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PER-057-14

A SINGLE ARM, OPEN-LABEL, LONG-TERM EFFICACY AND SAFETY STUDY OF ROMIPLOSTIM IN THROMBOCYTOPENIC PEDIATRIC SUBJECTS WITH IMMUNE THROMBOCYTOPENIA (ITP)

Base di dati: WHO (Importata da 29.03.2024)
Cambiato: 8 set 2023, 01:00
Categoria di malattie:

Health conditions (Fonte di dati: WHO)

-D693 Idiopathic thrombocytopenic purpura-D694 Other primary thrombocytopenia-D695 Secondary thrombocytopenia-D696 Thrombocytopenia, unspecified
Idiopathic thrombocytopenic purpura
Other primary thrombocytopenia
Secondary thrombocytopenia
Thrombocytopenia, unspecified;D693;D694;D695;D696;Idiopathic thrombocytopenic purpura;Other primary thrombocytopenia;Secondary thrombocytopenia;Thrombocytopenia, unspecified

Interventions (Fonte di dati: WHO)

Romiplostim is supplied in a 5 mL single-use vial as a sterile, white, preservative-free, lyophilized powder containing a protein concentration of 0.5 mg/mL of 10 mM histidine, 4.0% mannitol, 2.0% sucrose, 0.004% polysorbate 20, and a pH 5.0 when reconstituted with 1.2 mL of sterile water for injection. IP will be administered as a subcutaneous injection. The starting dose of romiplostim will be 1 μg/kg based on the subject?s recorded screening weight. Initially, IP will be administered in the clinic by a qualified health care provider and subjects will return to the clinic weekly to provide blood samples for platelet counts and undergo any dose titrations under the supervision of the treating physician. Subjects who receive their first 8 doses in the clinic and achieve a stable dose of romiplostim for at least 4 weeks may be allowed to self-inject romiplostim or have the injection administered by a caregiver. Weekly dose increases will continue in increments of 1 μg/kg up to a maximum dose of 10 μg/kg in an attempt to reach a target platelet count of ≥50 x 109/L. Dose adjustments will be allowed to maintain a platelet count between ≥ 50 x 109/L and ≤ 200 x 109/L. Dose adjustments will be evaluated every 12 weeks due to potential body weight changes.

Inclusion/Exclusion Criteria (Fonte di dati: WHO)

Gender: Both
Maximum age: 17
Minimum age: 1
Inclusion criteria:
Inclusion Criteria
4.1.1 Diagnosis of primary ITP according to The American Society of Hematology (ASH) Guidelines (Neunert et al, 2011) at least 6 months before screening, regardless of splenectomy status
4.1.2 Age ≥ 1 year and < 18 years at the time of providing informed consent
4.1.3 Subject must be refractory to a prior ITP therapy, having relapsed after at least 1 prior ITP therapy, or be ineligible for other ITP therapies Examples of prior therapy include but are not limited to: corticosteroids, IVIG, anti-D immunoglobulin, and platelet transfusions. Subjects who have failed a splenectomy are eligible for study participation
4.1.4 Subject has a documented platelet count ≤ 30 x109/L or is experiencing bleeding that is uncontrolled with conventional therapies
4.1.5 Subject?s legally acceptable representative (or subject, if applicable) has provided informed consent before any study-specific procedure; and subject has provided assent, where required by the IRB/IEC

Exclusion criteria:
Exclusion Criteria
4.2.1 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)
4.2.2 Prior bone marrow transplant or peripheral blood progenitor cell transplant
4.2.3 Known active or prior malignancy except non-melanoma skin cancers within the last 5 years
4.2.4 Known history of myelodysplastic syndrome
4.2.5 Known history of bleeding diathesis
4.2.6 Known history of congenital thrombocytopenia
4.2.7 Known history of hepatitis B, hepatitis C or human immunodeficiency virus
4.2.8 Known history of systemic lupus erythematosus, Evans syndrome, or autoimmune neutropenia
4.2.9 Known history of antiphospholipid antibody syndrome or known positive for lupus anticoagulant
4.2.10 Known history of disseminated intravascular coagulation, hemolytic uremic syndrome, or thrombotic thrombocytopenic purpura
4.2.11 History of venous thromboembolism or thrombotic events
4.2.12 Previous use of romiplostim or eltrombopag
4.2.13 Previous use of PEG-rHuMGDF, recombinant human thrombopoietin (rHuTPO) or any other platelet producing agent
4.2.14 Rituximab (for any indication) or 6-mercaptopurine within 8 weeks of enrollment, or anticipated use at any time during the study
4.2.15 Splenectomy within 4 weeks of the screening visit
4.2.16 Alkylating agents within 8 weeks before the screening visit or anticipated use during the time of the proposed study
4.2.17 Vaccinations known to decrease platelet counts within 8 weeks before the screening visit
4.2.18 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)
4.2.19 Subject will have investigational procedures performed while enrolled in
this clinical study
4.2.20 Female subject of child bearing potential (defined as having first menses) is 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
4.2.21 Subject is pregnant or breast feeding, or might become pregnant within 1 month after the end of treatment
4.2.22 Subject has known hypersensitivity to any recombinant Escherichia coli derived product (eg, Infergen, Neupogen, somatropin, and Actimmune?)
4.2.23 Subject has previously enrolled into this study
4.2.24 Subject will not be available for protocol-required study visits or procedures, to the best of the subject?s and investigator?s knowledge
4.2.25 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

Altri dati sulla sperimentazione nel registro primario dell’OMS

https://www.ins.gob.pe/ensayosclinicos/rpec/recuperarECPBNuevoEN.asp?numec=057-14

Altri dati sulla sperimentazione dalla banca dati dell’OMS (ICTRP)

https://trialsearch.who.int/Trial2.aspx?TrialID=PER-057-14
Altre informazioni sulla sperimentazione

Data di registrazione della sperimentazione

6 gen 2015

Inserimento del primo partecipante

16 gen 2015

Stato di reclutamento

Pending

Titolo scientifico (Fonte di dati: WHO)

A SINGLE ARM, OPEN-LABEL, LONG-TERM EFFICACY AND SAFETY STUDY OF ROMIPLOSTIM IN THROMBOCYTOPENIC PEDIATRIC SUBJECTS WITH IMMUNE THROMBOCYTOPENIA (ITP)

Tipo di sperimentazione (Fonte di dati: WHO)

Interventional

Disegno della sperimentazione (Fonte di dati: WHO)

This is a phase 3b single arm, open label, multicenter study describing the percentage of time
pediatric subjects with ITP have a platelet response while receiving romiplostim, defined as a
platelet count ≥ 50 x 109/L and in the absence of ITP rescue medications in the past 4 weeks.
This protocol will provide open label romiplostim to thrombocytopenic pediatric subjects with ITP
diagnosed for at least 6 months and who have received at least 1 prior ITP therapy (excluding
romiplostim) or are ineligible for ITP therapies.
The study design consists of a 4-week screening period, up to a 3-year treatment period, an end
of treatment (EOT) visit, and an end of study (EOS) visit.

Fase (Fonte di dati: WHO)

III

Contatto per informazioni (Fonte di dati: WHO)

AMGEN Inc

Risultati della sperimentazione (Fonte di dati: WHO)

Sintesi dei risultati

ancora nessuna informazione disponibile

Collegamento ai risultati nel registro primario

ancora nessuna informazione disponibile

Informazioni sulla disponibilità dei dati dei singoli partecipanti

ancora nessuna informazione disponibile

Siti di esecuzione della sperimentazione

Paesi di esecuzione (Fonte di dati: WHO)

Belgium, Brazil, Bulgaria, Czech Republic, Denmark, France, Germany, Greece, Hungary, Ireland, Israel, Italy, Lithuania, Netherlands, Peru, Poland, Russian Federation, Slovakia, South Africa, Spain, Switzerland, Turkey, United Kindgdom

Contatto per maggiori informazioni sulla sperimentazione

Contatto per informazioni generali (Fonte di dati: WHO)

Gabriela Celina
Loyola
AV. REPUBLICA DE PANAMA Nro. 3533, Int. 1404, Limatambo, San Isidro
IQVIA RDS Peru S.R.L
379 0122
gabriela.loyola@quintiles.com

Contatto per informazioni scientifiche (Fonte di dati: WHO)

Gabriela Celina
Loyola
AV. REPUBLICA DE PANAMA Nro. 3533, Int. 1404, Limatambo, San Isidro
IQVIA RDS Peru S.R.L
379 0122
gabriela.loyola@quintiles.com
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