Clinical Trials Register

Summary
EudraCT Number:	2020-005032-30
Sponsor's Protocol Code Number:	FLT201-01
National Competent Authority:	Spain - AEMPS
Clinical Trial Type:	EEA CTA
Trial Status:	Ongoing
Date on which this record was first entered in the EudraCT database:	2021-08-26
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

Spain - AEMPS

A.2

EudraCT number

2020-005032-30

A.3

Full title of the trial

A Phase 1/2, open-label, safety, tolerability and efficacy study of FLT201 in adult patients with Gaucher disease Type 1 (Galileo-1)

Estudio abierto de fase I/II, para evaluar la seguridad, tolerabilidad y eficacia de FLT201 en pacientes adultos con enfermedad de Gaucher de tipo 1 (GALILEO-1)

A.3.1

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

A Gene therapy study in patients with Gaucher Disease (Galileo-1)

Estudio en terapia génica en pacientes con enfermedad de Gaucher (GALILEO -1)

A.3.2

Name or abbreviated title of the trial where available

Galileo-1

GALILEO-1

A.4.1

Sponsor's protocol code number

FLT201-01

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	Freeline Therapeutics Limited
B.1.3.4	Country	United Kingdom
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	Freeline Therapeutics Limited
B.4.2	Country	United Kingdom
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Freeline Therapeutics Limited
B.5.2	Functional name of contact point	Thorold Guy
B.5.3	Address:
B.5.3.1	Street Address	Stevenage Bioscience Catalyst, Gunnels Wood Road,
B.5.3.2	Town/ city	Stevenage, Hertfordshire
B.5.3.3	Post code	SG1 2FX
B.5.3.4	Country	United Kingdom
B.5.4	Telephone number	+34900834 223
B.5.6	E-mail	RegistroEspanolDeEstudiosClinicos@druginfo.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	FLT201
D.3.2	Product code	FLT201
D.3.4	Pharmaceutical form	Solution 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.3.8	INN - Proposed INN	Not yet available
D.3.9.2	Current sponsor code	FLT201
D.3.9.3	Other descriptive name	FLT201
D.3.10	Strength
D.3.10.1	Concentration unit	Vector genome
D.3.10.2	Concentration type	range
D.3.10.3	Concentration number	1500000000000 to 3500000000000
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.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

Type 1 Gaucher Disease

Enfermedad de Gaucher tipo 1

E.1.1.1

Medical condition in easily understood language

Gaucher disease is caused by the deficiency of an enzyme. Due to this, fatty substances build up in the cells of the body, especially in liver, spleen and bone marrow.

La enfermedad de Gaucher es causada por la deficiencia de una enzima. Debido a esto, las sustancias grasas se acumulan en las células del cuerpo, especialmente en el hígado, el bazo y la médula ósea.

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	20.0
E.1.2	Level	SOC
E.1.2	Classification code	10010331
E.1.2	Term	Congenital, familial and genetic disorders
E.1.2	System Organ Class	10010331 - Congenital, familial and genetic disorders

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	10075697
E.1.2	Term	Gaucher's disease type I
E.1.2	System Organ Class	10010331 - Congenital, familial and genetic 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 assess the safety and tolerability of a single intravenous administration of FLT201 in adults with Gaucher disease Type 1

Evaluar la seguridad y la tolerabilidad de una administración intravenosa (IV) única de FLT201 en adultos con enfermedad de Gaucher de tipo 1.

E.2.2

Secondary objectives of the trial

• To investigate the relationship of FLT201 dose to endogenous production of GCase
• To investigate the clearance of LysoGb1
• To assess the impact of FLT201 on
- haemoglobin
- platelet count
- spleen size
- liver size
• To assess viral shedding after systemic administration of FLT201
• To describe the immune response to FLT201 transgene product

• Investigar la relación de la dosis de FLT201 con la producción endógena de GCasa
• Investigar la depuración de Lyso-Gb1.
• Evaluar el efecto de FLT201 sobre:
-hemoglobina
-recuento de plaquetas
-tamaño esplénico
-tamaño hepático
• Evaluar la diseminación viral tras la administración sistémica de FLT201.
• Describir la respuesta inmunitaria al producto transgénico FLT201.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Adult ≥ 18 years of age
2. Diagnosis of Gaucher Disease Type 1 with deficient GCase enzyme activity ≤30% of normal in leukocytes at diagnosis
Part 1 only: previously treated patients (PTP):
3. Treatment status at Screening (Screening period is 12 weeks):
a. Off-treatment with enzyme replacement therapy (ERT)/substrate replacement therapy (SRT) for at least 9 months prior to Screening, or
b. On treatment with SRT, with no change in regimen for at least 3 months prior to Screening, or
c. On-treatment with ERT, with no change in regimen for at least 3 months prior to Screening, and ERT dose ≥15 U/kg and ≤60 U/kg every other week (or equivalent) for ≥24 consecutive months

Part 2 only: previously untreated (naïve i.e. never received ERT/SRT) patients (PUP):
4. Patient has a haemoglobin (Hb) level ≥1 g/dL below the lower limit of normal adjusted for age and sex, and at least one of the following at Screening:
a. Platelet count <120X109/L
b. Hepatomegaly on abdominal magnetic resonance imaging (MRI)
c. Splenomegaly on abdominal MRI.

1. Adultos >= 18 años de edad.
2. Diagnóstico de enfermedad de Gaucher de tipo 1 con una actividad deficiente de la enzima GCasa < = 30 % de la normal en los leucocitos en el momento del diagnóstico.
En la parte 1 exclusivamente: pacientes tratados previamente (PTP)
3. Situación del tratamiento en la selección (el período de selección es de 12 semanas):
a. ausencia de tratamiento con tratamiento de reposición enzimática (TRE)/tratamiento de reposición de sustratos (TRS) durante al menos 9 meses antes de la selección, o
b. en tratamiento con TRS, sin modificación de la pauta durante al menos 3 meses antes de la selección.
c. en tratamiento con TRE, sin modificación de la pauta durante al menos 3 meses antes de la selección y con una dosis de TRE >= 15 U/kg y <= 60 U/kg en semanas alternas (o equivalente) durante >= 24 meses consecutivos.

En la parte 2 exclusivamente: pacientes no tratados previamente (es decir, no tratados nunca con TRE/TRS) (PNTP):
4. El paciente presenta una concentración de hemoglobina (Hb) >= 1 g/dl por debajo del límite inferior de la normalidad ajustada respecto a la edad y el sexo, y al menos una de las siguientes condiciones en la selección:
a. Recuento de plaquetas < 120 × 109/l
b. Hepatomegalia en una resonancia magnética (RM) abdominal
c. Esplenomegalia en una RM abdominal

E.4

Principal exclusion criteria

1. Diagnosed or suspected Type 2 or Type 3 Gaucher disease
2. Positive for neutralising antibodies to AAVS3 at Screening
3. Evidence of significant liver dysfunction at Sceening defined as >1.5x upper limit of normal (ULN) in ALT, AST or total bilirubin
4. Evidence of any of the following at Screening:
(a) Hb <8g/dL
(b) Platelets <45,000/mm3
(c) Pulmonary hypertension
(d) New osteonecrosis within 12 months of screening
(e) Fragility fracture or bone crisis within 12 months of screening
5. History of splenectomy (partial or total)

1. Diagnóstico o sospecha de enfermedad de Gaucher de tipo 2 o 3 .
2. Positividad para anticuerpos neutralizantes contra AAVS3 en la selección.
3. Signos de disfunción hepática importante y persistente en la selección, definida como > 1,5 veces el límite superior de la normalidad (LSN) en la alanina aminotransferasa (ALT), la aspartato aminotransferasa (AST) o la bilirrubina total.
4. Indicios de cualquiera de las siguientes condiciones en la selección:
a. Hb < 8 g/dl.
b. Plaquetas < 45 000/mm3.
c. Hipertensión pulmonar.
d. Osteonecrosis nueva en los 12 meses previos a la selección.
e. Fractura por fragilidad o crisis ósea en los 12 meses previos a la selección.
5. Antecedentes de esplenectomía (parcial o total).

E.5 End points

E.5.1

Primary end point(s)

• Incidence of treatment emergent adverse events (TEAEs) including DLTs.

• Incidencia de acontecimientos adversos surgidos durante el tratamiento (AAST).

E.5.1.1

Timepoint(s) of evaluation of this end point

Throughout the study

Durante el estudio

E.5.2

Secondary end point(s)

1. Efficacy - Change from baseline to each assessment point in:
• LysoGb1 in plasma
• Spleen volume by MRI
• Liver volume by MRI
• Haemoglobin
• Platelet count

2. Pharmacokinetic
• Change from baseline to each assessment point in plasma and leukocyte GCase activity
3. Viral shedding
• Clearance of vg in blood, urine, saliva, stool and semen
4. Immune response to Gcase transgene product.
• Change from baseline to each assessment point in anti-GCase antibody titre and neutralising antibody titre

1. Eficacia, cambio desde el momento basal a cada momento de evaluación:
•Lyso-Gb1 en plasma
•volumen esplénico mediante MR
• volumen hepatico mediante MR
• Hemoglobina
• Contaje de plaquetas
2. Farmacocinetica
• Variación de la actividad de la GCasa en plasma y leucocitos entre el momento basal y cada momento de evaluación.
3. Eliminación de virus
• Eliminación de vg en sangre, orina, saliva, heces y semen
4. Respuesta inmune al producto transgénico Gcase.
• Cambio desde el momento basal hasta cada punto de evaluación en el título de anticuerpos anti-GCasa y el título de anticuerpos neutralizantes

E.5.2.1

Timepoint(s) of evaluation of this end point

• Patients to be evaluated at baseline, throughout the study, and at the end of the study at week 38.

• Pacientes serán evaluados en el momento basal, durante el estudio y al final del estudio en la semana 38

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

Yes

E.6.7

Pharmacodynamic

Yes

E.6.8

Bioequivalence

E.6.9

Dose response

Yes

E.6.10

Pharmacogenetic

Yes

E.6.11

Pharmacogenomic

E.6.12

Pharmacoeconomic

E.6.13

Others

Yes

E.6.13.1

Other scope of the trial description

Immunogenicity
Health-related quality of life (HRQOL) questionnaire

Inmunogenicidad
Cuestionario de calidad de vida relacionada con la salud (HRQoL)

E.7

Trial type and phase

E.7.1

Human pharmacology (Phase I)

Yes

E.7.1.1

First administration to humans

Yes

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

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

Brazil

Israel

Paraguay

Tunisia

Germany

Italy

Spain

United Kingdom

Argentina

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 last visit by the last subject.

Ultima visita del último paciente

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

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)

At the end of the study, treatment of subjects with Gaucher disease will be transferred to a long term follow-up study.

TAl final del estudio, los pacientes tratados con la enfermedad de Gaucher serán transferidos a un estudio de seguimiento a largo plazo.

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

2023-03-13

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2023-03-02

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

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