NCT02591862

Coversin in Paroxysmal Nocturnal Haemoglobinuria (PNH) in Patients With Resistance to Eculizumab Due to Complement C5 Polymorphisms

Completed Phase 2 Results posted Last updated 20 June 2023

What this trial tests

Phase 2 trial testing Coversin in Paroxysmal Nocturnal Haemoglobinuria (PNH) in 1 participant. Completed in 20 March 2018.

Timeline

1 February 2016

Primary endpoint
20 March 2018

20 March 2018

Quick facts

Lead sponsor	AKARI Therapeutics
Phase	Phase 2
Status	Completed
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	1
Start date	1 February 2016
Primary completion	20 March 2018
Estimated completion	20 March 2018
Sites	1 location across Netherlands

Drugs / interventions tested

• Coversin — full drug profile →

Conditions studied

• Paroxysmal Nocturnal Haemoglobinuria (PNH) — all drugs for Paroxysmal Nocturnal Haemoglobinuria (PNH) →

Sponsor

AKARI Therapeutics — full company profile →

Who can join

Adults 18 to 80, any sex, with Paroxysmal Nocturnal Haemoglobinuria (PNH). Patients with the condition only — healthy volunteers not accepted.

What's being measured

Primary outcomes are the specific endpoints the trial is designed to prove or disprove.

• Measurement of Ratio of LDH to the Upper Limit of Normal (ULN)
  Time frame: Day 0 and Day 28
  LDH is an indicator of disease progression in patients with PNH and is expected to fall to within 2x upper limit of normal (ULN) within 28 days in successfully treated patients. Units are measured in ratio of LDH:ULN, where the ULN is 250 U/L. The primary efficacy endpoint was the LDH AUC from Day 0 to 28 compared with 28 days pretreatment. Data for the 28 days pre-treatment was not collected, and
• Number and Type of Adverse Events (AE)
  Time frame: 2 years
  The number and type of reported AEs will be recorded as well as the opinion of the Principle Investigator (PI) as to their possible relationship to the study drug.

Sponsor's own description

Coversin in Paroxysmal Nocturnal Haemoglobinuria (PNH) in patients with resistance to Eculizumab due to complement C5 polymorphisms.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. The renaissance of complement therapeutics.
   Ricklin D, Mastellos DC, Reis ES, Lambris JD. · · 2018 · cited 305× · PMID 29199277 · DOI 10.1038/nrneph.2017.156
2. Anti-complement Treatment for Paroxysmal Nocturnal Hemoglobinuria: Time for Proximal Complement Inhibition? A Position Paper From the SAAWP of the EBMT.
   Risitano AM, Marotta S, Ricci P, Marano L, et al · · 2019 · cited 155× · PMID 31258525 · DOI 10.3389/fimmu.2019.01157
3. Incomplete inhibition by eculizumab: mechanistic evidence for residual C5 activity during strong complement activation.
   Harder MJ, Kuhn N, Schrezenmeier H, Höchsmann B, et al · · 2017 · cited 115× · PMID 28028023 · DOI 10.1182/blood-2016-08-732800
4. Diseases of complement dysregulation-an overview.
   Wong EKS, Kavanagh D. · · 2018 · cited 80× · PMID 29327071 · DOI 10.1007/s00281-017-0663-8
5. New milestones ahead in complement-targeted therapy.
   Ricklin D, Lambris JD. · · 2016 · cited 73× · PMID 27321574 · DOI 10.1016/j.smim.2016.06.001
6. Targeting the complement system in neuromyelitis optica spectrum disorder.
   Asavapanumas N, Tradtrantip L, Verkman AS. · · 2021 · cited 64× · PMID 33513036 · DOI 10.1080/14712598.2021.1884223
7. The Use of Tick Salivary Proteins as Novel Therapeutics.
   Chmelař J, Kotál J, Kovaříková A, Kotsyfakis M. · · 2019 · cited 42× · PMID 31297067 · DOI 10.3389/fphys.2019.00812
8. Complement factor 5 blockade reduces porcine myocardial infarction size and improves immediate cardiac function.
   Pischke SE, Gustavsen A, Orrem HL, Egge KH, et al · · 2017 · cited 34× · PMID 28258298 · DOI 10.1007/s00395-017-0610-9