Long-term Study of Romiplostim in Thrombocytopenic Pediatric Patients With Immune Thrombocytopenia (ITP)

NCT ID: NCT02279173

Last Updated: 2022-06-23

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 203 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2014-12-10
• Study Completion Date: 2019-08-08

Brief Summary

This is a phase 3b single arm, open label, multicenter study describing the percentage of time pediatric participants with ITP have a platelet response while receiving romiplostim, defined as a platelet count ≥ 50 x 10^9/L in the absence of ITP rescue medications for the past 4 weeks.

Conditions

Immune Thrombocytopenia

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Romiplostim

Participants received romiplostim administered weekly by subcutaneous injection for up to 3 years. The starting dose was 1 µg/kg titrated in 1 µg/kg increments up to a maximum of 10 µg/kg to reach a target platelet count ≥ 50 x 10⁹/L.

Group Type: EXPERIMENTAL

Romiplostim

Intervention Type: DRUG

Romiplostim subcutaneous weekly injection

Interventions

Romiplostim

Romiplostim subcutaneous weekly injection

Intervention Type: DRUG

Other Intervention Names

Nplate

Eligibility Criteria

Inclusion Criteria

• Diagnosis of primary ITP according to The American Society of Hematology (ASH) Guidelines at least 6 months before screening, regardless of splenectomy status
• Age ≥ 1 year and < 18 years of age
• Refractory to prior ITP therapy, relapsed after at prior ITP therapy, or be ineligible for other therapies. Examples of prior therapy include: corticosteroids, intravenous Immunoglobulin (IVIG), anti-D immunoglobulin, platelet transfusions.
• Platelet count ≤ 30 x10^9/L or is experiencing uncontrolled bleeding
• Has provided informed consent before any study-specific procedure;
• Adequate hematologic, renal, and liver function during screening:

• Hemoglobin > 10.0 g/dL
• Serum creatinine ≤ 1.5 x the upper limit of normal (ULN)
• Total serum bilirubin ≤ 1.5 x the ULN
• Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 3.0 x the ULN
• For the EU, Switzerland and Turkey protocol supplement, subject must agree to a scheduled bone marrow biopsy and aspirate at Year 1 or Year 2 following romiplostim treatment and any unscheduled biopsies if clinically indicated
• For the EU, Switzerland and Turkey protocol supplement, a reticulin grade of 0, 1, 2, or 3 according to the modified Bauermeister grading scale, as assessed by central laboratory from a bone marrow biopsy performed within 1 year prior to planned first dose of romiplostim or consent to a pre-treatment bone marrow biopsy and aspirate prior to planned first dose of romiplostim

Exclusion Criteria

• History of a bone marrow stem cell disorder (Any abnormal bone marrow findings other than those typical of ITP must be approved by Amgen before a subject may be enrolled)
• Prior bone marrow transplant or peripheral blood progenitor cell transplant
• Active or prior malignancy except non-melanoma skin cancers within the last 5 years
• History of myelodysplastic syndrome
• History of bleeding diathesis
• History of congenital thrombocytopenia
• History of Hepatitis B, Hepatitis C or human immunodeficiency virus (HIV)
• History of systemic lupus erythematosus, Evans syndrome, or autoimmune neutropenia
• History of antiphospholipid antibody syndrome or known positive for lupus anticoagulant
• History of disseminated intravascular coagulation, hemolytic uremic syndrome, or thrombotic thrombocytopenic purpura
• History of venous thromboembolism or thrombotic events
• Previous use of romiplostim or previous use of eltrombopag within 4 weeks of enrollment
• Previous use of pegylated recombinant human megakaryocyte growth and development factor (PEG-rHuMGDF), recombinant human thrombopoietin (rHuTPO) or any other platelet producing agent
• Rituximab (for any indication) or 6-mercaptopurine within 8 weeks of enrollment, or anticipated use at any time during the study
• Splenectomy within 4 weeks of the screening visit
• Alkylating agents within 8 weeks before the screening visit or anticipated use during the time of the proposed study
• Vaccinations known to decrease platelet counts within 8 weeks before the screening visit
• Currently enrolled in another investigational device or drug study, or less than 30 days since ending investigational study
• Will have investigational procedures while enrolled on study
• Female subject of child bearing potential (defined as having first menses) not willing to use, in combination with her partner highly effective methods of birth control during treatment and for 1 month after the end of treatment
• Subject is pregnant or breast feeding, or might become pregnant within 1 month after the end of treatment
• Subject has known hypersensitivity to any recombinant Escherichia coli derived product (eg, Infergen®, Neupogen®, somatropin, and Actimmune®)
• Has previously enrolled into this study
• Will not be available for protocol-required study visits or procedures, to the best of the subject's and investigator's knowledge
• Any kind of disorder that, may compromise the subject to give written informed consent and/or to comply with all required study procedures

• Minimum Eligible Age: 1 Year
• Maximum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Amgen

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

MD

Role: STUDY_DIRECTOR

Amgen

Locations

Research Site

Roseville, California, United States

Research Site

Atlanta, Georgia, United States

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Chicago, Illinois, United States

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Peoria, Illinois, United States

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Indianapolis, Indiana, United States

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Iowa City, Iowa, United States

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Kansas City, Missouri, United States

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Las Vegas, Nevada, United States

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New York, New York, United States

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Cincinnati, Ohio, United States

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Columbus, Ohio, United States

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Pittsburgh, Pennsylvania, United States

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Nashville, Tennessee, United States

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Dallas, Texas, United States

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Fort Worth, Texas, United States

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Houston, Texas, United States

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Randwick, New South Wales, Australia

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South Brisbane, Queensland, Australia

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Parkville, Victoria, Australia

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Brussels, , Belgium

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Brussels, , Belgium

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Ghent, , Belgium

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Leuven, , Belgium

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Liège, , Belgium

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Belém, Pará, Brazil

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Jaú, São Paulo, Brazil

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São Paulo, São Paulo, Brazil

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São Paulo, São Paulo, Brazil

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Hamilton, Ontario, Canada

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Toronto, Ontario, Canada

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Montreal, Quebec, Canada

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Montreal, Quebec, Canada

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Olomouc, , Czechia

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Ostrava-Poruba, , Czechia

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Prague, , Czechia

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Montpellier, , France

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Nice, , France

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Paris, , France

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Vandœuvre-lès-Nancy, , France

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Budapest, , Hungary

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Debrecen, , Hungary

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Szeged, , Hungary

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Beersheba, , Israel

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Haifa, , Israel

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Jerusalem, , Israel

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Petach Tikvah, , Israel

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Tel Aviv, , Israel

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Tel Litwinsky, , Israel

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Monterrey, Nuevo León, Mexico

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Bydgoszcz, , Poland

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Lodz, , Poland

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Olsztyn, , Poland

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Zabrze, , Poland

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Krasnodar, , Russia

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Moscow, , Russia

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Moscow, , Russia

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Saint Petersburg, , Russia

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Saratov, , Russia

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Volgograd, , Russia

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Johannesburg, Gauteng, South Africa

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Parktown, Gauteng, South Africa

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Durban, KwaZulu-Natal, South Africa

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Tygerberg, Western Cape, South Africa

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Barcelona, Catalonia, Spain

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Esplugues de Llobregat, Catalonia, Spain

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Valencia, Valencia, Spain

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Madrid, , Spain

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Madrid, , Spain

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Basel, , Switzerland

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Sankt Gallen, , Switzerland

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Zurich, , Switzerland

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Adana, , Turkey (Türkiye)

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Antalya, , Turkey (Türkiye)

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Izmir, , Turkey (Türkiye)

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Birmingham, , United Kingdom

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Edinburgh, , United Kingdom

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London, , United Kingdom

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Manchester, , United Kingdom

Countries

United States; Australia; Belgium; Brazil; Canada; Czechia; France; Hungary; Israel; Mexico; Poland; Russia; South Africa; Spain; Switzerland; Turkey (Türkiye); United Kingdom

References

Tarantino MD, Despotovic J, Roy J, Grainger J, Cooper N, Beam D, Raj A, Maschan A, Kim J, Eisen M. Romiplostim treatment for children with immune thrombocytopenia: Results of an integrated database of five clinical trials. Pediatr Blood Cancer. 2020 Nov;67(11):e28630. doi: 10.1002/pbc.28630. Epub 2020 Sep 9.

Reference Type: BACKGROUND

PMID: 32902132 (View on PubMed)

Grainger J, Bussel J, Tarantino M, Cooper N, Beam D, Despotovic J, Maschan A, Wang K, Eisen M, Bowers C. A single-arm, long-term efficacy and safety study of subcutaneous romiplostim in children with immune thrombocytopenia. Blood Adv. 2023 Feb 14;7(3):396-405. doi: 10.1182/bloodadvances.2021006014.

Reference Type: BACKGROUND

PMID: 35413092 (View on PubMed)

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Related Links

http://www.amgentrials.com

AmgenTrials clinical trials website

Other Identifiers

2011-005019-96

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

20101221

Identifier Type: -

Identifier Source: org_study_id