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Study of Ravulizumab in Pediatric Participants With HSCT-TMA

Primary Purpose

Thrombotic Microangiopathy

Status
Recruiting
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Ravulizumab
Best Supportive Care
Sponsored by
Alexion
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Thrombotic Microangiopathy focused on measuring Thrombotic Microangiopathy (TMA), Ultomiris, Ravulizumab, Hematopoietic Stem Cell Transplant

Eligibility Criteria

28 Days - 17 Years (Child)All SexesDoes not accept healthy volunteers

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.
  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

Sites / Locations

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Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Ravulizumab plus Best Supportive Care

Arm Description

Participants will receive ravulizumab plus Best Supportive Care as background therapy.

Outcomes

Primary Outcome Measures

TMA Response

Secondary Outcome Measures

Time To TMA Response
TMA Relapse
Overall Survival
Hematologic Response
Hematologic Response as assessed by blood tests to measure lactate dehydrogenase (LDH) and platelet count. 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 Normalization of LDH and absence of schistocytes

Full Information

First Posted
September 2, 2020
Last Updated
May 4, 2023
Sponsor
Alexion
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1. Study Identification

Unique Protocol Identification Number
NCT04557735
Brief Title
Study of Ravulizumab in Pediatric Participants With HSCT-TMA
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)
Study Type
Interventional

2. Study Status

Record Verification Date
May 2023
Overall Recruitment Status
Recruiting
Study Start Date
November 6, 2020 (Actual)
Primary Completion Date
June 28, 2024 (Anticipated)
Study Completion Date
August 11, 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Alexion

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
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Thrombotic Microangiopathy
Keywords
Thrombotic Microangiopathy (TMA), Ultomiris, Ravulizumab, Hematopoietic Stem Cell Transplant

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
40 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Ravulizumab plus Best Supportive Care
Arm Type
Experimental
Arm Description
Participants will receive ravulizumab plus Best Supportive Care as background therapy.
Intervention Type
Drug
Intervention Name(s)
Ravulizumab
Other Intervention Name(s)
Ultomiris
Intervention Description
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.
Intervention Type
Other
Intervention Name(s)
Best Supportive Care
Intervention Description
Participants will receive medications, therapies, and interventions per standard hospital treatment protocols (unless specifically prohibited by the protocol).
Primary Outcome Measure Information:
Title
TMA Response
Time Frame
26 weeks (treatment period)
Secondary Outcome Measure Information:
Title
Time To TMA Response
Time Frame
26 weeks (treatment period) and through 52 weeks (includes treatment period and off-treatment follow- up period)
Title
TMA Relapse
Time Frame
Follow Up period (183-365 Days after start of study medication)
Title
Overall Survival
Time Frame
26 weeks and 52 weeks
Title
Hematologic Response
Description
Hematologic Response as assessed by blood tests to measure lactate dehydrogenase (LDH) and platelet count. 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 Normalization of LDH and absence of schistocytes
Time Frame
26 weeks and 52 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
28 Days
Maximum Age & Unit of Time
17 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: ≥ 28 days of age up to < 18 years of age at the time of signing the informed consent. Received HSCT within the past 12 months. Diagnosis of TMA that persists for at least 72 hours despite initial management. A TMA diagnosis based on meeting the select criteria during the Screening Period and/or <=14 days prior to the Screening Period. Body weight ≥ 5 kilograms at Screening or ≤7 days prior to the start of the Screening Period (date of consent). Female participants of childbearing potential and male participants with female partners of childbearing potential must use highly effective contraception. Participants must be vaccinated against meningococcal infections if clinically feasible. Participants who cannot receive meningococcal vaccine should receive antibiotic prophylaxis. Participants or their legally authorized representative must be capable of giving signed informed consent or assent Exclusion Criteria: Thrombotic thrombocytopenic purpura (TTP) evidenced by ADAMTS13 deficiency. Shiga toxin producing Escherichia coli infection. Positive direct Coombs test Clinical diagnosis of disseminated intravascular coagulation (DIC) Known bone marrow/graft failure. Diagnosis of veno-occlusive disease (VOD), regardless of severity. Human immunodeficiency virus (HIV) infection Unresolved meningococcal disease. Presence or suspicion of sepsis (treated or untreated). Pregnancy or breastfeeding. Respiratory failure requiring mechanical ventilation Previously or currently treated with a complement inhibitor Participation in an interventional treatment study of any therapy for TMA
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Alexion Pharmaceuticals Inc.
Phone
855-752-2356
Email
clinicaltrials@alexion.com
Facility Information:
Facility Name
Clinical Trial Site
City
Birmingham
State/Province
Alabama
ZIP/Postal Code
35233
Country
United States
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85016
Country
United States
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Tucson
State/Province
Arizona
ZIP/Postal Code
85724
Country
United States
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Duarte
State/Province
California
ZIP/Postal Code
91010
Country
United States
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
San Francisco
State/Province
California
ZIP/Postal Code
94158
Country
United States
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30322
Country
United States
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60611
Country
United States
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Louisville
State/Province
Kentucky
ZIP/Postal Code
40202
Country
United States
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55414
Country
United States
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55455
Country
United States
Individual Site Status
Active, not recruiting
Facility Name
Clinical Trial Site
City
Valhalla
State/Province
New York
ZIP/Postal Code
10595
Country
United States
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Charlotte
State/Province
North Carolina
ZIP/Postal Code
28203
Country
United States
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Akron
State/Province
Ohio
ZIP/Postal Code
44308
Country
United States
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44195
Country
United States
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
Country
United States
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Dallas
State/Province
Texas
ZIP/Postal Code
75235
Country
United States
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Fort Worth
State/Province
Texas
ZIP/Postal Code
76104
Country
United States
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84112
Country
United States
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Madison
State/Province
Wisconsin
ZIP/Postal Code
53792
Country
United States
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Bron
Country
France
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Nantes
Country
France
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Paris
Country
France
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Strasbourg
Country
France
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Vandœuvre-lès-Nancy
Country
France
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Berlin
Country
Germany
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Freiburg
Country
Germany
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Halle
Country
Germany
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Tuebingen
Country
Germany
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Jerusalem
Country
Israel
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Petach-Tikva
Country
Israel
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Ramat Gan
Country
Israel
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Bologna
Country
Italy
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Brescia
Country
Italy
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Firenze
Country
Italy
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Genova
Country
Italy
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Monza
Country
Italy
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Pavia
Country
Italy
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Rome
Country
Italy
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Torino
Country
Italy
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Verona
Country
Italy
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Fukuoka
Country
Japan
Individual Site Status
Recruiting
Facility Name
Clinical Study Site
City
Fukushima
Country
Japan
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Kobe
Country
Japan
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Nagoya
Country
Japan
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Osakasayama
Country
Japan
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Osaka
Country
Japan
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Saitama
Country
Japan
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Setagaya-Ku
Country
Japan
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Goyang
Country
Korea, Republic of
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Seoul
Country
Korea, Republic of
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Barcelona
Country
Spain
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Esplugues De Llobregat
Country
Spain
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Madrid
Country
Spain
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Salamanca
Country
Spain
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Valencia
Country
Spain
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Birmingham
Country
United Kingdom
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Bristol
Country
United Kingdom
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Leeds
Country
United Kingdom
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
London
Country
United Kingdom
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Newcastle
Country
United Kingdom
Individual Site Status
Recruiting
Facility Name
Clinical Trial Site
City
Wuerzburg
Country
United Kingdom
Individual Site Status
Recruiting

12. IPD Sharing Statement

Plan to Share IPD
No

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Study of Ravulizumab in Pediatric Participants With HSCT-TMA

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