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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05037760




Registration number
NCT05037760
Ethics application status
Date submitted
23/08/2021
Date registered
8/09/2021

Titles & IDs
Public title
Study to Evaluate KER-050 as a Monotherapy or in Combination With Ruxolitinib in Myelofibrosis
Scientific title
A Phase 2 Open-Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Efficacy of KER-050 as Monotherapy or in Combination With Ruxolitinib in Participants With Myelofibrosis
Secondary ID [1] 0 0
KER050-MF-301
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Myelofibrosis 0 0
Condition category
Condition code
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Cancer 0 0 0 0
Leukaemia - Chronic leukaemia

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - KER-050 monotherapy
Treatment: Drugs - KER-050 in combination with ruxolitinib

Experimental: Arm 1a - Dose Escalation KER-050 (SC, solution for injection, every 4 weeks) monotherapy

Experimental: Arm 1b - Dose Escalation KER-050 (SC, solution for injection, every 4 weeks) in combination with standard of care ruxolitinib (oral, tablet, twice daily)

Experimental: Arm 2a - Dose Expansion KER-050 (SC, solution for injection, every 4 weeks) monotherapy

Experimental: Arm 2b - Dose Expansion KER-050 (SC, solution for injection, every 4 weeks) in combination with standard of care ruxolitinib (oral, tablet, twice daily)


Treatment: Drugs: KER-050 monotherapy
KER-050 administered (SC) for up to 13 cycles

Treatment: Drugs: KER-050 in combination with ruxolitinib
KER-050 administered (SC) for up to 13 cycles in combination with standard of care ruxolitinib
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Incidence of adverse events (Safety and Tolerability)
Timepoint [1] 0 0
64 Weeks
Secondary outcome [1] 0 0
Subgroup of transfusion-independent participants
Timepoint [1] 0 0
24 weeks
Secondary outcome [2] 0 0
Subgroup of participants with anemia requiring red blood cell (RBC) transfusions
Timepoint [2] 0 0
24 weeks
Secondary outcome [3] 0 0
Proportion of participants with decrease in spleen volume of =35% from baseline as measured by computed tomography (CT) (excluding participants status post splenectomy or splenic irradiation)
Timepoint [3] 0 0
At Week 24 and at Week 52
Secondary outcome [4] 0 0
Proportion of participants with improvement in the Myelofibrosis Symptom Assessment Form Total Symptom Score (MF-SAF-TSS) of =50% from baseline
Timepoint [4] 0 0
At Week 24 and at Week 52
Secondary outcome [5] 0 0
Proportion of participants with progression to AML (bone marrow blasts >20%)
Timepoint [5] 0 0
At Week 24 and at Week 52
Secondary outcome [6] 0 0
Proportion of participants with progression to accelerated MF (bone marrow blasts =10%)
Timepoint [6] 0 0
At Week 24 and at Week 52

Eligibility
Key inclusion criteria
Key

* Male or female =18 years of age, at the time of signing informed consent.
* Diagnosis of PMF, post-PV MF, or post-ET MF according to the 2017 World Health Organization criteria.

Arm-specific criteria:

Arm 1A:

* Previously treated with JAK inhibitor(s) or Participant is ineligible for JAK inhibitor(s)
* Anemia, defined as hemoglobin =10 g/dL during screening, or receiving RBC transfusions

Arm 2A:

* Previously treated with JAK inhibitor(s) or Participant is ineligible for JAK inhibitor(s)
* Anemia, defined as hemoglobin =10 g/dL during screening, or receiving RBC transfusions

Arm-specific criteria for 1B and 2B:

* Has been receiving ruxolitinib for =8 weeks prior to C1D1 and on a stable dose for =4 weeks prior to C1D1.
* Anemia, defined as hemoglobin =10 g/dL during screening, or receiving RBC transfusions

Key
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Presence of the following cardiac conditions:

1. New York Heart Association Class 3 or 4 heart failure
2. QTcF >500 msec on the screening or C1D1 electrocardiogram (ECG)
3. Uncontrolled clinically significant arrhythmia (participants with rate-controlled atrial fibrillation are not excluded)
4. Acute myocardial infarction or unstable angina pectoris <6 months prior to C1D1
* History of stroke, deep venous thrombosis, or arterial embolism within 6 months prior to C1D1.
* Any malignancy other than PMF, post-ET MF, or post-PV MF that has not been in remission and/or has required systemic therapy including radiation, chemotherapy, hormonal therapy, or surgery, within 1 year prior to C1D1. In-situ cancers, squamous cell, and basal cell carcinomas which have been fully excised, and monoclonal gammopathy of unclear significance are allowed at the discretion of the Investigator.
* History of solid organ or hematological transplantation.
* Presence of uncontrolled hypertension, defined as systolic blood pressure =150 mm Hg or diastolic blood pressure =100 mm Hg despite adequate treatment.
* Diagnosis of hemolytic anemia, active bleeding, hemoglobinopathies, or congenital disorders as a cause of the participant's anemia.
* CTCAE Grade =2 bleeding events within the 3 months prior to C1D1.
* Bone marrow blast percentage >2%. Participants with blast % between 2-5% are allowed if at least 2 bone marrows >6 months apart demonstrate stability of blast percentage, these participants must be reviewed with the Medical Monitor prior to study entry.
* Prior treatment with luspatercept, sotatercept, or other commercially available or investigational TGF-ß inhibitors (all Arms)
* Treatment within 28 days prior to C1D1 with:

1. Erythropoiesis stimulating agent (ESA)
2. Granulocyte colony-stimulating factor (G-CSF)
3. Granulocyte-macrophage colony-stimulating factor (GM-CSF)
4. Thrombopoietin agonists (TPO)
5. Immunomodulator imide drugs (IMiDs; e.g., thalidomide, pomalidomide, lenalidomide)
6. Interferon

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Other
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
16/12/2021
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/06/2025
Actual
Sample size
Target
110
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,SA,VIC
Recruitment hospital [1] 0 0
The Tweed Hospital - Tweed Heads
Recruitment hospital [2] 0 0
Flinders Medical Centre - Woodville South
Recruitment hospital [3] 0 0
St. Vincent's Hospital Melbourne - Fitzroy
Recruitment hospital [4] 0 0
Royal Melbourne Hospital - Melbourne
Recruitment hospital [5] 0 0
Ballarat Oncology & Hematology Service - Wendouree
Recruitment postcode(s) [1] 0 0
2485 - Tweed Heads
Recruitment postcode(s) [2] 0 0
5042 - Woodville South
Recruitment postcode(s) [3] 0 0
3355 - Fitzroy
Recruitment postcode(s) [4] 0 0
3050 - Melbourne
Recruitment postcode(s) [5] 0 0
3355 - Wendouree

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Keros Therapeutics, Inc.
Address
Country
Other collaborator category [1] 0 0
Commercial sector/industry
Name [1] 0 0
IQVIA Biotech
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Rachael Ryzman
Address 0 0
Country 0 0
Phone 0 0
603-548-3907
Fax 0 0
Email 0 0
KER050-MF-301@kerostx.com
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT05037760