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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.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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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
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
Are there results?
Yes, posted in the registrySummary in the doctors' view

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
Show all 20Show fewer
  • 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.

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 groups in NCT00515203
Study groupRoleWhat they receive
II.Placebo comparator5 thrombocytopenic (as defined per protocol) subjects
Receives: Drug: Placebo
I.Experimental15 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.

  1. Adverse Events

    Occurrence of one or more adverse events in the participant during the 12-week treatment period

    Measured: 12 weeks

Secondary outcomes (5)

  1. 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

  2. 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)

  3. 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

  4. 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

  5. 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.

  1. First posted13 Aug 2007 (estimated)
  2. Study startJul 2007
  3. Primary completionMar 2009
  4. Study completionAug 2009

Resources

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)
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

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 21502541

  • 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.

    PubMed 21910213

Questions about this trial

What is this clinical trial studying?

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.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 7 inclusion and 20 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 25 Jul 2014. Status can change between updates, so confirm with the study contact before making plans.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 1 Mar 2011). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Adverse Events (time frame: 12 weeks). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00515203, which also lists 2 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Jul 2014.

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