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

Teijin

Status and phase: Completed; Phase 2; Phase 1

Conditions: Lumbar Disc Herniation

Treatments: Drug: KTP-001

Study type: Interventional

Funder types: Industry

Identifiers

NCT01978912

KTP-001-CL-101

Details and patient eligibility

About

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

Full description

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.

Enrollment: 24 patients

Sex: All

Ages: 30 to 70 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Sequential Assignment

Masking: None (Open label)

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24 participants in 4 patient groups

Cohort 1

Experimental group

Description:

one time 5 μg/disc dose of KTP-001 by intradiscal injection

Treatment:

Drug: KTP-001

Cohort 2

Experimental group

Description:

one time 15 μg/disc dose of KTP-001 by intradiscal injection

Treatment:

Drug: KTP-001

Cohort 3

Experimental group

Description:

one time 50 μg/disc dose of KTP-001 by intradiscal injection

Treatment:

Drug: KTP-001

Cohort 4

Experimental group

Description:

one time 150 μg/disc dose of KTP-001 by intradiscal injection

Treatment:

Drug: KTP-001