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Safety and Efficacy Clinical Study of Severe Hemophilia B Treatment With KL001

NCT06125756 · Status: NOT_YET_RECRUITING · Phase: EARLY_PHASE1 · Type: INTERVENTIONAL · Enrollment: 9

Last updated 2023-11-09

No results posted yet for this study

Summary

This is an open- label, non- randomized, uncontrolled, dose-escalation pilot study to evaluate the safety and efficacy of KL001 injection solution in hemophilia B subjects with ≤2 IU/dl residual FIX levels.

Conditions

  • Hemophilia B Without Inhibitor

Interventions

DRUG

Low dose KL001

Subjects will be dosed with single dose of KL001 at 2.5x10\^12 vg/kg.

DRUG

Middle dose KL001

Subjects will be dosed with single dose of KL001 at 5.0x10\^12 vg/kg.

DRUG

High dose KL001

Subjects will be dosed with single dose of KL001 at 1.0x10\^13 vg/kg.

Sponsors & Collaborators

  • Affiliated Hospital of Guangdong Medical University

    lead OTHER

Principal Investigators

  • Liang Liang, MD · Affiliated Hospital of Guangdong Medical University

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
12 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-12-20
Primary Completion
2024-09-20
Completion
2025-10-30

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06125756 on ClinicalTrials.gov