Back to resultsCoversin in PNH in Patients With Resistance to Eculizumab Due to Complement C5 Polymorphisms (CONSENTII)
Primary Purpose
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Status
Enrolling by invitation
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Coversin
Sponsored by
About this trial
This is an interventional treatment trial for Paroxysmal Nocturnal Hemoglobinuria (PNH)
Eligibility Criteria
Inclusion Criteria:
- Patients with known PNH.
- Aged 18 and above. No upper age limit.
- Lactate dehydrogenase (LDH) ≥1.5 upper limit of normal.
- Must agree to use two methods of contraception that are ≥99% effective in preventing pregnancy.
- Resistance to eculizumab (Soliris®).
- Voluntary written informed consent.
- Willing to self-inject Coversin daily.
- Willing to receive appropriate prophylaxis against Neisseria infection.
- Willing to avoid prohibited medications for duration of study.
Exclusion Criteria:
- Subjects with body weight <50 kg (110 lb) or >100 kg (220 lb).
- Pregnancy or breast feeding (females).
- Known allergy to ticks or severe reaction to arthropod venom (e.g. bee or wasp venom).
- Unresolved Neisseria meningitidis infection.
- Patients who have not received adequate immunization against Neisseria meningitides.
- Impaired hepatic function.
- Patients with impaired renal function.
- Failure to satisfy the Principal Investigator (PI) of fitness to participate for any other reason.
Sites / Locations
- Memorial Sloan Kettering Cancer Center
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Coversin treatment
Arm Description
Coversin - 22.5mg followed by 45mg for 6 months.
Outcomes
Primary Outcome Measures
Measurement of serum lactate dehydrogenase (LDH)
Measurement of serum lactate dehydrogenase (LDH)
Secondary Outcome Measures
Change in lactate dehydrogenase (LDH)
Change in lactate dehydrogenase (LDH)
Change in mean haemoglobin (Hb)
Change in mean haemoglobin (Hb)
Change in score on Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F) questionnaire
The FACIT Measurement System is a collection of quality of life questionnaires targeted to the management of chronic illness. Change in FACIT-F score, measured on a scale from 0 - 4 (0 = Not at all, 1 = A little bit, 2 = Some-what, 3 = Quite a bit, 4 = Very much).
Change in EuroQol 5 Dimensional 5 Level (EQ-5D-5L) score
The descriptive questionnaire comprises of five sections: Mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each section has 5 levels: No problems, slight problems, moderate problems, severe problems and extreme problems. The patient is asked to indicate his/her health state by ticking the box next to the most appropriate statement in each of the five dimensions. This decision results in a 1-digit number that expresses the level selected for that section. The digits for the five sections can be combined into a 5-digit number that describes the patient's health state.
Number of blood transfusions
Number of blood transfusions
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT03427060
Brief Title
Coversin in PNH in Patients With Resistance to Eculizumab Due to Complement C5 Polymorphisms
Acronym
CONSENTII
Official Title
CONSENT II: Coversin in Paroxysmal Nocturnal Hemoglobinuria (PNH) in Patients With Resistance to Eculizumab Due to Complement C5 Polymorphisms
Study Type
Interventional
2. Study Status
Record Verification Date
June 2022
Overall Recruitment Status
Enrolling by invitation
Study Start Date
May 14, 2018 (Actual)
Primary Completion Date
June 2024 (Anticipated)
Study Completion Date
June 2024 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
AKARI Therapeutics
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Paroxysmal Nocturnal Hemoglobinuria (PNH)
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
6 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Coversin treatment
Arm Type
Experimental
Arm Description
Coversin - 22.5mg followed by 45mg for 6 months.
Intervention Type
Drug
Intervention Name(s)
Coversin
Intervention Description
Coversin - 22.5mg followed by 45mg for 6 months.
Primary Outcome Measure Information:
Title
Measurement of serum lactate dehydrogenase (LDH)
Description
Measurement of serum lactate dehydrogenase (LDH)
Time Frame
Baseline to Day 180
Secondary Outcome Measure Information:
Title
Change in lactate dehydrogenase (LDH)
Description
Change in lactate dehydrogenase (LDH)
Time Frame
Day 28 to Day 180
Title
Change in mean haemoglobin (Hb)
Description
Change in mean haemoglobin (Hb)
Time Frame
Baseline to Day 180
Title
Change in score on Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F) questionnaire
Description
The FACIT Measurement System is a collection of quality of life questionnaires targeted to the management of chronic illness. Change in FACIT-F score, measured on a scale from 0 - 4 (0 = Not at all, 1 = A little bit, 2 = Some-what, 3 = Quite a bit, 4 = Very much).
Time Frame
Baseline to Day 180
Title
Change in EuroQol 5 Dimensional 5 Level (EQ-5D-5L) score
Description
The descriptive questionnaire comprises of five sections: Mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each section has 5 levels: No problems, slight problems, moderate problems, severe problems and extreme problems. The patient is asked to indicate his/her health state by ticking the box next to the most appropriate statement in each of the five dimensions. This decision results in a 1-digit number that expresses the level selected for that section. The digits for the five sections can be combined into a 5-digit number that describes the patient's health state.
Time Frame
Baseline to Day 180
Title
Number of blood transfusions
Description
Number of blood transfusions
Time Frame
Baseline to Day 180
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Patients with known PNH.
Aged 18 and above. No upper age limit.
Lactate dehydrogenase (LDH) ≥1.5 upper limit of normal.
Must agree to use two methods of contraception that are ≥99% effective in preventing pregnancy.
Resistance to eculizumab (Soliris®).
Voluntary written informed consent.
Willing to self-inject Coversin daily.
Willing to receive appropriate prophylaxis against Neisseria infection.
Willing to avoid prohibited medications for duration of study.
Exclusion Criteria:
Subjects with body weight <50 kg (110 lb) or >100 kg (220 lb).
Pregnancy or breast feeding (females).
Known allergy to ticks or severe reaction to arthropod venom (e.g. bee or wasp venom).
Unresolved Neisseria meningitidis infection.
Patients who have not received adequate immunization against Neisseria meningitides.
Impaired hepatic function.
Patients with impaired renal function.
Failure to satisfy the Principal Investigator (PI) of fitness to participate for any other reason.
Facility Information:
Facility Name
Memorial Sloan Kettering Cancer Center
City
New York
State/Province
New York
ZIP/Postal Code
10065
Country
United States
12. IPD Sharing Statement
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Coversin in PNH in Patients With Resistance to Eculizumab Due to Complement C5 Polymorphisms
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