Age: 18+
Sex: Any
Travel: May be covered
Time Reimbursement: Varies
Trial Phase: Phase 2
Recruiting
Sponsor: Alexander Hawkins
No Placebo Group
Prior Safety Data
Approved in 2 jurisdictions
Trial Summary
What is the purpose of this trial?The average success rate for healing and remission of complex perianal fistulas, idiopathic or Crohn's-related, is approximately 50%. These abnormal connections between the rectum and the outside skin remain a major clinical challenge in need of new treatments aimed at tissue repair. Platelet-derived growth factor drives wound healing and tissue regeneration, and manufactured PDGF is currently used to heal diabetic foot ulcers and regenerate bone in periodontal and orthopedic patients. Manufactured recombinant human PDGF has the potential to improve the success rate for complete healing of complex perianal fistulas, reduce the recurrence rate due to reopening of the fistula tract, and avoid complications associated with routine surgical interventions.
Will I have to stop taking my current medications?
The trial protocol does not specify whether you need to stop taking your current medications. However, if you are currently enrolled in another drug or device trial, you must wait 30 days after the last investigational treatment before participating in this trial.
What data supports the effectiveness of the treatment RhPDGF-BB for perianal fistula healing?
Research shows that RhPDGF-BB is effective in promoting wound healing and tissue regeneration in various conditions, such as periodontal defects and chronic diabetic foot ulcers, by stimulating cell growth and tissue repair.
12345Is RhPDGF-BB safe for use in humans?
Research shows that RhPDGF-BB is generally safe for humans, with studies indicating it is biocompatible and has no systemic or local toxicity when used alone or with other materials, supporting its safe use in wound healing.
16789How is the drug RhPDGF-BB unique in treating perianal fistulas?
RhPDGF-BB is unique because it is a growth factor that stimulates the healing process by promoting the growth and movement of cells needed for tissue repair, which is different from traditional treatments that may not directly enhance cellular regeneration.
12357Eligibility Criteria
This trial is for individuals with complex perianal fistulas, which can be idiopathic or related to Crohn's disease. Participants should have a history of these fistulas that are difficult to heal and may have tried other treatments without success.Inclusion Criteria
I have a type of anal fistula that cannot be treated with simple surgery.
I currently have a draining seton in place for my fistula.
I am over 21 years old.
Exclusion Criteria
I am not pregnant, breastfeeding, nor planning to become pregnant during the trial.
I have cancer at the site where the treatment will be applied.
I have an abnormal connection between my genital and urinary tracts.
I have an ileal anal pouch.
My condition involves specific types of abnormal connections between organs.
I have an infection where the treatment would be applied.
I have had radiation therapy on the area being considered for the trial.
Participant Groups
The study is testing RhPDGF-BB, a manufactured protein known to aid in wound healing and tissue regeneration. It's being used in the form of an enhanced collagen plug inserted into the fistula tract to promote healing.
2Treatment groups
Experimental Treatment
Active Control
Group I: rhPDGF-BB-enhanced collagen plugExperimental Treatment1 Intervention
Participants receive collagen plug saturated with rhPDGF-BB.
Group II: Routine careActive Control1 Intervention
Participants receive routine care for their fistula.
RhPDGF-BB is already approved in United States, European Union for the following indications:
๐บ๐ธ Approved in United States as rhPDGF-BB for:
- Diabetic foot ulcers
- Periodontal bone regeneration
- Orthopedic bone regeneration
๐ช๐บ Approved in European Union as rhPDGF-BB for:
- Diabetic foot ulcers
- Periodontal bone regeneration
Find A Clinic Near You
Research locations nearbySelect from list below to view details:
Vanderbilt University Medical CenterNashville, TN
Loading ...
Who is running the clinical trial?
Alexander HawkinsLead Sponsor
Lynch Regenerative Medicine, LLCCollaborator
References
Periodontal regeneration in humans using recombinant human platelet-derived growth factor-BB (rhPDGF-BB) and allogenic bone. [2022]Purified recombinant human platelet-derived growth factor BB (rhPDGF-BB) is a potent wound healing growth factor and stimulator of the proliferation and recruitment of both periodontal ligament (PDL) and bone cells. The hypothesis tested in this study was that application of rhPDGF-BB incorporated in bone allograft would induce regeneration of a complete new attachment apparatus, including bone, periodontal ligament, and cementum in human interproximal intrabony defects and molar Class II furcation lesions.
Using recombinant platelet-derived growth factor to facilitate wound healing. [2018]Recombinant human platelet-derived growth factor (rhPDGF-BB) is a bioactive protein that has been combined with bone graft materials in an effort to regenerate the periodontium to its prediseased state more predictably. The combination of a biocompatible scaffold with this highly purified, recombinant human growth factor capitalizes on PDGF's stimulatory effects on cellular migration and proliferation, with subsequent matrix formation, to drive the healing process toward regeneration. When using a growth factor such as rhPDGF-BB with broad wound healing activity, an added clinical benefit is the more rapid soft tissue wound healing pattern observed at the surgical site. This also may be seen as a patient-centered benefit because it allows dentists to provide for a patient's prosthetic care on a timely basis, leading to a faster return to normal function. Also, the patient may resume plaque control efforts earlier, which further facilitates the wound-healing process. The purpose of these 2 case reports is to document the use of rhPDGF-BB as a wound-healing agent in 2 clinical situations and to provide a rationale for its use in this capacity.
Treatment of avascular ulcers with cytokine-induced tissue generation and skin grafting. [2003]Recombinant platelet-derived growth factor (rPDGF-BB) stimulates migration and proliferation of fibroblasts and smooth muscle cells and induces the rapid development of granulation tissue. This study investigated the use of rPDGF-BB and skin grafting to heal avascular ulcers using a rabbit model. We further investigated the effect of a hyaluronic acid carrier and a vascular pedicle on rPDGF-induced tissue generation in this model.
Platelet-derived growth factor promotes periodontal regeneration in localized osseous defects: 36-month extension results from a randomized, controlled, double-masked clinical trial. [2022]Recombinant human platelet-derived growth factor (rhPDGF) is safe and effective for the treatment of periodontal defects in short-term studies up to 6 months in duration. We now provide results from a 36-month extension study of a multicenter, randomized, controlled clinical trial evaluating the effect and long-term stability of PDGF-BB treatment in patients with localized severe periodontal osseous defects.
The evolution of rhPDGF-BB in musculoskeletal repair and its role in foot and ankle fusion surgery. [2018]Platelet-derived growth factor (PDGF) is one of the most thoroughly studied proteins in the body. Research has progressively highlighted the role of PDGF during wound healing and in the bone repair cascade. This research has resulted in FDA approval of 2 products containing a recombinant version of the protein, rhPDGF-BB, for treating chronic diabetic foot ulcers and periodontal bone defects. This article reviews the applicable basic science and mechanisms of action of PDGF, with attention to the increasingly defined role of rhPDGF-BB in initiating bone regeneration. The most recent data from prospective clinical trials evaluating the use of rhPDGF-BB in combination with beta tricalcium phosphate as a substitute for autogenous bone graft in hindfoot and ankle arthrodesis are also summarized.
Preclinical Toxicology Studies of Recombinant Human Platelet-Derived Growth Factor-BB Either Alone or in Combination with Beta-Tricalcium Phosphate and Type I Collagen. [2021]Human platelet-derived growth factor-BB (hPDGF-BB) is a basic polypeptide growth factor released from platelets at the injury site. It is a multifunctional molecule that regulates DNA synthesis and cell division and induces biological effects that are implicated in tissue repair, atherosclerosis, inflammatory responses, and neoplastic diseases. This paper is an overview of the toxicology data generated from a broad testing platform to determine bone, soft tissue, and systemic responses following administration of rhPDGF-BB. Moreover, the systemic and local toxicity of recombinant human PDGF-BB (rhPDGF-BB) in combination with either beta-tricalcium phosphate (ฮฒ-TCP) or collagen combined with ฮฒ-TCP was studied to determine dermal sensitization, irritation, intramuscular tissue responses, pyrogenicity, genotoxicity, and hemolytic properties. All data strongly suggest that rhPDGF-BB either alone or in combination with ฮฒ-TCP or collagen with ฮฒ-TCP is biocompatible and has neither systemic nor local toxicity, supporting its safe use in enhancing wound healing in patients.
Recombinant Human Platelet-Derived Growth Factor: A Systematic Review of Clinical Findings in Oral Regenerative Procedures. [2021]The use of recombinant human platelet-derived growth factor-BB (rhPDGF) has received Food and Drug Administration approval for the treatment of periodontal and orthopedic bone defects and dermal wound healing. Many studies have investigated its regenerative potential in a variety of other oral clinical indications. The aim of this systematic review was to assess the efficacy, safety, and clinical benefit of recombinant human platelet-derived growth factor (rhPDGF) use for alveolar bone and/or soft tissue regeneration.
Recombinant human platelet-derived growth factor for refractory nondiabetic ulcers: a retrospective series. [2022]Recombinant human platelet-derived growth factor-BB (rhPDGF-BB or rhPDGF gel 0.01%) has been approved by the U.S. Food and Drug Administration (FDA) for topical use on lower extremity, neuropathic, diabetic ulcers to enhance healing and has been reported to be effective in expediting the healing of full-thickness chronic pressure ulcers. Published clinical data using rhPDGF gel to treat nondiabetic and nonpressure-related ulcers have been limited.
Transgenic expression and functional characterization of human platelet derived growth factor BB (hPDGF-BB) in tobacco (Nicotiana tabacum L.). [2023]Recombinant human platelet derived growth factor BB (rhPDGF-BB) is clinically approved for treating diabetic neuropathic ulcers. Plant-based expression systems offer less expensive ways of producing recombinant drugs, which do not require purification for clinical use. From this perspective, rhPDGF-BB is an ideal candidate for expression in plants as it can be applied topically. Here, we report a proof of concept study, in which rhPDGF-BB was expressed in tobacco plants, and its biological activity was tested in vitro. The mature human platelet derived growth factor BB (hPDGF-BB) gene was codon-optimized for tobacco and fused with ER targeting and retention signals, 5' and 3' UTRs of arc5-1 gene along with CaMV 35S promoter, and then, transferred by Agrobacterium-mediated transformation. Gene and protein expression of hPDGF-BB were confirmed by PCR and immunoblot studies. Bioactivity of hPDGF-BB expressed protein was determined by in vitro assays such as proliferation and migration in NIH3T3 cells. Our data reveals that total soluble proteins containing hPDGF-BB from transgenic plants showed a 4.5-fold increase in fibroblast proliferation compared to non-transgenic plants. Furthermore, plant-made rhPDGF-BB induced chemotaxis of treated cells and promoted wound healing in vitro. These results clearly demonstrate that functionally active rhPDGF-BB protein can be produced in plants and might have therapeutic benefits.