CONSENT II: Coversin in Paroxysmal Nocturnal Hemoglobinuria (PNH) in Patients With Resistance to Eculizumab Due to Complement C5 Polymorphisms

AK585

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

2018-05-14

2024-06-01

2024-06-01

Early phase I

6

Inclusion Criteria: 1. Patients with known PNH. 2. Aged 18 and above. No upper age limit. 3. Lactate dehydrogenase (LDH) ≥1.5 upper limit of normal. 4. Must agree to use two methods of contraception that are ≥99% effective in preventing pregnancy. 5. Resistance to eculizumab (Soliris®). 6. Voluntary written informed consent. 7. Willing to self-inject Coversin daily. 8. Willing to receive appropriate prophylaxis against Neisseria infection. 9. Willing to avoid prohibited medications for duration of study. Exclusion Criteria: 1. Subjects with body weight \<50 kg (110 lb) or \>100 kg (220 lb). 2. Pregnancy or breast feeding (females). 3. Known allergy to ticks or severe reaction to arthropod venom (e.g. bee or wasp venom). 4. Unresolved Neisseria meningitidis infection. 5. Patients who have not received adequate immunization against Neisseria meningitides. 6. Impaired hepatic function. 7. Patients with impaired renal function. 8. Failure to satisfy the Principal Investigator (PI) of fitness to participate for any other reason.

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE2'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 6, 'type': 'ESTIMATED'}}

2024-02-08