korea, Republic of
Investigational Site Number 410004
๐ฐ๐ทSeoul, Korea, Republic of
Investigational Site Number 410001
๐ฐ๐ทSeoul, Korea, Republic of
Investigational Site Number 410007
๐ฐ๐ทSeoul, Korea, Republic of
Investigational Site Number 440002
๐ฑ๐นKlaipeda, Lithuania
Investigational Site Number 484001
๐ฒ๐ฝQueretaro, Mexico
Investigational Site Number 616005
๐ต๐ฑBrzozow, Poland
Investigational Site Number 616002
๐ต๐ฑGdansk, Poland
Investigational Site Number 616006
๐ต๐ฑLodz, Poland
Investigational Site Number 616010
๐ต๐ฑWarszawa, Poland
Investigational Site Number 616003
๐ต๐ฑWroclaw, Poland
Investigational Site Number 620005
๐ต๐นCoimbra, Portugal
Investigational Site Number 620004
๐ต๐นLisboa, Portugal
Investigational Site Number 620001
๐ต๐นLisboa, Portugal
Investigational Site Number 642003
๐ท๐ดBrasov, Romania
Investigational Site Number 642004
๐ท๐ดBucharest, Romania
Investigational Site Number 642006
๐ท๐ดBucuresti, Romania
Investigational Site Number 643009
๐ท๐บMoscow, Russian Federation
Investigational Site Number 642001
๐ท๐ดTimisoara, Romania
Investigational Site Number 643008
๐ท๐บPetrozavodsk, Russian Federation
Investigational Site Number 643004
๐ท๐บSt-Petersburg, Russian Federation
Investigational Site Number 643010
๐ท๐บNizhny Novgorod, Russian Federation
Investigational Site Number 643005
๐ท๐บSt.-Petersburg, Russian Federation
Investigational Site Number 702002
๐ธ๐ฌSingapore, Singapore
Investigational Site Number 702001
๐ธ๐ฌSingapore, Singapore
Investigational Site Number 710003
๐ฟ๐ฆJohannesburg, South Africa
Investigational Site Number 724001
๐ช๐ธBarcelona, Spain
Investigational Site Number 710002
๐ฟ๐ฆParktown, South Africa
Investigational Site Number 724002
๐ช๐ธMadrid, Spain
Investigational Site Number 158002
๐จ๐ณChanghua, Taiwan
Investigational Site Number 752001
๐ธ๐ชStockholm, Sweden
Investigational Site Number 158003
๐จ๐ณKaohsiung, Taiwan
Investigational Site Number 752002
๐ธ๐ชUddevalla, Sweden
Investigational Site Number 158001
๐จ๐ณTaipei, Taiwan
Investigational Site Number 826006
๐ฌ๐งBelfast, United Kingdom
Investigational Site Number 826007
๐ฌ๐งManchester, United Kingdom
Investigational Site Number 826002
๐ฌ๐งGlasgow, United Kingdom
Investigational Site Number 826001
๐ฌ๐งLondon, United Kingdom
Investigational Site Number 826004
๐ฌ๐งLeeds, United Kingdom
Investigational Site Number 826005
๐ฌ๐งLondon, United Kingdom
Investigational Site Number 380007
๐ฎ๐นBologna, Italy
Investigational Site Number 380001
๐ฎ๐นPavia, Italy
Investigational Site Number 380006
๐ฎ๐นPavia, Italy
Investigational Site Number 380003
๐ฎ๐นVarese, Italy
Investigational Site Number 250001
๐ซ๐ทVillejuif Cedex, France
Investigational Site Number 826003
๐ฌ๐งBirmingham, United Kingdom
Investigational Site Number 826008
๐ฌ๐งNewcastle Upon Tyne, United Kingdom
Investigational Site Number 826009
๐ฌ๐งOxford, United Kingdom
Investigational Site Number 826010
๐ฌ๐งSouthampton, United Kingdom
Investigational Site Number 410006
๐ฐ๐ทSeoul, Korea, Republic of
Investigational Site Number 036001
๐ฆ๐บBox Hill, Australia