Multicenter Trial Evaluating the Safety and Efficacy of Autologous Volar Fibroblast Injection Into the Terminal Limb of Amputees.

NCT04839497 | Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: W81XWH1820055
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

Brief Summary

The study will enroll 20 adults ages 18-75 with a transtibial amputation with mature residual limbs who are ambulatory prosthesis users. Participants will be randomized to either treatment with low dose volar fibroblast injections (n=10) or to vehicle control (n=10). Participants will undergo a biopsy to harvest volar skin for fibroblast expansion and tattooing to identify injection sites on the residual limb. Fibroblasts will be processed at the Hopkins Cellular Therapy Core Lab and volar cells primed for injection will be sent to participating centers for administration. Participants randomized to the treatment group will be treated with low-dose cells. Injections will be administered on at least 1 and up to three separate days over the course of one week. Participants randomized to the vehicle control group will receive injection of cryoprotectant. All participants will be followed at 2 weeks, 1, 2, and 3 months after the last injection. These visits will include a clinical evaluation for complications, non-invasive assessments of skin firmness and thickness, skin appearance, and patient-reported outcomes. After the final monitoring visit, individuals randomized to the vehicle control group will have the opportunity to receive the volar fibroblast injections and will be followed for an additional 3 months. The investigators hypothesize that (1) There will be no difference in the rate of serious adverse events among patients treated with volar fibroblast injections compared with patients treated with vehicle control, and (2) Patients treated with volar fibroblast injections will have firmer skin on the residual limb compared with patients tread with vehicle alone.

Conditions

Amputation

Outcome Measures

Primary Outcomes (2)

• Safety assessment of complications and adverse events associated with Volar Fibroblast injections

  Safety will be evaluated by routinely assessing all complications at each study visit. Minor and expected complications may include local injection reactions like bruising and erythema, swelling and pain. Minor complications that are not expected include local small hemorrhage (x\<1mL), edema, nodules, papules, irritation, dermatitis, pruritus, and cellulitis. Serious Adverse Events are defined as unexpected AND serious AND related or possibly related to the study treatment. These may include but are not limited to osteomyelitis.

  3 months from the injection visit

• Efficacy of autologous volar fibroblasts for increasing skin firmness in individuals with a transtibial amputation.

  Efficacy will be determined by treatment group difference in the pre- post-change in skin firmness at three months post injection. Skin firmness is the level of resistance as measured by a non-invasive hand-held durometer

  Baseline through 3 months from the injection visit

Study Arms (2)

Volar Fibroblast Treatment

EXPERIMENTAL

Volar fibroblasts are injected into the residual limb of transtibial amputees

Combination Product: Autologous Volar Fibroblast Injection into the Terminal Limb of Amputees

Cryoprotectant

NO INTERVENTION

Vehicle Control. Interdermal injection of cryoprotectant

Interventions

Autologous Volar Fibroblast Injection into the Terminal Limb of Amputees COMBINATION_PRODUCT

Autologous Volar Fibroblasts Treatment- harvested from patient's biopsied volar skin. Intradermal injection of Volar fibroblasts in the residual limb of transtibial amputees.

Also known as: Autologous Volar Fibroblast Treatment

Volar Fibroblast Treatment

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Ages 18 to 75, inclusive
• Must have a transtibial amputation
• Must be using a prosthesis within the vicinity of 3 months or have had osteo-integration of a prosthetic in place for 1 month
• In the opinion of the investigator, must be medically able to undergo the administration of study material determined by laboratory tests obtained up to 14 days before baseline for which the investigator identified no clinically significant abnormality.
• Able to provide written informed consent
• Females of childbearing potential must: have a negative pregnancy test at screening agree to not become pregnant or breastfeed for the period of the study through 1 month after completion of the study be willing to use a reliable form of contraception during the study.
• Has healthy skin on the residual limb and is free of severe and active skin disease (excluding those on the residual limb) such as extreme and active eczema or psoriasis, active non-healing wounds, lichen planus, a history of keloid scare formation or lupus as determined by the investigator or study nurse.
• Willing and able to comply with the scheduled visits, biopsy/injection procedures, wound care instructions treatment plan, and other study procedures for the duration of the study.

You may not qualify if:

• A skin erosion deeper than the skin dermis.
• Ulcers exceeding 4cm in diameter or 2cm in radius
• Non circular ulcers that cannot tolerate a 1cm margin at all sides
• Having received any investigational drug within 30 days prior to study entry
• An allergy history to any study materials including local anesthetic, dimethyl sulfoxide, human albumin, or bovine constituents, hetastarch, or EMLA (lidocaine 2.5% and prilocaine 2.5%).
• Pregnant, lactating, or trying to become pregnant
• A history of keloid formation
• Having a significant medical history that the investigator feels is not safe for study participation (for example, some forms of autoimmune conditions, metastatic cancer, infectious diseases such as HIV, HTLV I/II, Hepatitis B, Hepatitis C).
• Autoimmune diseases affecting the skin such as lupus.
• Presence of necrotic ischemic tissue on any stump ulcers, and/or capillary refill on stump skin of greater than 3 seconds
• Active infection of the residual limb (e.g. osteomyelitis, wound or skin condition with surrounding swelling, erythema, drainage, pain, gangrenous tissue or fever).
• Active smoker during the study (this includes e cigarettes and any type of tobacco use)
• We will also exclude those who are on chronic immunosuppressive therapies such as oral steroids, but also those on chronic topical steroids in the area of investigation.
• Amputees with symptomatic neuromas of the terminal limb within the last 3 months.
• Known bleeding disorder.

Sponsors & Collaborators

• Major Extremity Trauma Research Consortium: lead
• United States Department of Defense: collaborator

Study Sites (1)

Johns Hopkins Hospital

Baltimore, Maryland, 21287, United States

RECRUITING

Central Study Contacts

Dana Alkhoury

CONTACT

410- 955-7498; dalkhou1@jhu.edu

Luis Garza, MD

CONTACT

lag@jhmi.edu

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: PARTICIPANT
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: Cross over design

Study Record Dates

• First Submitted: March 23, 2021
• First Posted: April 9, 2021
• Study Start: September 26, 2022
• Primary Completion (Estimated): August 1, 2026
• Study Completion (Estimated): January 1, 2027
• Last Updated: October 14, 2025

Record last verified: 2025-10

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)