Clinical Trials Register

Summary
EudraCT Number:	2011-006322-25
Sponsor's Protocol Code Number:	SPD602-301(FBS0701-CTP-15)
National Competent Authority:	Italy - Italian Medicines Agency
Clinical Trial Type:	EEA CTA
Trial Status:	Prematurely Ended
Date on which this record was first entered in the EudraCT database:	2012-09-03
Trial results	View 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

Expand All Collapse All

A. Protocol Information

A.1

Member State Concerned

Italy - Italian Medicines Agency

A.2

EudraCT number

2011-006322-25

A.3

Full title of the trial

A PHASE 3, OPEN-LABEL, MILTICENTRE, EXTENSION SAFETY AND TOLERABILITY STUDY FOR TRANSFUSIONALLY IRON OVERLOADED CHILDRE, ADOLISCENTS AND ADULTS USING FBS0701 (SSP-004184)

STUDIO CLINICO MULTICENTRICO, DI FASE 3, OPEN LABEL, DI ESTENSIONE PER VALUTARE LA SICUREZZA E LA TOLLERABILITA' DI FBS0701 (SSP-004184)IN BAMBINI, ADOLESCENTI ED ADULTI CON SOVRACCARICO DI FERRO TRASFUSIONE-DIPENDENTE.

A.3.1

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

Extension to the treatment of iron overload with chelation therapy

Estensione al trattamento del sovraccarico di ferro con terapia chelante

A.3.2

Name or abbreviated title of the trial where available

SPD602-301 (FBS0701 - CTP - 15)

A.4.1

Sponsor's protocol code number

SPD602-301(FBS0701-CTP-15)

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	FERROKIN BIOSCIENCES INC.
B.1.3.4	Country	United States
B.3.1 and B.3.2	Status of the sponsor	Commercial
B.4 Source(s) of Monetary or Material Support for the clinical trial:
B.4.1	Name of organisation providing support	Ferrokin Bioscience Inc (consociata Shire LLC)
B.4.2	Country	United States
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	FERROKIN BIOSCIENCE LTD
B.5.2	Functional name of contact point	CLINICAL RESEARCH DPT
B.5.3	Address:
B.5.3.1	Street Address	311, SHOREHAM STREET
B.5.3.2	Town/ city	SHEFFIELD
B.5.3.3	Post code	S2 4FA
B.5.3.4	Country	United Kingdom
B.5.4	Telephone number	+44 (0) 7780113652
B.5.5	Fax number	N/A
B.5.6	E-mail	amber.jones@ferrokin.com

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	No
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	Yes
D.2.5.1	Orphan drug designation number	EU/3/09/647
D.3 Description of the IMP
D.3.1	Product name	FBS0701 CAPSULE
D.3.2	Product code	FBS0701/SSP004184
D.3.4	Pharmaceutical form	Capsule, hard
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	iron chelating agents
D.3.9.1	CAS number	1173092-59-2
D.3.9.2	Current sponsor code	FBS0701/SSP004184
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	500
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	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
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	Yes
D.3.11.13.1	Other medicinal product type	a novel iron chelator, FBS0701 is a magnesium salt of a desferrithiocin analogue, (S)-3-(OH)-DADFT-P
D.IMP: 2
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	No
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	Yes
D.2.5.1	Orphan drug designation number	EU/3/09/647
D.3 Description of the IMP
D.3.1	Product name	FBS0701 CAPSULE
D.3.2	Product code	FBS0701/SSP004184
D.3.4	Pharmaceutical form	Capsule, hard
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	IRON CHELATING AGENTS
D.3.9.1	CAS number	1173092-59-5
D.3.9.2	Current sponsor code	FBS0701/SSP004184
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	50
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	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
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	Yes
D.3.11.13.1	Other medicinal product type	a novel iron chelator FBS0701 is a magnesium salt of a desferrithiocin analogue, (S)-3-(OH)-DADFT-PE
D.IMP: 3
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	No
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	Yes
D.2.5.1	Orphan drug designation number	EU/3/09/647
D.3 Description of the IMP
D.3.1	Product name	FBS0701 CAPSULE
D.3.2	Product code	FBS0701/SSP004184
D.3.4	Pharmaceutical form	Capsule, hard
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	IRON CHELATING AGENTS
D.3.9.1	CAS number	1173092-59-5
D.3.9.2	Current sponsor code	FBS0701/SSP004184
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	100
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	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
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	Yes
D.3.11.13.1	Other medicinal product type	A NOVEL IRON CHELATO, FBS0701 IS A MAGNESIUM SALT OF A DESFERRTHIOCIN ANALOGUE, (S)-3-(OH)-DADFT-PE
D.IMP: 4
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	No
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	Yes
D.2.5.1	Orphan drug designation number	EU/3/09/647
D.3 Description of the IMP
D.3.1	Product name	FBS0701 CAPSULE
D.3.2	Product code	FBS0701/SSP004184
D.3.4	Pharmaceutical form	Capsule, hard
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	IRON CHELATING AGENTS
D.3.9.1	CAS number	1173092-59-5
D.3.9.2	Current sponsor code	FBS0701/SSP004184
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	200
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	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
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	Yes
D.3.11.13.1	Other medicinal product type	A NOVEL IRON CHELATOR, FBS0701 IS A MAGNESIUM SALT OF A DESFERRITHIOCIN ANALOGUE,(S)-3-(OH)-DADFT-PE
D.IMP: 5
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	No
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	Yes
D.2.5.1	Orphan drug designation number	EU/3/09/647
D.3 Description of the IMP
D.3.1	Product name	FBS0701 CAPSULE
D.3.2	Product code	FBS0701
D.3.4	Pharmaceutical form	Capsule, hard
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	IRON CHELATING AGENTS
D.3.9.1	CAS number	1173092-59-5
D.3.9.2	Current sponsor code	FBS0701/SSP004184
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	375
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	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
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	Yes
D.3.11.13.1	Other medicinal product type	A NOVEL IRON CHELATOR, FBS0701 IS A MAGNESIUM SALT OF DESFERRITHION ANALOGUE,(S)-3-(OH)-DADFT-PE
D.IMP: 6
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	No
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	Yes
D.2.5.1	Orphan drug designation number	EU/3/09/647
D.3 Description of the IMP
D.3.1	Product name	FBS0701 CAPSULE
D.3.2	Product code	FBS0701/SSP004184
D.3.4	Pharmaceutical form	Capsule, hard
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	IRON CHELATING AGENTS
D.3.9.1	CAS number	1173092-59-5
D.3.9.2	Current sponsor code	FBS0701/SSP004184
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	250
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	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
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	Yes
D.3.11.13.1	Other medicinal product type	A NOVEL IRON CHELATOR, FBS0701 IS A MAGNESIUM SALT OF DESFERRITHIOCIN ANALOGUE,(S)-3-(OH)-DADFT-PE

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

ADULT PATIENTS SUFFERING FROM IRON OVERLOAD OF TRANSFUSION-DEPENDENT DOCUMENTED, IN WHICH THE FOLLOWING PRIMARY DIAGNOSIS: ANEMIA HEREDITARY (EG sickle cell anemia), thalassemia and Diamond-Blackfan anemia

PAZIENTI ADULTI AFFETTI DA SOVRACCARICO DI FERRO TRASFUSIONE-DIPENDENTE DOCUMENTATO, CHE PRESENTANO LE SEGUENTI DIAGNOSI PRIMARIE: ANEMIA EREDITARIA (AD ESEMPIO ANEMIA FALCIFORME), TALASSEMIA E ANEMIA DI DIAMOND BLACKFAN

E.1.1.1

Medical condition in easily understood language

ADULT PATIENTS SUFFERING FROM IRON OVERLOAD OF TRANSFUSION DEPENDENT

PAZIENTI ADULTI AFFETTI DA SOVRACCARICO DI FERRO TRASFUSIONE DIPENDENTE

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	15.0
E.1.2	Level	LLT
E.1.2	Classification code	10065974
E.1.2	Term	Chronic iron overload
E.1.2	System Organ Class	10027433 - Metabolism and nutrition disorders

E.1.3

Condition being studied is a rare disease

Yes

E.2 Objective of the trial

E.2.1

Main objective of the trial

MEASURE THE EXTENT AND DURABILITY OF FBS0701/SSP004184 TREATMENT EFFECTS, CONTINUE TO ASSESS THE SAFETY,TOLERABILITY AND EFFICACY OF FBS0701/SSP004184 IN A PRIOR FERROKIN SPONSORED STUDY, ALLOW ACCESS AND ASSESS RESPONSE TO FBS0701/SSP004184 IN SUBJECTS RANDOMIZED TO ANOTHER CHELATING THERAPY IN A PRIOR FERROKIN-SPONMSORED FBS0701/SSP004184 STUDY.

VALUTARE IL GRADO E LA DURATA DEGLI EFFETTI TERAPEUTICI DI FBS0701/SSP-004184; CONTINUARE A VALUTARE LA SICUREZZA, LA TOLLERABILITA' E L'EFFICACIA DI FBS0701/SSP004184 IN SOGGETTI CUI è STATO SOMMINISTRATO FBS0701/SSP004184 IN UN PRECEDENTE STUDIO DI CUI ERA PROMOTORE FERROKIN;CONSENTIRE L'ACCESSO E VALUTARE LA RISPOSTA ALL'FBS0701/SSP004184 IN SOGGETTI RANDOMIZZATI SOTTOPOSTI AD UN'ALTRA TERAPIA CHELANTE IN UN PRECEDENTE STUDIO SU FBS0701/SSP004184 DI CUI ERA PROMOTORE FERROKIN.

E.2.2

Secondary objectives of the trial

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1.SUBJECTS AND/OR PARENTS WILLING AND ABLE TO SIGN THE APPROVED INFORMED CONSENT AND/OR ASSENT (BASED ON ISTITUTIONAL GUIDELINES) 2,AN UNDERSTANDING, ABILITY, AND WILLINGNESS TO FULLY COMPLY WITH STUDY PROCEDURES AND RESTRICTION 3.HAS COMPLETED ONE OF THE FBS/SSP004184 FEEDER PROTOCOLS 4.MALE,OR NON PREGNANT, NON LACTATING FEMALE WHO FEMALE WHO AGREES TO COMPLY WITH ANY APPLICABLE CONTRACEPTIVE REQUIREMENTS OF THE PROTOCOL OR FEMALES OF NON -CHILDBEARING POTENTIAL. SUBJECT, WHO IS FEMALE OF CHILD-BEARING POTENTIAL (FOCP), MUST HAVE A NEGATIVE URINE PREGNANCY TEST AT THE QUALIFICATION AND ENROLLMENT VISIT PRIOR TO DOSING ON DAY1 AND AGREE TO COMPLY WITH ANY APPLICABLE CONTRACEPTIVE REQUIREMENTS OF THE PROTOCOL.

1.SOGGETTI E/O GENITORI DISPOSTI E IN GRADO DI FIRMARE IL MODULO DI CONSENSO INFORMATO APPROVATO E/O L'ASSENSO (IN BASE ALLE LINEE GUIDA ISTITUZIONALI) 2.COMPRENSIONE, CAPACITA' E VOLONTA' DI ATTENERSI PIENAMENTE ALLE PROCEDURE ED AI VINCOLI PREVISTI DALLO STUDIO 3.SOGGETTO CHE ABBIA COMPLETATO UNO DEI PROTOCOLLI ORIGINARI FBS0701/SSP00418 4.UOMO O DONNA NON IN GRAVIDANZA O IN ALLATTAMENTO CHE ACCETTI DI ATTENERSI A TUTTE LE PRATICHE CONTRACETTIVE PREVISTE DAL PROTOCOLLO O DONNE IN ETA' FERTILE.IL SOGGETTO, SE DONNA IN ETA' POTENZIALMENTE FERTILE, DOVRA' RISULTARE NEGATIVA ALL'ESAME DELLE URINE DEL TEST DI GRAVIDANZA NELLA VISITA DI QUALIFICAZIONE E ARRUOLAMENTO PRIMA DI ASSUMERE IL DOSAGGIO NEL GIORNO 1 ED ACCETTARE DI ATTENERSI A TUTTE LE PRATICHE CONTRACETTIVE PREVISTE DAL PROTOCOLLO.

E.4

Principal exclusion criteria

1.Unwilling to remain off all existing chelation therapies during FBS0701 (SSP-004184) dosing and for 1 week after last dose. 2.A history of non-compliance in a prior FerroKin-sponsored FBS0701 (SSP-004184) study (excluding dose suspensions that were medically warranted). 3.Cardiac MRI T2* <8 milliseconds at the time of last MRI prior to study entry. 4.Has restarted prior chelation therapy. (NOTE: Should there be an administrative need to interrupt study treatment, the subject may be allowed to remain off all chelator therapy for a maximum of 3 weeks prior to starting the extension study, should the Medical Monitor and the subject’s Principal Investigator deem it safe and clinically reasonable to do so.) 5.Evidence of clinically relevant oral, cardiovascular, gastrointestinal, endocrine, pulmonary, neurologic, psychiatric, immunologic, bone marrow, dermatologic, hepatic or renal dysfunction where, in the opinion of the PI or the Medical Monitor, treatment with FBS0701 (SSP-004184) is relatively contraindicated. 6.Platelet count below 100,000/µL or absolute neutrophil count less than 1500/mm3 in the last assessment prior to study entry. 7.Known sensitivity to any ingredient in the FBS0701 (SSP-004184) formulation. 8.Pregnant or lactating females.

1.Il soggetto non vuole essere escluso da tutte le terapie chelanti durante l’assunzione di FBS0701 (SSP-004184) e per 1 settimana dopo l’ultima somministrazione. 2.Anamnesi di non-compliance in un precedente studio con FBS0701 (SSP-004184) di cui era promotore FerroKin (escludendo le interruzioni della somministrazione clinicamente giustificate). 3.T2* della MRI cardiaca <8 millisecondi al momento dell’ultima MRI prima dell’ingresso nello studio. 4.Soggetto che abbia ripreso una precedente terapia chelante. (NOTA: Nel caso vi dovesse essere un’esigenza amministrativa di interrompere la terapia in studio, al soggetto sarebbe permesso di rimanere senza alcuna terapia chelante per un massimo di 3 settimane prima di iniziare il protocollo di estensione dello studio, nel caso che il Medical Monitor e lo Sperimentatore Principale del soggetto lo ritenessero sicuro e clinicamente ragionevole.) 5.Evidenza di una disfunzione clinicamente rilevante orale, cardiovascolare, gastrointestinale, endocrina, polmonare, neurologica, psichiatrica, immunologica, midollare, dermatologica, epatica o renale in cui, in base all’opinione dello Sperimentatore Principale o del Medical Monitor, la terapia con FBS0701 (SSP-004184) sia relativamente controindicata. 6.Conta piastrinica inferiore a 100,000/µL o conta assoluta dei neutrofili inferiore a 1500/mm3 nell’ultimo controllo antecedente all’entrata nello studio.

E.5 End points

E.5.1

Primary end point(s)

SEE THE PRIMARY OBJECTIVES

VEDI OBIETTIVI PRINCIPALI

E.5.1.1

Timepoint(s) of evaluation of this end point

5 YEARS

5 ANNI

E.5.2

Secondary end point(s)

NAN

E.5.2.1

Timepoint(s) of evaluation of this end point

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

E.8.1.2

Open

Yes

E.8.1.3

Single blind

E.8.1.4

Double blind

E.8.1.5

Parallel group

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)

E.8.2.2

Placebo

E.8.2.3

Other

Yes

E.8.2.3.1

Comparator description

STESSO FARMACO ALLO STESSO DOSAGGIO

SAME DRUG AT SAME DOSAGE

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

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

United States

E.8.7

Trial has a data monitoring committee

Yes

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

database lock

chiusura database

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

Yes

F.1.1

Number of subjects for this age range:

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)

Yes

F.1.1.5.1

Number of subjects for this age range:

F.1.1.6

Adolescents (12-17 years)

Yes

F.1.1.6.1

Number of subjects for this age range:

F.1.2

Adults (18-64 years)

Yes

F.1.2.1

Number of subjects for this age range:

F.1.3

Elderly (>=65 years)

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

F.4.2.2

In the whole clinical trial

1500

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)

After the last dose of FBS0701 (SSP-004184) subjects 28 day safety follow up period,at the end of which time subjects will undergo final study assesments. Patients will be follow according to normal clinical practice.

dopo l'ultima dose di FBS0701(SSP004184)i soggetti entreranno in un periodo di follow up di sicurezza di 28 giorni, alla conclusione del quale i soggetti saranno sottoposti ai controlli finali dello studio. In seguito verranno sottoposti ai normali controlli previsti dalla pratica clinica.

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

2012-08-20

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2012-07-23

P. End of Trial
P.	End of Trial Status	Prematurely Ended

Select Country:	Select Age Range:
Select Trial Status:	Select Trial Phase:
Select Gender:
Select Date Range: to
Select Rare Disease:
IMP with orphan designation in the indication
Orphan Designation Number:
Results Status:
Clear advanced search filters

Austria
Belgium
Bulgaria
Croatia
Cyprus
Czechia
Denmark
Estonia
Finland
France
Germany
Greece
Hungary
Iceland
Ireland
Italy
Latvia
Liechtenstein
Lithuania
Luxembourg
Malta
Netherlands
Norway
Poland
Portugal
Romania
Slovakia
Slovenia
Spain
Sweden
United Kingdom
Outside EU/EEA

Clinical trials