Clinical Trials Register

Summary
EudraCT Number:	2021-006970-22
Sponsor's Protocol Code Number:	2021-006970-22
National Competent Authority:	Italy - Italian Medicines Agency
Clinical Trial Type:	EEA CTA
Trial Status:	Ongoing
Date on which this record was first entered in the EudraCT database:	2023-02-08
Trial results

Index
A. PROTOCOL INFORMATION
B. SPONSOR INFORMATION
C. APPLICANT IDENTIFICATION
D. IMP IDENTIFICATION
D.8 INFORMATION ON PLACEBO
E. GENERAL INFORMATION ON THE TRIAL
F. POPULATION OF TRIAL SUBJECTS
G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
P. END OF TRIAL

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A. Protocol Information

A.1

Member State Concerned

Italy - Italian Medicines Agency

A.2

EudraCT number

2021-006970-22

A.3

Full title of the trial

A multicentre, randomized, open-label study of romiplostim plus dexamethasone vs dexamethasone in patients with newly diagnosed primary immune thrombocytopenia

Studio multicentrico, randomizzato ed in aperto di romiplostim più desametasone verso desametasone in pazienti con trombocitopenia immune primaria di nuova diagnosi

A.3.1

Title of the trial for lay people, in easily understood, i.e. non-technical, language

Clinical study in order to compare the effectiveness and safety of two different treatments in patients with newly diagnosed primary immune thrombocytopenia

Studio clinico per confrontare l'efficacia e la sicurezza di due diversi trattamenti in pazienti con trombocitopenia immune primaria di nuova diagnosi

A.3.2

Name or abbreviated title of the trial where available

RODEX

A.4.1

Sponsor's protocol code number

2021-006970-22

A.7

Trial is part of a Paediatric Investigation Plan

A.8

EMA Decision number of Paediatric Investigation Plan

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	FISEVI - Fundación Pública Andaluza para la Gestión de la Investigación en Salud de Sevilla
B.1.3.4	Country	Spain
B.3.1 and B.3.2	Status of the sponsor	Non-Commercial
B.4 Source(s) of Monetary or Material Support for the clinical trial:
B.4.1	Name of organisation providing support	AMGEN S.A.
B.4.2	Country	Spain
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Unidad de Investigación Clínica y apoyo a Ensayos Clínicos
B.5.2	Functional name of contact point	UICEC
B.5.3	Address:
B.5.3.1	Street Address	Avenida Manuel Siurot
B.5.3.2	Town/ city	Siviglia
B.5.3.3	Post code	41013
B.5.3.4	Country	Spain
B.5.4	Telephone number	+34955013414
B.5.5	Fax number	+34955095338
B.5.6	E-mail	claram.rosso.sspa@juntadeandalucia.es

D. IMP Identification
D.IMP: 1
D.1.2 and D.1.3	IMP Role	Test
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	Yes
D.2.1.1.1	Trade name	Romiplostim
D.2.1.1.2	Name of the Marketing Authorisation holder	Amgen Europe B.V
D.2.1.2	Country which granted the Marketing Authorisation	Spain
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	Romiplostim
D.3.2	Product code	[Romiplostim]
D.3.4	Pharmaceutical form	Powder for injection
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Subcutaneous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	ROMIPLOSTIM
D.3.9.1	CAS number	267639-76-9
D.3.9.2	Current sponsor code	IMP-1
D.3.9.4	EV Substance Code	SUB27756
D.3.10	Strength
D.3.10.1	Concentration unit	µg microgram(s)
D.3.10.2	Concentration type	range
D.3.10.3	Concentration number	250 to 500
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	No
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	Yes
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	Information not present in EudraCT
D.3.11.3.2	Gene therapy medical product	Information not present in EudraCT
D.3.11.3.3	Tissue Engineered Product	Information not present in EudraCT
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	Information not present in EudraCT
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	Information not present in EudraCT
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	No
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No
D.IMP: 2
D.1.2 and D.1.3	IMP Role	Comparator
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	Yes
D.2.1.1.1	Trade name	Desametasone 40mg
D.2.1.1.2	Name of the Marketing Authorisation holder	TAD Pharma GmbH
D.2.1.2	Country which granted the Marketing Authorisation	Spain
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	Dexamethasone
D.3.2	Product code	[IMP-2]
D.3.4	Pharmaceutical form	Tablet
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Oral use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	DESAMETASONE
D.3.9.1	CAS number	50-02-2
D.3.9.2	Current sponsor code	84.416
D.3.9.4	EV Substance Code	SUB07017MIG
D.3.10	Strength
D.3.10.1	Concentration unit	mg milligram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	40
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	Yes
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	No
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	Information not present in EudraCT
D.3.11.3.2	Gene therapy medical product	Information not present in EudraCT
D.3.11.3.3	Tissue Engineered Product	Information not present in EudraCT
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	Information not present in EudraCT
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	Information not present in EudraCT
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	No
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No

D.8 Information on Placebo

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Primary immune thrombocytopenia

Trombocitopenia immune primaria

E.1.1.1

Medical condition in easily understood language

Primary immune thrombocytopenia is an autoimmune blood disease characterized by the presence of low platelet levels.

La trombocitopenia immune primaria è una malattia del sangue autoimmune che comporta bassi livelli di piastrine.

E.1.1.2

Therapeutic area

Diseases [C] - Blood and lymphatic diseases [C15]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	23.0
E.1.2	Level	PT
E.1.2	Classification code	10083842
E.1.2	Term	Immune thrombocytopenia
E.1.2	System Organ Class	10005329 - Blood and lymphatic system disorders

E.1.3

Condition being studied is a rare disease

E.2 Objective of the trial

E.2.1

Main objective of the trial

To evaluate the superiority of romiplostim plus dexamethasone vs dexamethasone alone after 6 months (>/=180 days) from treatment cessation in patients with newly primary immune thrombocytopenia (ITP) in terms of sustained response off any ITP treatment (6mSROT-50) and without WHO grade 2 or more bleeding.

Valutare la superiorità di romiplostim più desametasone verso solo desametasone a 6 mesi (>/=180 giorni) dopo l'interruzione del trattamento in pazienti con trombocitopenia immune primaria (TIP) di nuova diagnosi in termini di risposta sostenuta senza trattamenti per la TIP (6mSROT-50) e senza sanguinamenti di grado WHO 2 o superiore.

E.2.2

Secondary objectives of the trial

To evaluate the superiority of experimental arm after 6 and 12 months from treatment cessation in terms of sustained response off any ITP treatment(6mSROT-30,12mSROT-30 and 12mSROT-50)and without WHO grade 2 or more bleeding
To evaluate the superiority of experimental arm in terms of complete response,response,global response and response within the target range
To compare the time to first response, the proportion of patients with early response and initial response
To compare the duration of platelet response, the time to loss of response in patients who achieved response, the proportion of patients requiring any rescue treatment along the study period
To evaluate and compare proportion of treatment failure and time to treatment failure
To evaluate the safety and tolerability
To compare the difference between study arms in the mean change in patients’ bleeding and the change in patients’ quality of life
To describe and compare healthcare resources use and loss of productivity

Valutare superiorità del braccio sperimentale dopo 6 e 12 mesi dall'interruzione del trattamento in termini di risposta sostenuta da qualsiasi trattamento per la TIP (6mSROT-30, 12mSROT-30 e-50) e senza sanguinamenti di grado WHO >/=2
Valutare la superiorità del braccio sperimentale in termini di risposta completa, risposta, risposta globale e risposta nell’intervallo di riferimento
Confrontare il tempo alla prima risposta, la proporzione di pazienti con risposta precoce e risposta iniziale
Confrontare la durata della risposta piastrinica, tempo alla perdita di risposta nei pazienti che hanno risposto, proporzione di pazienti che hanno necessitato un trattamento di soccorso durante lo studio
Valutare e confrontare la % di fallimento del trattamento e tempo al fallimento
Valutare sicurezza e tollerabilità
Confrontare differenza tra i bracci dello studio nel cambiamento medio di sanguinamento e qualità di vita
Descrivere e confrontare l'uso di risorse sanitarie e perdita di produttività

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1.Age = 18 years of age at the time of signing informed consent.
2.Newly diagnosis of primary ITP according to the International Working Group assessment [1] and previously untreated for ITP.
3.Platelet counts <30x10^9/L or ITP with platelet counts <50x10^9/L and concomitant bleeding symptoms.
4.Serum creatinine concentration </= 1.5 mg/dL.

1. Età = 18 anni al momento di firma del consenso informato.
2. Nuova diagnosi di TIP in base alla valutazione del “International Working Group” e senza precedente trattamento per la TIP.
3. Conta piastrinica <30x10^9/L o TIP con <50x10^9/L e sintomi emorragici concomitanti.
4. Concentrazione di creatinina sierica </= 1,5 mg/dl.

E.4

Principal exclusion criteria

1. WHO performance status >2.
2. Previous therapy with rituximab (within 3 months previous of study enrollment), corticosteroids, therapy with other immunomodulating agents within 1 month of enrolment, hematopoietic analogs and fostamatinib for any other reason despite ITP.
3. Previous use of romiplostim, PEG-recombinant human (rHu) megakaryocyte growth and development factor, eltrombopag, recombinant human anti-thrombopoietin (rHuTPO), or any platelet-producing agent.
4. Alkylating agents within 8 weeks before the screening visit or anticipated use during the time of the proposed study.
5. Splenectomy within 3 months of the screening visit or planned splenectomy during study period.
6. Abnormal renal function (serum creatinine > 1.5 mg/dL).
7. Active hepatic disease (evidenced by alanine aminotransferase [ALT] or aspartate aminotransferase [AST] levels >5 times the upper limit of normal (it will only be necessary to determine one of the two transaminases).
8. Severe chronic liver disease as evidenced by, but not limited to, any of the following: International Normalized Ratio (INR) > 1.4, hypoalbuminemia, portal vein hypertension including presence of otherwise unexplained splenomegaly and history of esophageal varices.
9.Pregnancy or lactation.
10.Patients with known IgM seropositive tests for cytomegalovirus and/or Epstein-Barr virus in the previous month.
11.Patients with known serum-positivity and a positive test for an active viral infection at screening with: Hepatitis B Virus (HBV), Hepatitis C Virus (HCV), detectable virus charge of HIV.
12.Intolerance to dexamethasone.
13.History of a bone marrow stem cell disorder.
14.Active or prior malignancy except adequately treated (ie, complete surgical excision with negative margins) basal cell carcinoma.
15.History of Heliobacter pylori by urea breath test or stool antigen test within 6 months of enrollment, if available.
16.History of myelodysplastic syndrome, systemic lupus erythematosus, or autoimmune cytopenia.
17.History of antiphospholipid antibody syndrome.
18.History of disseminated intravascular coagulation, hemolytic uremic syndrome, or thrombotic thrombocytopenic purpura.
19.History of deep or superficial venous thromboembolism in the last 12 months or stroke, acute ischaemic heart disease or acute peripheral vascular disese in the last 6 months.
20.Hypersensitivity to any recombinant Escherichia coli-derived product (eg, Infergen, Neupogen, Somatropin, and Actimmune) or known sensitivity to any of the products to be administered during dosing
21.Currently enrolled in another investigational device or drug study or < 30 days since ending another investigational device or drug studies, or receiving other investigational agents.
22.Will have any other investigational procedures performed while enrolled in this clinical study.
23.Pregnant or breastfeeding, or planning to become pregnant or breastfeed during treatment or within 1 month after the end of treatment.
24.Female subject of childbearing potential is not willing to use, in combination with her partner, an acceptable method of effective contraception during treatment and for 1 month after the end of treatment (see annex 5 for additional contraception information). Females of childbearing potential should only be included after a negative, highly sensitive urine pregnancy test.
25.Will not be available for protocol-required study visits, to the best of the subject’s and investigator’s knowledge.
26.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.
27.Other serious comorbidities at investigator criteria.

1. Performance status WHO > 2.
2. Terapia precedente con rituximab (nei 3 mesi precedenti l’arruolamento nello studio), corticosteroidi, terapia con altri agenti immunomodulatori nel mese precedente all’arruolamento, analoghi ematopoietici e fostamatinib per qualsiasi motivo oltre alla TIP.
3. Uso precedente di romiplostim, fattore di crescita e sviluppo di megacariociti umani PEG-ricombinanti (rHu), eltrombopag, antitrombopoietina umana ricombinante (rHuTPO) o qualsiasi agente che produce piastrine.
4. Agenti alchilanti nelle 8 settimane precedenti alla visita di screening o uso anticipato durante il periodo dello studio proposto.
5. Splenectomia nei 3 mesi precedenti alla visita di screening o splenectomia pianificata durante il periodo dello studio.
6. Funzionalità renale anormale (creatinina sierica > 1,5 mg/dL).
7. Malattia epatica attiva: livelli di alanina aminotransferasi [ALT] o aspartato aminotransferasi [AST] > 5 volte il limite superiore di normalità (è necessario determinare una sola delle due transaminasi).
8. Malattia epatica cronica grave evidenziata da, tra l’altro, qualsiasi dei seguenti: Rapporto Internazionale Normalizzato (INR) > 1,4, ipoalbuminemia, ipertensione della vena porta, inclusa la presenza di splenomegalia senza causa apparente e storia di varici esofagee.
9. Gravidanza o allattamento.
10. Pazienti con nota sieropositività ai test IgM per citomegalovirus e/o virus di Epstein-Barr nel mese precedente.
11. Pazienti con nota sieropositività e test positivo per un’infezione virale attiva allo screening di: virus dell’epatite B (HBV), virus dell’epatite C (HCV), carica virale rilevabile del virus dell’immunodeficienza umana (HIV).
12. Intolleranza al desametasone.
13. Storia di disordini delle cellule staminali del midollo osseo.
14. Neoplasia maligna attiva o precedente, eccetto il carcinoma basocellulare adeguatamente trattato (ovvero escissione chirurgica completa con margini negativi).
15. Storia di Helicobacter pylori al test del respiro dell’urea o test antigenico delle feci, nei 6 mesi precedenti l’arruolamento, se disponibile.
16. Storia di sindrome mielodisplastica, lupus eritematoso sistemico, o citopenia autoimmune.
17. Storia di sindrome da anticorpi anti-fosfolipidi.
18. Storia di coagulazione intravascolare disseminata, sindrome uremica emolitica, porpora trombotica trombocitopenica.
19. Storia di tromboembolismo venoso profondo o superficiale negli ultimi 12 mesi o ictus, cardiopatia ischemica acuta o malattia vascolare periferica acuta negli ultimi 6 mesi.
20. Ipersensibilità a qualsiasi prodotto ricombinante Escherichia coli-derivato (per esempio Infergen, Neupogen, Somatropina e Actimmune) o nota sensibilità a qualsiasi dei prodotti da somministrare durante il trattamento.
21. Paziente attualmente arruolato in un altro studio con dispositivo medico o farmaco o paziente che ha terminato altri studi su dispositivo medico o farmaco o altri agenti sperimentali negli ultimi 30 giorni.
22. Paziente che verrà sottoposto a qualsiasi altra procedura sperimentale mentre è arruolato in questo studio clinico.
23. Gravidanza o allattamento o intenzione a iniziare una gravidanza o allattamento durante il trattamento o nel mese successivo alla fine del trattamento.
24. Soggetto di sesso femminile in età fertile che non è disposto ad utilizzare, insieme al suo partner, un accettabile metodo contraccettivo efficace durante il trattamento e per 1 mese dopo la fine del trattamento (vedi Appendice 5 per ulteriori informazioni sulla contraccezione). Donne in età fertile dovrebbero essere incluse nello studio solo dopo un test di gravidanza su urine ad alta sensibilità negativo.
25. Pazienti che non sono in grado di eseguire le visite di studio richieste dal protocollo secondo il giudizio del soggetto e dello sperimentatore.
26. Qualsiasi tipo di disordine che, nell’opinione dello sperimentatore, può compromettere la capacità del soggetto di fornire il consenso informato scritto e/o di ottemperare alle procedure di studio richieste.
27. Altre comorbidità serie secondo il giudizio dello sperimentatore.

E.5 End points

E.5.1

Primary end point(s)

To evaluate the difference between study arms in the proportion of patients achieving 6mSROT-50 at 6 months from treatment cessation.

Valutare la differenza tra i due bracci di studio in termini di proporzione di pazienti che raggiungo l'endpoint 6mSROT-50 a 6 mesi dopo l'interruzione del trattamento (6mSROT-50 = conta piastrinica >/= a 50x10^9/L in assenza di trattamenti per la TIP incluso qualsiasi trattamenti di soccorso per almeno 6 mesi consecutivi (>/= 180 giorni) dopo l'interruzione del trattamento e senza sanguinamenti di grado WHO >/= 2).

E.5.1.1

Timepoint(s) of evaluation of this end point

At 6 months from treatment cessation.

A 6 mesi dopo la cessazione del trattamento.

E.5.2

Secondary end point(s)

To evaluate the superiority of romiplostim plus dexamethasone vs dexamethasone alone in terms of complete response (CR), response (R), global response (GR) and response within the target range (TR) at 6 months (Day 180), at 12 months (Day 365) from randomisation and End of study Visit (visit 12 months after the last dose of study treatment). We will evaluate them in the who trial cohort but also in a subgroup that ecludes patients receiving any rescue treatment. Or an additional sensitivity analysis will exclude patients receiving rescue treatment.; To compare the time to first response defined as the time from randomization to first response (R) in both arms. We will evaluate this in the total sample and in the absence of any rescue treatment.
To compare the proportion of patients with early response (ER) and initial response (IR) in both arms.
To compare the duration of platelet response assessed as: the maximum number of consecutive days with platelet response (CR, R, GR and TR) and, the total number of days along the study period with platelet response (CR, R, GR and TR) in both arms. We will evaluate them in the total sample and in the absence of any rescue treatment.
To compare the time to loss of response (LoR) in patients who achieved response in both arms.
To compare the proportion of patients requiring any rescue treatment along the study period in both arms
To evaluate and compare proportion of treatment failure and time to treatment failure.
To evaluate the safety and tolerability of study treatments in both arms.
To compare the difference between study arms in the mean change in patients’ bleeding.
To describe and compare healthcare resources use (HRU) and loss of productivity in both study arms.; To compare the change in patients’ quality of life from baseline Day 1 (the day of administration of the first dose of study medication) to 2 (Week 8), Month 6 (Day 180), Month 12 (Day 365) and End of study Visit (visit 12 months after the last dose of study treatment) assessed with SF-36 v2, FACIT-F and ITP-PAQ in both arms.; To evaluate the superiority of romiplostim plus dexamethasone vs dexamethasone alone after 6 months (=180 days) from treatment cessation in patients with newly primary immune thrombocytopenia in terms of sustained response off any ITP treatment (6mSROT-30) from treatment cessation and without WHO grade 2 or more bleeding.; To evaluate the superiority of romiplostim plus dexamethasone vs dexamethasone alone after 12 months (=365 days) from treatment cessation, if data available, in patients with newly primary immune
thrombocytopenia in terms of sustained response off any ITP treatment (12mSROT-30 and 12mSROT-50) from treatment cessation and without WHO grade 2 or more bleeding.

Valutare la superiorità di romiplostim più desametasone verso solo desametasone in termini di risposta completa (CR), risposta (R), risposta globale (GR) e risposta nell’intervallo di riferimento (TR) a 6 mesi (Giorno 180), a 12 mesi (giorno 365) dalla randomizzazione e alla Visita di fine studio (visita a 12 mesi dopo l’ultima dose di trattamento). Saranno valutati nella coorte WHO dello studio, ma anche in un sottogruppo che esclude i pazienti che ricevono qualsiasi trattamento di soccorso. O un’analisi aggiuntiva di sensibilità escluderà i pazienti che ricevono qualsiasi trattamento di soccorso.; Confrontare in entrambe le braccia il tempo alla prima risposta definito come il tempo dalla randomizzazione fino alla prima risposta (R). Verrà valutato nel campione totale e nel campione che non ha assunto trattamenti di soccorso.
Confrontare la proporzione di pazienti con risposta precoce (RP) e risposta iniziale (RI) in entrambe le braccia.
Confrontare la durata della risposta piastrinica valutata come: il numero massimo di giorni consecutivi con risposta piastrinica (CR, R, GR e TR) e, il numero totale di giorni durante il periodo dello studio con risposta piastrinica (CR, R, GR e TR) in entrambe le braccia. Verranno valutati nel campione totale e in assenza di qualsiasi trattamento di soccorso.
Confrontare il tempo alla perdita della risposta (LoR) in pazienti che hanno ottenuto una risposta in entrambe le braccia.
Confrontare la proporzione di pazienti che hanno necessitato di un trattamento di soccorso durante il periodo dello studio in entrambe le braccia.
Valutare e confrontare la proporzione di fallimento terapeutico ed il tempo al fallimento terapeutico.
Valutare la sicurezza e la tollerabilità dei trattamenti di studio in entrambe le braccia.
Confrontare la differenza media di sanguinamenti nei pazienti in entrambe le braccia.
Descrivere e confrontare l’utilizzo delle risorse sanitarie (HRU) e la perdita di produttività in entrambe le braccia.; Confrontare il cambio nella qualità della vita dei pazienti dal basale Giorno 1 (giorno della somministrazione della prima dose del farmaco di studio) al Mese 2 (settimana 8), al Mese 6 (Day 180) e al Mese 12 (Giorno 365) ed alla visita di fine studio (Visita a 12 mesi dopo l’ultima dose del trattamento di studio), tramite i questionari SF-36 v2, FACIT-F e ITP-PAQ, in entrambe le braccia.; Valutare la superiorità di romiplostim più desametasone verso solo desametasone 6 mesi (=180 giorni) dopo l’interruzione del trattamento in pazienti con trombocitopenia immune primaria di nuova diagnosi in termini di risposta sostenuta senza trattamenti per la TIP (6mSROT-30) e senza emorragie di grado WHO 2 o superiore.; Valutare la superiorità di romiplostim più desametasone verso solo desametasone 12 mesi (=365 giorni) dopo l’interruzione del trattamento (se ci sono dati disponibili) in pazienti con trombocitopenia immune primaria di nuova diagnosi in termini di risposta sostenuta senza trattamenti per la TIP (12mSROT-30 e 12mSROT-50) e senza sanguinamenti di grado WHO 2 o superiore.

E.5.2.1

Timepoint(s) of evaluation of this end point

At 6 months (Day 180), at 12 months (Day 365) from randomisation and End of study Visit (visit 12 months after the last dose of study treatment).; Tthroughout the entire study.; At Month 2 (Week 8), Month 6 (Day 180), Month 12 (Day 365) and End of study Visit (visit 12 months after the last dose of study treatment).; At 6 months from treatment cessation.; At 12 months from treatment cessation.

A 6 mesi (Giorno 180) e a 12 mesi (giorno 365) dalla randomizzazione e alla Visita di fine studio (visita a 12 mesi dopo l’ultima dose di trattamento).; Durante il corso di studio; Al Mese 2 (settimana 8), al Mese 6 (Day 180) e al Mese 12 (Giorno 365) ed alla visita di fine studio (Visita a 12 mesi dopo l’ultima dose del trattamento di studio).; A 6 mesi dopo la cessazione del trattamento.; A 12 mesi dopo la cessazione del trattamento.

E.6 and E.7 Scope of the trial

E.6

Scope of the trial

E.6.1

Diagnosis

E.6.2

Prophylaxis

E.6.3

Therapy

Yes

E.6.4

Safety

Yes

E.6.5

Efficacy

Yes

E.6.6

Pharmacokinetic

E.6.7

Pharmacodynamic

E.6.8

Bioequivalence

E.6.9

Dose response

E.6.10

Pharmacogenetic

E.6.11

Pharmacogenomic

E.6.12

Pharmacoeconomic

E.6.13

Others

E.7

Trial type and phase

E.7.1

Human pharmacology (Phase I)

E.7.1.1

First administration to humans

E.7.1.2

Bioequivalence study

E.7.1.3

Other

E.7.1.3.1

Other trial type description

E.7.2

Therapeutic exploratory (Phase II)

E.7.3

Therapeutic confirmatory (Phase III)

Yes

E.7.4

Therapeutic use (Phase IV)

E.8 Design of the trial

E.8.1

Controlled

Yes

E.8.1.1

Randomised

Yes

E.8.1.2

Open

Yes

E.8.1.3

Single blind

E.8.1.4

Double blind

E.8.1.5

Parallel group

Yes

E.8.1.6

Cross over

E.8.1.7

Other

E.8.2

Comparator of controlled trial

E.8.2.1

Other medicinal product(s)

Yes

E.8.2.2

Placebo

E.8.2.3

Other

E.8.2.4

Number of treatment arms in the trial

E.8.3

The trial involves single site in the Member State concerned

E.8.4

The trial involves multiple sites in the Member State concerned

Yes

E.8.4.1

Number of sites anticipated in Member State concerned

E.8.5

The trial involves multiple Member States

Yes

E.8.5.1

Number of sites anticipated in the EEA

E.8.6 Trial involving sites outside the EEA

E.8.6.1

Trial being conducted both within and outside the EEA

Yes

E.8.6.2

Trial being conducted completely outside of the EEA

Information not present in EudraCT

E.8.6.3

If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned

Spain

Italy

United Kingdom

E.8.7

Trial has a data monitoring committee

E.8.8

Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial

LVLS

E.8.9 Initial estimate of the duration of the trial

E.8.9.1

In the Member State concerned years

E.8.9.1

In the Member State concerned months

E.8.9.1

In the Member State concerned days

E.8.9.2

In all countries concerned by the trial years

E.8.9.2

In all countries concerned by the trial months

E.8.9.2

In all countries concerned by the trial days

F. Population of Trial Subjects

F.1 Age Range

F.1.1

Trial has subjects under 18

F.1.1.1

In Utero

F.1.1.2

Preterm newborn infants (up to gestational age < 37 weeks)

F.1.1.3

Newborns (0-27 days)

F.1.1.4

Infants and toddlers (28 days-23 months)

F.1.1.5

Children (2-11years)

F.1.1.6

Adolescents (12-17 years)

F.1.2

Adults (18-64 years)

Yes

F.1.2.1

Number of subjects for this age range:

100

F.1.3

Elderly (>=65 years)

Yes

F.1.3.1

Number of subjects for this age range:

F.2 Gender

F.2.1

Female

Yes

F.2.2

Male

Yes

F.3 Group of trial subjects

F.3.1

Healthy volunteers

F.3.2

Patients

Yes

F.3.3

Specific vulnerable populations

Yes

F.3.3.1

Women of childbearing potential not using contraception

F.3.3.2

Women of child-bearing potential using contraception

Yes

F.3.3.3

Pregnant women

F.3.3.4

Nursing women

F.3.3.5

Emergency situation

F.3.3.6

Subjects incapable of giving consent personally

F.3.3.7

Others

F.4 Planned number of subjects to be included

F.4.1

In the member state

F.4.2

For a multinational trial

F.4.2.1

In the EEA

100

F.4.2.2

In the whole clinical trial

126

F.5

Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)

None

Nessuno

G. Investigator Networks to be involved in the Trial

N. Review by the Competent Authority or Ethics Committee in the country concerned

Competent Authority Decision

Authorised

Date of Competent Authority Decision

2023-05-29

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2023-05-24

P. End of Trial
P.	End of Trial Status	Ongoing

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