Ropeginterferon Alfa-2b (P1101) vs. Anagrelide in Essential Thrombocythemia Patients With Hydroxyurea Resistance or Intolerance (SURPASS ET)
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|ClinicalTrials.gov Identifier: NCT04285086|
Recruitment Status : Recruiting
First Posted : February 26, 2020
Last Update Posted : January 14, 2022
|Condition or disease||Intervention/treatment||Phase|
|Essential Thrombocythemia||Biological: Ropeginterferon alfa-2b Drug: Anagrelide||Phase 3|
PharmaEssentia Corporation is developing a pegylated (PEG) IFN-α product, P1101, for the treatment of ET.
Available clinical data and experience with P1101 in PV shows that the compound, with proper dose modifications, is effective in controlling disease in a significant proportion of subjects with ET. Further, its increased serum half-life presents distinct advantages for ET treatment over that of standard IFN-α and other available PEG IFN-α therapy. This pivotal Phase 3 study will establish the efficacy and safety of P1101 in ET subjects.
The enrolled subjects will be randomized into two arms, the test arm is P1101, the control arm is ANA. The overall duration for each eligible patient is 14 months, including screening (1 month), treatment (12 months) and follow-up (1 month) period. Efficacy evaluations, safety assessments, and PK and immunogenicity evaluations of P1101 will be performed.
Evaluation of efficacy will include clinical laboratory assessments, allelic burden measurements of CALR, JAK-2, and MPL, spleen size measurements, bone marrow sampling, EQ-5D-3L, and MPN-SAF TSS completion.
Evaluation of safety will include assessing vital signs, clinical safety laboratory tests, physical examinations, ECG evaluation, heart ECHO, lung X-ray, ECOG performance status, ocular examination, and AEs.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||160 participants|
|Intervention Model:||Parallel Assignment|
|Intervention Model Description:||Experimental Drug (Biological): Ropeginterferon alfa-2b (P1101), Q2W, SC injection Control Drug: Anagrelide, capsules, daily, p.o.|
|Masking:||None (Open Label)|
|Official Title:||A Phase 3, Open-Label, Multicenter, Randomized, Active-controlled Study to Assess Pharmacokinetics and Compare the Efficacy, Safety, and Tolerability of P1101 vs Anagrelide as Second Line Therapy for Essential Thrombocythemia|
|Actual Study Start Date :||August 25, 2020|
|Estimated Primary Completion Date :||December 2023|
|Estimated Study Completion Date :||January 2024|
Experimental: Ropeginterferon alfa-2b (P1101)
Pre-filled Syringe, Q2W, SC injection
Biological: Ropeginterferon alfa-2b
Ropeginterferon alfa-2b (P1101) dosage: from 250 mcg to 500 mcg
Other Name: P1101
Active Comparator: Anagrelide
Capsules, Daily, p.o.
Anagrelide dosage: 0.5 mg per capsule, according to label and physician's judgement
- Peripheral blood count remission [ Time Frame: month 9 and month 12 ]platelets ≤400 x 10^9/L AND white blood cells (WBC) <9.5 x 10^9/L
- Improvement or non-progression in disease-related signs [ Time Frame: month 9 and month 12 ]splenomegaly
- Large symptoms improvement or maintain non-progression [ Time Frame: month 9 and month 12 ]based on the Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MPN-SAF TSS)
- Absence of hemorrhagic or thrombotic events [ Time Frame: month 9 and month 12 ]absence of hemorrhagic or thrombotic events
- Durable response [ Time Frame: month 3 and month 6 ]measure durable response at month 3 and 6
- Longitudinal rate [ Time Frame: over the 12 months ]measure longitudinal rate of change in the ELN response rates over the 12 months
- Response rates [ Time Frame: 3, 6, 9, and 12 months ]measure response rate based on peripheral blood count remission, no signs of progressive disease, and absence of any hemorrhagic or thrombotic events
- Occurrence of thromboembolic events [ Time Frame: over the 12 months ]measure occurrence of thromboembolic events over the 12 months
- Time to first peripheral blood count remission response [ Time Frame: over the 12 months ]measure time to first peripheral blood count remission response over the 12 months
- Duration of peripheral blood count remission response [ Time Frame: over the 12 months ]measure duration of peripheral blood count remission response over the 12 months
- Symptomatic improvement assessed by the EuroQOL 5 dimensions 3 level version (EQ-5D-3L) questionnaire [ Time Frame: over the 12 months ]measure symptomatic improvement assessed by the EuroQOL 5 dimensions 3 level version (EQ-5D-3L) questionnaire over the 12 months
- Symptomatic improvement assessed by the 10-item MPN-SAF TSS [ Time Frame: over the 12 months ]measure symptomatic improvement assessed by the 10-item MPN-SAF TSS over the 12 months
- Change of CALR, MPL, and JAK-2 allelic burden over time [ Time Frame: over the 12 months ]measure change of CALR, MPL, and JAK-2 allelic burden over time
- Improvement or non-progression of spleen size assessment [ Time Frame: over the 12 months ]measure spleen size over time
- Bone marrow histological remission [ Time Frame: over the 12 months ]the disappearance of megakaryocyte hyperplasia and absence of >grade 1 reticulin fibrosis
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04285086
|Contact: Toshiaki Sato, MD/PhD||+81 3 firstname.lastname@example.org|
|Contact: TingFang Wang, MS/MHA||+886 2 26557688 ext email@example.com|
|Study Director:||Toshiaki Sato, MD/PhD||PharmaEssentia Japan K.K.|
|Study Director:||Craig Zimmerman, PhD||PharmaEssentia USA Corp.|