Clinical Trials Register

Summary
EudraCT Number:	2020-003087-45
Sponsor's Protocol Code Number:	MYLOX-1
National Competent Authority:	Germany - BfArM
Clinical Trial Type:	EEA CTA
Trial Status:	Completed
Date on which this record was first entered in the EudraCT database:	2020-11-30
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

Germany - BfArM

A.2

EudraCT number

2020-003087-45

A.3

Full title of the trial

An open-label, phase IIa study of the safety, tolerability, pharmacokinetics and pharmacodynamics of oral GB2064 (a LOXL2 inhibitor) in participants with myelofibrosis (The MYLOX-1 study).

Eine offene Phase IIa Studie für Patienten mit primärer oder sekundärer Myelofibrose zur Bewertung der Sicherheit, Verträglichkeit, Pharmakokinetik und Pharmakodynamik des LOXL2 Inhibitors GB2064 über einen Behandlungszeitraum von 9 Monaten. (MYLOX-1)

A.3.1

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

Study of the safety, tolerability, pharmacokinetics and pharmacodynamics of oral GB2064 in participants with myelofibrosis.

Untersuchung der Sicherheit, Verträglichkeit, Pharmakokinetik und Pharmakodynamik von oralem GB2064 bei Teilnehmern mit Myelofibrose.

A.3.2

Name or abbreviated title of the trial where available

The MYLOX-1 study

A.4.1

Sponsor's protocol code number

MYLOX-1

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	Galecto Biotech AB
B.1.3.4	Country	Denmark
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	Galecto Biotech AB
B.4.2	Country	Denmark
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	OPIS s.r.l.
B.5.2	Functional name of contact point	Dipartimento Medico
B.5.3	Address:
B.5.3.1	Street Address	Palazzo Aliprandi - Via Matteotti, 10
B.5.3.2	Town/ city	Desio (MB)
B.5.3.3	Post code	20832
B.5.3.4	Country	Italy
B.5.4	Telephone number	003903626331
B.5.5	Fax number	00390362633633
B.5.6	E-mail	info.studiclinici@opis.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	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	GB2064
D.3.2	Product code	PAT-1251
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	GB2064
D.3.9.3	Other descriptive name	(3-((4-(aminomethyl)-6-(trifluoromethyl)-pyridin-2-yl)oxy)phenyl)(3R,4R)-3-fluoro-4-hydroxypyrrolidin-1-yl)methanone methansulfonate
D.3.9.4	EV Substance Code	SUB219269
D.3.10	Strength
D.3.10.1	Concentration unit	mg milligram(s)
D.3.10.3	Concentration number	250
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

Primary (PMF) or secondary (SMF) myelofibrosis.

E.1.1.1

Medical condition in easily understood language

Primary (PMF) or secondary (SMF) myelofibrosis.

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	20.0
E.1.2	Level	PT
E.1.2	Classification code	10077161
E.1.2	Term	Primary myelofibrosis
E.1.2	System Organ Class	10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)

E.1.2 Medical condition or disease under investigation

E.1.2	Version	21.1
E.1.2	Level	LLT
E.1.2	Classification code	10074691
E.1.2	Term	Post polycythaemia vera myelofibrosis
E.1.2	System Organ Class	10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)

E.1.2 Medical condition or disease under investigation

E.1.2	Version	21.0
E.1.2	Level	LLT
E.1.2	Classification code	10074690
E.1.2	Term	Post essential thrombocythemia myelofibrosis
E.1.2	System Organ Class	10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)

E.1.3

Condition being studied is a rare disease

E.2 Objective of the trial

E.2.1

Main objective of the trial

To assess the safety, tolerability, pharmacokinetics, pharmacodynamics and clinical effects of 1000 mg BID GB2064 in participants with PMF or SMF.

E.2.2

Secondary objectives of the trial

- To assess the pharmacokinetics (PK) in blood of GB2064 in participants with PMF or SMF;
- To assess the effect of GB2064 on clinical parameters of disease activity in participants with PMF or SMF;
- To assess the effect of GB2064 on MFrelated symptoms and quality of life (QoL);
- To assess the effect of GB2064 on the bone marrow.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

The main inclusion criteria include a diagnosis of PMF or SMF, Eastern Cooperative Oncology Group (ECOG) performance status 0-2, and clinical laboratory parameters within appropriate limits for participants with MF.

E.4

Principal exclusion criteria

Participants are excluded if they are taking or have taken a JAK inhibitor (ruxolitinib or fedratinib) within two weeks of enrolment or chronic (> 14 days) treatment with corticosteroids at a dose up to 60 mg prednisone per day (or its glucocorticoid equivalent) for a maximum of 4 weeks once enrolled in the study, during which the dose should be tapered to ≤10mg per day, if needed for maintenance, before potentially being discontinued at the Investigators discretion. The dosage of corticosteroids for JAK inhibitor naïve participants should not exceed 10 mg per day should it be necessary while the participant is on the study.

E.5 End points

E.5.1

Primary end point(s)

Adverse events (AE), serious adverse events (SAE), clinical laboratory assessments, vital signs, ECG, physical examination, body weight.

E.5.1.1

Timepoint(s) of evaluation of this end point

At screening and during the study.

E.5.2

Secondary end point(s)

1. Pre-dose and post-dose plasma levels of GB2064
2. Clinical parameters including, but not limited to changes in anaemia, platelets and transfusion dependence, splenic volume.
3. Clinical Benefit Rate (CBR) and Overall Survival (OS) (Tefferi et al. 2013)
4. Patient-reported assessments of symptoms and quality of life, as measured by MPN10 and EQ-5D-5L
5. Histopathological examination of bone marrow biopsy tissue including the extent of fibrosis

E.5.2.1

Timepoint(s) of evaluation of this end point

1. Each month during the study.
2. At screening and during the study.
3. During the study.
4. On Day 1 and Month, 3, 6 and 9.
5. On Day 1 and Month, 3, 6 and 9.

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

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

Yes

E.6.13.1

Other scope of the trial description

Tolerability

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

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

Australia

United States

Germany

Italy

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

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

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)

Participants who derive benefit may continue therapy for up to 3 years.

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

2021-03-19

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2021-04-22

P. End of Trial
P.	End of Trial Status	Completed
P.	Date of the global end of the trial	2024-09-22

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