Clinical trial NCT03699943: Autologous Bone Marrow Concentrate in Treatment of Erectile Dysfunction.
Official title: Feasibility Study of Intra-cavernosal Administration of Non-Expanded Autologous Bone Marrow Concentrate in Treatment of Erectile Dysfunction.
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Erectile Dysfunction
Lead sponsor: Creative Medical Health, Inc.
Sponsor class: INDUSTRY
First posted: 2018-10-09
Enrollment: 40
Sex: MALE
Minimum age (years): 18
Maximum age (years): 85
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SEQUENTIAL
Intervention 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
Primary purpose: TREATMENT

Brief summary: 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: 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)).

Eligibility criteria: Inclusion Criteria:

1. Chronic organic ED duration at least 0.5 years
2. Diagnosis of ED based on Doppler Ultrasound and/or dynamic infusion cavernosonometry.
3. Baseline International Index of Erectile Function (IIEF-5) score of \< 21
4. Oral medications and intracavernous pharmacological approaches have been deemed ineffective, contraindicated or cannot be tolerated.
5. Concurrently undergoing treatment with testosterone.
6. Willing to forego any other treatments for ED over the course of the study.

Exclusion Criteria:

1. 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.
2. Subjects using herbal remedies for addressing erectile dysfunction within one month of study initiation.
3. Subjects with penile prosthesis or other urinary prosthesis.
4. Subjects with penile anatomical deformities (e.g. Peyronie's disease) or history of priapism.
5. Previous penile surgeries for erectile dysfunction, premature ejaculation or penile enlargement.
6. Diagnosis of psychogenic ED as determined by nocturnal tumenscence testing.
7. Presenting with uncontrolled or severe disease, including cardiovascular disease, diabetes, liver disease.
8. Uncontrolled hypertension or hypotension (systolic blood pressure \> 170 or \< 90 mm Hg, and diastolic blood pressure \> 100 or \< 50 mm Hg)
9. Suffered a cardiovascular event within 6 months prior to study initiation.
10. Current or previous malignancy other than non-melanoma skin cancer (successfully treated or treatable by curative excision or other local curative therapy).
11. Diagnosis of a systemic autoimmune disorder.
12. Receiving immunosuppressant medications.

Primary outcomes:
measure: Improvement in erectile function as measured by total score in the International Index of Erectile Function
timeFrame: 6 months
measure: Rate of Adverse Events
timeFrame: 6 months
Secondary outcomes:
measure: Change in Doppler Measurements
timeFrame: 6 months
Other outcomes:
Locations:
city: Torrance
country: United States
facility: Harbor - UCLA Medical Center
state: California
Study officials:
affiliation: UCLA/Cedar
name: Alex Gershman
role: STUDY_CHAIR
affiliation: University of California, Los Angeles
name: Jacob Rajfer
role: STUDY_DIRECTOR
References:
citation: Bieri M, Said E, Antonini G, Dickerson D, Tuma J, Bartlett CE, Patel AN, Gershman A. Phase I and registry study of autologous bone marrow concentrate evaluated in PDE5 inhibitor refractory erectile dysfunction. J Transl Med. 2020 Jan 14;18(1):24. doi: 10.1186/s12967-019-02195-w.