Idiopathic Thrombocytopenic PurpuraPhase 1/2NCT00515203
Safety and Efficacy Study of Romiplostim (AMG 531) to Treat ITP in Pediatric Subjects
A Phase 1/2 study testing AMG 531 in people with Idiopathic Thrombocytopenic Purpura and Thrombocytopenia in Pediatric Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP), run by Amgen.
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). Read more about this study
- Who is it for?
- Ages 1 year to 17 years
- Conditions the study is for
- Idiopathic Thrombocytopenic Purpura, Thrombocytopenia in Pediatric Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP), Thrombocytopenia in Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP)
- What is being tested?
- AMG 531
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 22 participants
- Where
- No sites listed yet
- Study dates
- Jul 2007to Aug 2009
- Phase
- Phase 1/2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 year to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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
Who cannot join
- 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
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- 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
About this study
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
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.
- Conditions
- Idiopathic Thrombocytopenic Purpura, Thrombocytopenia in Pediatric Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP), Thrombocytopenia in Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP)
- Being tested
- Placebo, AMG 531
- Keywords
- Immune (Idiopathic) Thrombocytopenic Purpura, Pediatric Idiopathic Thrombocytopenic Purpura
- Medical subject headings
- Purpura, Thrombocytopenic, Idiopathic, Purpura, Thrombocytopenic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PlaceboDrug
Starting dose of 1.0 ug/kg. Dose adjustments are made throughout the study based on individual platelet counts.
AMG 531Drug
Also called romiplostim
Starting dose of 1.0 ug/kg. Dose adjustments are made throughout the study based on individual platelet counts.
| Study group | Role | What they receive |
|---|---|---|
| II. | Placebo comparator | 5 thrombocytopenic (as defined per protocol) subjects Receives: Drug: Placebo |
| I. | Experimental | 15 thrombocytopenic (as defined per protocol) subjects Receives: Drug: AMG 531 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse Events
Occurrence of one or more adverse events in the participant during the 12-week treatment period
Measured: 12 weeks
Secondary outcomes (5)
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.
Measured: 12-week treatment period
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)
Measured: 12-week treatment period (Weeks 2 - 13)
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.
Measured: 12-week treatment period
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.
Measured: 12-week treatment period
Requirement for Rescue Therapy (as Defined Per Protocol)
Participant required rescue therapy (as defined per protocol) during the 12 week treatment period.
Measured: 12-week treatment period
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Aug 2007 (estimated)
- Study startJul 2007
- Primary completionMar 2009
- Study completionAug 2009
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Idiopathic Thrombocytopenic PurpuraStudies of the same condition, open and under way.
- More trials run by AmgenThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Investigator, Outcomes assessor
- Enrolment
- 22 participants (actual)
Sponsor and investigators
- Lead sponsor
- Amgen
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- MD, Study director, Amgen
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00515203
- Sponsor's protocol ID
- 20060195
- Status last verified
- Jul 2014
- First submitted
- 9 Aug 2007
- Last updated
- 25 Jul 2014
- Results posted
- Yes, on ClinicalTrials.gov
Publications
Bussel JB, Buchanan GR, Nugent DJ, Gnarra DJ, Bomgaars LR, Blanchette VS, Wang YM, Nie K, Jun S. A randomized, double-blind study of romiplostim to determine its safety and efficacy in children with immune thrombocytopenia. Blood. 2011 Jul 7;118(1):28-36. doi: 10.1182/blood-2010-10-313908. Epub 2011 Apr 18.
PubMed 21502541Klaassen 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.
PubMed 21910213