- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT04419649
A Study of KER-050 to Treat Anemia Due to Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes
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)
Study Overview
Status
Conditions
Intervention / Treatment
Detailed Description
Study Type
Enrollment (Estimated)
Phase
- Phase 2
Contacts and Locations
Study Contact
- Name: Clinical Study Team
- Phone Number: +1 (617) 314-6297
- Email: ker050-md-201@kerostx.com
Study Locations
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New South Wales
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Albury, New South Wales, Australia, 2640
- Recruiting
- Border Medical Oncology Research Unit
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Contact:
- Anish Puliyayil
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Tweed Heads, New South Wales, Australia, 2485
- Recruiting
- The Tweed Hospital
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Contact:
- Alejandro Arbelaez
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Westmead, New South Wales, Australia, 2145
- Recruiting
- Westmead Hospital
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Contact:
- John Kwan
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Queensland
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Douglas, Queensland, Australia, 4814
- Recruiting
- Townsville University Hospital
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Contact:
- Joel Wight
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South Australia
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Adelaide, South Australia, Australia, 5000
- Recruiting
- Royal Adelaide Hospital
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Contact:
- Devendra Hiwase
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Bedford Park, South Australia, Australia, 5042
- Recruiting
- Flinders Medical Centre
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Contact:
- David Ross, MD
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Victoria
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Box Hill, Victoria, Australia, 3128
- Recruiting
- Box Hill Hospital
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Contact:
- Tse-Chieh Teh
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Geelong, Victoria, Australia, 3220
- Recruiting
- University Hospital Geelong
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Contact:
- Hannah Rose
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Heidelberg, Victoria, Australia, 3084
- Recruiting
- Austin Health
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Contact:
- Chun Fong
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Melbourne, Victoria, Australia, 3050
- Recruiting
- Royal Melbourne Hospital
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Contact:
- Lynette Chee
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Melbourne, Victoria, Australia, 3065
- Recruiting
- St Vincent's Hospital Melbourne
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Contact:
- Shuh Ying Tan
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Wendouree, Victoria, Australia, 3355
- Recruiting
- Ballarat Oncology and Haematology Service
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Contact:
- George Kannourakis, MD
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Brno, Czechia
- Recruiting
- Fakultní Nemocnice Brno
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Contact:
- Jiri Mayer
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Praha, Czechia
- Recruiting
- Fakultni Nemocnice Kralovske Vinohrady
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Contact:
- Olga Cerna
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Praha, Czechia
- Recruiting
- Vseobecna Fakultni Nemocnice Praha
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Contact:
- Anna Jonasova
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Angers, France
- Recruiting
- CHU Angers - Hôpital Hôtel Dieu
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Contact:
- Sylvain Thepot
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Nantes, France
- Recruiting
- CHU de Nantes - Hotel Dieu
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Contact:
- Alice Garnier
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Nice, France
- Recruiting
- CHU Nice - Hôpital de l'Archet
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Contact:
- Thomas Cluzeau
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Paris, France
- Recruiting
- Hopital Saint-Louis
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Contact:
- Lionel Ades
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Pontoise, France
- Recruiting
- CH René-Dubos
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Contact:
- Riad Benramdane
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Talence, France
- Recruiting
- CHU de Bordeaux - Hôpital Haut-Lévêque
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Contact:
- Sophie Dimicoli Salazar
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Épagny, France
- Recruiting
- Centre Hospitalier de la Région d'Annecy
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Contact:
- Natacha Mauz
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Contact:
- Charlotte Doublet
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Bayreuth, Germany
- Recruiting
- Klinikum Bayreuth GmbH
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Contact:
- Alexander Kiani
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Düsseldorf, Germany
- Recruiting
- Marien Hospital Dusseldorf GMBH
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Contact:
- Aristoteles Giagounidis
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Düsseldorf, Germany
- Recruiting
- Universitaetsklinikum Duesseldorf AoeR
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Contact:
- Ulrich Germing
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Esslingen, Germany
- Recruiting
- Klinikum Esslingen GmbH
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Contact:
- Swen Wessendorf
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Leipzig, Germany
- Recruiting
- Universitaetsklinikum Leipzig AoeR
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Contact:
- Anne Kubasch
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Mainz, Germany
- Recruiting
- Universitaetsmedizin der Johannes Gutenberg-Universitaet Mainz
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Contact:
- Daniel Sasca
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Rostock, Germany
- Recruiting
- Universitaetsmedizin Rostock
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Contact:
- Christoph Wittke
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Ramat Gan, Israel, 52621
- Recruiting
- Sheba Medical Center
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Contact:
- Drorit Merkel
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Tel Aviv, Israel, 6423906
- Recruiting
- Tel-Aviv Sourasky Medical Center
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Contact:
- Yakir Moshe
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Auckland, New Zealand, 2025
- Recruiting
- Middlemore Hospital
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Contact:
- James Liang
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Barcelona, Spain
- Recruiting
- Hospital Universitario Vall d'Hebron
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Contact:
- David V Ferreiras
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Barcelona, Spain
- Recruiting
- ICO l'Hospitalet - Hospital Duran i Reynals
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Contact:
- Montserrat Sangerman
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Barcelona, Spain
- Recruiting
- Hospital Universitario Central de Asturias
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Contact:
- Teresa Bernal del Castillo
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Salamanca, Spain
- Recruiting
- Hospital Universitario de Salamanca
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Contact:
- Maria Campelo
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Sevilla, Spain
- Recruiting
- Hospital Universitario Virgen del Rocio
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Contact:
- Jose Gonzalez
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Valencia, Spain
- Recruiting
- Hospital Universitari i Politecnic La Fe
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Contact:
- Guillermo Santillana
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California
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Duarte, California, United States, 91010
- Recruiting
- City of Hope National Medical Center
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Principal Investigator:
- Andrew Artz
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Florida
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Miami, Florida, United States, 33136
- Recruiting
- University of Miami, Sylvester Comprehensive Cancer Center
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Contact:
- Mikkael A Sekeres, MD
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Principal Investigator:
- Mikkael A Sekeres, MD
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Tampa, Florida, United States, 33612
- Not yet recruiting
- H. Lee Moffitt Cancer Center and Research Center
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Contact:
- Rami Komrokji
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Michigan
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Lansing, Michigan, United States, 48910
- Not yet recruiting
- Karmanos Cancer Institute at McLaren Greater Lansing
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Principal Investigator:
- Daniel Isaac
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Pennsylvania
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Pittsburgh, Pennsylvania, United States, 15213
- Not yet recruiting
- University of Pittsburgh Medical Health Center
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Contact:
- Robert Redner
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Description
Key Inclusion Criteria:
- 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.
- < 5% blasts in bone marrow.
- Peripheral blood white blood cell count <13,000/µL.
Anemia defined as:
- In non-transfused participants, having received no red blood cell (RBC) transfusions within 8 weeks Hgb concentration ≤ 10.0 g/dL OR
- In LTB participants, having received 1 to 3 units RBCs for Hgb ≤ 9.0 g/dL within 8 weeks OR
- In HTB participants, having received ≥ 4 units of RBCs for Hgb ≤ 9.0 g/dL within 8 weeks
- Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2 (if related to anemia.
- Females of child-bearing potential and sexually active males must agree to use effective methods of contraception.
Key Exclusion Criteria:
- 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.
- 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).
- Vitamin B12 deficiency.
- Prior treatment with azacitidine, decitabine, lenalidomide, luspatercept, or sotatercept.
Treatment within 28 days prior to Cycle 1 Day 1 with:
- Erythropoiesis stimulating agent (ESA) OR
- Granulocyte colony-stimulating factor (G-CSF) OR
- Granulocyte-macrophage colony-stimulating factor (GM-CSF)
- Iron chelation therapy if initiated within 8 weeks prior to Cycle 1 Day 1.
- Vitamin B12 therapy within 8 weeks prior to Cycle 1 Day 1.
- 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.
- Platelet count > 450 x 10*9/L or < 30 x 10*9/L.
- Transferrin saturation < 15%.
- Ferritin < 50 µg/L.
- Folate < 4.5 nmol/L (< 2.0 ng/mL).
- Vitamin B12 < 148 pmol/L (< 200 pg/mL).
- Estimated glomerular filtration rate (GFR) < 40 mL/min/1.73 m2 (as determined by the Chronic Kidney Disease Epidemiology Collaboration [CKD-EPI].
- Pregnant or lactating females
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Non-Randomized
- Interventional Model: Sequential Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
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.
|
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.
|
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.
|
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.
|
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.
|
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.
|
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.
|
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.
|
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.
|
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.
|
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.
|
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.
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Incidence of adverse events (AEs) and serious adverse events (SAEs).
Time Frame: From treatment initiation to end of study, approximately 2 years
|
Type, frequency, severity of AEs and relationship of AEs to KER-050
|
From treatment initiation to end of study, approximately 2 years
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Maximum concentrations of KER-050
Time Frame: Measured at multiple timepoints over the course of study from study day 1 to end of study, approximately 2 years
|
Pharmacokinetics of KER-050
|
Measured at multiple timepoints over the course of study from study day 1 to end of study, approximately 2 years
|
Erythroid response in low-transfusion burden (LTB) and high-transfusion burden (HTB) participants.
Time Frame: Measured at multiple timepoints over the course of study from study day 1 to end of study, approximately 2 years
|
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. |
Measured at multiple timepoints over the course of study from study day 1 to end of study, approximately 2 years
|
Incidence of progression to higher risk MDS or AML per World Health Organization 2016 criteria
Time Frame: Measured at multiple timepoints over the course of study from study day 1 to end of study, approximately 2 years
|
Change in baseline laboratory assessments prior to treatment with KER-050 and after treatment with KER-050
|
Measured at multiple timepoints over the course of study from study day 1 to end of study, approximately 2 years
|
Incidence of progression to higher risk MDS or AML per World Health Organization 2016 criteria
Time Frame: Measured at multiple timepoints over the course of study from study day 1 to end of study, approximately 2 years
|
Change in baseline bone marrow assessments prior to treatment with KER-050 and after treatment with KER-050
|
Measured at multiple timepoints over the course of study from study day 1 to end of study, approximately 2 years
|
Modified 2006 International Working Group (IWG) Hematologic Improvement Erythroid (HI-E) response
Time Frame: Measured at multiple timepoints over the course of study from study day 1 to end of study, approximately 2 years
|
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. |
Measured at multiple timepoints over the course of study from study day 1 to end of study, approximately 2 years
|
Mean change from baseline in hemoglobin
Time Frame: Measured at multiple timepoints over the course of study from study day 1 to end of study, approximately 2 years
|
Change in baseline hemoglobin prior to treatment with KER-050 and after treatment with KER-050
|
Measured at multiple timepoints over the course of study from study day 1 to end of study, approximately 2 years
|
Time to erythroid response and modified 2006 IWG HI-E response
Time Frame: Measured at multiple timepoints over the course of study from study day 1 to end of study, approximately 2 years
|
Time to erythroid response prior to treatment with KER-050 and after treatment with KER-050
|
Measured at multiple timepoints over the course of study from study day 1 to end of study, approximately 2 years
|
Duration of erythroid response and modified 2006 IWG HI-E response
Time Frame: Measured at multiple timepoints over the course of study from study day 1 to end of study, approximately 2 years
|
Duration of erythroid response prior to treatment with KER-050 and after treatment with KER-050
|
Measured at multiple timepoints over the course of study from study day 1 to end of study, approximately 2 years
|
Frequency of red blood cell (RBC) transfusions and rate of RBC transfusion independence ≥ 8 weeks
Time Frame: Measured at multiple timepoints over the course of study from study day 1 to end of study, approximately 2 years
|
Frequency and rate of RBC transfusions prior to treatment with KER-050 and after treatment with KER-050
|
Measured at multiple timepoints over the course of study from study day 1 to end of study, approximately 2 years
|
Change from Baseline in RBC counts and reticulocytes
Time Frame: Measured at multiple timepoints over the course of study from study day 1 to end of study, approximately 2 years
|
Change in baseline RBC counts and reticulocytes prior to treatment with KER-050 and after treatment with KER-050
|
Measured at multiple timepoints over the course of study from study day 1 to end of study, approximately 2 years
|
Collaborators and Investigators
Sponsor
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Estimated)
Study Completion (Estimated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- KER050-MD-201
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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