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Nomacopan (rVA576) in Transplant Associated Thrombotic Microangiopathy

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04784455
Recruitment Status : Recruiting
First Posted : March 5, 2021
Last Update Posted : February 8, 2024
Sponsor:
Information provided by (Responsible Party):
AKARI Therapeutics

Study Description
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Brief Summary:
Multicentre Study of nomacopan in Paediatric Haematopoietic Stem-Cell Transplant Associated Thrombotic Microangiopathy

Condition or disease Intervention/treatment Phase
Thrombotic Microangiopathies Drug: nomacopan (rVA576) Phase 3

Detailed Description:

This is an open-label, multi-centre study of two-parts, Part A and B, includes 24 weeks of treatment, safety follow up after 30 days.

Part A: dose algorithm, safety and efficacy

Part B: safety and efficacy

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 50 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Intervention Model Description: Open-label, uncontrolled, multi-centre two-part study. Part A: dose algorithm, safety and efficacy Part B: safety and efficacy
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Multicentre Study of Nomacopan (rVA576) in Paediatric Haematopoietic Stem-Cell Transplant Associated Thrombotic Microangiopathy
Actual Study Start Date : February 1, 2021
Estimated Primary Completion Date : June 2025
Estimated Study Completion Date : December 2025

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: nomacopan (rVA576)
The study population will consist of paediatric patients who have undergone allogeneic or autologous HSCT and develop HSCT-TMA within a year of HSCT
 Drug: nomacopan (rVA576)
The study population will consist of paediatric patients who have undergone allogeneic or autologous HSCT and develop HSCT-TMA within a year of HSCT


Outcome Measures
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Primary Outcome Measures :
  1. RBC transfusion independence for ≥ 28 days immediately prior to any scheduled clinical visit up to Week 24 [ Time Frame: 24 weeks ]
    Transfusion independence is defined as no RBC or platelet transfusion attributable to, or required to manage, thrombotic microangiopathy (TMA). Transfusions required for causes other than TMA will not be considered within the evaluation of the primary efficacy endpoints.

  2. Urine protein creatinine ratio ≤ 2 mg/mg [ Time Frame: 24 weeks ]
    Urine protein creatinine ratio ≤ 2 mg/mg


Secondary Outcome Measures :
  1. Renal Function Improvement [ Time Frame: 24 weeks ]
    Percentage of patients who achieve the primary endpoint of urine protein creatinine ratio ≤ 2 mg/mg (the nephrotic threshold) for ≥ 28 days

  2. Platelet transfusion independence [ Time Frame: 24 weeks ]
    Platelet transfusion independence for ≥ 28 days within the 24 week timeframe

  3. Normalisation of lab parameters [ Time Frame: 24 weeks ]
    Plasma sC5b-9 ≤ ULN

  4. Normalisation of lab parameters [ Time Frame: 24 weeks ]
    Lactate dehydrogenase (LDH) ≤ULN

  5. Normalisation of lab parameters [ Time Frame: 24 weeks ]
    Normalization of haptoglobin

  6. Safety and tolerability of nomacopan [ Time Frame: 28 weeks ]
    New or worsening AEs after dosing of investigational product will be recorded in the eCRF.

  7. Safety and tolerability of nomacopan [ Time Frame: 28 weeks ]
    Listings of subjects who have an SAE.

  8. Safety and tolerability of nomacopan [ Time Frame: 28 weeks ]
    Listings of subjects who discontinue from the study due to an AE.

  9. Safety and tolerability of nomacopan [ Time Frame: 28 weeks ]
    Occurrence of significant laboratory abnormalities will be summarized.


Eligibility Criteria
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Ages Eligible for Study:   6 Months to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Aged ≥ 0.5 and < 18 years at the time of diagnosis of TMA.
  2. Undergone allogeneic or autologous HSCT.
  3. TMA diagnosis within a year of their allogeneic or autologous HSCT.
  4. Clinical or histological diagnosis of TMA
  5. Provision of written informed consent.
  6. Provision of informed assent

Exclusion Criteria:

  1. Patients weighing less than 5 kg.
  2. Patients with a positive direct Coombs' test.
  3. Patients who do not receive nomacopan within 21 days of the initial diagnosis of TMA.
  4. Patients having an active systemic or organ system bacterial or fungal infection or progressive severe infection at the time of diagnosis of TMA
  5. Grade 4 Acute GVHD
  6. Received eculizumab or any other complement blocker therapy at any time.
  7. Known hypersensitivity to the active ingredient or excipients

If an enrolled patient has a positive ADAMTS13 test (<10%) returned from their screening assessment, the patient should be withdrawn from the study

Contacts and Locations
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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): NCT04784455


Contacts
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Contact: Wynne W Davies, MD 02080040268 medical.monitors@akaritx.com

Locations
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United States, California
Children's Hospital Los Angeles Recruiting
Los Angeles, California, United States, 90027
Contact: Neena Kapoor       medical.monitors@akaritx.com   
Principal Investigator: Neena Kapoor         
Stanford Children's Hospital Recruiting
Palo Alto, California, United States, 94304
Contact: David Shyr       medical.monitors@akaritx.com   
Principal Investigator: David Shyr         
United States, North Carolina
Duke University Medical Center, Children's Health Center Recruiting
Durham, North Carolina, United States, 27710
Contact: Vinod Prasad       medical.monitors@akaritx.com   
Principal Investigator: Vinod Prasad         
United States, Pennsylvania
Children's Hospitall of Philadelphia Recruiting
Philadelphia, Pennsylvania, United States, 19104
Contact: Timothy Olson       medical.monitors@akaritx.com   
Principal Investigator: Timothy Olson         
Poland
Uniwersytecki Szpital Kliniczny im. Jana Mikulicza Radeckiego we Wroclawiu Recruiting
Wrocław, Poland, 50556
Contact: Krzysztof Kalwak       medical.monitors@akaritx.com   
Principal Investigator: Krzysztof Kalwak         
United Kingdom
Great Ormond Street Hospital (GOSH) Recruiting
London, United Kingdom, WC1N3JH
Contact: Robert Chiesa       medical.monitors@akaritx.com   
Principal Investigator: Robert Chiesa         
Royal Manchester Children's Hospital Recruiting
Manchester, United Kingdom, M139WL
Contact: Robert Wynn       medical.monitors@akaritx.com   
Principal Investigator: Robert Wynn         
Sponsors and Collaborators
AKARI Therapeutics
More Information
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Responsible Party: AKARI Therapeutics
ClinicalTrials.gov Identifier: NCT04784455    
Other Study ID Numbers: AK901
First Posted: March 5, 2021    Key Record Dates
Last Update Posted: February 8, 2024
Last Verified: February 2024
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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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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Vascular Diseases
Thrombotic Microangiopathies
Cardiovascular Diseases
Thrombocytopenia
 Blood Platelet Disorders
Hematologic Diseases
Cytopenia