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

Phase 1

• Conditions: Primary (PMF) or secondary (SMF) myelofibrosis.; MedDRA version: 20.0Level: PTClassification code 10077161Term: Primary myelofibrosisSystem Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps); MedDRA version: 21.1Level: LLTClassification code 10074691Term: Post polycythaemia vera myelofibrosisSystem Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps); MedDRA version: 21.0Level: LLTClassification code 10074690Term: Post essential thrombocythemia myelofibrosisSystem Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps); Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2020-003087-45-IT
• Lead Sponsor: Galecto Biotech AB

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2021-05-24
• Last Update Posted: 17 August 2021

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 24

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

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 more than 10mg of prednisone (or its glucocorticoid equivalent) per day.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
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;<br>- To assess the effect of GB2064 on clinical parameters of disease activity in participants with PMF or SMF;<br>- To assess the effect of GB2064 on MFrelated symptoms and quality of life (QoL);<br>- 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.

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): 1. Pre-dose and post-dose plasma levels of GB2064<br>2. Clinical parameters including, but not limited to changes in anaemia, platelets and transfusion dependence, splenic volume.<br>3. Clinical Benefit Rate (CBR) and Overall Survival (OS) (Tefferi et al. 2013)<br>4. Patient-reported assessments of symptoms and quality of life, as measured by MPN10 and EQ-5D-5L<br>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.<br>2. At screening and during the study.<br>3. During the study.<br>4. On Day 1 and Month, 3, 6 and 9.<br>5. On Day 1 and Month, 3, 6 and 9.