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Clinical Trial
. 2024 Aug 16;95(9):845-854.
doi: 10.1136/jnnp-2023-333112.

Efficacy, safety and tolerability of rozanolixizumab in patients with chronic inflammatory demyelinating polyradiculoneuropathy: a randomised, subject-blind, investigator-blind, placebo-controlled, phase 2a trial and open-label extension study

Luis Querol  1   2 , Jérôme De Sèze  3 , Tina Dysgaard  4 , Todd Levine  5 , T Hemanth Rao  6 , Michael Rivner  7 , Hans-Peter Hartung  8   9   10 , Peter Kiessling  11 , Saori Shimizu  12 , Dominika Marmol  13 , Ali Bozorg  14 , Anny-Odile Colson  15 , Ute Massow  11 , Filip Eftimov  16 ; CIDP01 Study Investigators
Collaborators, Affiliations
Clinical Trial

Efficacy, safety and tolerability of rozanolixizumab in patients with chronic inflammatory demyelinating polyradiculoneuropathy: a randomised, subject-blind, investigator-blind, placebo-controlled, phase 2a trial and open-label extension study

Luis Querol et al. J Neurol Neurosurg Psychiatry. .

Abstract

Background: Chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) is a peripheral nerve disorder characterised by weakness and sensory loss. We assessed the neonatal Fc receptor inhibitor rozanolixizumab for CIDP management.

Methods: CIDP01 (NCT03861481) was a randomised, subject-blind, investigator-blind, placebo-controlled, phase 2a study. Adults with definite or probable CIDP receiving subcutaneous or intravenous immunoglobulin maintenance therapy were randomised 1:1 to 12 once-weekly subcutaneous infusions of rozanolixizumab 10 mg/kg or placebo, stratified according to previous immunoglobulin administration route. Investigators administering treatment and assessing efficacy, and patients, were blinded. The primary outcome was a change from baseline (CFB) to day 85 in inflammatory Rasch-built Overall Disability Scale (iRODS) score. Eligible patients who completed CIDP01 entered the open-label extension CIDP04 (NCT04051944).

Results: In CIDP01, between 26 March 2019 and 31 March 2021, 34 patients were randomised to rozanolixizumab or placebo (17 (50%) each). No significant difference in CFB to day 85 in iRODS centile score was observed between rozanolixizumab (least squares mean 2.0 (SE 3.2)) and placebo (3.4 (2.6); difference -1.5 (90% CI -7.5 to 4.5)). Overall, 14 (82%) patients receiving rozanolixizumab and 13 (76%) receiving placebo experienced a treatment-emergent adverse event during the treatment period. Across CIDP01 and CIDP04, rozanolixizumab was well tolerated over up to 614 days; no clinically meaningful efficacy results were seen. No deaths occurred.

Conclusions: Rozanolixizumab did not show efficacy in patients with CIDP in this study, although this could be due to a relatively high placebo stability rate. Rozanolixizumab was well tolerated over medium-to-long-term weekly use, with an acceptable safety profile.

Keywords: FC RECEPTOR; MOVEMENT DISORDERS; MYASTHENIA; NEUROMUSCULAR; RANDOMISED TRIALS.

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Conflict of interest statement

Competing interests: LQ received research grants from Instituto de Salud Carlos III–Ministry of Economy and Innovation (Spain), CIBERER, GBS-CIDP Foundation International, Grifols, Roche and UCB Pharma; provided expert testimony to Annexon, argenx, Avilar Therapeutics, CSL Behring, Johnson and Johnson, Lundbeck, Lumanity, Merck, Novartis, Octapharma, Roche, Takeda, Sanofi Genzyme and UCB Pharma; serves on the Clinical Trial Steering Committee for Sanofi Genzyme. JDS, TD, TL and THR: report no disclosures relevant to the manuscript. MR served as a consultant and received research support from Alexion and Allergan; received research support from Anelixis Therapeutics, Apellis Pharmaceuticals, Biohaven, Catalyst, MediciNova, Millennium Pharmaceuticals, Momenta (now Johnson and Johnson), Orion, Ra Pharmaceuticals (now UCB Pharma), Seikagaku, Shire Takeda, UCB Pharma and Viela Bio (now Horizon Therapeutics). H-PH received honoraria for serving on steering and data monitoring committees of Bayer Healthcare, Biogen, BMS Celgene, GeNeuro, Merck, Novartis, Octapharma, Roche, TG Therapeutics and UCB Pharma. PK was an employee and shareholder of UCB Pharma during the conduct of the study but is now retired. SS is an employee and shareholder of UCB Pharma. DM was a contractor employed by UCB Pharma during the conduct of this study, but is currently employed by IQVIA. AB was an employee and shareholder of UCB Pharma during the conduct of the study but is currently employed at Otsuka Pharmaceutical Commercialization and Development. A-OC and UM are employees and shareholders of UCB Pharma. FE reports grants from ZonMw (Dutch Governmental Agency) and Prinses Beatrix Spierfonds; he also reports grants from CSL Behring, the GBS-CIDP Foundation, Grifols, Kedrion, Takeda Pharmaceutical Company and Terumo BCT, all outside the submitted work; grants were paid to his institution and were used for investigator-initiated studies within INCbase, an international CIDP registry; in addition, he has received consultancy fees from CSL Behring, Grifols and UCB Pharma, paid to his institution.

Figures

Figure 1
Figure 1
CIDP01 and CIDP04 trial profiles. (A) CIDP01. (B) CIDP04. *One patient relapsed and one patient did not want to continue to CIDP04. †One COVID-19 pandemic circumstance. ‡Study ended once a managed access programme was in place, to allow patients in the optional second 52-week treatment period to continue receiving rozanolixizumab if wished. iRODS, inflammatory Rasch-built Overall Disability Scale.
Figure 2
Figure 2
CIDP01 mean change from baseline. (A) iRODS centile metric score. (B) Adjusted INCAT disability score. (C) Maximum grip strength. Error bars are 90% CI. *Denotes an observation visit (all other visits were treatment visits). INCAT, Inflammatory Neuropathy Cause and Treatment; iRODS, inflammatory Rasch-built Overall Disability Scale.
Figure 3
Figure 3
Time to CIDP relapse (iRODS, adjusted INCAT disability scale and maximum grip strength combined assessments). *Patients who did not experience a relapse while under observation and completed the treatment period are censored at day 85. CIDP, chronic inflammatory demyelinating polyneuropathy; INCAT, Inflammatory Neuropathy Cause and Treatment; iRODS, inflammatory Rasch-built Overall Disability Scale.
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References

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