TY - JOUR
T1 - A randomised, multi-centre phase III study of 3 different doses of intravenous immunoglobulin 10% in patients with chronic inflammatory demyelinating polyradiculoneuropathy (ProCID trial)
T2 - Study design and protocol
AU - Cornblath, David R.
AU - Hartung, Hans Peter
AU - Katzberg, Hans D.
AU - Merkies, Ingemar S.J.
AU - van Doorn, Pieter A.
N1 - Funding Information:
information Octapharma Pharmazeutika Produktionsgesm. b.H., Vienna, AustriaWe thank Sarah Greig, PhD, of Springer Healthcare Communications who prepared the outline and subsequent drafts of this manuscript. We also thank Doris Hinterberger and Elisabeth Clodi of Octapharma Pharmazeutika Produktionsges.m.b.H for their valuable input. Funding for this study, the medical writing assistance, and Open Access for this article was provided by Octapharma Pharmazeutika Produktionsges.m.b.H. H.-P.H. has received honoraria for consulting, serving on steering committees and speaking from Baxter, Bayer Healthcare, CSL Behring, Kedrion, LFB, Octapharma, Syntimmune; H.D.K. has received honoraria for consulting, serving on steering committees and data safety monitoring boards, and has received research support from CSL Behring, Grifols Biotherapeutics, Sanofi Genzyme, Flexpharma and Octapharma; P.A.D. has received honoraria for consulting and serving on steering committees from Baxter, CSL Behring, Octapharma and Kedrion, and research support from Baxter, Grifols and Sanquin Blood supply; D.R.C. serves as consultant to Acetylon Pharmaceuticals Inc., Alcobra Pharma, Alnylam Pharmaceuticals, Annexon Biosciences, Akros Pharma, Biotest Pharmaceuticals Inc., Boehringer Ingelheim, Cigna Health Management Inc., CSL Behring, DP Clinical Inc., GlaxoSmithKline, Grifols SA, Karos Pharmaceuticals Inc., Neurocrine Biosciences, Novartis Corp., Octapharma AG, Pharnext SAS and Sun Pharmaceuticals, Syntimmune; is on a Data Safety Monitoring Board for Pfizer Inc., Ionis Pharmaceuticals, GlaxoSmithKline, Axovant Sciences Ltd.; has technology licencing agreements through Johns Hopkins University with Acetylon Pharmaceuticals Inc., AstraZeneca Pharmaceuticals, Calithera Biosciences, Genentech Inc., Neurocrine Biosciences, Merrimack Pharmaceuticals Inc., Seattle Genetics Inc., Shire Development LLC; and serves on the Board of Directors for the GBS-CIDP Foundation International, Foundation for Peripheral Neuropathy, and The Peripheral Nerve Society; ISJ Merkies has received honoraria for serving on the steering committee for Talecris, Biotest, LFB Biopharmaceuticals, CSL Behring, Novartis and Grifols; and has received grants from the Talecris Talents Program, GBS CIDP Foundation International, Prinses Beatrix Spierfonds and the European Union Seventh Framework Programme. The sponsor designed the clinical study in close cooperation with the steering committee (D.R.C., H.-P.H., I.S.J.M., and P.A.D.) and the coordinating investigator (H.D.K.). This manuscript has been written in collaboration with the members of the steering committee and the coordinating investigator based on the study protocol (amended version 03; March 9, 2017). The authors obtained approval from the sponsor but made the final decision to submit the manuscript. All authors have reviewed the manuscript and approved the final version to be published.
Funding Information:
We thank Sarah Greig, PhD, of Springer Healthcare Communications who prepared the outline and subsequent drafts of this manuscript. We also thank Doris Hinterberger and Elisabeth Clodi of Octapharma Pharmazeutika Produktionsges.m.b.H for their valuable input. Funding for this study, the medical writing assistance, and Open Access for this article was provided by Octapharma Pharmazeutika Produktionsges. m.b.H.
Funding Information:
H.-P.H. has received honoraria for consulting, serving on steering committees and speaking from Baxter, Bayer Healthcare, CSL Behring, Kedrion, LFB, Octapharma, Syntimmune; H.D.K. has received honoraria for consulting, serving on steering committees and data safety monitoring boards, and has received research support from CSL Behring, Grifols Biotherapeutics, Sanofi Genzyme, Flexpharma and Octapharma; P.A.D. has received honoraria for consulting and serving on steering committees from Baxter, CSL Behring, Octa-pharma and Kedrion, and research support from Baxter, Grifols and Sanquin Blood supply; D.R.C. serves as consultant to Acetylon Pharmaceuticals Inc., Alcobra Pharma, Alnylam Pharmaceuticals, Annexon Biosciences, Akros Pharma, Biotest Pharmaceuticals Inc., Boehringer Ingelheim, Cigna Health Management Inc., CSL Behring, DP Clinical Inc., GlaxoSmithKline, Grifols SA, Karos Pharmaceuticals Inc., Neurocrine Biosciences, Novartis Corp., Octapharma AG, Pharnext SAS and Sun Pharmaceuticals, Syntimmune; is on a Data Safety Monitoring Board for Pfizer Inc., Ionis Pharmaceuticals, GlaxoSmithKline, Axovant Sciences Ltd.; has technology licencing agreements through Johns Hopkins University with Acetylon Pharmaceuticals Inc., Astra-Zeneca Pharmaceuticals, Calithera Biosciences, Genentech Inc., Neurocrine Biosciences, Merrimack Pharmaceuticals Inc., Seattle Genetics Inc., Shire Development LLC; and serves on the Board of Directors for the GBS-CIDP Foundation International, Foundation for Peripheral Neuropathy, and The Peripheral Nerve Society; ISJ Merkies has received honoraria for serving on the steering committee for Talecris, Biotest, LFB Biopharmaceuticals, CSL Behring, Novartis and Grifols; and has received grants from the Talecris Talents Program, GBS CIDP Foundation International, Prinses Beatrix Spierfonds and the European Union Seventh Framework Programme.
Publisher Copyright:
© 2018 The Authors. Journal of the Peripheral Nervous System published by Wiley Periodicals, Inc. on behalf of Peripheral Nerve Society.
PY - 2018/6
Y1 - 2018/6
N2 - Patients with chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) show varying degrees of response to intravenous immunoglobulin (IVIg) therapy. This randomised phase III study in patients with CIDP (ProCID trial) will compare the efficacy and safety of 3 different doses (0.5, 1.0, and 2.0 g/kg) of IVIg 10% (panzyga) administered every 3 weeks for 24 weeks. The primary efficacy endpoint is the rate of treatment response, defined as a decrease in adjusted inflammatory neuropathy cause and treatment disability score of ≥1 point, in the IVIg 1.0 g/kg arm at week 24. Patients with definite or probable CIDP according to European Federation of Neurological Sciences/Peripheral Nerve Society criteria with IVIg or corticosteroid dependency and active disease are eligible. All potentially eligible patients will undergo IVIg or corticosteroid dose reduction (washout phase) over ≤12 weeks or until deterioration of CIDP (active disease). Patients with deterioration during the washout phase will be randomised to receive study treatment during a dose-evaluation phase starting with a loading dose of IVIg 2.0 g/kg followed by maintenance treatment with IVIg 0.5, 1.0, or 2.0 g/kg every 3 weeks. Rescue medication (2 doses of IVIg 2.0 g/kg given 3 weeks apart) will be administered to patients in the IVIg 0.5 and 1.0 g/kg groups who deteriorate after week 3 and before week 18 or who do not improve at week 6. Safety, tolerability and quality of life will be assessed. The ProCID study will provide new information on the best maintenance dose of IVIg for patients with CIDP.
AB - Patients with chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) show varying degrees of response to intravenous immunoglobulin (IVIg) therapy. This randomised phase III study in patients with CIDP (ProCID trial) will compare the efficacy and safety of 3 different doses (0.5, 1.0, and 2.0 g/kg) of IVIg 10% (panzyga) administered every 3 weeks for 24 weeks. The primary efficacy endpoint is the rate of treatment response, defined as a decrease in adjusted inflammatory neuropathy cause and treatment disability score of ≥1 point, in the IVIg 1.0 g/kg arm at week 24. Patients with definite or probable CIDP according to European Federation of Neurological Sciences/Peripheral Nerve Society criteria with IVIg or corticosteroid dependency and active disease are eligible. All potentially eligible patients will undergo IVIg or corticosteroid dose reduction (washout phase) over ≤12 weeks or until deterioration of CIDP (active disease). Patients with deterioration during the washout phase will be randomised to receive study treatment during a dose-evaluation phase starting with a loading dose of IVIg 2.0 g/kg followed by maintenance treatment with IVIg 0.5, 1.0, or 2.0 g/kg every 3 weeks. Rescue medication (2 doses of IVIg 2.0 g/kg given 3 weeks apart) will be administered to patients in the IVIg 0.5 and 1.0 g/kg groups who deteriorate after week 3 and before week 18 or who do not improve at week 6. Safety, tolerability and quality of life will be assessed. The ProCID study will provide new information on the best maintenance dose of IVIg for patients with CIDP.
KW - chronic inflammatory demyelinating polyradiculoneuropathy
KW - intravenous immunoglobulin
KW - randomised-controlled trial
KW - study design
KW - trial protocol
UR - http://www.scopus.com/inward/record.url?scp=85045924081&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=85045924081&partnerID=8YFLogxK
U2 - 10.1111/jns.12267
DO - 10.1111/jns.12267
M3 - Article
C2 - 29603842
AN - SCOPUS:85045924081
SN - 1085-9489
VL - 23
SP - 108
EP - 114
JO - Journal of the Peripheral Nervous System
JF - Journal of the Peripheral Nervous System
IS - 2
ER -