Clinical Trials Register

Summary
EudraCT Number:	2020-004568-25
Sponsor's Protocol Code Number:	BLAST
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:	2021-08-17
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

2020-004568-25

A.3

Full title of the trial

Open-label, prospective, phase II descriptive pilot trial of belimumab therapy for refractory and/or non-criteria
manifestations of Antiphospholipid Syndrome.

Studio pilota in aperto, prospettico, di fase II della terapia con belimumab in pazienti con sindrome da antifosfolipidi refrattari e/o con manifestazioni non-criteria.

A.3.1

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

Belimumab in patients with Antiphospholipid Syndrome

Belimumab in pazienti con Sindrome da Anticorpi Antifosfolipidi

A.3.2

Name or abbreviated title of the trial where available

BLAST

A.4.1

Sponsor's protocol code number

BLAST

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	DIPARTIMENTO DI SCIENZE CLINICHE E BIOLOGICHE (UNITO)
B.1.3.4	Country	Italy
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	GSK
B.4.2	Country	Italy
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Università di Torino
B.5.2	Functional name of contact point	Dario Roccatello
B.5.3	Address:
B.5.3.1	Street Address	Piazza del Donatore di Sangue 3
B.5.3.2	Town/ city	Torino
B.5.3.3	Post code	10154
B.5.3.4	Country	Italy
B.5.6	E-mail	dario.roccatello@unito.it

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	Benlysta
D.2.1.1.2	Name of the Marketing Authorisation holder	GSK
D.2.1.2	Country which granted the Marketing Authorisation	Italy
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	Belimumab
D.3.2	Product code	[041381029]
D.3.4	Pharmaceutical form	Powder and solvent for concentrate for solution for infusion
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Enteral use (Noncurrent)
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.9.1	CAS number	356547-88-1
D.3.9.2	Current sponsor code	01
D.3.9.3	Other descriptive name	University of Turin
D.3.10	Strength
D.3.10.1	Concentration unit	1X 100 milligrams/millilitre
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	4
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.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

APS

E.1.1.1

Medical condition in easily understood language

Antiphospholipid Syndrome

Sindrome da Anticorpi Antifosfolipidi

E.1.1.2

Therapeutic area

Diseases [C] - Musculoskeletal Diseases [C05]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	20.0
E.1.2	Level	PT
E.1.2	Classification code	10002817
E.1.2	Term	Antiphospholipid syndrome
E.1.2	System Organ Class	10005329 - Blood and lymphatic system 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

to evaluate the safety and tolerability of belimumab treatment in patients with APS at 24 months of treatment

valutare la sicurezza e tollerabilità del trattamento con belimumab nei pazienti con APS a 24 mesi di trattamento

E.2.2

Secondary objectives of the trial

changes in antibodies titre

variazioni nel titolo anticorpale

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

Positive aPL profile
Clinical features attributable to aPL that are resistant to warfarin and/or heparin:
o Recurrent thrombosis despite ongoing anticoagulation and/or
o Persistent thrombocytopenia and/or
o Persistent autoimmune hemolytic anemia and/or
o Cardiac valve disease and/or
o Chronic skin ulcers and/or
o Renal thrombotic microangiopathy and/or
o Cognitive dysfunction with/without white matter changes

Profilo aPL positivo
Caratteristiche cliniche attribuibili ad aPL resistenti a warfarin e / o eparina:
o Trombosi ricorrente nonostante l'anticoagulazione in corso e / o
o Trombocitopenia persistente e / o
o Anemia emolitica autoimmune persistente e / o
o Malattia della valvola cardiaca e / o
o ulcere cutanee croniche e / o
o Microangiopatia trombotica renale e / o
o Disfunzione cognitiva con / senza alterazioni della sostanza bianca

E.4

Principal exclusion criteria

• >=4/11 American College of Rheumatology Classification Criteria for SLE
• Acute thrombosis (arterial or venous acute thrombosis diagnosis less than 30 days
before study screening)
• History of stroke Acute or chronic pancreatitis
• Pregnancy
• Have a history of malignant neoplasm within the last 5 years except basal cell or
squamous cell carcinoma of the skin treated with local resection only or carcinoma in
situ of the uterine cervix treated locally and with no evidence of metastatic disease
for 3 years
• Have evidence of serious suicide risk including any history of suicidal behaviour in the
last 6 months and/or any suicidal ideation in the last 2 months or who in the
investigator's judgment, poses a significant suicide risk
• Have a history of a primary immunodeficiency
• Have a significant IgG deficiency (IgG level < 400 mg/dL)
• Have an IgA deficiency (IgA level < 10 mg/dL)
• Known active bacterial, viral fungal mycobacterial, or other infection
• Infection history:
• Currently on any suppressive therapy for a chronic infection (such as tuberculosis,
pneumocystis, cytomegalovirus, herpes simplex virus, herpes zoster and atypical
mycobacteria)
• Hospitalization for treatment of infection within 60 days of Day 0.
• Use of parenteral (IV or IM) antibiotics (anti-bacterial, antiviral, anti-fungal, or antiparasitic
agents) within 60 days of Day 0
• Have current drug or alcohol abuse or dependence, or a history of drug or alcohol
abuse or dependence within 365 days prior to Day 0
• Have a historically positive HIV test or test positive at screening for HIV
• Hepatitis status:
• Serologic evidence of current or past Hepatitis B (HB) infection based on the
results of testing for HBsAg and HBcAb as follows:
• Patients positive for HBsAg or HBcAb are excluded
• Positive test for Hepatitis C antibody
• Have a history of an anaphylactic reaction to parenteral administration of contrast
agents, human or murine proteins or monoclonal antibodies
• Have any other clinically significant abnormal laboratory value in the opinion of the
investigator
• If Women of Child-Bearing Potential (WCBP) are included, please see special
instructions below.

•> = 4/11 Criteri di classificazione dell'American College of Rheumatology per il LES
• Trombosi acuta (diagnosi di trombosi acuta arteriosa o venosa inferiore a 30 giorni
prima dello screening dello studio)
• Storia di ictus Pancreatite acuta o cronica
• Gravidanza
• Avere una storia di neoplasia maligna negli ultimi 5 anni ad eccezione delle cellule basali o
carcinoma a cellule squamose della pelle trattata solo con resezione locale o carcinoma in
situ della cervice uterina trattata localmente e senza evidenza di malattia metastatica
per 3 anni
• Avere prove di un grave rischio di suicidio, inclusa qualsiasi storia di comportamento suicidario in
ultimi 6 mesi e / o qualsiasi idea suicida negli ultimi 2 mesi o chi nel
giudizio dell'investigatore, pone un rischio di suicidio significativo
• Avere una storia di immunodeficienza primaria
• Presenta una significativa carenza di IgG (livello di IgG <400 mg / dL)
• Ha un deficit di IgA (livello di IgA <10 mg / dL)
• Nota infezione batterica attiva, micobatterica fungina virale o altra infezione
• Storia dell'infezione:
• Attualmente in terapia soppressiva per un'infezione cronica (come la tubercolosi,
pneumocystis, citomegalovirus, virus herpes simplex, herpes zoster e atipico
micobatteri)
• Ricovero per il trattamento dell'infezione entro 60 giorni dal Giorno 0.
• Uso di antibiotici parenterali (EV o IM) (antibatterici, antivirali, antifungini o antiparassitari
agenti) entro 60 giorni dal Giorno 0
• Avere abuso o dipendenza da droghe o alcol, o una storia di droga o alcol
abuso o dipendenza entro 365 giorni prima del giorno 0
• Avere un test HIV storicamente positivo o risultare positivo allo screening per l'HIV
• Stato dell'epatite:
• Evidenza sierologica di infezione da epatite B (HB) attuale o passata basata su
risultati dei test per HBsAg e HBcAb come segue:
• I pazienti positivi per HBsAg o HBcAb sono esclusi
• Test positivo per l'anticorpo dell'epatite C.
• Avere una storia di reazione anafilattica alla somministrazione parenterale di contrasto
agenti, proteine ¿¿umane o murine o anticorpi monoclonali
• Possedere qualsiasi altro valore di laboratorio anormale clinicamente significativo secondo il parere di
investigatore
• Se sono incluse le donne in età fertile (WCBP), vedere l'apposito
istruzioni di seguito.

E.5 End points

E.5.1

Primary end point(s)

Number of Participants Experiencing Adverse Events [Time Frame: 104 weeks]

Numero di partecipanti che hanno manifestato eventi avversi [lasso di tempo: 104 settimane]

E.5.1.1

Timepoint(s) of evaluation of this end point

104 weeks

104 settimane

E.5.2

Secondary end point(s)

Antibodies titre

Titolo anticorpale

E.5.2.1

Timepoint(s) of evaluation of this end point

104 weeks

104 settimane

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

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)

Yes

E.7.3

Therapeutic confirmatory (Phase III)

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

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

Yes

E.8.4

The trial involves multiple sites in the Member State concerned

E.8.4.1

Number of sites anticipated in Member State concerned

E.8.5

The trial involves multiple Member States

E.8.6 Trial involving sites outside the EEA

E.8.6.1

Trial being conducted both within and outside the EEA

E.8.6.2

Trial being conducted completely outside of the EEA

Information not present in EudraCT

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

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:

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 For clinical trials recorded in the database before the 10th March 2011 this question read: "Women of childbearing potential" and did not include the words "not using contraception". An answer of yes could have included women of child bearing potential whether or not they would be using contraception. The answer should therefore be understood in that context. This trial was recorded in the database on 2021-08-17.

Yes

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

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)

Clinical and laboratory follow up

Prosecuzione monitoraggio clinico e laboratoristico

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

2021-08-18

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2021-05-10

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

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IMP with orphan designation in the indication
Orphan Designation Number:
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