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

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). - The MYLOX-1 study

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2020-003087-45-DE

Enrollment

Registered

2020-11-30

Start date

2021-04-22

Completion date

Unknown

Last updated

2025-09-15

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Primary (PMF) or secondary (SMF) myelofibrosis. MedDRA version: 20.0 Level: PT Classification code 10077161 Term: Primary myelofibrosis System Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) MedDRA version: 21.1 Level: LLT Classification code 10074691 Term: Post polycythaemia vera myelofibrosis System Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) MedDRA version: 21.0 Level: LLT Classification code 100746

Interventions

Product Name: GB2064 Product Code: PAT-1251 Pharmaceutical Form: Tablet Current Sponsor code: GB2064 Other descriptive name: (3-((4-(aminomethyl)-6-(trifluoromethyl)-pyridin-2-yl)oxy)phenyl)(3R,4R)-3-

Eligibility

Sex/Gender

All

Inclusion criteria

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. Are the trial subjects under 18? no 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 16 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 8

Exclusion criteria

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.

Design outcomes

Primary

Measure	Time frame
Main Objective: To assess the safety, tolerability, pharmacokinetics, pharmacodynamics and clinical effects of 1000 mg BID GB2064 in participants with PMF or SMF.;Secondary Objective: - 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.;Primary end point(s): Adverse events (AE), serious adverse events (SAE), clinical laboratory assessments, vital signs, ECG, physical examination, body weight.;Timepoint(s) of evaluation of this end point: At screening and during the study.	—

Measure

Time frame

Main Objective: To assess the safety, tolerability, pharmacokinetics, pharmacodynamics and clinical effects of 1000 mg BID GB2064 in participants with PMF or SMF.;Secondary Objective: - 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.;Primary end point(s): Adverse events (AE), serious adverse events (SAE), clinical laboratory assessments, vital signs, ECG, physical examination, body weight.;Timepoint(s) of evaluation of this end point: At screening and during the study.

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Secondary

Measure	Time frame
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;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.	—

Measure

Time frame

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;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.

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Countries

Australia, Germany, Italy, United States

Contacts

Public ContactDipartimento Medico

OPIS s.r.l.

info.studiclinici@opis.it003903626331

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026