Clinical Trials Register

Summary
EudraCT Number:	2017-004045-25
Sponsor's Protocol Code Number:	GBT440-034
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:	2021-01-18
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

2017-004045-25

A.3

Full title of the trial

An Open Label Extension Study of GBT440 Administered Orally to Patients with Sickle Cell Disease Who Have Participated in GBT440 Clinical Trials

Studio di estensione in aperto di GBT440 somministrato per via orale a pazienti affetti da anemia falciforme che hanno partecipato a sperimentazioni cliniche di GBT440

A.3.1

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

An Open Label Extension Study of GBT440 Administered Orally to Patients
with Sickle Cell Disease Who Have Participated in GBT440 Clinical Trials

Studio di estensione in aperto di GBT440 somministrato per via orale a pazienti affetti da anemia falciforme che hanno partecipato a sperimentazioni cliniche di GBT440

A.3.2

Name or abbreviated title of the trial where available

A.4.1

Sponsor's protocol code number

GBT440-034

A.5.2

US NCT (ClinicalTrials.gov registry) number

NCT03573882

A.5.4

Other Identifiers

Name:

Number:

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	GLOBAL BLOOD THERAPEUTICS, 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	Global Blood Therapeutics, 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	Global Blood Therapeutics, Inc.
B.5.2	Functional name of contact point	Clinical Trial Manager
B.5.3	Address:
B.5.3.1	Street Address	171 OYSTER POINT BLVD, SUITE 300
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	00000000000
B.5.5	Fax number	000000000000
B.5.6	E-mail	mhottmann@globalbloodtx.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/3/16/1769 - EMA/OD/187/16
D.3 Description of the IMP
D.3.1	Product name	GBT440 300 mg compresse
D.3.2	Product code	[GBT440]
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.3.9.2	Current sponsor code	GBT440
D.3.9.3	Other descriptive name	Voxelotor
D.3.9.4	EV Substance Code	AS3
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	300
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

Sickle Cell Disease

Anemia falciforme

E.1.1.1

Medical condition in easily understood language

Sickle cell anaemia is a serious inherited blood disorder where the red blood cells, develop abnormally and become rigid and shaped like a crescent (or sickle).

L'anemia falciforme è una grave malattia ereditaria del sangue in cui i globuli rossi, si sviluppano in modo anomalo e diventano rigidi e a forma di mezzaluna (o falce).

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	20.1
E.1.2	Level	LLT
E.1.2	Classification code	10002077
E.1.2	Term	Anaemia sickle cell
E.1.2	System Organ Class	100000004850

E.1.2 Medical condition or disease under investigation

E.1.2	Version	20.1
E.1.2	Level	LLT
E.1.2	Classification code	10055200
E.1.2	Term	Anemia sickle cell
E.1.2	System Organ Class	100000004850

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

The objective of this open-label extension (OLE) study is to assess the effect of long-term treatment with GBT440 in participants who have completed treatment in Study GBT440-031, using the following
parameters:
• Hemolysis markers as measured by hematological laboratory
parameters (e.g. hemoglobin, reticulocytes and unconjugated bilirubin).
• Frequency of sickle cell-related complications
• Safety based upon adverse events, clinical laboratory tests, physical
examinations (PE) and other clinical measures.

L’obiettivo di questo studio di estensione in aperto (OLE) è valutare l’effetto del trattamento a lungo termine con GBT440 in partecipanti che hanno completato il trattamento nello studio GBT440-031, utilizzando i seguenti parametri:
1. Marcatori di emolisi, misurati mediante parametri ematologici di laboratorio (ad es., emoglobina, reticolociti e bilirubina non coniugata).
2. Frequenza di complicazioni correlate all’anemia falciforme.
3. Sicurezza in base a eventi avversi (EA), esami clinici di laboratorio, esami obiettivi (PE) e altre misure cliniche.

E.2.2

Secondary objectives of the trial

not applicable

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Male or female study participants with SCD who participated and received study treatment in Study GBT440-031.
Note: Participants in GBT440-031 who discontinued study drug due to an AE, but who remained on study may be eligible for treatment in this study provided the AE does not pose a risk for treatment with GBT440.
2. Females of child-bearing potential are required to have a negative urine pregnancy test prior to dosing on Day 1.
3. Female participants of child-bearing potential must use highly effective methods of contraception to 30 days after the last dose of study drug. Male participants must continue to use barrier methods of
contraception to 30 days after the last dose of study
drug.
4. Participant has provided written informed consent or assent (the ICF must be reviewed and signed by each participant; in the case of pediatric participants, both the consent of the participant's legal representative or legal guardian, and the participant's assent must be obtained).

1. Partecipanti allo studio ambosessi con SCD che hanno partecipato e ricevuto il trattamento in studio nell’ambito dello studio GBT440-031.
Nota: i partecipanti allo studio GBT440-031, che hanno interrotto il farmaco dello studio a causa di un EA, ma che sono rimasti nello studio, possono essere idonei al trattamento in questo studio a condizione che l’EA non comporti un rischio per il trattamento con GBT440.
2. Le donne fertili devono risultare negative al test di gravidanza sulle urine prima della somministrazione del Giorno 1.
3. Le partecipanti fertili devono adottare metodi contraccettivi altamente efficaci fino a 30 giorni dopo l’ultima dose di farmaco dello studio. I partecipanti di sesso maschile devono continuare a usare metodi contraccettivi barriera fino a 30 giorni dopo l’ultima dose di farmaco dello studio.
4. Il partecipante ha fornito il consenso o assenso informato scritto (il modulo di consenso informato [ICF] deve essere esaminato e firmato da ciascun partecipante; in caso di partecipanti in età pediatrica, è necessario ottenere sia il consenso del rappresentante legale o tutore legale del partecipante, sia l’assenso del partecipante stesso).

E.4

Principal exclusion criteria

1. Female who is breast-feeding or pregnant.
2. Participant withdrew consent from Study GBT440-031.
3. Participant was lost to follow-up from Study GBT440-031.
4. Participant requiring chronic dialysis.
5. Any medical, psychological, safety, or behavioral conditions, which, in the opinion of the Investigator, may confound safety interpretation, interfere with compliance, or preclude informed consent.

1. Soggetto di sesso femminile in gravidanza o in allattamento.
2. Partecipante che ha ritirato il consenso dallo studio GBT440-031.
3. Partecipante perso al follow-up nello studio GBT440-031.
4. Partecipante che necessiti di dialisi cronica.
5. Qualsiasi condizione medica, psicologica, di sicurezza o comportamentale che, a giudizio dello sperimentatore, potrebbe confondere l’interpretazione della sicurezza, interferire con la conformità o precludere il consenso informato.

E.5 End points

E.5.1

Primary end point(s)

1. Safety endpoints include AEs, clinical laboratory tests, physical examinations and other clinical measures.
2. Frequency of SCD-related complications of long-term dosing with GBT440.
3. Hemoglobin and measures of hemolysis (e.g. reticulocytes and unconjugated bilirubin).

1. Gli endpoint di sicurezza includono eventi avversi, test clinici di laboratorio, esami fisici e altre misure cliniche.
2. Frequenza delle complicanze associate a SCD di dosaggio a lungo termine con GBT440.
3. Emoglobina e parametri di emolisi (ad esempio reticolociti e bilirubina non coniugata).

E.5.1.1

Timepoint(s) of evaluation of this end point

Day 1, Weeks 4, 8, 12, 24, 48, then Q24 weeks until EOS visit (last dose, 4 Wks ± 7 days)

Giorno 1, Settimane 4, 8, 12, 24, 48, poi Q24 settimane fino alla visita EOS (ultima dose, 4 settimane ± 7 giorni)

E.5.2

Secondary end point(s)

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

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

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

Yes

E.8.1.7.1

Other trial design description

randomizzazione - solo per un sottogruppo di pazienti

randomisation - only for one patient sub-group

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

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

Canada

Egypt

Jamaica

Kenya

Lebanon

Oman

Turkey

United States

France

Italy

Netherlands

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

The study will end when GBT440 obtains an EU marketing authorization.

Lo studio terminerà quando GBT440 otterrà un'autorizzazione all'immissione in commercio in UE

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.1.1.1

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)

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)

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

F.4.2.2

In the whole clinical trial

120

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)

The trial is intended to last until the IMP (GBT440) obtains marketing authorisation or a managed access program. Patients who withdraw from the study will be referred to standard of care treatment.

Lo studio è destinato a durare fino a quando l'IMP (GBT440) ottiene l'autorizzazione all'immissione in commercio o un programma di accesso gestito. I pazienti che si ritirano dallo studio faranno riferimento allo standard di trattamento di cura.

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

2018-03-21

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2018-04-26

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

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