A Study to Evaluate Safety and Exploratory Efficacy of KTP-001 in Subjects With Lumbar Disc Herniation

Phase 2CompletedNCT01978912

Teijin America, Inc.24 enrolled

Overview

The purpose of this study is to evaluate the safety and exploratory efficacy of KTP-001 in subjects with lumbar disc herniation.

This study was a first-in-human, open-label, non-controlled single ascending dose study of KTP-001 in male and female subjects between the ages of 30 and 70 years with a single herniated lumbar disc. After obtaining informed consent, subjects were evaluated during a screening period of no more than 3 weeks (21 days). This study was conducted in 10 centers in the US. Subjects that met all screening requirements and inclusion criteria and none of the exclusion criteria were enrolled into the study. Overall, 24 subjects were enrolled and treated: 6 subjects in each cohort. Cohort 1 received a 5 μg/disc dose of KTP-001 by intradiscal injection. Following administration of study drug, subjects were confined to the study center for 24 hours to collect data for safety and efficacy measures and collect blood samples for safety, PK evaluation, exploratory PD and anti-KTP-001 antibody and then returned for further assessments at various intervals from weeks 1 through to month 24. After all subjects in Cohort 1 had received study drug, safety measures were evaluated by a Data and Safety Monitoring Board (DSMB) to determine whether to escalate KTP-001 administration to the next dose level. If appropriate, Cohort 2 subjects received 15 μg/disc of KTP-001, Cohort 3 subjects received 50 μg/disc of KTP-001, and Cohort 4 subjects received 150 μg/disc of KTP-001 by intradiscal injection. All safety, PK, and exploratory efficacy assessments were performed for the subjects in the subsequent cohorts as were performed for Cohort 1.

Study Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Lumbar Disc Herniation

Interventions

KTP-001DRUG

KTP-001 is one time dose intradiscally.

Eligibility

Sex: ALLMin age: 30 YearsMax age: 70 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Subject has had a single contained or noncontained (extruded) lumbar disc herniation (L3-L4, L4-L5 or L5-S1) diagnosed by clinical symptoms and/or physical findings and confirmed by MRI. * Subject has leg pain with a documented positive straight leg raise (SLR) test or femoral stretch test (FST). * Subject has experiences herniated disc symptoms for at least 6 weeks prior to the study without relief with pain medications and other therapies. * Subject has a BMI of 18 to 35 kg/m2 Exclusion Criteria: * Subject has a sequestered lumbar disc herniation or intrathecal herniation confirmed by MRI * Subject has two or more symptomatic lumbar disc herniations * Previous intradiscal therapeutic intervention or has had any lumbar surgery * Presence of lumbar spine disease and/or deformity other than a lumbar disc herniation * Active smoker or is unable to abstain from tobacco use for 2 weeks prior to study injection * Subject has a history or presence of significant cardiovascular, pulmonary, hepatic, renal, hematologic, gastrointestinal, endocrine, immunologic, dermatologic, neurologic, or psychiatric disease

Locations (10)

Alabama Clinical Therapeutics, LLC

Birmingham, Alabama, United States

HOPE Research Institute, LLC

Phoenix, Arizona, United States

CORE Orthopaedic Medical Center

Encinitas, California, United States

Outcomes

Primary Outcomes

Safety Assessed by Adverse Events, Magnetic Resonance Imaging (MRI), X-ray Imaging, Physical Examination, Neurologic Examination and Vital Signs

Any clinically significant changes were recorded as adverse events. They are described in the adverse events section of the results. AEs related to MRI, X-ray Imaging, Physical examination, and Neurologic examination are considered as adverse events of special interest (AESI). A treatment-emergent AE (TEAE) was defined as an AE that was not present prior to treatment with study drug, but appeared following treatment or was present at treatment initiation but worsened in severity during treatment.

Time frame: 24 months

Number of Participants With Change in 12-lead Electrocardiogram (ECG) and Clinical Laboratory Tests (CLT)

Assessment of the number of participants with change in 12-lead ECG and CLT were assessed from baseline, 24 hours and 13 weeks.

Time frame: 13 weeks

Secondary Outcomes

Number of Participants Serum Concentrations of KTP-001 Below the Limit of Quantification (BLQ)

The serum concentrations of KTP-001 were below the limit of quantification (BLQ) (\<100 ng/mL) at all time points in all participants

Time frame: 13 weeks

Number of Participants With Anti-KTP-001 Antibody

Time frame: 13 weeks

Data from ClinicalTrials.gov

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