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Trial details imported from ClinicalTrials.gov

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




Registration number
NCT04419649
Ethics application status
Date submitted
3/06/2020
Date registered
5/06/2020
Date last updated
9/06/2020

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
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 (SC) every 4 weeks for up to 4 cycles

Experimental: KER-050 Cohort 2 - Escalating doses of KER-050 administered subcutaneously (SC) every 4 weeks for up to 4 cycles

Experimental: KER-050 Cohort 3 - Escalating doses of KER-050 administered subcutaneously (SC) every 4 weeks for up to 4 cycles

Experimental: KER-050 Cohort 4 - Escalating doses of KER-050 administered subcutaneously (SC) every 4 weeks for up to 4 cycles

Experimental: KER-050 Dose Confirmation Cohort - Participants to receive KER-050 administered subcutaneously (SC) every 4 weeks for up to 4 cycles


Treatment: Drugs: KER-050
KER-050 administered (SC) every 4 weeks for up to 4 cycles

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). - Type, frequency, severity of AEs and relationship of AEs to KER-050
Timepoint [1] 0 0
From treatment initiation to End of Study visit (approximately 25 weeks)
Secondary outcome [1] 0 0
Maximum concentrations of KER-050 - Pharmacokinetics of KER-050
Timepoint [1] 0 0
Measured at multiple timepoints over the course of treatment from study day 1 to approximately 15 weeks
Secondary outcome [2] 0 0
Erythroid response in low-transfusion burden (LTB) and high-transfusion burden (HTB) participants. - In LTB participants, the proportion of participants who have a hemoglobin increase of = 1.5 g/dL from Baseline for = 14 days (in the absence of RBC transfusions).
In HTB participants, the proportion of participants having a reduction of = 50% or = 4 RBC units transfused compared to pretreatment over an 8-week period.
Timepoint [2] 0 0
Measured over the course of study, up to approximately 25 weeks from study day 1
Secondary outcome [3] 0 0
Modified 2006 International Working Group (IWG) Hematologic Improvement Erythroid (HI-E) response - In LTB participants, the proportion of participants with an increase of = 1.5 g/dL from pretreatment hemoglobin during any 8-week period on study compared to Baseline.
In HTB participants, the proportion of participants having a reduction by = 4 units of RBCs transfused (for a hemoglobin = 9.0 g/dL) during any 8-week period on study, compared with the 8-week period prior to study Cycle 1 Day 1.
Timepoint [3] 0 0
Measured over the course of study, up to approximately 25 weeks from study day 1
Secondary outcome [4] 0 0
Mean change from baseline in hemoglobin
Timepoint [4] 0 0
Measured over the course of study, up to approximately 25 weeks from study day 1
Secondary outcome [5] 0 0
Time to erythroid response and modified 2006 IWG HI-E response
Timepoint [5] 0 0
From treatment initiation to End of Study visit (approximately 25 weeks)
Secondary outcome [6] 0 0
Duration of erythroid response and modified 2006 IWG HI-E response
Timepoint [6] 0 0
From treatment initiation to End of Study visit (approximately 25 weeks)
Secondary outcome [7] 0 0
Frequency of red blood cell (RBC) transfusions and rate of RBC transfusion independence = 8 weeks
Timepoint [7] 0 0
From treatment initiation to End of Study visit (approximately 25 weeks)
Secondary outcome [8] 0 0
Change from Baseline in RBC counts and reticulocytes
Timepoint [8] 0 0
From treatment initiation to End of Study visit (approximately 25 weeks)

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 (WBC) count < 13,000/µL.

4. Anemia defined as:

- In LTB participants (defined as having received < 4 units of RBCs within 8
weeks), mean hemoglobin concentration < 10.0 g/dL of two measurements (one
performed within 1 day prior to Cycle 1 Day 1 and the other performed 7-28 days
prior, not influenced by RBC transfusion within 7 days of measurement) OR

- In HTB participants (defined as requiring > or = 4 units of RBCs for hemoglobin <
or = 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
Gender
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 (ie, MDS that is 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) < 60 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
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
SA
Recruitment hospital [1] 0 0
Keros Investigative Site - Bedford Park
Recruitment postcode(s) [1] 0 0
5042 - Bedford Park

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Keros Therapeutics
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
The purpose of this study is to evaluate the effects of KER-050 on anemia in patients with
very low, low or intermediate risk MDS.
Trial website
https://clinicaltrials.gov/show/NCT04419649
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
Rachel Barger
Address 0 0
Country 0 0
Phone 0 0
603 548 3907
Fax 0 0
Email 0 0
clinicalstudies@kerostx.com
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT04419649