Autologous Bone Marrow Concentrate in Treatment of Erectile Dysfunction.

Study Overview
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Brief Summary
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This study assessed the safety and efficacy of autologous bone marrow concentrate and injected intra-cavernously into patients with erectile dysfunction (ED). Specifically, this study will evaluate ED treatment >18 year old men, a demographic where the etiology of ED is attributable primarily to the loss of corporal smooth muscle in the penis. Study endpoints will evaluate the safety and efficacy of intracavernosal bone marrow concentrate administration for treating ED patients.

Detailed Description
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Erectile dysfunction (ED) is characterized by abnormalities of the vascular system; most commonly, venous leakage (or veno-occlusive dysfunction) resulting from loss of integrity of the surrounding corporal smooth muscle. In the penile vascular system, the corporal smooth muscle is responsible for trapping the blood delivered to the corpora cavernosa via the arterial system. Bone marrow is enriched for cells with regenerative potential, including mesenchymal stem cells, which accelerate healing of damaged tissue. The possibility of using bone marrow cells in the treatment of ED is enticing since stem/progenitor cell populations are known to secrete various growth factors, possess anti-inflammatory activities, and can differentiate into cells of the penile architecture. This study will evaluate safety and efficacy of autologous bone marrow concentrate generated by a closed system device and injected intra-cavernously in 40 patients aged > 18 years of age diagnosed with erectile dysfunction with low dose 30 cc (20 patients) or high dose 60 cc (20 patients). Safety and efficacy will be evaluated at baseline (prior to treatment) and at 1,3,6 and 12-month follow up visits. The study will determine whether injection of bone marrow cells intra-cavernously is a clinically feasible, safe and reproducible approach for treating erectile dysfunction. A clinical registry will also be enrolling treating the same patient population (100 patients (20 cc)).

Official Title
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Feasibility Study of Intra-cavernosal Administration of Non-Expanded Autologous Bone Marrow Concentrate in Treatment of Erectile Dysfunction.

Conditions
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Erectile Dysfunction

Intervention / Treatment
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* Procedure: CaverStem


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Chronic organic ED duration at least 0.5 years Diagnosis of ED based on Doppler Ultrasound and/or dynamic infusion cavernosonometry. Baseline International Index of Erectile Function (IIEF-5) score of < 21 Oral medications and intracavernous pharmacological approaches have been deemed ineffective, contraindicated or cannot be tolerated. Concurrently undergoing treatment with testosterone. Willing to forego any other treatments for ED over the course of the study. Exclusion Criteria: Subjects using any medications/drugs with known effects on erectile function within 4 weeks of the study period, including certain antidepressants, antihistamines, diuretics, and beta-blockers. Subjects using herbal remedies for addressing erectile dysfunction within one month of study initiation. Subjects with penile prosthesis or other urinary prosthesis. Subjects with penile anatomical deformities (e.g. Peyronie's disease) or history of priapism. Previous penile surgeries for erectile dysfunction, premature ejaculation or penile enlargement. Diagnosis of psychogenic ED as determined by nocturnal tumenscence testing. Presenting with uncontrolled or severe disease, including cardiovascular disease, diabetes, liver disease. Uncontrolled hypertension or hypotension (systolic blood pressure > 170 or < 90 mm Hg, and diastolic blood pressure > 100 or < 50 mm Hg) Suffered a cardiovascular event within 6 months prior to study initiation. Current or previous malignancy other than non-melanoma skin cancer (successfully treated or treatable by curative excision or other local curative therapy). Diagnosis of a systemic autoimmune disorder. Receiving immunosuppressant medications.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 85 Years

Sexes Eligible for Study
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Male

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: Non-Randomized
Intervention Model: Sequential Assignment
Interventional Model Description: Caverstem 1.0 - Low dose - 30 cc - 20 patients Caverstem 1.0 - High dose - 60 cc - 20 patients Caverstem 2.0 - 20 cc - 100 patients
Masking: Single


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Active Comparator: CaverStem 1.0 - Low<br>Intra-cavernosal injection of autologous bone marrow concentrate for the treatment of Erectile Dysfunction. low dose 30 cc | Procedure: CaverStem<br> <br> |
| Active Comparator: CaverStem 1.0 - High<br>Intra-cavernosal injection of autologous bone marrow concentrate for the treatment of Erectile Dysfunction. high dose 60 cc | Procedure: CaverStem<br> <br> |
| Active Comparator: Caverstem 2.0 - Clinical Registry<br>Intra-cavernosal injection of autologous bone marrow concentrate for the treatment of Erectile Dysfunction. 20 cc | Procedure: CaverStem<br> <br> |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Improvement in erectile function as measured by total score in the International Index of Erectile Function | IIEF-5 scale | 6 months | 
| Rate of Adverse Events | bruising, infection, pain | 6 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in Doppler Measurements | ml/s | 6 months |