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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04419649




Registration number
NCT04419649
Ethics application status
Date submitted
3/06/2020
Date registered
5/06/2020

Titles & IDs
Public title
A Study of KER-050 to Treat Anemia Due to Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes
Scientific title
A Phase 2, Open-Label, Ascending Dose Study of KER-050 for the Treatment of Anemia in Patients With Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes (MDS)
Secondary ID [1] 0 0
KER050-MD-201
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Myelodysplastic Syndromes 0 0
Cytopenia 0 0
Condition category
Condition code
Blood 0 0 0 0
Haematological diseases
Blood 0 0 0 0
Other blood disorders
Blood 0 0 0 0
Haematological diseases
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - KER-050

Experimental: KER-050 Cohort 1 - Escalating doses of KER-050 administered subcutaneously every 4 weeks for up to 4 cycles. Participants have the option to continue to receive KER-050 once 4 cycles have been completed for up to 24 cycles. Eligible participants may be able to continue to receive subcutaneously administered KER-050 after completing 24 cycles.

Experimental: KER-050 Cohort 2 - Escalating doses of KER-050 administered subcutaneously every 4 weeks for up to 4 cycles. Participants have the option to continue to receive KER-050 once 4 cycles have been completed for up to 24 cycles. Eligible participants may be able to continue to receive subcutaneously administered KER-050 after completing 24 cycles.

Experimental: KER-050 Cohort 3 - Escalating doses of KER-050 administered subcutaneously every 4 weeks for up to 24 cycles. Eligible participants may be able to continue to receive subcutaneously administered KER-050 after completing 24 cycles.

Experimental: KER-050 Cohort 4 - Escalating doses of KER-050 administered subcutaneously every 4 weeks for up to 24 cycles. Eligible participants may be able to continue to receive subcutaneously administered KER-050 after completing 24 cycles.

Experimental: KER-050 Cohort 5 - Escalating doses of KER-050 administered subcutaneously every 4 weeks for up to 24 cycles. Eligible participants may be able to continue to receive subcutaneously administered KER-050 after completing 24 cycles.

Experimental: KER-050 Dose Confirmation Cohort - Participants to receive KER-050 administered subcutaneously every 4 weeks for up to 24 cycles. Eligible participants may be able to continue to receive subcutaneously administered KER-050 after completing 24 cycles.


Treatment: Drugs: KER-050
KER-050 administered subcutaneously every 4 weeks for up to 24 cycles. Eligible participants may be able to continue to receive subcutaneously administered KER-050 after completing 24 cycles in a Long-Term Extension.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Incidence of adverse events (AEs) and serious adverse events (SAEs).
Timepoint [1] 0 0
From treatment initiation to end of study, approximately 2 years
Secondary outcome [1] 0 0
Maximum concentrations of KER-050
Timepoint [1] 0 0
Measured at multiple timepoints over the course of study from study day 1 to end of study, approximately 2 years
Secondary outcome [2] 0 0
Erythroid response in low-transfusion burden (LTB) and high-transfusion burden (HTB) participants.
Timepoint [2] 0 0
Measured at multiple timepoints over the course of study from study day 1 to end of study, approximately 2 years
Secondary outcome [3] 0 0
Incidence of progression to higher risk MDS or AML per World Health Organization 2016 criteria
Timepoint [3] 0 0
Measured at multiple timepoints over the course of study from study day 1 to end of study, approximately 2 years
Secondary outcome [4] 0 0
Incidence of progression to higher risk MDS or AML per World Health Organization 2016 criteria
Timepoint [4] 0 0
Measured at multiple timepoints over the course of study from study day 1 to end of study, approximately 2 years
Secondary outcome [5] 0 0
Modified 2006 International Working Group (IWG) Hematologic Improvement Erythroid (HI-E) response
Timepoint [5] 0 0
Measured at multiple timepoints over the course of study from study day 1 to end of study, approximately 2 years
Secondary outcome [6] 0 0
Mean change from baseline in hemoglobin
Timepoint [6] 0 0
Measured at multiple timepoints over the course of study from study day 1 to end of study, approximately 2 years
Secondary outcome [7] 0 0
Time to erythroid response and modified 2006 IWG HI-E response
Timepoint [7] 0 0
Measured at multiple timepoints over the course of study from study day 1 to end of study, approximately 2 years
Secondary outcome [8] 0 0
Duration of erythroid response and modified 2006 IWG HI-E response
Timepoint [8] 0 0
Measured at multiple timepoints over the course of study from study day 1 to end of study, approximately 2 years
Secondary outcome [9] 0 0
Frequency of red blood cell (RBC) transfusions and rate of RBC transfusion independence = 8 weeks
Timepoint [9] 0 0
Measured at multiple timepoints over the course of study from study day 1 to end of study, approximately 2 years
Secondary outcome [10] 0 0
Change from Baseline in RBC counts and reticulocytes
Timepoint [10] 0 0
Measured at multiple timepoints over the course of study from study day 1 to end of study, approximately 2 years

Eligibility
Key inclusion criteria
Key

1. Diagnosis of MDS according to World Health Organization (WHO)/French American British (FAB) classification that meets Revised International Prognostic Scoring System (IPSS-R) classification of very low, low, or intermediate risk disease.
2. < 5% blasts in bone marrow.
3. Peripheral blood white blood cell count <13,000/µL.
4. Anemia defined as:

1. In non-transfused participants, having received no red blood cell (RBC) transfusions within 8 weeks Hgb concentration = 10.0 g/dL OR
2. In LTB participants, having received 1 to 3 units RBCs for Hgb = 9.0 g/dL within 8 weeks OR
3. In HTB participants, having received = 4 units of RBCs for Hgb = 9.0 g/dL within 8 weeks
5. Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2 (if related to anemia.
6. Females of child-bearing potential and sexually active males must agree to use effective methods of contraception.

Key
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Any active infection requiring parenteral antibiotic therapy within 28 days prior to Cycle 1 Day 1 or oral antibiotics within 14 days of Cycle 1 Day 1.
2. Diagnosis of secondary MDS (i.e., MDS known to have arisen as the result of chemical injury or treatment with chemotherapy and/or radiation for other diseases).
3. Vitamin B12 deficiency.
4. Prior treatment with azacitidine, decitabine, lenalidomide, luspatercept, or sotatercept.
5. Treatment within 28 days prior to Cycle 1 Day 1 with:

1. Erythropoiesis stimulating agent (ESA) OR
2. Granulocyte colony-stimulating factor (G-CSF) OR
3. Granulocyte-macrophage colony-stimulating factor (GM-CSF)
6. Iron chelation therapy if initiated within 8 weeks prior to Cycle 1 Day 1.
7. Vitamin B12 therapy within 8 weeks prior to Cycle 1 Day 1.
8. Treatment with another investigational drug or device or approved therapy for investigational use < or = 28 days prior to Cycle 1 Day 1, or if the half-life of the previous product is known, within 5 times the half-life prior to Cycle 1 Day 1, whichever is longer.
9. Platelet count > 450 x 10*9/L or < 30 x 10*9/L.
10. Transferrin saturation < 15%.
11. Ferritin < 50 µg/L.
12. Folate < 4.5 nmol/L (< 2.0 ng/mL).
13. Vitamin B12 < 148 pmol/L (< 200 pg/mL).
14. Estimated glomerular filtration rate (GFR) < 40 mL/min/1.73 m2 (as determined by the Chronic Kidney Disease Epidemiology Collaboration [CKD-EPI].
15. Pregnant or lactating females

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
19/08/2020
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
30/11/2025
Actual
Sample size
Target
140
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,QLD,SA,VIC
Recruitment hospital [1] 0 0
Border Medical Oncology Research Unit - Albury
Recruitment hospital [2] 0 0
The Tweed Hospital - Tweed Heads
Recruitment hospital [3] 0 0
Westmead Hospital - Westmead
Recruitment hospital [4] 0 0
Townsville University Hospital - Douglas
Recruitment hospital [5] 0 0
Royal Adelaide Hospital - Adelaide
Recruitment hospital [6] 0 0
Flinders Medical Centre - Bedford Park
Recruitment hospital [7] 0 0
Box Hill Hospital - Box Hill
Recruitment hospital [8] 0 0
University Hospital Geelong - Geelong
Recruitment hospital [9] 0 0
Austin Health - Heidelberg
Recruitment hospital [10] 0 0
Royal Melbourne Hospital - Melbourne
Recruitment hospital [11] 0 0
St Vincent's Hospital Melbourne - Melbourne
Recruitment hospital [12] 0 0
Ballarat Oncology and Haematology Service - Wendouree
Recruitment postcode(s) [1] 0 0
2640 - Albury
Recruitment postcode(s) [2] 0 0
2485 - Tweed Heads
Recruitment postcode(s) [3] 0 0
2145 - Westmead
Recruitment postcode(s) [4] 0 0
4814 - Douglas
Recruitment postcode(s) [5] 0 0
5000 - Adelaide
Recruitment postcode(s) [6] 0 0
5042 - Bedford Park
Recruitment postcode(s) [7] 0 0
3128 - Box Hill
Recruitment postcode(s) [8] 0 0
3220 - Geelong
Recruitment postcode(s) [9] 0 0
3084 - Heidelberg
Recruitment postcode(s) [10] 0 0
3050 - Melbourne
Recruitment postcode(s) [11] 0 0
3065 - Melbourne
Recruitment postcode(s) [12] 0 0
3355 - Wendouree
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Florida
Country [3] 0 0
United States of America
State/province [3] 0 0
Michigan
Country [4] 0 0
United States of America
State/province [4] 0 0
Pennsylvania
Country [5] 0 0
Czechia
State/province [5] 0 0
Brno
Country [6] 0 0
Czechia
State/province [6] 0 0
Praha
Country [7] 0 0
France
State/province [7] 0 0
Angers
Country [8] 0 0
France
State/province [8] 0 0
Nantes
Country [9] 0 0
France
State/province [9] 0 0
Nice
Country [10] 0 0
France
State/province [10] 0 0
Paris
Country [11] 0 0
France
State/province [11] 0 0
Pontoise
Country [12] 0 0
France
State/province [12] 0 0
Talence
Country [13] 0 0
France
State/province [13] 0 0
Épagny
Country [14] 0 0
Germany
State/province [14] 0 0
Bayreuth
Country [15] 0 0
Germany
State/province [15] 0 0
Düsseldorf
Country [16] 0 0
Germany
State/province [16] 0 0
Esslingen
Country [17] 0 0
Germany
State/province [17] 0 0
Leipzig
Country [18] 0 0
Germany
State/province [18] 0 0
Mainz
Country [19] 0 0
Germany
State/province [19] 0 0
Rostock
Country [20] 0 0
Israel
State/province [20] 0 0
Ramat Gan
Country [21] 0 0
Israel
State/province [21] 0 0
Tel Aviv
Country [22] 0 0
New Zealand
State/province [22] 0 0
Auckland
Country [23] 0 0
Spain
State/province [23] 0 0
Barcelona
Country [24] 0 0
Spain
State/province [24] 0 0
Salamanca
Country [25] 0 0
Spain
State/province [25] 0 0
Sevilla
Country [26] 0 0
Spain
State/province [26] 0 0
Valencia

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Keros Therapeutics, Inc.
Address
Country

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
Clinical Study Team
Address 0 0
Country 0 0
Phone 0 0
+1 (617) 314-6297
Fax 0 0
Email 0 0
[email protected]
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/NCT04419649