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

Kanako 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

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Abstract

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/m2 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.

Original languageEnglish
Article numbere007330
JournalBMJ open
Volume5
Issue number1
DOIs
Publication statusPublished - Jan 1 2015

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POEMS Syndrome
Thalidomide
Clinical Protocols
Placebos
Safety
Dexamethasone
Vascular Endothelial Growth Factor A
Japan
Randomized Controlled Trials
Paraproteinemias
Research Ethics Committees
Neurology
Ethics
Publications
Therapeutics
Transplantation
Equipment and Supplies
Skin
Serum
Pharmaceutical Preparations

All Science Journal Classification (ASJC) codes

  • Medicine(all)

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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. / Katayama, Kanako; Misawa, Sonoko; Sato, Yasunori; Sobue, Gen; Yabe, Ichiro; Watanabe, Osamu; Nishizawa, Masatoyo; Kusunoki, Susumu; Kikuchi, Seiji; Nakashima, Ichiro; Ikeda, Shu Ichi; Kohara, Nobuo; Kanda, Takashi; Kira, Jun-Ichi; Hanaoka, Hideki; Kuwabara, Satoshi.

In: BMJ open, Vol. 5, No. 1, e007330, 01.01.2015.

Research output: Contribution to journalArticle

Katayama, K, Misawa, S, Sato, Y, Sobue, G, Yabe, I, Watanabe, O, Nishizawa, M, Kusunoki, S, Kikuchi, S, Nakashima, I, Ikeda, SI, Kohara, N, Kanda, T, Kira, J-I, Hanaoka, H & Kuwabara, S 2015, 'Japanese POEMS syndrome with thalidomide ( J-POST) trial: Study protocol for a phase II/III multicentre, randomised, double-blind, placebo-controlled trial', BMJ open, vol. 5, no. 1, e007330. https://doi.org/10.1136/bmjopen-2014-007330
Katayama, Kanako ; Misawa, Sonoko ; Sato, Yasunori ; Sobue, Gen ; Yabe, Ichiro ; Watanabe, Osamu ; Nishizawa, Masatoyo ; Kusunoki, Susumu ; Kikuchi, Seiji ; Nakashima, Ichiro ; Ikeda, Shu Ichi ; Kohara, Nobuo ; Kanda, Takashi ; Kira, Jun-Ichi ; Hanaoka, Hideki ; Kuwabara, Satoshi. / Japanese POEMS syndrome with thalidomide ( J-POST) trial : Study protocol for a phase II/III multicentre, randomised, double-blind, placebo-controlled trial. In: BMJ open. 2015 ; Vol. 5, No. 1.
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abstract = "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/m2 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.",
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AU - Sobue, Gen

AU - Yabe, Ichiro

AU - Watanabe, Osamu

AU - Nishizawa, Masatoyo

AU - Kusunoki, Susumu

AU - Kikuchi, Seiji

AU - Nakashima, Ichiro

AU - Ikeda, Shu Ichi

AU - Kohara, Nobuo

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