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).

NCT ID: NCT05927012

Last Updated: 2023-11-24

Basic Information

• Recruitment Status: WITHDRAWN
• Clinical Phase: PHASE2
• Study Classification: INTERVENTIONAL
• Study Start Date: 2023-11-30
• Study Completion Date: 2026-01-04

Brief Summary

This study aims to explore the safety and preliminary efficacy of a response-guided dose titration of KER-047 in the treatment of functional IDA (Iron deficiency anemia) in MDS (Myelodysplastic syndrome), MF(Myelofibrosis), and MDS/MPN (Myeloproliferative neoplasm) overlap syndromes.

Detailed Description

This is a Phase 2 multicenter, open-label study being conducted to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary efficacy of response-guided dose titration of KER-047 in adult participants with functional iron deficiency anemia (IDA) associated with myelodysplastic syndrome (MDS), myelofibrosis (MF), and myelodysplastic syndrome/myeloproliferative neoplasm (MDS/MPN) overlap syndromes. Approximately 20 patients will be enrolled. Dosing of KER-047 may be adjusted based on safety/tolerability and treatment response. The study will be conducted in 2 parts: Part 1 Initial Titration Strategy and Part 2 Cohort Expansion or Alternate Titration Strategy.

The total planned duration of participation for an individual participant is approximately 32 weeks (4-week screening phase, 24-week treatment period, and 4-week follow-up period). For participants in the extension phase, the maximum duration of participation would be approximately 104 weeks (2 years) (4-week screening phase, 24-week treatment period, 18 month [72 weeks] extension period, and 4-week follow-up period).

Conditions

Iron Deficiency Anemia

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: SEQUENTIAL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Part 1 (Initial Titration Strategy)

KER-047(30 mg, 60mg or 80mg) oral tablet daily (or every other day) for up to 24 weeks.

Group Type: EXPERIMENTAL

KER-047

Intervention Type: DRUG

Oral tablet, daily (or every other day) administration

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.

Group Type: EXPERIMENTAL

KER-047

Intervention Type: DRUG

Oral tablet, daily (or every other day) administration

Interventions

KER-047

Oral tablet, daily (or every other day) administration

Intervention Type: DRUG

Eligibility Criteria

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.

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.

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Keros Therapeutics, Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Royal Adelaide Hospital

Adelaide, South Australia, Australia

Hadassah University Medical Center

Jerusalem, , Israel

Galilee Medical Center

Nahariya, , Israel

Laniado Hospital - Sanz Medical Center

Netanya, , Israel

Shamir Medical Center (Assaf Harofeh Medical Center)

Zrifin, , Israel

Countries

Australia; Israel

Other Identifiers

KER047-IR-202

Identifier Type: -

Identifier Source: org_study_id