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Study of Ravulizumab in Pediatric Participants With HSCT-TMA

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: NCT04557735
Recruitment Status : Recruiting
First Posted : September 22, 2020
Last Update Posted : May 6, 2023
Sponsor:
Information provided by (Responsible Party):
Alexion

Study Description
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Brief Summary:
This study will evaluate the safety, efficacy, pharmacokinetics, and pharmacodynamics of ravulizumab administered by intravenous infusion to pediatric participants, from 1 month to < 18 years of age, with HSCT-TMA. The treatment period is 26 weeks, followed by a 26-week off-treatment follow-up period.

Condition or disease Intervention/treatment Phase
Thrombotic Microangiopathy Drug: Ravulizumab Other: Best Supportive Care Phase 3

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 40 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 3, Open-label, Single Arm, Multicenter Study of Ravulizumab in Addition to Best Supportive Care in Pediatric Participants With Thrombotic Microangiopathy (TMA) After Hematopoietic Stem Cell Transplantation (HSCT)
Actual Study Start Date : November 6, 2020
Estimated Primary Completion Date : June 28, 2024
Estimated Study Completion Date : August 11, 2025

Resource links provided by the National Library of Medicine

Drug Information available for: Ravulizumab

Arms and Interventions
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Arm Intervention/treatment
Experimental: Ravulizumab plus Best Supportive Care
Participants will receive ravulizumab plus Best Supportive Care as background therapy.
 Drug: Ravulizumab
Weight-based doses of ravulizumab will be administered intravenously as a loading dose regimen followed by maintenance dosing every 4 or 8 weeks, depending upon weight.
Other Name: Ultomiris

Other: Best Supportive Care
Participants will receive medications, therapies, and interventions per standard hospital treatment protocols (unless specifically prohibited by the protocol).


Outcome Measures
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Primary Outcome Measures :
  1. TMA Response [ Time Frame: 26 weeks (treatment period) ]

Secondary Outcome Measures :
  1. Time To TMA Response [ Time Frame: 26 weeks (treatment period) and through 52 weeks (includes treatment period and off-treatment follow- up period) ]
  2. TMA Relapse [ Time Frame: Follow Up period (183-365 Days after start of study medication) ]
  3. Overall Survival [ Time Frame: 26 weeks and 52 weeks ]
  4. Hematologic Response [ Time Frame: 26 weeks and 52 weeks ]

    Hematologic Response as assessed by blood tests to measure lactate dehydrogenase (LDH) and platelet count.

    1. If baseline platelet count ≤ 50,000/mm3, all of the following criteria must be met:

      - Absolute platelet count > 50,000/mm3 without platelet transfusion support during the prior 7 days [or]

      If baseline platelet count > 50,000/mm3, all of the following criteria must be met:

      - ≥ 50% increase in platelet count compared to baseline value

    2. Normalization of LDH and absence of schistocytes


Eligibility Criteria
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Ages Eligible for Study:   28 Days to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. ≥ 28 days of age up to < 18 years of age at the time of signing the informed consent.
  2. Received HSCT within the past 12 months.
  3. Diagnosis of TMA that persists for at least 72 hours despite initial management.
  4. A TMA diagnosis based on meeting the select criteria during the Screening Period and/or <=14 days prior to the Screening Period.
  5. Body weight ≥ 5 kilograms at Screening or ≤7 days prior to the start of the Screening Period (date of consent).
  6. Female participants of childbearing potential and male participants with female partners of childbearing potential must use highly effective contraception.
  7. Participants must be vaccinated against meningococcal infections if clinically feasible. Participants who cannot receive meningococcal vaccine should receive antibiotic prophylaxis.
  8. Participants or their legally authorized representative must be capable of giving signed informed consent or assent

Exclusion Criteria:

  1. Thrombotic thrombocytopenic purpura (TTP) evidenced by ADAMTS13 deficiency.
  2. Shiga toxin producing Escherichia coli infection.
  3. Positive direct Coombs test
  4. Clinical diagnosis of disseminated intravascular coagulation (DIC)
  5. Known bone marrow/graft failure.
  6. Diagnosis of veno-occlusive disease (VOD), regardless of severity.
  7. Human immunodeficiency virus (HIV) infection
  8. Unresolved meningococcal disease.
  9. Presence or suspicion of sepsis (treated or untreated).
  10. Pregnancy or breastfeeding.
  11. Respiratory failure requiring mechanical ventilation
  12. Previously or currently treated with a complement inhibitor
  13. Participation in an interventional treatment study of any therapy for TMA
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): NCT04557735


Contacts
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Contact: Alexion Pharmaceuticals Inc. 855-752-2356 clinicaltrials@alexion.com

Locations
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Sponsors and Collaborators
Alexion
More Information
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Responsible Party: Alexion
ClinicalTrials.gov Identifier: NCT04557735    
Other Study ID Numbers: ALXN1210-TMA-314
2020-000761-16 ( EudraCT Number )
First Posted: September 22, 2020    Key Record Dates
Last Update Posted: May 6, 2023
Last Verified: May 2023
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
Keywords provided by Alexion:
Thrombotic Microangiopathy (TMA)
Ultomiris
Ravulizumab
Hematopoietic Stem Cell Transplant
Additional relevant MeSH terms:
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Vascular Diseases
Thrombotic Microangiopathies
Cardiovascular Diseases
Thrombocytopenia
Blood Platelet Disorders
Hematologic Diseases
 Ravulizumab
Complement Inactivating Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs