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Trial record 9 of 73 for:    "Paroxysmal Nocturnal Hemoglobinuria"

Treating Paroxysmal Nocturnal Haemoglobinuria Patients With rVA576 (CAPSTONE)

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ClinicalTrials.gov Identifier: NCT03588026
Recruitment Status : Recruiting
First Posted : July 17, 2018
Last Update Posted : February 21, 2019
Sponsor:
Information provided by (Responsible Party):
AKARI Therapeutics

Brief Summary:
rVA576 for patients with Paroxysmal Nocturnal Hemoglobinuria (PNH).

Condition or disease Intervention/treatment Phase
Paroxysmal Nocturnal Hemoglobinuria (PNH) Drug: rVA576 Other: Standard of care (SOC) Phase 3

Detailed Description:

rVA576, 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.

Patients will be treated with rVA576 by daily subcutaneous injection 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 rVA576 and being entered into the long term follow-up study.


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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 30 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Investigational Product ; Coversin. Phase III Safety and Efficacy in Three-Part, Two-Arm, Randomised Open Label Evaluation in Patients With Paroxysmal Nocturnal Haemoglobinuria (PNH)
Actual Study Start Date : July 9, 2018
Estimated Primary Completion Date : January 2020
Estimated Study Completion Date : January 2020


Arm Intervention/treatment
Experimental: Arm 1 - 9 months of treatment (rVA576 plus SOC)
6 months (SOC plus rVA576), Followed by a further 3 months of (SOC plus rVA576).
Drug: rVA576
6 months of treatment, rVA576 plus SOC. Followed by a further 3 months of rVA576 plus SOC. In total, 9 months on rVA576 plus SOC.

Other: Standard of care (SOC)
6 months on SOC followed by 3 months of treatment with rVA576 plus SOC. In total, 3 months on rVA576 plus SOC.

Experimental: Arm 2 - 6 months on SOC
6 months on SOC only. Followed by 3 months (SOC plus rVA576).
Drug: rVA576
6 months of treatment, rVA576 plus SOC. Followed by a further 3 months of rVA576 plus SOC. In total, 9 months on rVA576 plus SOC.

Other: Standard of care (SOC)
6 months on SOC followed by 3 months of treatment with rVA576 plus SOC. In total, 3 months on rVA576 plus SOC.




Primary Outcome Measures :
  1. HB (Haemoglobin) stabilisation rate and the avoidance of packed red blood cells (PRBC) transfusions [ Time Frame: 9 months ]
    Haemoglobin stabilisation rate defined as haemoglobin greater than the set point for each patient during the pre-study randomisation period and the avoidance of PRBC transfusions during the treatment period.


Secondary Outcome Measures :
  1. Number of units of packed red blood cells (PRBC) transfused [ Time Frame: Day 1 to Day 180 ]
    Number of units of packed red blood cells (PRBC) transfused from Baseline Day 1 to Day 180

  2. Percentage of patients who achieve transfusion avoidance [ Time Frame: Day 1 to Day 180 ]
    Percentage of patients who achieve transfusion avoidance

  3. Change in (QOl) Quality of Life score [ Time Frame: Day 1 to Day 180 ]
    Change in Quality of Life score

  4. AUC (LDH) [ Time Frame: Day 1 to Day 180 ]
    AUC (Area under the curve) (LDH) Lactate Dehydrogenase

  5. CH50 [ Time Frame: Day 1 to Day 180 ]
    CH50 (Classical haemolytic 50% lysis)



Information from the National Library of Medicine

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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. Willing to give informed consent to treatment with rVA576
  2. Diagnosed with paroxysmal nocturnal haemoglobinuria (PNH)
  3. Have not received any complement inhibitor within the 4 months prior to screening
  4. ≥ 18 years of age at the time of screening
  5. Weight ≥50kg
  6. Complete transfusion medical history for 12 months
  7. Transfusion dependent
  8. LDH ≥1.5 x the ULN
  9. Willing to receive appropriate prophylaxis against Neisseria meningitidis infection, by both immunisation and continuous or intermittent antibiotics
  10. Willing to avoid prohibited medications such as other complement inhibitors and chemotherapeutic agents
  11. Patients must agree to avoid pregnancy and fathering children from the time of signing the Informed Consent Form until 90 days after the last dose of rVA576.
  12. Patients who are on erythropoietin and/or immunosuppressant treatment should be on stable doses for at least 6 months.
  13. Patients who are taking systemic corticosteroids should be on a stable dose for at least 4 weeks.
  14. Patients on anticoagulant therapy should be well-controlled prior to entry.
  15. Patients taking iron and/or folic acid supplements should be on a stable dose for at least 4 weeks

Exclusion Criteria:

  1. Patients whose mean haemoglobin level over the previous 12 months prior to screening was greater than 105 g/L (10.5g/dL)
  2. Severe bone marrow failure
  3. Patients with a platelet count of ≤ 70 x 109/L
  4. Patients with known or suspected acquired somatic mutations affecting the bone marrow (e.g. acute myeloid leukaemia) which may be associated with PNH
  5. Chemotherapy within 3 months of screening visit
  6. History of recurrent bacterial infections or suspicion of active bacterial infections requiring antibiotic therapy
  7. Planned or actual pregnancy or breast feeding (females)
  8. Known allergy to ticks or severe reaction to arthropod venom (e.g. bee or wasp venom)
  9. Unresolved N. meningitidis infection.
  10. Patients who are not willing to receive adequate immunisation against N. meningitidis unless, in the opinion of the investigator, the risks of delaying therapy outweigh the risks of developing a meningococcal infection
  11. Impaired hepatic function unless, in the opinion of the investigator, the risks of delaying therapy outweigh the risks of treatment in the presence of impaired hepatic function
  12. Patients with a glomerular filtration rate (GFR) of <30mL/min/1.73m2 unless, in the opinion of the investigator, the risks of delaying therapy outweigh the risks of treatment in the presence of impaired renal function
  13. Participation in other clinical trials within 4 weeks of signing the consent form
  14. History of active systemic autoimmune diseases.
  15. Any other systemic disorders that could interfere with the evaluation of the study treatment
  16. Failure to comply with protocol requirements
  17. Known Hepatitis B or Hepatitis C

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): NCT03588026


Contacts
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Contact: Wynne Weston-Davies 020 8004 0967 ext 0268 wynne.weston-davies@akaritx.com
Contact: Rohini Verma 020 8004 0271 ext 0271 rohini.verma@akaritx.com

Locations
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Kazakhstan
Almaty City Hospital No.7 Recruiting
Almaty, Microdistrict Kalkaman, Kazakhstan, 050006
Contact: Dalimantas Glodenis    +370 604 666 20    Dalimantas.Glodenis@pharm-olam.com   
Principal Investigator: Gulzhan Sabyrbayeva, MBBS         
Lithuania
Vilnius University Hospital Santaros Klinikos , Santariškių St. 2, LT-08661, Recruiting
Vilnius, Lithuania, LT-08661
Contact: Dalimantas Glodenis    +370 604 666 20    Dalimantas.Glodenis@pharm-olam.com   
Principal Investigator: Andrius Degulys, MBBS         
Sri Lanka
University of Kelaniya, Faculty of Medicine, Thalagolla Road Recruiting
Colombo, Sri Lanka, Ragama/11010
Contact: Arka Moitra    +91-80-6718 2214    arka.moitra@pharm-olam.com   
Principal Investigator: Senani Williams, MBBS         
Sponsors and Collaborators
AKARI Therapeutics
Investigators
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Principal Investigator: Andrius Degulys, MBBS Vilnius University Hospital Santaros Klinikos

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Responsible Party: AKARI Therapeutics
ClinicalTrials.gov Identifier: NCT03588026     History of Changes
Other Study ID Numbers: AK580
First Posted: July 17, 2018    Key Record Dates
Last Update Posted: February 21, 2019
Last Verified: February 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: 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