Clinical Trials Register

Summary
EudraCT Number:	2021-002172-39
Sponsor's Protocol Code Number:	VX21-CTX001-141
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:	2021-09-06
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

Expand All Collapse All

A. Protocol Information

A.1

Member State Concerned

Italy - Italian Medicines Agency

A.2

EudraCT number

2021-002172-39

A.3

Full title of the trial

A Phase 3 Study to Evaluate the Safety and Efficacy of a Single Dose of CTX001 in Pediatric Subjects With Transfusion-Dependent ß-Thalassemia

Studio di fase 3 per valutare la sicurezza e l'efficacia di una dose singola di CTX001 in soggetti pediatrici affetti da ß-talassemia trasfusione dipendente

A.3.1

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

A study to learn about the safety and efficacy of CTX001 (the "study drug product") to treat beta-thalassemia in pediatric subjects

Studio per comprendere la sicurezza e l'efficacia di CTX001 (il "prodotto in studio") nel trattamento della ß-talassemia in soggetti pediatrici

A.3.2

Name or abbreviated title of the trial where available

Evaluation of Safety and Efficacy of CTX001 in Pediatric Subjects With Transfusion-Dependent ß-Thala

Valutazione della sicurezza e dell’efficacia di CTX001 in soggetti pediatrici affetti da ß-talassemi

A.4.1

Sponsor's protocol code number

VX21-CTX001-141

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	VERTEX PHARMACEUTICALS INCORPORATED
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	Vertex Pharmaceuticals Incorporated
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	Vertex Pharmaceuticals Incorporated
B.5.2	Functional name of contact point	Clinical Trials and Medical Info
B.5.3	Address:
B.5.3.1	Street Address	50 Northern Avenue
B.5.3.2	Town/ city	Boston
B.5.3.3	Post code	MA 02210
B.5.3.4	Country	United States
B.5.4	Telephone number	+18776348789
B.5.5	Fax number	000000000
B.5.6	E-mail	medicalinfo@vrtx.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	Yes
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	filgrastim
D.3.2	Product code	[filgrastim]
D.3.4	Pharmaceutical form	Solution for injection
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Intravenous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	FILGRASTIM
D.3.9.2	Current sponsor code	filgrastim
D.3.10	Strength
D.3.10.1	Concentration unit	µg/ml microgram(s)/millilitre
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	600
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	Yes
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	Yes
D.2.5.1	Orphan drug designation number	EU/3/19/2210
D.3 Description of the IMP
D.3.1	Product name	CTX001
D.3.2	Product code	[CTX001]
D.3.4	Pharmaceutical form	Dispersion for infusion
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Intravenous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.9.2	Current sponsor code	CTX001
D.3.9.4	EV Substance Code	SUB192451
D.3.10	Strength
D.3.10.1	Concentration unit	Other
D.3.10.2	Concentration type	not less then
D.3.10.3	Concentration number	3
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)	No
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	Yes
D.3.11.3.1	Somatic cell therapy medicinal product	No
D.3.11.3.2	Gene therapy medical product	Yes
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	Yes
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: 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	Yes
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	Plerixafor
D.3.2	Product code	[Plerixafor]
D.3.4	Pharmaceutical form	Solution 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	PLERIXAFOR
D.3.9.1	CAS number	110078-46-1
D.3.9.2	Current sponsor code	Plerixafor
D.3.10	Strength
D.3.10.1	Concentration unit	mg/l milligram(s)/litre
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	20
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.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	Yes
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	Busulfan
D.3.2	Product code	[Busulfan]
D.3.4	Pharmaceutical form	Concentrate for solution for injection/infusion
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Intravenous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	BUSULFAN
D.3.9.2	Current sponsor code	Busulfan
D.3.10	Strength
D.3.10.1	Concentration unit	mg/ml milligram(s)/millilitre
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	6
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

Transfusion-Dependent ß Thalassemia

ß-talassemia trasfusione-dipendente

E.1.1.1

Medical condition in easily understood language

Transfusion-Dependent ß Thalassemia

ß-talassemia trasfusione-dipendente

E.1.1.2

Therapeutic area

Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

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	10054660
E.1.2	Term	Thalassemia beta
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

Evaluate the efficacy of a single dose of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs; CTX001) in pediatric subjects with Transfusion-Dependent ß- Thalassemia (TDT)

Valutare l’efficacia di una dose singola di cellule staminali e progenitrici ematopoietiche umane (hHSPC) CD34+ autologhe modificate con il sistema CRISPR-Cas9 (CTX001) in soggetti pediatrici affetti da ß-talassemia trasfusionedipendente (TDT)

E.2.2

Secondary objectives of the trial

• Evaluate the safety and tolerability of a single dose of CTX001
• Quantify percentage of edited alleles in peripheral blood and CD34+ cells of the bone marrow
• Assess the production of HbF post-CTX001 infusion
• Assess the effects of infusion of CTX001 on disease-specific events and clinical status

• Valutare la sicurezza e la tollerabilità di una dose singola di CTX001
• Quantificare la percentuale di alleli modificati nel sangue periferico e in cellule CD34+ del midollo osseo
• Valutare la produzione di emoglobina fetale (HbF) post-infusione di CTX001
• Valutare gli effetti dell’infusione di CTX001 su eventi specifici della malattia e stato clinico

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

• Subjects 2 through 11 years of age, inclusive, on the date of informed consent.
• Diagnosis of transfusion-dependent ß-thalassemia (TDT) as defined by:
a. Documented homozygous or compound heterozygous ß-thalassemia including ß-thalassemia/hemoglobin E (HbE). Subjects can be enrolled based on historical data, but a confirmation of the genotype using the study central laboratory will be required before start of busulfan conditioning.
b. History of at least 100 mL/kg/year of packed RBC transfusions in the prior 24 months before signing of consent (or the last rescreening for patients going through repeat screening) or, for subjects initiating transfusion therapy <24 months before signing of consent, requirement for packed RBC transfusion at least every 3 to 4 weeks for =6 months.
• Eligible for autologous stem cell transplant as per investigator's judgment.
Other protocol defined inclusion criteria may apply.

• Soggetti di età compresa tra 2 e 11 anni inclusi, alla data del consenso informato.
• Diagnosi di ß-talassemia trasfusione-dipendente (TDT) come definita da:
A. ß-talassemia omozigote o eterozigote composta documentata inclusa ß-talassemia/emoglobina E (HbE). I soggetti possono essere arruolati sulla base dei dati storici, ma sarà richiesta una conferma del genotipo utilizzando il laboratorio centrale dello studio prima dell'inizio del condizionamento con busulfan.
B. Storia di almeno 100 ml/kg/anno di trasfusioni di globuli rossi concentrati nei 24 mesi precedenti la firma del consenso (o l'ultimo nuovo screening per i pazienti sottoposti a screening ripetuto) o, per i soggetti che hanno iniziato la terapia trasfusionale <24 mesi prima della firma del consenso, requisito per trasfusione di globuli rossi concentrati almeno ogni 3-4 settimane per =6 mesi.
• Idoneo al trapianto di cellule staminali autologhe secondo il giudizio dello sperimentatore.
Possono essere applicati altri criteri di inclusione definiti dal protocollo.

E.4

Principal exclusion criteria

• An available 10/10 Human Leukocyte Antigen (HLA)-matched related donor.
• Prior allo-HSCT.
• Subjects with associated a-thalassemia and >1 alpha chain deletion or alpha multiplications.
• Subjects with sickle cell beta thalassemia variant.
• Clinically significant and active bacterial, viral, fungal, or parasitic infection as determined by the investigator.
• White blood cell (WBC) count <3 × 10^9/L or platelet count <150 × 10^9/L not related to hypersplenism.
Other protocol defined exclusion criteria may apply.

• Un donatore correlato disponibile 10/10 dell'antigene leucocitario umano (HLA).
• Precedente allo-HSCT.
• Soggetti con a-talassemia associata e >1 delezione della catena alfa o moltiplicazioni alfa.
• Soggetti con variante della beta talassemia falciforme.
• Infezione batterica, virale, fungina o parassitaria clinicamente significativa e attiva come determinato dallo sperimentatore.
• Conta leucocitaria (WBC) <3 × 10^9/L o conta piastrinica <150 × 10^9/L non correlata all'ipersplenismo.
Possono essere applicati altri criteri di esclusione definiti dal protocollo.

E.5 End points

E.5.1

Primary end point(s)

Proportion of subjects who achieve TI12. A subject will be considered to have achieved TI12 if he/she has maintained weighted average Hb >=9 g/dL without RBC transfusions for at least 12 consecutive months any time after CTX001 infusion. The evaluation of TI12 starts 60 days after last RBC transfusion for post-transplant support or TDT disease management.

Percentuale di soggetti che raggiungono l’indipendenza da trasfusioni per 12 mesi (TI12). Si riterrà che un soggetto abbia raggiunto la TI12 se ha mantenuto un livello medio ponderato di emoglobina (Hb) >=9 g/dl senza trasfusioni di globuli rossi per almeno 12 mesi consecutivi in qualsiasi momento dopo l’infusione di CTX001. La valutazione della TI12 inizia 60 giorni dopo l’ultima trasfusione di globuli rossi per il supporto post-trapianto o la gestione della malattia TDT.

E.5.1.1

Timepoint(s) of evaluation of this end point

Up to 24 months

fino a 24 mesi

E.5.2

Secondary end point(s)

• Safety and tolerability of CTX001 based on adverse events (AEs), clinical laboratory values, vital signs, neutrophil engraftment, platelet engraftment, transplant-related mortality (TRM), and all-cause mortality.
• Proportion of subjects who achieve TI6. A subject will be considered to have achieved TI6 if he/she has maintained weighted average Hb >=9 g/dL without RBC transfusions for at least 6 consecutive months any time after CTX001 infusion. The evaluation of TI6 starts 60 days after last RBC transfusion for post-transplant support or TDT disease management.
• Proportion of subjects achieving at least 95%, 90%, 85%, 75%, 50% reduction from baseline in annualized transfusions up to 24 months starting 60 days after CTX001 infusion.
• Relative change from baseline in transfusions up to 24 months starting 60 days after CTX001 infusion.
• Transfusion free duration for subjects who achieve TI12.
• Proportion of alleles with intended genetic modification present in peripheral blood over time.
• Proportion of alleles with intended genetic modification present in CD34+ cells of the bone marrow over time.
• Fetal hemoglobin concentration (pre-transfusion) over time.
• Total hemoglobin concentration (pre-transfusion) over time.
• Change in proportion of circulating erythrocytes expressing fetal hemoglobin (F-cells) from baseline (pre-transfusion) over time

• Sicurezza e tollerabilità di CTX001 basate su eventi avversi (EA), valori clinici di laboratorio, segni vitali, attecchimento dei neutrofili, attecchimento delle piastrine, mortalità correlata al trapianto (TRM) e mortalità per tutte le cause.
• Percentuale di soggetti che raggiungono la l’indipendenza da trasfusioni per 6 mesi (TI6). Si riterrà che un soggetto abbia raggiunto la TI6 se ha mantenuto un livello medio ponderato di emoglobina (Hb) >=9 g/dl senza trasfusioni di globuli rossi per almeno 6 mesi consecutivi in qualsiasi momento dopo l’infusione di CTX001. La valutazione della TI6 inizia 60 giorni dopo l’ultima trasfusione di globuli rossi per il supporto post-trapianto o la gestione della malattia TDT.
• Percentuale di soggetti che raggiungono una riduzione di almeno il 95%, il 90%, l’85%, il 75%, il 50% rispetto al basale nelle trasfusioni annualizzate fino a 24 mesi a partire da 60 giorni dopo l’infusione di CTX001.
• Variazione relativa rispetto al basale nelle trasfusioni fino a 24 mesi a partire da 60 giorni dopo l’infusione di CTX001.
• Durata del periodo senza trasfusioni per i soggetti che raggiungono la TI12.
• Percentuale di alleli con modificazione genetica prevista, presenti nel sangue periferico, nel tempo.
• Percentuale di alleli con modificazione genetica prevista, presenti nelle cellule CD34+ del midollo osseo, nel tempo.
• Concentrazione di emoglobina fetale (pre-trasfusione) nel tempo.
• Concentrazione di emoglobina totale (pre-trasfusione) nel tempo.
• Variazione nella percentuale di eritrociti circolanti che esprimono l’emoglobina fetale (cellule F) dal basale (pre-trasfusione) nel tempo.

E.5.2.1

Timepoint(s) of evaluation of this end point

Up to 24 months

fino a 24 mesi

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

Yes

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

E.8.1.2 - In aperto è Si

E.8.1.2 - open is Yes

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

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

United States

Germany

Italy

United Kingdom

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 end of study is defined as the last scheduled visit (or contact) of the last subject, which is the date the last subject completes the 24- month follow-up period or date of early withdrawal from the study.

La fine dello studio è definita come l'ultima visita programmata (o contatto) dell'ultimo soggetto, che è la data in cui l'ultimo soggetto completa il periodo di follow-up di 24 mesi o la data di ritiro anticipato dallo studio.

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)

Yes

F.1.1.5.1

Number of subjects for this age range:

F.1.1.6

Adolescents (12-17 years)

F.1.2

Adults (18-64 years)

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

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)

All subjects infused with CTX001 will be asked to enroll in the long term follow-up study (Study VX18 CTX001 131).

A tutti i soggetti infusi con CTX001 verrà chiesto di arruolarsi allo studio di followup a lungo termine (Studio VX18 CTX001 131).

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

2021-10-05

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2021-09-15

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

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