Autologous Bone Marrow Concentrate in Treatment of Erectile Dysfunction.

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

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.

Study Overview

• Status: Completed
• Conditions: Erectile Dysfunction
• Intervention / Treatment: Procedure: CaverStem

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)).

• Study Type: Interventional
• Enrollment (Actual): 40
• Phase: Not Applicable

Contacts and Locations

Study Locations

• United States
  • California
    • Torrance, California, United States, 90502
      • Harbor - UCLA Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years to 83 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: Male

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Sequential Assignment
• Masking: Single

Number of Arms

3

Arms and Interventions

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

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: Creative Medical Health, Inc.
• Investigators: Study Chair: Alex Gershman, UCLA/Cedar; Study Director: Jacob Rajfer, University of California, Los Angeles

Publications and helpful links

General Publications

• 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.

Study record dates

Study Major Dates

• Study Start (Actual): December 8, 2015
• Primary Completion (Actual): August 31, 2018
• Study Completion (Actual): August 31, 2019

Study Registration Dates

• First Submitted: December 12, 2015
• First Submitted That Met QC Criteria: October 5, 2018
• First Posted (Actual): October 9, 2018

Study Record Updates

• Last Update Posted (Actual): September 6, 2019
• Last Update Submitted That Met QC Criteria: September 4, 2019
• Last Verified: September 1, 2019

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Mental Disorders; Sexual Dysfunctions, Psychological; Sexual Dysfunction, Physiological; Erectile Dysfunction
• Other Study ID Numbers: 21511-01

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: Yes
• IPD Plan Description: Data will be shared among other investigators through secure clinical registry.
• IPD Sharing Time Frame: 1 year
• IPD Sharing Supporting Information Type: Statistical Analysis Plan (SAP); Informed Consent Form (ICF); Clinical Study Report (CSR)

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No