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

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ClinicalTrials.gov Identifier: NCT03427060
Recruitment Status : Recruiting
First Posted : February 9, 2018
Last Update Posted : December 20, 2018
Sponsor:
Information provided by (Responsible Party):
AKARI Therapeutics

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

Condition or disease Intervention/treatment Phase
Paroxysmal Nocturnal Hemoglobinuria (PNH) Drug: Coversin Phase 2

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 6 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: CONSENT II: Coversin in Paroxysmal Nocturnal Hemoglobinuria (PNH) in Patients With Resistance to Eculizumab Due to Complement C5 Polymorphisms
Actual Study Start Date : May 14, 2018
Estimated Primary Completion Date : January 2019
Estimated Study Completion Date : January 2019


Arm Intervention/treatment
Experimental: Coversin treatment
Coversin - 22.5mg followed by 45mg for 6 months.
Drug: Coversin
Coversin - 22.5mg followed by 45mg for 6 months.




Primary Outcome Measures :
  1. Measurement of serum lactate dehydrogenase (LDH) [ Time Frame: Baseline to Day 180 ]
    Measurement of serum lactate dehydrogenase (LDH)


Secondary Outcome Measures :
  1. Change in lactate dehydrogenase (LDH) [ Time Frame: Day 28 to Day 180 ]
    Change in lactate dehydrogenase (LDH)

  2. Change in mean haemoglobin (Hb) [ Time Frame: Baseline to Day 180 ]
    Change in mean haemoglobin (Hb)

  3. Change in score on Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F) questionnaire [ Time Frame: Baseline to Day 180 ]
    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).

  4. Change in EuroQol 5 Dimensional 5 Level (EQ-5D-5L) score [ Time Frame: Baseline to Day 180 ]
    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.

  5. Number of blood transfusions [ Time Frame: Baseline to Day 180 ]
    Number of blood transfusions



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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03427060


Contacts
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Contact: Beth Hoover 646.888.8570 hoovere@mskcc.org
Contact: Taylor W Borrill 646.888.8575 borrillt@mskcc.org

Locations
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United States, New York
Memorial Sloan Kettering Cancer Center Recruiting
New York, New York, United States, 10065
Contact: Beth Hoover    646-888-8570    hoovere@mskcc.org   
Contact: Taylor W Borrill    646.888.8575    borrillt@mskcc.org   
Sponsors and Collaborators
AKARI Therapeutics

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Responsible Party: AKARI Therapeutics
ClinicalTrials.gov Identifier: NCT03427060     History of Changes
Other Study ID Numbers: AK585
First Posted: February 9, 2018    Key Record Dates
Last Update Posted: December 20, 2018
Last Verified: December 2018

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
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Hemoglobinuria
Hemoglobinuria, Paroxysmal
Proteinuria
Urination Disorders
Urologic Diseases
Urological Manifestations
Signs and Symptoms
Anemia, Hemolytic
Anemia
Hematologic Diseases
Myelodysplastic Syndromes
Bone Marrow Diseases
Complement System Proteins
Complement C5
Immunologic Factors
Physiological Effects of Drugs