Coversin in Paroxysmal Nocturnal Haemoglobinuria (PNH)

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02591862
• Date of registration: 10/09/2015
• Prospective Registration: Yes
• Primary sponsor: AKARI Therapeutics
• Public title: Coversin in Paroxysmal Nocturnal Haemoglobinuria (PNH)
• Scientific title: Coversin in Paroxysmal Nocturnal Haemoglobinuria (PNH) in Patients With Resistance to Eculizumab Due to Complement C5 Polymorphisms
• Date of first enrolment: February 2016
• Target sample size: 6
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02591862
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

Netherlands

Contacts

• Name: Saskia Dr Langemeijer
• Affiliation: Radboud University

• Name: Petra Dr Muus
• Affiliation: Radboud University

Key inclusion & exclusion criteria

Inclusion Criteria:

- Patients with known Paroxysmal Nocturnal Haemoglobinuria (PNH)

- LDH >=1.5 Upper Limit of Normal (ULN)

- Resistance to Eculizumab proven by both a recognised C5 polymorphism on genetic
screening and complement inhibition on CH50 ELISA of <100% at concentrations of
Eculizumab in excess of 5mug/mL

- Willing to self-inject Coversin daily or to receive daily subcutaneous injections by a
home nurse or in a doctor's office or hospital clinic

- Males or females taking adequate contraceptive precautions if of childbearing
potential, 18 - 80 years of age

- Body weight =50kg and = 100kg

- The patient has provided written informed consent.

- Willing to avoid prohibited medications for duration of study

- Must agree to take appropriate prophylactic precautions against Neisseria infection.

- Must be counselled regarding the possible reproductive risks of using Coversin and be
advised to use an adequate method of contraception pending further data on
reproductive toxicology.

Exclusion Criteria:

- Body weight <50kg or>100kg

- Pregnancy (females)

- Failure to satisfy the PI of fitness to participate for any other reason

- Known allergy to ticks or severe reaction to arthropod venom (e.g., bee or wasp venom)



Age minimum: 18 Years
Age maximum: 80 Years
Gender: All

Health Condition(s) or Problem(s) studied

Paroxysmal Nocturnal Haemoglobinuria (PNH)

Intervention(s)

Drug: Coversin

Primary Outcome(s)

Measurement of Serum Lactic Dehydrogenase (LDH) from Day 0 (pre-dose) to Day 28 (AUC) compared with 28 days pre-treatment [Time Frame: Day 0 to Day 28]

Secondary Outcome(s)

Measurement of Haptoglobin (Hp) [Time Frame: Day 28 and Day 180]

Change in proportion of PNH [Time Frame: Day 0 - Day 90]

Measurement of Haemoglobin (Hb) [Time Frame: Day 28 and Day 180]

Change in Functional Assessment of Chronic Illness Therapy (FACIT) Score [Time Frame: Day 0 - Day 180]

Change in Quality Of Life Questionnaire (QOQ) Score [Time Frame: Day 0 - Day 180]

Measurement of Change in LDH [Time Frame: Day 0 (pre-dose) at monthly intervals up to 1 year]

Number and type of adverse events (AE) [Time Frame: 6 months]

Secondary ID(s)

AK578

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Radboud University