Coversin in PNH in Patients With Resistance to Eculizumab Due to Complement C5 Polymorphisms

NCT ID: NCT03427060

Last Updated: 2025-05-04

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 1 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2018-05-14
• Study Completion Date: 2021-02-03

Brief Summary

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

Detailed Description

Coversin, a small protein complement C5 inhibitor which prevents the cleavage of C5 by C5 convertase into C5a and C5b, will be used in an open label, non-comparative clinical trial in patients with PNH and proven resistance to eculizumab due to C5 polymorphisms. Patients will be treated with Coversin by daily subcutaneous injection for 6 months in order to determine the safety and efficacy of the drug in these circumstances. If satisfactory control of the PNH is achieved, and at the discretion of the Principal Investigator (PI), patients will have the option of remaining on Coversin and being entered into the long term follow-up study.

Conditions

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Coversin treatment

Coversin - 22.5mg followed by 45mg for 6 months.

Group Type: EXPERIMENTAL

Coversin

Intervention Type: DRUG

Coversin - 22.5mg followed by 45mg for 6 months.

Interventions

Coversin

Coversin - 22.5mg followed by 45mg for 6 months.

Intervention Type: DRUG

Eligibility Criteria

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.

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

AKARI Therapeutics

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Memorial Sloan Kettering Cancer Center

New York, New York, United States

Countries

United States

Provided Documents

Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

AK585

Identifier Type: -

Identifier Source: org_study_id