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A Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral GB2064 in Participants With Myelofibrosis

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04679870
Recruitment Status : Recruiting
First Posted : December 22, 2020
Last Update Posted : January 19, 2022
Sponsor:
Collaborator:
OPIS s.r.l
Information provided by (Responsible Party):
Galecto Biotech AB

Brief Summary:
This study is an open label, phase IIa trial in subjects with Myelofibrosis

Condition or disease Intervention/treatment Phase
Myelofibrosis Drug: GB2064 Phase 2

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

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 21 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Intervention Model Description: All subjects eligible for the study will receive GB2064 1000mg, twice a day
Masking: None (Open Label)
Primary Purpose: Treatment
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)
Actual Study Start Date : June 9, 2021
Estimated Primary Completion Date : December 31, 2022
Estimated Study Completion Date : December 31, 2022


Arm Intervention/treatment
Experimental: GB2064
GB2064 will be administered orally as 4 x 250 mg tablets twice a day.
Drug: GB2064
GB2064 (formerly PAT-1251) is a high-affinity, selective, mechanism-based, small molecule inhibitor of LOXL2, administered twice a day




Primary Outcome Measures :
  1. Safety and tolerability of GB2064: AE [ Time Frame: 9 Months ]
    Incidence and severity of adverse events as reported by investigators



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

Participants must satisfy all of the following criteria at the Screening visit:

  1. Adult male or female participants ≥ 18 years of age at enrolment:

    1. 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.
    2. 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.
  2. 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.
  3. 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).
  4. Eastern Cooperative Oncology Group (ECOG) performance status 0-2.
  5. Required baseline laboratory status:

    1. Absolute platelet count (APC) ≥ 50 x 109/L
    2. Absolute neutrophil count (ANC) ≥ 1.5 x 109/L (1500/mm3)
    3. Serum direct bilirubin ≤ 2.0 x ULN (upper limit of normal)
    4. 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
    5. Estimated Glomerular Filtration Rate (eGFR) or creatinine clearance (CrCl) (CrCl calculated by the Cockroft and Gault method) ≥ 30 ml/min/1.73 m2.
    6. Peripheral blood blasts <10%
  6. Treatment-related toxicities from prior therapies must have resolved to Common Terminology Criteria for Adverse Events (CTCAE) Grade ≤ 1.
  7. Participants must have a documented history of transfusion records (if there have been any such transfusions) in the preceding 12 weeks to Day 1.

Exclusion Criteria:

  1. 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.
  2. Positive hepatitis panel and/or positive HIV test.
  3. 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
  4. Impaired cardiac function or clinically significant cardiac diseases, including any of the following:

    1. History or presence of ventricular tachyarrhythmia.
    2. 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.
    3. Clinically significant resting bradycardia (< 50 bpm) and use of a cardiac pacemaker or implantable cardioverter defibrillator.
    4. Angina pectoris or acute myocardial infarction ≤ 90 days prior to starting study drug.
    5. 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).
  5. 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.
  6. Participants with impairment of gastrointestinal (GI) function or GI disease that may significantly alter the absorption of GB2064 as per physician's opinion.
  7. 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.
  8. 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.
  9. Presence of clinically meaningful active bacterial, fungal, parasitic or viral infection which requires therapy.
  10. Previous history of Progressive Multifocal Leuko-encephalopathy (PML).
  11. 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.
  12. 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.
  13. 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.
  14. Hypersensitivity to GB2064 and/or its excipients.
  15. Participants unable or unwilling to comply with protocol requirements.
  16. Participants related to PI/site staff.
  17. Participants who have had a hematopoietic stem cell transplantation.
  18. Participants who are eligible, have a donor and are willing to undergo a hematopoietic stem cell transplantation.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04679870


Contacts
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Contact: Bertil Lindmark, MD +4570705210 Clinicaltrials@galecto.com

Locations
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United States, Texas
MD Andersson Cancer Hospital Recruiting
Houston, Texas, United States, 77030
Contact: Srdan Verstovsek, MD         
Australia
Woden Dermatology Recruiting
Canberra, Australia, 2605
Contact: Dipti Talaulikar, MD         
Germany
Heinrich-Heine-University Dusseldorf Recruiting
Düsseldorf, Germany, 40225
Contact: Norbert Gattermann, MD         
Universitätsklinikum Heidelberg Recruiting
Heidelberg, Germany, 69120
Contact: Richard Schlenk, MD         
Universität Leipzig Recruiting
Leipzig, Germany, 04103
Contact: Georg-Nikolaus Franke, MD         
Praxis für Haemathologie and Onkologie am Isartor Recruiting
München, Germany, 80331
Contact: Petro Petrides, MD         
Klinikum rechts der Isar der Technischen Universitaet Munchen Recruiting
München, Germany, 81675
Contact: Peter Herhaus, MD         
Italy
University of Bologna Sant Orsola Malpighi Recruiting
Bologna, Italy, 40138
Contact: Francesca Palandri, MD         
Azienda Ospedaliero-Universitaria Careggi Recruiting
Firenze, Italy, 50134
Contact: Francesco Mannelli, MD         
ASST Grande Ospedale Metropolitano Niguarda Recruiting
Milano, Italy, 20162
Contact: Marianna Caramella, MD         
Azienda Ospedaliero-Universitaria San Luigi Gonzaga di Orbassano Recruiting
Orbassano, Italy, 10043
Contact: Daniela Cilloni, MD         
Azienda Socio-Sanitaria Territoriale dei Sette Laghi Recruiting
Varese, Italy, 21100
Contact: Francesco Passamonti, MD         
Sponsors and Collaborators
Galecto Biotech AB
OPIS s.r.l
Investigators
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Principal Investigator: Srdan Verstovsek, MD, PhD The University of Texas MD Anderson Cancer Center, Houston, TX
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Responsible Party: Galecto Biotech AB
ClinicalTrials.gov Identifier: NCT04679870    
Other Study ID Numbers: MYLOX-1
2020-003087-45 ( EudraCT Number )
First Posted: December 22, 2020    Key Record Dates
Last Update Posted: January 19, 2022
Last Verified: January 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Galecto Biotech AB:
GB2064
Myelofibrosis
LOXL-2
PAT1251
Additional relevant MeSH terms:
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Primary Myelofibrosis
Myeloproliferative Disorders
Bone Marrow Diseases
Hematologic Diseases