Clinical Trials Register

Summary
EudraCT Number:	2013-005454-30
Sponsor's Protocol Code Number:	C-935788-049
National Competent Authority:	Italy - Italian Medicines Agency
Clinical Trial Type:	EEA CTA
Trial Status:	Completed
Date on which this record was first entered in the EudraCT database:	2014-05-14
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

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A. Protocol Information

A.1

Member State Concerned

Italy - Italian Medicines Agency

A.2

EudraCT number

2013-005454-30

A.3

Full title of the trial

A Phase 3 Open Label Extension Study of Fostamatinib Disodium in the Treatment of Persistent/Chronic Immune Thrombocytopenic Purpura

A.3.1

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

A Study of Fostamatinib Disodium in the Treatment of Thrombocytopenic Purpura

A.4.1

Sponsor's protocol code number

C-935788-049

A.5.2

US NCT (ClinicalTrials.gov registry) number

NCT02077192

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	Rigel Pharmaceuticals, 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	Rigel Pharmaceuticals, Inc.
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	Rigel Pharmaceuticals, Inc.
B.5.2	Functional name of contact point	Ann M. Lowe, MD
B.5.3	Address:
B.5.3.1	Street Address	1180 Veterans Boulevard
B.5.3.2	Town/ city	South San Francisco
B.5.3.3	Post code	CA 94080
B.5.3.4	Country	United States
B.5.4	Telephone number	1 415 309 7672
B.5.5	Fax number	1 650 624 1282
B.5.6	E-mail	alowe@rigel.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	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	fostamatinib disodium tablets
D.3.2	Product code	R935788
D.3.4	Pharmaceutical form	Film-coated 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	Fostamatinib disodium
D.3.9.1	CAS number	914295-16-2
D.3.9.2	Current sponsor code	R935788 disodium hexahydrate
D.3.9.3	Other descriptive name	FOSTAMATINIB DISODIUM
D.3.9.4	EV Substance Code	SUB32625
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	No
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	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	fostamatinib disodium tablets
D.3.2	Product code	R935788
D.3.4	Pharmaceutical form	Film-coated 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	fostamatinib disodium
D.3.9.1	CAS number	914295-16-2
D.3.9.2	Current sponsor code	R935788 disodium hexahydrate
D.3.9.3	Other descriptive name	FOSTAMATINIB DISODIUM
D.3.9.4	EV Substance Code	SUB32625
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	150
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	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

Chronic Immune Thrombocytopenic Purpura

E.1.1.1

Medical condition in easily understood language

Thrombocytopenic Purpura

E.1.1.2

Therapeutic area

Diseases [C] - Immune System Diseases [C20]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	17.1
E.1.2	Level	LLT
E.1.2	Classification code	10067644
E.1.2	Term	Immune-mediated thrombocytopenic purpura
E.1.2	System Organ Class	100000004851

E.1.3

Condition being studied is a rare disease

E.2 Objective of the trial

E.2.1

Main objective of the trial

The primary objective of this study is to assess the long term safety of fostamatinib in subjects with persistent/chronic ITP

E.2.2

Secondary objectives of the trial

The secondary objectives of this study are to establish the long-term efficacy of fostamatinib in achieving and maintaining a stable platelet count in subjects who complete the treatment phase of Study C935788-047 or Study C935788-048, and to assess the pharmacokinetic (PK) profile of fostamatinib in subjects with persistent/chronic ITP.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Subject must be willing and able to give written informed consent by signing an IRB-approved Informed Consent Form prior to participating in any study-specific procedures.
2. Subjects must have completed the Week 24 evaluation of Study C-935788-047 or C-935788-048 or have discontinued early (starting at Week 12) due to lack of response. No more than 7 days may have elapsed between the last day of treatment on the Study C-935788-047 or C-935788-048 and initial dosing (Day 1) in Study C-935788-049.
3. Male or female at least 18 years of age.
4. Females must be either post-menopausal for at least 1 year or surgically sterile; or if female of child-bearing potential, must not be pregnant or lactating and must agree to use an acceptable method of birth control throughout the duration of the trial and for 30 days following the last dose. Acceptable methods of birth control are defined as: hormonal contraception (pill, injection or implant) used consistently for at least 30 days prior toenrollment, intrauterine device (IUD), double-barrier (ie, condom and spermicide, or condom and diaphragm), or complete abstinence.
5. In the Investigator’s opinion, the subject has the ability to understand the nature of the study and any hazards of participation and to communicate satisfactorily with the Investigator.

E.4

Principal exclusion criteria

1. Any subject who discontinued participation in Study C-935788-047 or C-935788-048 prior to Week 12 or for any reason other than lack of response. Exceptions may be considered on a case-by-case basis after consultation between the Investigator and Sponsor with the specific reason for the exception noted.
2. Subjects with poorly controlled hypertension during Study C-935788-047 or C-935788-048, defined as persistent or repeated systolic ≥ 140 mmHg, or diastolic ≥ 90 mmHg whether or not the subject is receiving anti-hypertensive treatment.
3. Subjects with the following laboratory abnormalities at the time of enrollment (Day 1): a leukocyte count < 2,000/μL, a neutrophil count of < 1,000/μL, lymphocyte count < 750/μL, Hgb < 10 g/dL, or transaminase levels (ALT, AST) > 1.5x ULN, bilirubin > 1.5x ULN, or estimated glomerular filtration rate (eGFR) < 30 mL/min.
4. Subjects who have a significant infection, an acute infection such as influenza, or known inflammatory process at the time of enrollment into this study.

E.5 End points

E.5.1

Primary end point(s)

Version 1: The first version of the primary efficacy endpoint is for the purpose of assessing efficacy among all patients while they are on active treatment in one of the prior studies, in the current extension study, or in both. This version of the primary efficacy endpoint is the achievement and maintenance of a stable platelet count defined as follows:
1) Achievement of a platelet count of at least 50,000/μL within 12 weeks of beginning active treatment.
2) Achievement of a sustained stable platelet response; defined as no two visits, at least 4 weeks apart, with a platelet count < 50,000/μL, without an intervening visit with a platelet count of ≥ 50,000/μL unrelated to rescue therapy, within a period of 12 months following initial achievement of the target platelet count (see above).
Version 2: The second version of the primary efficacy endpoint is for the purpose of a within-subject, between-study comparison of fostamatinib and placebo among subjects randomized to placebo in either of the prior studies, This version of the primary efficacy endpoint is the achievement and maintenance of a stable platelet count defined as follows:
1) Achievement of a platelet count of at least 50,000/μL within 12 weeks of beginning treatment (placebo treatment in the prior study and fostamatinib treatment in the present study) and
2) Achievement of a sustained stable platelet response; defined as no two visits, at least 4 weeks apart, with a platelet count < 50,000/ μL, without an intervening visit with a platelet count of ≥ 50,000/ μL unrelated to rescue therapy, within a period of 12 weeks following initial achievement of the target platelet count (see above).

E.5.1.1

Timepoint(s) of evaluation of this end point

- within 12 weeks of beginning active treatment
- within a period of 12 weeks following initial achievement of the target platelet count

E.5.2

Secondary end point(s)

• The duration of any stable platelet response.
• Proportion of subjects in whom a reduction in the dose of concomitant ITP therapy can be achieved while maintaining an adequate platelet count.

E.5.2.1

Timepoint(s) of evaluation of this end point

various study visits

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

E.6.4

Safety

Yes

E.6.5

Efficacy

Yes

E.6.6

Pharmacokinetic

Yes

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

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

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

Austria

Bulgaria

Canada

Czech Republic

Denmark

France

Germany

Hungary

Italy

Netherlands

Poland

Spain

United Kingdom

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

The study will end upon completion of all protocol procedures

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:

120

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

150

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

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

2015-04-09

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2015-10-06

P. End of Trial
P.	End of Trial Status	Completed

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