Ignite Creation Date:
2025-12-25 @ 3:16 AM
Ignite Modification Date:
2025-12-26 @ 1:55 AM
Study NCT ID:
NCT07285005
Status:
NOT_YET_RECRUITING
Last Update Posted:
2025-12-19
First Post:
2025-11-19
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
A Study to Investigate Efficacy and Safety of KP-001 Compared With Placebo in Patients Aged ≥2 Years With Common VM, Common LM, or KTS/CLOVES Syndrome
Sponsor:
Kaken Pharmaceutical
Organization:
Study Overview
Official Title:
A Phase 3, Randomized, Parallel, Multicenter, Double-blind, Placebo-controlled Study to Investigate Efficacy and Safety of KP 001 in Patients Aged ≥2 Years With Common Venous Malformations, Common Lymphatic Malformations, or KTS/CLOVES Syndrome
Status:
NOT_YET_RECRUITING
Status Verified Date:
2025-12
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
S-KY
Brief Summary:
This is a phase 3, double-blind, randomized, placebo-controlled, parallel group, adaptive, multicenter study planned to be conducted at multiple sites in North America, Canada, Taiwan and South Korea.
The purpose of this study is to measure the efficacy and safety of KP-001 compared with placebo in patients aged ≥2 years with common VM, common LM, or KTS/CLOVES syndrome.
An independent data monitoring committee (DMC) will be established to determine whether to discontinue or continue the study. It will also determine the redesign of the number of cases based on the result of the interim analysis.
The study will comprise the following:
* Screening Period: Up to 42 days prior to the first dose of study intervention.
* Treatment Period 1: This is a double-blind period in which KP-001 100 mg (or lower dose depending on their body weight) or placebo will be administered to patients once daily after breakfast until Week 24.
* Treatment Period 2: After 24 weeks of double blind treatment, all patients will switch to the KP-001 open label extension and treated up to Week 52.
* Follow-up Visit: This visit will occur 30 days after the last dose of study intervention, and assessments will be performed per the SoA.
* Discontinuation Visit: Patients who discontinue study intervention will be requested to continue participating in the study and assessments will be performed per the SoA. If the patients request to withdraw from the study, all tests and evaluations when possible will be performed at Discontinuation visit.
The purpose of this study is to measure the efficacy and safety of KP-001 compared with placebo in patients aged ≥2 years with common VM, common LM, or KTS/CLOVES syndrome.
An independent data monitoring committee (DMC) will be established to determine whether to discontinue or continue the study. It will also determine the redesign of the number of cases based on the result of the interim analysis.
The study will comprise the following:
* Screening Period: Up to 42 days prior to the first dose of study intervention.
* Treatment Period 1: This is a double-blind period in which KP-001 100 mg (or lower dose depending on their body weight) or placebo will be administered to patients once daily after breakfast until Week 24.
* Treatment Period 2: After 24 weeks of double blind treatment, all patients will switch to the KP-001 open label extension and treated up to Week 52.
* Follow-up Visit: This visit will occur 30 days after the last dose of study intervention, and assessments will be performed per the SoA.
* Discontinuation Visit: Patients who discontinue study intervention will be requested to continue participating in the study and assessments will be performed per the SoA. If the patients request to withdraw from the study, all tests and evaluations when possible will be performed at Discontinuation visit.
Detailed Description:
Safety and Exploratory Assessments Vital signs (blood pressure, heart rate, respiratory rate, and body temperature) will be monitored at each scheduled visit for safety.
Safety laboratory assessments will include hematology, serum chemistry, coagulation parameters (e.g., PT/INR, aPTT, fibrinogen), and urinalysis.
Coagulation biomarkers other than D-dimer will be collected for exploratory/safety purposes only and will not be included in the reported outcome measures.
Exploratory analyses may include the relationship between changes in Symptom Numeric Rating Scale (NRS) scores and Patient Global Impression of Severity (PGI-S) scores.
Safety laboratory assessments will include hematology, serum chemistry, coagulation parameters (e.g., PT/INR, aPTT, fibrinogen), and urinalysis.
Coagulation biomarkers other than D-dimer will be collected for exploratory/safety purposes only and will not be included in the reported outcome measures.
Exploratory analyses may include the relationship between changes in Symptom Numeric Rating Scale (NRS) scores and Patient Global Impression of Severity (PGI-S) scores.
Study Oversight
Has Oversight DMC:
True
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
Secondary ID Infos
| Secondary ID | Type | Domain | Link | View |
|---|---|---|---|---|
| undecided | REGISTRY | test test test test | View |