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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05927012




Registration number
NCT05927012
Ethics application status
Date submitted
8/06/2023
Date registered
3/07/2023

Titles & IDs
Public title
A Study to Evaluate the Safety and Preliminary Efficacy of a Response-guided Dose Titration of KER-047 in the Treatment of Functional IDA (Iron Deficiency Anemia).
Scientific title
A Phase 2, Intrapatient Dose Titration Study of KER-047 in Participants With Functional Iron Deficiency Anemia Associated With Myelodysplastic Syndromes, Myelofibrosis, and Myelodysplastic Syndrome/Myeloproliferative Neoplasm Overlap Syndromes
Secondary ID [1] 0 0
KER047-IR-202
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Iron Deficiency Anemia 0 0
Condition category
Condition code
Blood 0 0 0 0
Anaemia
Blood 0 0 0 0
Haematological diseases
Blood 0 0 0 0
Other blood disorders
Diet and Nutrition 0 0 0 0
Other diet and nutrition disorders
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - KER-047

Experimental: Part 1 (Initial Titration Strategy) - KER-047(30 mg, 60mg or 80mg) oral tablet daily (or every other day) for up to 24 weeks.

Experimental: Part 2 (Cohort Expansion or Alternate Titration Strategy) - The starting dose regimen and titration schedule of KER-047 oral tablet will be based on the SRC (Safety Review Committee) recommendation from Part 1.


Treatment: Drugs: KER-047
Oral tablet, daily (or every other day) administration
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Percentage of participants experiencing Treatment-emergent adverse events (TEAEs)
Timepoint [1] 0 0
Up to 29 weeks in Part 1 or up to 101 weeks if continuing in the treatment extension
Primary outcome [2] 0 0
Dose limiting toxicities (DLTs)
Timepoint [2] 0 0
Up to 29 weeks in Part 1 or up to 101 weeks if continuing in the treatment extension
Primary outcome [3] 0 0
Percentage of participants experiencing Treatment-related AEs (Adverse events)
Timepoint [3] 0 0
Up to 29 weeks in Part 1 or up to 101 weeks if continuing in the treatment extension
Primary outcome [4] 0 0
Number of participants discontinuing due to AEs (Adverse events)
Timepoint [4] 0 0
Up to 29 weeks in Part 1 or up to 101 weeks if continuing in the treatment extension
Primary outcome [5] 0 0
Change from Baseline in clinical laboratory values
Timepoint [5] 0 0
Up to 29 weeks in Part 1 or up to 101 weeks if continuing in the treatment extension
Primary outcome [6] 0 0
Systolic Blood Pressure
Timepoint [6] 0 0
Up to 29 weeks in Part 1 or up to 101 weeks if continuing in the treatment extension
Primary outcome [7] 0 0
Diastolic Blood Pressure
Timepoint [7] 0 0
Up to 29 weeks in Part 1 or up to 101 weeks if continuing in the treatment extension
Primary outcome [8] 0 0
Respiratory rate
Timepoint [8] 0 0
Up to 29 weeks in Part 1 or up to 101 weeks if continuing in the treatment extension
Primary outcome [9] 0 0
Heart rate
Timepoint [9] 0 0
Up to 29 weeks in Part 1 or up to 101 weeks if continuing in the treatment extension
Primary outcome [10] 0 0
Body temperature
Timepoint [10] 0 0
Up to 29 weeks in Part 1 or up to 101 weeks if continuing in the treatment extension
Primary outcome [11] 0 0
Fridericia corrected QT interval via 12-lead Electrocardiogram (ECG)
Timepoint [11] 0 0
Up to 29 weeks in Part 1 or up to 101 weeks if continuing in the treatment extension
Primary outcome [12] 0 0
QT interval via 12-lead Electrocardiogram (ECG)
Timepoint [12] 0 0
Up to 29 weeks in Part 1 or up to 101 weeks if continuing in the treatment extension
Primary outcome [13] 0 0
QRS interval via 12-lead Electrocardiogram (ECG)
Timepoint [13] 0 0
Up to 29 weeks in Part 1 or up to 101 weeks if continuing in the treatment extension
Primary outcome [14] 0 0
PR interval via 12-lead Electrocardiogram (ECG)
Timepoint [14] 0 0
Up to 29 weeks in Part 1 or up to 101 weeks if continuing in the treatment extension
Primary outcome [15] 0 0
Body weight (in kg)
Timepoint [15] 0 0
Up to 29 weeks in Part 1 or up to 101 weeks if continuing in the treatment extension
Secondary outcome [1] 0 0
Change from baseline in reticulocyte hemoglobin content (RET-He)
Timepoint [1] 0 0
Up to 29 weeks in Part 1 or up to 101 weeks if continuing in the treatment extension
Secondary outcome [2] 0 0
Change from baseline in hepcidin concentration
Timepoint [2] 0 0
Up to 29 weeks in Part 1 or up to 101 weeks if continuing in the treatment extension
Secondary outcome [3] 0 0
Change from baseline in hemoglobin (Hgb)
Timepoint [3] 0 0
Up to 29 weeks in Part 1 or up to 101 weeks if continuing in the treatment extension
Secondary outcome [4] 0 0
Proportion of participants who have Hgb increase of =1.0 g/dL (0.6 mmol/L)
Timepoint [4] 0 0
Up to 29 weeks in Part 1 or up to 101 weeks if continuing in the treatment extension
Secondary outcome [5] 0 0
Proportion of participants who have Hgb increase of =1.5 g/dL (0.9 mmol/L)
Timepoint [5] 0 0
Up to 29 weeks in Part 1 or up to 101 weeks if continuing in the treatment extension
Secondary outcome [6] 0 0
Proportion of RBC-transfused participants who achieve =8 weeks of transfusion independence during any consecutive period up to End of Treatment
Timepoint [6] 0 0
Up to 29 weeks or up to 101 weeks if in the treatment extension
Secondary outcome [7] 0 0
Plasma KER-047 and any metabolites concentration, summarized by time point
Timepoint [7] 0 0
Week 1 and Week 13 in Part 1 and 2
Secondary outcome [8] 0 0
Estimated peak plasma concentration (Cmax)
Timepoint [8] 0 0
Week 1 and Week 13 in Part 1 and 2
Secondary outcome [9] 0 0
Time to peak plasma concentration (Tmax)
Timepoint [9] 0 0
Week 1 and Week 13 in Part 1 and 2
Secondary outcome [10] 0 0
Area under the plasma KER-047 concentration curve (AUClast)
Timepoint [10] 0 0
Week 1 and Week 13 in Part 1 and 2
Secondary outcome [11] 0 0
Mean trough (Ctrough) plasma KER-047 and metabolites of interest concentration
Timepoint [11] 0 0
Up to 25 weeks
Secondary outcome [12] 0 0
Plasma KER-047 and metabolites of interest accumulation (Rac)
Timepoint [12] 0 0
Up to 25 weeks
Secondary outcome [13] 0 0
Determination of steady-state (as appropriate)
Timepoint [13] 0 0
Up to 25 weeks

Eligibility
Key inclusion criteria
* Male or female =18 years of age, at the time of signing informed consent.
* One of the following:

1. Diagnosis of MDS according to the 2016 World Health Organization (WHO) classification that meets Revised International Prognostic Scoring System (IPSS-R) classification of very low, low, or intermediate risk disease with bone marrow blast percentage <5% within 6 months prior to Day 1 (D1).
2. Diagnosis of primary myelofibrosis, post polycythemia vera MF, or post-essential thrombocytopenia MF according to the 2017 WHO criteria with bone marrow and peripheral blood blast percentage <2%, or stable between 2% to 5% over 6 months.
3. Diagnosis of MDS/MPN overlap syndromes according to the 2016 WHO classification, with bone marrow blast percentage <5% within 6 months prior to D1.
* Anemia with iron-restricted erythropoiesis as assessed by laboratory criteria during screening.
* Ability to understand the purpose and risks of the study and provide signed and dated informed consent and authorization to use protected health information in accordance with national and local study participant privacy regulations.
* Females of childbearing potential and sexually active males must meet the contraception requirements as outlined in the protocol.
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Active infection within 14 days of D1.
* IPSS-R score indicating high or very high risk MDS, accelerated myelofibrosis (defined as >10% blasts), or diagnosis of acute leukemia.
* Diagnosis of hemolytic anemia.
* Diagnosis of porphyria.
* Anemia due to blood loss 28 days prior to D1.
* Diagnosis of thalassemia, thalassemia trait, or other hemoglobinopathy.
* History of drug or alcohol abuse, as defined by the Investigator, within the past 2 years.
* History of stroke, arterial embolism, or deep venous thrombosis within 6 months prior to D1.
* Known positive for human immunodeficiency virus, active infectious hepatitis B virus or active infectious hepatitis C virus.

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
Withdrawn
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
30/11/2023
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
4/01/2026
Actual
Sample size
Target
Accrual to date
Final
0
Recruitment in Australia
Recruitment state(s)
SA
Recruitment hospital [1] 0 0
Royal Adelaide Hospital - Adelaide
Recruitment postcode(s) [1] 0 0
5000 - Adelaide
Recruitment outside Australia
Country [1] 0 0
Israel
State/province [1] 0 0
Jerusalem
Country [2] 0 0
Israel
State/province [2] 0 0
Nahariya
Country [3] 0 0
Israel
State/province [3] 0 0
Netanya
Country [4] 0 0
Israel
State/province [4] 0 0
Zrifin

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
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Undecided
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/NCT05927012