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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04485260




Registration number
NCT04485260
Ethics application status
Date submitted
21/07/2020
Date registered
24/07/2020
Date last updated
9/05/2022

Titles & IDs
Public title
An Open-Label, Multicenter, Phase 1b/2 Study of the Safety and Efficacy of KRT-232 Combined With Ruxolitinib in Patients With Primary Myelofibrosis (PMF), Post-Polycythemia Vera MF (Post-PV-MF), Or Post-Essential Thrombocythemia MF (Post ET-MF) Who Have a Suboptimal Response to Ruxolitinib
Scientific title
An Open-Label, Multicenter, Phase 1b/2 Study of the Safety and Efficacy of KRT-232 Combined With Ruxolitinib in Patients With Primary Myelofibrosis (PMF), Post-Polycythemia Vera MF (Post-PV-MF), Or Post-Essential Thrombocythemia MF (Post ET-MF) Who Have a Suboptimal Response to Ruxolitinib
Secondary ID [1] 0 0
KRT-232-109
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Myelofibrosis 0 0
Condition category
Condition code
Blood 0 0 0 0
Haematological diseases
Blood 0 0 0 0
Other blood disorders
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Cancer 0 0 0 0
Leukaemia - Chronic leukaemia

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - KRT-232
Treatment: Drugs - Ruxolitinib

Experimental: Part A, Arm 1, Cohort 1 - KRT-232 by mouth once daily for Days 1-7, off treatment for Days 8-28 (28 day cycle)


Treatment: Drugs: KRT-232
administered by mouth

Treatment: Drugs: Ruxolitinib
administered by mouth

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
For Phase 1: To determine the KRT-232 RP2D in combination with ruxolitinib
Timepoint [1] 0 0
15 months
Primary outcome [2] 0 0
For Phase 2:To determine the spleen volume reduction (SVR) at Week 24
Timepoint [2] 0 0
6 months after last patient enrolled
Secondary outcome [1] 0 0
To determine spleen response
Timepoint [1] 0 0
43 months
Secondary outcome [2] 0 0
To determine the change in Total Symptom Score (TSS) based Myelofibrosis Symptom Assessment Form version 4.0 (MFSAF v4.0)
Timepoint [2] 0 0
43 months

Eligibility
Key inclusion criteria
* Confirmed diagnosis of PMF, post-PV MF, or post-ET MF, as assessed by treating physician according to the World Health Organization (WHO)
* Treatment with ruxolitinib for =18 weeks prior to study entry, and on a stable dose of ruxolitinib in the 8 weeks prior to study entry
* Spleen =5 cm palpable below the LLCM or =450 cm3 by MRI or CT
* Patients must have at least 2 symptoms with a score of at least 1 on the MFSAF v4.0
* ECOG performance status of 0 to 2
Minimum age
18 Years
Maximum age
99 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Patients who are positive for TP53 mutations
* Documented disease progression or clinical deterioration any time while on ruxolitinib treatment
* Patients who have had a documented spleen response to ruxolitinib.
* Prior splenectomy
* Prior MDM2 inhibitor therapy or p53-directed therapy

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
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
Single group
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
UNKNOWN
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)
Recruitment hospital [1] 0 0
Royal Adelaide Hospital - Adelaide
Recruitment postcode(s) [1] 0 0
- Adelaide
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
Maryland
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
New York
Country [5] 0 0
United States of America
State/province [5] 0 0
Ohio
Country [6] 0 0
United States of America
State/province [6] 0 0
Pennsylvania
Country [7] 0 0
United States of America
State/province [7] 0 0
Texas
Country [8] 0 0
United States of America
State/province [8] 0 0
Washington
Country [9] 0 0
Bulgaria
State/province [9] 0 0
Pleven
Country [10] 0 0
Bulgaria
State/province [10] 0 0
Sofia
Country [11] 0 0
France
State/province [11] 0 0
Cedex 9
Country [12] 0 0
France
State/province [12] 0 0
Angers
Country [13] 0 0
France
State/province [13] 0 0
Le Mans
Country [14] 0 0
France
State/province [14] 0 0
Marseille
Country [15] 0 0
France
State/province [15] 0 0
Montpellier
Country [16] 0 0
France
State/province [16] 0 0
Paris
Country [17] 0 0
France
State/province [17] 0 0
Tours
Country [18] 0 0
Germany
State/province [18] 0 0
Aachen
Country [19] 0 0
Germany
State/province [19] 0 0
Halle
Country [20] 0 0
Germany
State/province [20] 0 0
Jena
Country [21] 0 0
Germany
State/province [21] 0 0
Mainz
Country [22] 0 0
Germany
State/province [22] 0 0
Mutlangen
Country [23] 0 0
Israel
State/province [23] 0 0
Zerifin
Country [24] 0 0
Italy
State/province [24] 0 0
Bologna
Country [25] 0 0
Italy
State/province [25] 0 0
Catania
Country [26] 0 0
Italy
State/province [26] 0 0
Firenze
Country [27] 0 0
Italy
State/province [27] 0 0
Meldola
Country [28] 0 0
Italy
State/province [28] 0 0
Varese
Country [29] 0 0
Poland
State/province [29] 0 0
Kraków
Country [30] 0 0
Poland
State/province [30] 0 0
Opole
Country [31] 0 0
Spain
State/province [31] 0 0
Barcelona
Country [32] 0 0
Spain
State/province [32] 0 0
Las Palmas De Gran Canaria
Country [33] 0 0
Spain
State/province [33] 0 0
Málaga
Country [34] 0 0
Spain
State/province [34] 0 0
Salamanca
Country [35] 0 0
Spain
State/province [35] 0 0
Zaragoza

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Kartos 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
John Mei
Address 0 0
Country 0 0
Phone 0 0
650-542-0136
Fax 0 0
Email 0 0
jmei@kartosthera.com
Contact person for scientific queries

No information has been provided regarding IPD availability


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.