MyelofibrosisPhase 2NCT04679870
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral GB2064 in Participants With Myelofibrosis
A Phase 2 study testing GB2064 in people with Myelofibrosis, run by Galecto Biotech AB at 11 sites in 4 countries.
Trial summary
This study is an open label, phase IIa trial in subjects with Myelofibrosis Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myelofibrosis
- What is being tested?
- GB2064
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 21 participants
- Where
- 11 sitesin 4 countries
- Study dates
- 9 Jun 2021to 30 Jun 2026
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Participants must satisfy all of the following criteria at the Screening visit:
- Adult male or female participants ≥ 18 years of age at enrolment:
- Female participants may be of non-childbearing potential defined as permanently sterile or postmenopausal, or female participants considered to be of childbearing potential who agree to use highly effective birth control methods until 90 days after the follow-up visit. Female participants should refrain from ova donation from the date of Enrolment (Day -1) until 90 days after the follow-up visit.
- Male participants will agree to use contraception throughout the study and until 90-days after the Follow-up visit. Male participants must agree to refrain from sperm donation from the date of Enrolment (Day -1) until 90 days after the follow-up visit.
- Diagnosis of PMF or SMF with intermediate -2 or high-risk disease according to the Dynamic International Prognostic Scoring System (DIPSS)-plus or if with low risk disease then with symptomatic splenomegaly as defined by sonographic assessment as spleen length of >12 cm or by physical examination as ≥ 5 cm below left costal margin.
- Participants who are not currently taking a Janus kinase (JAK) inhibitor (e.g. ruxolitinib or fedratinib) and are therefore refractory, intolerant or ineligible for a JAK inhibitor according to appropriate guidelines (including local guidelines).
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- Eastern Cooperative Oncology Group (ECOG) performance status 0-2.
- Required baseline laboratory status:
- Absolute platelet count (APC) ≥ 50 x 109/L
- Absolute neutrophil count (ANC) ≥ 1.5 x 109/L (1500/mm3)
- Serum direct bilirubin ≤ 2.0 x ULN (upper limit of normal)
- AST (SGOT) or ALT (SGPT) [if both measured, then this applies to both measurements] ≤ 2.5 x ULN, except for participants with MF involvement of the liver who must have levels ≤ 5 x ULN
- Estimated Glomerular Filtration Rate (eGFR) or creatinine clearance (CrCl) (CrCl calculated by the Cockroft and Gault method) ≥ 30 ml/min/1.73 m2.
- Peripheral blood blasts <10%
- Treatment-related toxicities from prior therapies must have resolved to Common Terminology Criteria for Adverse Events (CTCAE) Grade ≤ 1.
- Participants must have a documented history of transfusion records (if there have been any such transfusions) in the preceding 12 weeks to Day 1.
Who cannot join
- Current treatment with a JAK inhibitor (e.g. ruxolitinib or fedratinib) or a history of treatment with a JAK inhibitor within two weeks of enrolment.
- Positive hepatitis panel and/or positive HIV test.
- Any concurrent severe and/or uncontrolled medical conditions that could increase the participant's risk for toxicity while in the study or that could confound discrimination between disease- and study treatment-related toxicities. Any planned major surgery during the study period
- Impaired cardiac function or clinically significant cardiac diseases, including any of the following:
- History or presence of ventricular tachyarrhythmia.
- Presence of unstable atrial fibrillation (ventricular response > 100 bpm); Participants with stable atrial fibrillation are eligible, provided they do not meet any of the other cardiac exclusion criteria.
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- Clinically significant resting bradycardia (< 50 bpm) and use of a cardiac pacemaker or implantable cardioverter defibrillator.
- Angina pectoris or acute myocardial infarction ≤ 90 days prior to starting study drug.
- Other clinically significant heart disease (e.g., symptomatic congestive heart failure; uncontrolled arrhythmia or hypertension; history of labile hypertension or poor compliance with an antihypertensive regimen).
- Participants who are currently receiving chronic (> 14 days) treatment with corticosteroids at a dose > 10 mg of prednisone (or its glucocorticoid equivalent) per day, or any other chronic immunosuppressive treatment that cannot be discontinued prior to starting study drug.
- Participants with impairment of gastrointestinal (GI) function or GI disease that may significantly alter the absorption of GB2064 as per physician's opinion.
- Participants who received radiotherapy within the last month prior to screening procedures, or patients who received splenectomy in the previous three months or are scheduled for the procedure in the next three months.
- Participants who had a history of malignancy in the past 3 years, except for treated early stage squamous, basal cell carcinoma or treated, localised prostate cancer.
- Presence of clinically meaningful active bacterial, fungal, parasitic or viral infection which requires therapy.
- Previous history of Progressive Multifocal Leuko-encephalopathy (PML).
- Pregnant or breast feeding (lactating) women, where pregnancy is defined as the state of a female after conception and until the termination of gestation, confirmed by a positive β- HCG laboratory test.
- Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using highly effective methods of contraception during dosing and for 90 days after study treatment. Highly effective contraception methods must be used.
- Sexually active males must use a condom during intercourse while taking the drug and for 90 days after stopping study drug and should not father a child in this period. A condom is required to be used also by vasectomized men in order to prevent delivery of the drug via seminal fluid.
- Hypersensitivity to GB2064 and/or its excipients.
- Participants unable or unwilling to comply with protocol requirements.
- Participants related to PI/site staff.
- Participants who have had a hematopoietic stem cell transplantation.
- Participants who are eligible, have a donor and are willing to undergo a hematopoietic stem cell transplantation.
Trial locations and contacts
11 sites in 4 countries.
11 sites in 4 countries
Italy5 sites
University of Bologna Sant Orsola Malpighi
Bologna, 40138
Azienda Ospedaliero-Universitaria Careggi
Florence, 50134
ASST Grande Ospedale Metropolitano Niguarda
Milan, 20162
Azienda Ospedaliero-Universitaria San Luigi Gonzaga di Orbassano
Orbassano, 10043
Azienda Socio-Sanitaria Territoriale dei Sette Laghi
Varese, 21100
Germany4 sites
Heinrich-Heine-University Dusseldorf
Düsseldorf, 40225
Universitätsklinikum Heidelberg
Heidelberg, 69120
Universität Leipzig
Leipzig, 04103
Klinikum rechts der Isar der Technischen Universitaet Munchen
München, 81675
About this study
Official title: An Open-label, Phase IIa Study of the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral GB2064 (a LOXL2 Inhibitor) in Participants With Myelofibrosis (MF)
This study is an open label, phase IIa trial in subjects with Myelofibrosis
Read the detailed description
This study is designed to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of orally administered GB2064 a LOXL-2 inhibitor over 9 months. Subjects will receive doses of GB2064, given twice per day to participants with primary or secondary Myelofibrosis
- Conditions
- Myelofibrosis
- Being tested
- GB2064
- Keywords
- GB2064, Myelofibrosis, LOXL-2, PAT1251
- Medical subject headings
- Primary Myelofibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GB2064Drug
GB2064 (formerly PAT-1251) is a high-affinity, selective, mechanism-based, small molecule inhibitor of LOXL2, administered twice a day
| Study group | Role | What they receive |
|---|---|---|
| GB2064 | Experimental | GB2064 will be administered orally as 4 x 250 mg tablets twice a day. Receives: Drug: GB2064 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and tolerability of GB2064: AE
Incidence and severity of adverse events as reported by investigators
Measured: 9 Months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Dec 2020
- Study start9 Jun 2021
- Primary completion31 Dec 2023
- Study completion30 Jun 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for MyelofibrosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- All subjects eligible for the study will receive GB2064 1000mg, twice a day
- Masking
- None (open label)
- Enrolment
- 21 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Galecto Biotech AB
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- OPIS s.r.l
- Responsible party
- Sponsor
- Investigators
- Richard F Schlenk, MD, Principal investigator, Universitätsklinikum Heidelberg, Germany
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04679870
- Sponsor's protocol ID
- MYLOX-1
- Other IDs
- 2020-003087-45
- Status last verified
- Apr 2024
- First submitted
- 17 Dec 2020
- Last updated
- 3 Apr 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Apr 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label, Phase IIa Study of the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral GB2064 (a LOXL2 Inhibitor) in Participants With Myelofibrosis (MF)
- ClinicalTrials.gov ID
- NCT04679870
- Acronym / sponsor ID
- MYLOX-1
- Conditions
- Myelofibrosis
- Keywords
- GB2064, Myelofibrosis, LOXL-2, PAT1251
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 21 participants (estimated)
- Lead sponsor
- Galecto Biotech ABIndustry (pharma, biotech, devices)
- Collaborators
- OPIS s.r.l
- Study start
- 9 Jun 2021 (actual)
- Primary completion
- 31 Dec 2023 (actual)
- Study completion
- 30 Jun 2026 (estimated)
- First posted
- 22 Dec 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study is an open label, phase IIa trial in subjects with Myelofibrosis
Detailed description
This study is designed to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of orally administered GB2064 a LOXL-2 inhibitor over 9 months. Subjects will receive doses of GB2064, given twice per day to participants with primary or secondary Myelofibrosis
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- All subjects eligible for the study will receive GB2064 1000mg, twice a day
| Arm | Type | Receives |
|---|---|---|
| GB2064 | Experimental | Drug: GB2064GB2064 will be administered orally as 4 x 250 mg tablets twice a day. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (16 listed)
- Adult male or female participants ≥ 18 years of age at enrolment:
- Diagnosis of PMF or SMF with intermediate -2 or high-risk disease according to the Dynamic International Prognostic Scoring System (DIPSS)-plus or if with low risk disease then with symptomatic splenomegaly as defined by sonographic assessment as spleen length of >12 cm or by physical examination as ≥ 5 cm below left costal margin.
- Participants who are not currently taking a Janus kinase (JAK) inhibitor (e.g. ruxolitinib or fedratinib) and are therefore refractory, intolerant or ineligible for a JAK inhibitor according to appropriate guidelines (including local guidelines).
- Eastern Cooperative Oncology Group (ECOG) performance status 0-2.
- Required baseline laboratory status:
Key exclusion criteria (23 listed)
- Current treatment with a JAK inhibitor (e.g. ruxolitinib or fedratinib) or a history of treatment with a JAK inhibitor within two weeks of enrolment.
- Positive hepatitis panel and/or positive HIV test.
- Any concurrent severe and/or uncontrolled medical conditions that could increase the participant's risk for toxicity while in the study or that could confound discrimination between disease- and study treatment-related toxicities. Any planned major surgery during the study period
- Impaired cardiac function or clinically significant cardiac diseases, including any of the following:
- Participants who are currently receiving chronic (> 14 days) treatment with corticosteroids at a dose > 10 mg of prednisone (or its glucocorticoid equivalent) per day, or any other chronic immunosuppressive treatment that cannot be discontinued prior to starting study drug.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| GB2064GB2064 (formerly PAT-1251) is a high-affinity, selective, mechanism-based, small molecule inhibitor of LOXL2, administered twice a day | Drug | GB2064 |
Primary outcome
Safety and tolerability of GB2064: AE
Time frame: 9 Months
Incidence and severity of adverse events as reported by investigators
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Apr 2024.
- Individual participant dataSharing planned: No
Related trials for Myelofibrosis
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Primary Myelofibrosis (PMF), Post-polycythemia Vera Myelofibrosis (PPV-MF)
RecruitingPhase 3
A Study to Compare Elritercept to Placebo in Adults With Myelofibrosis and Anemia Who Are Taking Ruxolitinib
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RecruitingObservational study
Motor Development and Early Predictors of Psychomotor Outcomes in Preterm and Term Infants Assessed by MOS-R and Caregiver Questionnaire at 18 and 36 Months
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Active, not recruitingPhase 3
Study of Selinexor in Combination With Ruxolitinib in Myelofibrosis
Myelofibrosis
RecruitingPhase 2
Study of Momelotinib in Combination With Luspatercept in Participants With Transfusion Dependent Myelofibrosis
Primary Myelofibrosis, Myelofibrosis; Primary Myelofibrosis; Post-polycythemia Vera Myelofibrosis; Post-essential Thrombocythemia Myelofibrosis
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Richard F Schlenk, MD principal investigator
Universitätsklinikum Heidelberg, Germany
11 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing GB2064 in people with Myelofibrosis, run by Galecto Biotech AB at 11 sites in 4 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 16 inclusion and 23 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 3 Apr 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 11 sites in Italy, Germany, United States, Australia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety and tolerability of GB2064: AE (time frame: 9 Months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Apr 2024.