Phase I/II Study of KP-100IT in Acute Spinal Cord Injury

Study Overview
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Brief Summary
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This study is randomized, double-blind, placebo-controlled Phase I/II study designed to evaluate safety and efficacy of KP-100IT, code of Hepatocyte Growth Factor (HGF) formulation for intrathecal injection, as a treatment for acute spinal cord injury. The study is conducted at two clinical sites in Japan.

Official Title
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A Phase I/II Study to Evaluate the Safety and Efficacy of Intrathecal Injection of KP-100IT in Subjects With Acute Spinal Cord Injury

Conditions
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Spinal Cord Injuries

Intervention / Treatment
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* Drug: KP-100IT
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Age equal to or greater than 18 years and equal to or less than 75 years Cervical spinal cord injury, and Grade A, B1 or B2 in the modified Frankel Scale at 72 hours since the injury Subjects able to provide written informed concent, which may require a relative to sign if arm/hand function of the subject is compromised Exclusion Criteria: Spinal cord injury at C1-C2 0r C2-C3 level Patients not to able to start rehabilitation within a week by setup of respirator or other reason First dose of the study drug will not be given within 78 hours since the injury History of spinal cord injury or abnormality in spinal cavity. Or current considerable meningeal damage Outcome assessment will not be conducted adequately through damage on injuries other than the injury High-dose steroid therapy within 30 days before the entry Patients who have diseases such as serious liver disorder, renal disorder, hear disease, blood dyscrasia, metabolism disorder and infections History of malignant tumor Patients who participated in other clinical study within 30 days before the entry Patients who have allergies to drug scheduled to be used in the study Administration of the study drug to the area of spinal cord injury is not appropriate for example by intrathecal infections or intrathecal tumor Patients not able to understand informed consent properly Patients who are nursing or may be pregnant Investigator considers that the patient is not appropriate for participating in the study

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 75 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Active Comparator: KP-100IT<br>Intrathecal injection of 0.6 mg HGF starting at 72 hours since the injury and repeating weekly 5 times | Drug: KP-100IT<br> <br> * Other names: HGF;|
| Placebo Comparator: Placebo<br>Intrathecal injection of placebo starting at 72 hours since the injury and repeating weekly 5 times | Drug: Placebo<br> <br> |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number and degree of adverse events | Adverse events will be judged by general condition, vital sign, electrocardiogram, MRI, blood chemistry, hematology, urinalysis, cerebrospinal fluid examination, and antibody formation | 24 weeks | 
| Change of ASIA (American Spinal Injury Association) motor score from baseline at 24 weeks |  | 24 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change of ASIA motor score from baseline at 12 weeks |  | 12 weeks | 
| Time-dependent change of ASIA motor score from baseline |  | 24 weeks | 
| Time-dependent change of ASIA sensory score from baseline |  | 24 weeks | 
| Time-dependent grade change of modified Frankel scale from baseline |  | 24 weeks | 
| Time-dependent change of P-100 concentration in plasma and cerebrospinal fluid |  | 6 weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Hepatocyte Growth Factor, HGF, KP-100IT, Intrathecal injection