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Japanese POEMS syndrome with Thalidomide (J-POST) Trial: study protocol for a phase II/III multicentre, randomised, double-blind, placebo-controlled trial.

AbstractINTRODUCTION:
Polyneuropathy, organomegaly, endocrinopathy, M-protein and skin changes (POEMS) syndrome is a fatal systemic disorder associated with plasma cell dyscrasia and the overproduction of the vascular endothelial growth factor (VEGF). Recently, the prognosis of POEMS was substantially improved by introduction of therapeutic intervention for myeloma. However, no randomised clinical trial has been performed because of the rarity and severity of the disease.
METHODS AND ANALYSIS:
The Japanese POEMS syndrome with Thalidomide (J-POST) Trial is a phase II/III multicentre, double-blinded, randomised, controlled trial that aims to evaluate the efficacy and safety of a 24-week treatment with thalidomide in POEMS syndrome, with an additional 48-week open-label safety study. Adults with POEMS syndrome who have no indication for transplantation are assessed for eligibility at 12 tertiary neurology centres in Japan. Patients who satisfy the eligibility criteria are randomised (1:1) to receive thalidomide (100-300 mg daily) plus dexamethasone (12 mg/m(2) on days 1-4 of a 28-day cycle) or placebo plus dexamethasone. Both treatments were administered for 24 weeks (six cycles; randomised comparative study period). Patients who complete the randomised study period or show subacute deterioration during the randomised period participate in the subsequent 48-week open-label safety study (long-term safety period). The primary end point of the study is the reduction rate of serum VEGF levels at 24 weeks.
ETHICS AND DISSEMINATION:
The protocol was approved by the Institutional Review Board of each hospital. The trial was notified and registered at the Pharmaceutical and Medical Devices Agency, Japan (No. 22-1716). The J-POST Trial is currently ongoing and is due to finish in August 2015. The findings of this trial will be disseminated through peer-reviewed publications and conference presentations and will also be disseminated to participants.
TRIAL REGISTRATION NUMBER:
UMIN000004179 and JMA-IIA00046.
AuthorsKanako Katayama, Sonoko Misawa, Yasunori Sato, Gen Sobue, Ichiro Yabe, Osamu Watanabe, Masatoyo Nishizawa, Susumu Kusunoki, Seiji Kikuchi, Ichiro Nakashima, Shu-Ichi Ikeda, Nobuo Kohara, Takashi Kanda, Jun-Ichi Kira, Hideki Hanaoka, Satoshi Kuwabara, J-POST Trial study investigators
JournalBMJ open (BMJ Open) Vol. 5 Issue 1 Pg. e007330 (Jan 08 2015) ISSN: 2044-6055 [Electronic] England
PMID25573527 (Publication Type: Clinical Trial, Phase II, Clinical Trial, Phase III, Journal Article, Multicenter Study, Randomized Controlled Trial, Research Support, Non-U.S. Gov't)
CopyrightPublished by the BMJ Publishing Group Limited. For permission to use (where not already granted under a licence) please go to http://group.bmj.com/group/rights-licensing/permissions.
Chemical References
  • Hematologic Agents
  • Vascular Endothelial Growth Factor A
  • Thalidomide
Topics
  • Adult
  • Clinical Protocols
  • Double-Blind Method
  • Female
  • Hematologic Agents (therapeutic use)
  • Humans
  • Japan
  • Male
  • Multiple Myeloma (drug therapy)
  • Outcome Assessment, Health Care
  • POEMS Syndrome (blood, drug therapy)
  • Paraproteinemias (blood, drug therapy)
  • Research Design
  • Thalidomide (therapeutic use)
  • Vascular Endothelial Growth Factor A (blood)

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