Safety and Efficacy Study of Romiplostim (AMG 531) to Treat ITP in Pediatric Subjects

Idiopathic Thrombocytopenic Purpura Clinical Trial

Official title:

A Randomized, Double-Blind, Placebo-controlled Phase 1/2 Study to Determine the Safety and Efficacy of Romiplostim (AMG 531) in Thrombocytopenic Pediatric Subjects With Chronic Immune (Idiopathic) Thrombocytopenic Purpura

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00515203
• Other study ID #: 20060195
• Status: Completed
• Phase: Phase 1/Phase 2
• First received: August 9, 2007
• Last updated: July 18, 2014
• Start date: July 2007
• Est. completion date: August 2009

Study information

• Verified date: July 2014
• Source: Amgen
• Contact: n/a
• Is FDA regulated: No
• Health authority: Australia: Therapeutic Goods AdministrationCanada: Health CanadaFrance: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)Spain: Agencia Española de Medicamentos y Productos SanitariosUnited States: Food and Drug AdministrationAustralia: Department of Health and Ageing Therapeutic Goods AdministrationAustralia: Human Research Ethics CommitteeUnited States: Institutional Review Board
• Study type: Interventional

Clinical Trial Summary

The purpose of this study is to evaluate the safety and tolerability of romiplostim (AMG 531) in the treatment of thrombocytopenia in pediatric subjects with chronic ITP. We will also evaluate the efficacy of romiplostim (AMG 531) and characterize the pharmacokinetics of romiplostim (AMG 531). It is anticipated that romiplostim (AMG 531), when given at an effective dose and schedule, will be well tolerated treatment for thrombocytopenia among pediatric subjects with chronic ITP.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 22
• Est. completion date: August 2009
• Est. primary completion date: March 2009
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 1 Year to 17 Years

Eligibility

Inclusion Criteria:

• Before any study-specific procedure, the appropriate written informed consent must be obtained. In addition to the written informed consent, the assent of the child from those subjects capable of providing assent must also be obtained if requested by the IRB/IEC.

• Diagnosis of ITP according to The American Society of Hematology (ASH) Guidelines at least six months prior to screening

• Age = 12 months and < 18 years at enrollment

• The mean of two platelet counts taken during the screening period must be = 30 x 10^9/L with no single count >35 x 10^9/L

• A serum creatinine concentration = 1.5 times the laboratory normal range (for each age category)

• Adequate liver function; serum bilirubin = 1.5 times the laboratory normal range

• Hemoglobin >10.0 g/dL

Exclusion Criteria:

• Known 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 in the study)

• Known history of venous or arterial thrombotic or thromboembolic event

• Known history of congenital thrombocytopenia

• Known history of malignancy except basal cell carcinoma

• Known history of hepatitis B, hepatitis C, or HIV

• Known history of systemic lupus erythematosus, Evans Syndrome, or autoimmune neutropenia

• Known positive lupus anticoagulant or history of antiphospholipid antibody syndrome

• Known history of Disseminated Intravascular Coagulation, Hemolytic Uremic Syndrome, or Thrombotic Thrombocytopenic Purpura

• Currently receiving any treatment for ITP except for corticosteroids

• IV Ig or anti-D Ig within two weeks prior to the screening visit

• Rituximab (for any indication) within 14 weeks before the screening visit or anticipated use during the time of the proposed study

• Splenectomy within eight weeks of the screening visit

• Received hematopoietic growth factors including IL-11 (oprelvekin) within four weeks before the screening visit

• Received any alkylating agents within eight weeks before the screening visit or anticipated use during the time of the proposed study

• Subject is currently enrolled in or has not yet completed at least four weeks since ending other investigational device or drug trial(s), or subject is receiving investigational agent(s)

• Past or present participation in any study evaluating PEG-rHuMGDF, recombinant human thrombopoietin (rHuTPO), AMG 531, or related platelet product

• Pregnant (i.e. positive urine pregnancy test) or breast feeding

• Subject is not using adequate contraceptive precautions, if applicable.

• Known hypersensitivity to any recombinant E coli-derived product

• Subject has any kind of disorder that compromises the ability to comply with all study procedures

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Investigator, Outcomes Assessor), Primary Purpose: Treatment

Related Conditions & MeSH terms

• Idiopathic Thrombocytopenic Purpura
• Purpura
• Purpura, Thrombocytopenic
• Purpura, Thrombocytopenic, Idiopathic
• Thrombocytopenia
• Thrombocytopenia in Pediatric Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP)
• Thrombocytopenia in Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP)
• Thrombocytosis

Intervention

Drug:
• Placebo
Starting dose of 1.0 ug/kg. Dose adjustments are made throughout the study based on individual platelet counts.
• AMG 531
Starting dose of 1.0 ug/kg. Dose adjustments are made throughout the study based on individual platelet counts.

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Amgen

References & Publications (2)

Bussel J, Buchanan G, Nugent D, Gnarra D, Blanchette V, Nie K, Jun S. A randomized, double-blind, placebo-controlled phase 1/2 study to determine the safety and efficacy of romiplostim in children with chronic Immune (Idiopathic) Thrombocytopenic Purpura (ITP). Blood;2011;118:28-36

Klaassen RJ, Mathias SD, Buchanan G, Bussel J, Deuson R, Young NL, Collier A, Bomgaars L, Blanchette V. Pilot study of the effect of romiplostim on child health-related quality of life (HRQoL) and parental burden in immune thrombocytopenia (ITP). Pediatr Blood Cancer. 2012 Mar;58(3):395-8. doi: 10.1002/pbc.23312. Epub 2011 Sep 9. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Adverse Events	Occurrence of one or more adverse events in the participant during the 12-week treatment period	12 weeks	Yes
Secondary	Weeks With Platelet Count = 50 x 10^9/L	The number of weeks with platelet count = 50 x 10^9/L during the 12 week treatment period.	12-week treatment period	No
Secondary	Bleeding Events (Grade 2 or Higher)	Total number of bleeding events (Grade 2 or higher, i.e., mild to life-threatening, as defined in the protocol) for each participant during Weeks 2-13 (end-of-study visit for non-responders)	12-week treatment period (Weeks 2 - 13)	No
Secondary	Platelet Count = 50 x 10^9/L for Two Consecutive Weeks	Participant incidence of achieving a platelet count =50 x 10^9/L for two consecutive weeks during the 12 week treatment period.	12-week treatment period	No
Secondary	Increase in Platelet Count = 20 x 10^9/L Above Baseline for Two Consecutive Weeks	Participant incidence of achieving an increase in platelet count =20 x 10^9/L above baseline for two consecutive weeks during the 12 week treatment period.	12-week treatment period	No
Secondary	Requirement for Rescue Therapy (as Defined Per Protocol)	Participant required rescue therapy (as defined per protocol) during the 12 week treatment period.	12-week treatment period	No

External Links

•   AmgenTrials clinical trials website