A Single Ascending Dose Study of Safety and Tolerability of STA363 Compared to Placebo in 15 Patients With Chronic Discogenic Low Back Pain

Study Overview
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Brief Summary
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A phase I, randomized, double-blinded, placebo-controlled, single ascending dose study to investigate the safety, local tolerability and transformation of nucleus pulposus following intradiscal injection of STA363 or placebo in patients with discogenic low back pain. 15 patients will participate in either of 3 dose groups, each comprising 5 patients: Group 1: STA363 dose 1 (3 patients) or placebo (2 patients) Group 2: STA363 dose 2 (3 patients) or placebo (2 patients) Group 3: STA363 dose 3 (3 patients) or placebo (2 patients)

Official Title
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A Prospective, Randomised, Double-blinded, Placebo-controlled, Single Ascending Dose Study Investigating the Safety and Local Tolerability of STA363 Compared to Placebo in 15 Patients With Chronic Discogenic Low Back Pain

Conditions
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Low Back Pain

Intervention / Treatment
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* Drug: STA363
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Signed informed consent prior to any study-related procedures Chronic discogenic low back pain present for more than 6 months prior to the screening visit 20 to 60 years of age at the screening visit Insufficient response to at least 6 months of non-operative treatment (analgesics and/or antiinflammatory medication, physiotherapy etc.) A single lumbar disc appropriate for treatment at L3/4 to L5/S1, based on clinical evaluation by the investigator Pfirrmann grade II-III Ability to understand the written and verbal information about the study Exclusion Criteria: Treatment with any investigational product within 3 months prior to the screening visit More than one painful intervertebral disc A painful intervertebral disc above L3/4 level Current infection or prior history of spinal infection (e.g., discitis, septic arthritis, epidural abscess) or an active systemic infection Previous lumbar spine surgery Previous disc invasive treatment procedures at the affected level(s) (e.g., intradiscal electrothermal therapy, intradiscal radiofrequency thermocoagulation) Pfirrmann grade I, IV and V Evidence of prior lumbar vertebral body fracture or trauma Need for spinal decompression assessed by the investigator Presence of disc extrusion or sequestration Patients previously included in the study Patients suffering from psychosomatic pain in the opinion of the investigator Referred leg pain of compressive origin Known alcohol and/or drug abuse Severe intercurrent illness (e.g. rheumatic disease or chronic pain syndrome) or concomitant treatment (e.g. immunosuppressive drugs), which, in the opinion of the investigator, may put the patient at risk when participating in the study, or affect the patient's ability to take part in the study Clinically significant abnormalities in clinical chemistry or haematology parameters as assessed by the investigator Pregnant or lactating females or intention to become pregnant within the study period Known allergy to any of the components of the drug product or placebo Known opioid allergy or intolerance Patients requiring treatment with warfarin or other anticoagulant therapy Unwillingness to refrain from treatment with non-steroidal anti-inflammatory drugs (NSAIDs) within 5 days before the planned study treatment Body weight less than 50 kg

Ages Eligible for Study
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Minimum Age: 20 Years
Maximum Age: 60 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
Interventional Model Description: Following treatment of 2 patients in each dose group (injections with active drug and placebo, respectively), safety data will be reviewed. If no safety or tolerability concerns are identified, the next 3 patients in each dose group will be treated (active treatment or placebo [2:1]).
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: STA363 dose 1<br> | Drug: STA363<br>* STA363, containing 3 different doses of active ingredient, in a volume of 1.5 mL. Administered as intradiscal injection.<br>|
| Experimental: STA363 dose 2<br> | Drug: STA363<br>* STA363, containing 3 different doses of active ingredient, in a volume of 1.5 mL. Administered as intradiscal injection.<br>|
| Experimental: STA363 dose 3<br> | Drug: STA363<br>* STA363, containing 3 different doses of active ingredient, in a volume of 1.5 mL. Administered as intradiscal injection.<br>|
| Placebo Comparator: Placebo<br> | Drug: Placebo<br>* Matching placebo solution with identical appearance to the test product, used as reference treatment. Administered as intradiscal injection.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Local injection site reactions |  | Up to 4 weeks after injection | 
| Frequency and severity of adverse events |  | Up to 12 weeks after injection | 
| Changes in routine safety laboratory parameters |  | Up to 12 weeks after injection | 
| Visual analogue scale (VAS) pain (injection site) |  | Up to 15 minutes after injection | 
| Changes in vital signs |  | Up to 12 weeks after injection | 
| Changes in electrocardiogram (ECG) |  | 1 day after injection | 
| Changes in physical examination findings |  | 1 and 12 weeks after injection | 
| Changes in body weight |  | 12 weeks after injection | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Transformation of nucleus pulposus into connective tissue by magnetic resonance imaging (MRI) |  | 12 weeks after injection | 
| Disc height by MRI |  | 12 weeks after injection |