An Open Label Study Evaluating the Safety and Efficacy of Long-Term Dosing of Romiplostim in Thrombocytopenic Pediatric Subjects with immune (Idiopathic) Thrombocytopenia Purpura (ITP).------------------------------------------------ Estudio Abierto para Evaluar la Seguridad y Eficacia del Tratamiento a Largo Plazo con Romiplostim en Sujetos Pediátricos Trombocitopénicos con Púrpura Trombocitopénica Inmune (Idiopática) (PTI).

• Register: EUCTR
• Last refreshed on: 6 February 2017
• Main ID: EUCTR2009-016203-32-ES
• Date of registration: 20/10/2009
• Prospective Registration: Yes
• Primary sponsor: Amgen Inc
• Public title: An Open Label Study Evaluating the Safety and Efficacy of Long-Term Dosing of Romiplostim in Thrombocytopenic Pediatric Subjects with immune (Idiopathic) Thrombocytopenia Purpura (ITP).------------------------------------------------ Estudio Abierto para Evaluar la Seguridad y Eficacia del Tratamiento a Largo Plazo con Romiplostim en Sujetos Pediátricos Trombocitopénicos con Púrpura Trombocitopénica Inmune (Idiopática) (PTI).
• Scientific title: An Open Label Study Evaluating the Safety and Efficacy of Long-Term Dosing of Romiplostim in Thrombocytopenic Pediatric Subjects with immune (Idiopathic) Thrombocytopenia Purpura (ITP).------------------------------------------------ Estudio Abierto para Evaluar la Seguridad y Eficacia del Tratamiento a Largo Plazo con Romiplostim en Sujetos Pediátricos Trombocitopénicos con Púrpura Trombocitopénica Inmune (Idiopática) (PTI).
• Date of first enrolment: 19/11/2009
• Target sample size: 20
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2009-016203-32
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: no Open: yes Single blind: no Double blind: no Parallel group: no Cross over: no Other: yes Other trial design description: NA If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no

Countries of recruitment

Spain

Key inclusion & exclusion criteria

Inclusion criteria:
Ethical
- Subject or subject's legally acceptable representative has provided informed consent.
Demographic
- Subject is less than 18 years old at time of screening.
Disease-Related
- Subject completed a romiplostim study for the treatment of thrombocytopenia in pediatric subjects with ITP.
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
Disease Related
- Subject has or previously had any 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).
- Subject has any new active malignancy diagnosed since enrollment in the previous romiplostim ITP study.
Medications
- Subject received any alkylating agents within four weeks before the screening visit or anticipated use during the time of the proposed study.
General
- Other investigational medications are excluded.
- Currently enrolled in another investigational device or drug study, or less than 30 days since ending another investigational device or drug study(s), or receiving other investigational agent(s).
- Subject (male or female) is not willing to use highly effective contraception during treatment if and when sexually active and for 4 weeks (women) or 14 weeks (men) after the end of treatment.
- Subject is pregnant or breast feeding, or planning to become pregnant within 4 weeks after the end of treatment.
- Male subject with a pregnant partner who is not willing to use a condom if and when sexually active during treatment and for 14 weeks after the end of treatment.
- Subject has known sensitivity to any of the products to be administered during dosing.
- Subject previously has entered this study (this will depend on the type of study).
- Subject will not be available for protocol-required study visits, to the best of the subject and investigator?s knowledge.
- Subject has any kind of disorder that, in the opinion of the investigator, may compromise the ability of the subject to give written informed consent and/or to comply with all required study procedures.


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Thrombocytopenia in pediatric subjects with ITP---------Sujetos Pediátricos Trombocitopénicos con Púrpura Trombocitopénica Inmune (Idiopática) (PTI)
MedDRA version: 12.0 Level: LLT Classification code 10043558 Term: Thrombocytopenia purpura

Intervention(s)

Product Name: NPLATE
Pharmaceutical Form: Powder for solution for injection
INN or Proposed INN: ROMIPLOSTIM
Other descriptive name: ROMIPLOSTIM
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 0.5-

Primary Outcome(s)

Main Objective: The primary objective of this study is to evaluate the safety of romiplostim as a long-term treatment in pediatric thrombocytopenic subjects with immune (idiopathic) thrombocytopenic purpura (ITP).

Secondary Objective: This study will also evaluate the long-term platelet response to romiplostim as well as the possible reductions in the dose of concurrent ITP therapies while receiving romiplostim.

Primary end point(s): The primary endpoints are the subject incidence and exposure adjusted incidence of adverse events, including clinically significant changes in laboratory values and incidence of antibody formation.

Secondary ID(s)

20090340