Effect of SPD422 on Platelet Lowering and Safety in Japanese Adults With At Risk Essential Thrombocythaemia

A Phase 3, Open-label, Single-arm Study Evaluating the Effect of SPD422 on Platelet Lowering and Safety in Japanese Adults With At Risk Essential Thrombocythaemia Who Are Intolerant or Refractory to Current Cytoreductive Treatment

The purpose of this study is to investigate how effective and safe SPD422 (Anagrelide Hydrochloride) is in Japanese subjects, diagnosed with Essential Thrombocythemia, who's previously treatment has either not been effective or has caused unacceptable adverse reactions. The study will aim to show that platelet counts can be safely reduced in treated patients to below 600 x 10^9/L after a minimum of three months treatment. To demonstrate an positive effect platelet levels will need to remain below 600 x 10^9/L for at least 4 weeks.

Study Overview

• Status: Completed
• Conditions: Essential Thrombocythemia (ET)
• Intervention / Treatment: Drug: Anagrelide Hydrochloride

• Study Type: Interventional
• Enrollment (Actual): 53
• Phase: Phase 3

Contacts and Locations

Study Locations

• Japan
  • Tokyo, Japan, 13 160-8582
    • Keio University Hospital
  • Akita Prefecture
    • Akita-shi, Akita Prefecture, Japan, 05 010-8543
      • Akita University Hospital
  • Bunkyo-ku
    • Hongo 3-1-3, Bunkyo-ku, Japan, 13 113-8431
      • Juntendo University Hospital
    • Honkomagome 3-18-22, Bunkyo-ku, Japan, 13 113-8677
      • Tokyo Metropolitan Cancer and Infectious diseases Center Kom
    • Sendagi 1-1-5, Bunkyo-ku, Japan, 13 113-8603
      • Nippon Medical School Hospital
  • Chiba-shi
    • Chuo-ku Inohana 1-8-1, Chiba-shi, Japan, 12 260-8677
      • Chiba University Hospital
  • Chubu
    • Nagoya-shi, Chubu, Japan, 23 460-0001
      • NHO Nagoya Medical Center
  • Hokkaidō Prefecture
    • Sapporo-shi, Hokkaidō Prefecture, Japan, 01 060-8648
      • Hokkaido University Hospital
  • Izunokuni-shi
    • Nagaoka 1129, Izunokuni-shi, Japan, 22 410-2295
      • Juntendo University Shizuoka Hospital
  • Kanagawa Prefecture
    • Isehara-shi, Kanagawa Prefecture, Japan, 259-1143
      • Tokai University Hospital
  • Kansai
    • Osaka-shi, Kansai, Japan, 27 545-8586
      • Osaka City University Hospital
  • Maebashi-shi
    • Showa-machi 3-39-15, Maebashi-shi, Japan, 10 371-8511
      • Gunma University Hospital
  • Meguro-ku
    • Higashigaoka 2-5-1, Meguro-ku, Japan, 13 152-8902
      • NHO Tokyo Medical Center
  • Mie
    • Tsu-shi, 24, Mie, Japan, 514-8507
      • Mie University Hospital
  • Miyazaki Prefecture
    • Miyazaki-shi, Miyazaki Prefecture, Japan, 889-1692
      • University of Miyazaki Hospital
  • Niigata-shi
    • Chuo-ku Kawagishi-cho 2-15-3, Niigata-shi, Japan, 15 951-8566
      • Niigata Cancer Center Hospital
  • Okayama Prefecture
    • Okayama-shi, Okayama Prefecture, Japan, 33 700-8558
      • Okayama University Hospital
  • Osaka Prefecture
    • Suita-shi, Osaka Prefecture, Japan, 27 565-0871
      • Osaka University Hospital
  • Tokushima Perfecture
    • Tokushima-shi, Tokushima Perfecture, Japan, 770-8503
      • Tokushima University Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 20 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Subjects must have previously been treated with a cytoreductive therapy and been intolerant or refractory to that therapy.

Exclusion Criteria:

• Subjects should not have any other underlying conditions or medications that would confound the study analysis or interact with the study medication.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Anagrelide Hydrochloride	Drug: Anagrelide Hydrochloride; Subjects will be started at 1.0 mg per day and titrated as necessary.; Other Names: SPD422

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percentage of Subjects Who Responded in Platelet Count	A response was defined as platelet counts to <600x10^9/L across consecutive visits for at least 4 weeks following at least 3 months of treatment.	12 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percentage of Subjects With at Least 50% Reduction in Platelet Count	Subjects who achieved at least 50% reduction in platelet count from their baseline level across consecutive visits for at least 4 weeks and following 3 months of treatment.	12 months
Percentage of Subjects With Normalization in Platelet Count	Normalization was defined as platelet counts ≤400x10^9/L across consecutive visits for at least 4 weeks following at least 3 months of treatment.	12 months
Percentage of Subjects Who Responded in Platelet Count Whose Baseline Platelet Count Was ≥600x10^9/L	A response was defined as platelet counts to <600x10^9/L across consecutive visits for at least 4 weeks following at least 3 months of treatment.	12 months
Percentage of Subjects Who Responded in Platelet Count Whose Baseline Platelet Count Was <600x10^9/L	A response was defined as platelet counts to <600x10^9/L across consecutive visits for at least 4 weeks following at least 3 months of treatment.	12 months

Collaborators and Investigators

• Sponsor: Shire

Publications and helpful links

General Publications

• Kanakura Y, Miyakawa Y, Wilde P, Smith J, Achenbach H, Okamoto S. Phase III, single-arm study investigating the efficacy, safety, and tolerability of anagrelide as a second-line treatment in high-risk Japanese patients with essential thrombocythemia. Int J Hematol. 2014 Oct;100(4):353-60. doi: 10.1007/s12185-014-1631-x. Epub 2014 Aug 27.

Study record dates

Study Major Dates

• Study Start (Actual): October 27, 2010
• Primary Completion (Actual): October 24, 2012
• Study Completion (Actual): October 24, 2012

Study Registration Dates

• First Submitted: October 4, 2010
• First Submitted That Met QC Criteria: October 4, 2010
• First Posted (Estimate): October 5, 2010

Study Record Updates

• Last Update Posted (Actual): June 9, 2021
• Last Update Submitted That Met QC Criteria: May 24, 2021
• Last Verified: May 1, 2021

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Bone Marrow Diseases; Hematologic Diseases; Hemorrhagic Disorders; Myeloproliferative Disorders; Blood Coagulation Disorders; Blood Platelet Disorders; Thrombocytosis; Thrombocythemia, Essential; Molecular Mechanisms of Pharmacological Action; Fibrinolytic Agents; Fibrin Modulating Agents; Platelet Aggregation Inhibitors; Anagrelide
• Other Study ID Numbers: SPD422-308