RhPDGF-BB for Perianal Fistula Healing

Overseen By Cyndi R Clark, PhD

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Alexander Hawkins

Disqualifiers: Genito-urinary fistulization, Major GI surgery, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 7

Duration: 6 months

12 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores a new treatment for healing complex perianal fistulas, which are abnormal connections between the rectum and outside skin. The treatment uses RhPDGF-BB, a growth factor that aids wound healing, delivered through a special collagen plug. The goal is to enhance healing and reduce the likelihood of fistulas reopening, particularly for those with Crohn's disease or unidentified causes. Participants should have a single draining fistula and not be suitable for a common surgery called fistulotomy. As a Phase 2 trial, this research focuses on measuring the treatment's effectiveness in an initial, smaller group of people.

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.

Is there any evidence suggesting that rhPDGF-BB is likely to be safe for humans?

Previous research has shown that recombinant human platelet-derived growth factor-BB (rhPDGF-BB) is safe when combined with other treatments. These studies suggest that rhPDGF-BB is well-tolerated in therapies for wound healing and tissue repair. Reports of serious side effects are rare, indicating a positive safety profile. Additionally, rhPDGF-BB is already used to treat conditions such as healing diabetic foot ulcers and aiding bone regrowth, further supporting its safety in humans. For those considering joining a trial with this treatment, the existing evidence reassures about its safety. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for perianal fistulas?

Most treatments for perianal fistulas, such as surgery or seton placement, focus on managing symptoms and promoting drainage. But rhPDGF-BB acts differently, targeting the healing process at a cellular level. rhPDGF-BB is a growth factor that enhances tissue repair by stimulating cell growth and new blood vessel formation. Researchers are excited about this treatment because it has the potential to improve healing rates and reduce recurrence by directly promoting the body's natural repair mechanisms. This innovative approach could offer a more effective and less invasive option for patients.

What evidence suggests that rhPDGF-BB might be an effective treatment for perianal fistulas?

Research has shown that recombinant human platelet-derived growth factor BB (rhPDGF-BB) significantly aids tissue healing and repair. This growth factor has already been used successfully to treat diabetic foot sores and to promote bone healing in dental and bone surgeries. Studies have found that it enhances healing when combined with various graft materials. In this trial, participants will receive either routine care or a collagen plug saturated with rhPDGF-BB. For complex perianal fistulas, which typically have a healing success rate of about 50%, rhPDGF-BB could increase this rate by enhancing tissue repair. This treatment may also prevent the fistula from reopening, a common issue with standard surgery. ^{1 3 6 7 8}

Who Is on the Research Team?

Alexander T Hawkins

Principal Investigator

Vanderbilt University Medical Center

Are You a Good Fit for This Trial?

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

Stated willingness to comply with all study procedures and availability for the duration of the study

I currently have a draining seton in place for my fistula.

Willing and able to provide informed consent and to comply with study protocol and follow-up

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.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Baseline Procedure

Participants are randomized and scheduled for the baseline procedure, including seton placement and initial assessment

1 week

1 visit (in-person)

Treatment

Participants receive either the rhPDGF-BB-enhanced collagen plug or routine care, followed by assessments at 1, 3, and 6 months

6 months

3 visits (in-person) at 1, 3, and 6 months

Follow-up

Participants are monitored for safety and effectiveness after treatment, including assessments for fistula healing and recurrence

6 months

3 visits (in-person) at 1, 3, and 6 months

Open-label Extension (optional)

Participants in the control arm with non-healing fistulas at 3 months may crossover to receive the investigational intervention

3 months

What Are the Treatments Tested in This Trial?

Interventions

• RhPDGF-BB

Trial Overview 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.

How Is the Trial Designed?

Group I: rhPDGF-BB-enhanced collagen plug Experimental Treatment 1 Intervention

Participants receive collagen plug saturated with rhPDGF-BB.

Group II: Routine care Active Control 1 Intervention

Participants receive routine care for their fistula.

RhPDGF-BB is already approved in United States, European Union for the following indications:

Approved in United States as rhPDGF-BB for:

• Diabetic foot ulcers
• Periodontal bone regeneration
• Orthopedic bone regeneration

Approved in European Union as rhPDGF-BB for:

• Diabetic foot ulcers
• Periodontal bone regeneration

Who Is Running the Clinical Trial?

Alexander Hawkins

Lead Sponsor

Trials

1

Recruited

10+

Lynch Regenerative Medicine, LLC

Collaborator

Trials

2

Recruited

50+

Published Research Related to This Trial

Recombinant platelet-derived growth factor (rPDGF-BB) significantly enhances granulation tissue migration in avascular ulcers, with increases measured at 1.88 mm and 3.00 mm by days 5 and 7, respectively, compared to control treatments.

The combination of rPDGF-BB with a hyaluronic acid carrier and an intact vascular pedicle further boosts tissue generation and is essential for the survival of skin grafts, highlighting the importance of blood supply in effective wound healing.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Treatment of avascular ulcers with cytokine-induced tissue generation and skin grafting. Brown, DM., Chung, SH., Pasia, EN., et al.[2003]

Recombinant human platelet-derived growth factor-BB (rhPDGF) is safe for use in oral regenerative procedures, with no serious adverse effects reported in 63 studies involving a mean follow-up of 10.7 months.

rhPDGF shows significant clinical benefits when combined with bone allografts, xenografts, or alloplasts for treating periodontal defects and gingival recession, as well as for guided bone regeneration and alveolar ridge preservation.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Recombinant Human Platelet-Derived Growth Factor: A Systematic Review of Clinical Findings in Oral Regenerative Procedures. Tavelli, L., Ravidà, A., Barootchi, S., et al.[2021]

Recombinant human platelet derived growth factor BB (rhPDGF-BB) expressed in tobacco plants showed a significant increase in fibroblast proliferation (4.5-fold) compared to non-transgenic plants, indicating its potential efficacy in promoting wound healing.

The study confirmed that plant-based production of rhPDGF-BB retains its biological activity, as it not only enhanced cell proliferation but also induced chemotaxis, suggesting therapeutic benefits for treating diabetic neuropathic ulcers.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Transgenic expression and functional characterization of human platelet derived growth factor BB (hPDGF-BB) in tobacco (Nicotiana tabacum L.). Deepa, K., Rodionov, RN., Weiss, N., et al.[2023]

Recombinant human platelet-derived growth factor-BB (rhPDGF-BB) has been shown to be biocompatible and safe, with no systemic or local toxicity observed in extensive testing, making it a promising option for enhancing wound healing.

The study evaluated the effects of rhPDGF-BB alone and in combination with beta-tricalcium phosphate or collagen, confirming its safety in various applications related to tissue repair and inflammation.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Preclinical Toxicology Studies of Recombinant Human Platelet-Derived Growth Factor-BB Either Alone or in Combination with Beta-Tricalcium Phosphate and Type I Collagen. Young, CS., Bradica, G., Hart, CE., et al.[2021]

The study involved 9 adult patients with advanced periodontitis and demonstrated that using purified recombinant human platelet-derived growth factor BB (rhPDGF-BB) mixed with bone allograft led to significant periodontal regeneration, including new bone, periodontal ligament, and cementum in both interproximal intrabony defects and Class II furcation lesions.

There were no safety concerns during the study, and the treatment resulted in substantial clinical improvements, with an average clinical attachment level gain of 6.17 mm and a vertical probing depth reduction of 6.42 mm in interproximal defects, indicating effective wound healing and regeneration.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Periodontal regeneration in humans using recombinant human platelet-derived growth factor-BB (rhPDGF-BB) and allogenic bone. Nevins, M., Camelo, M., Nevins, ML., et al.[2022]

Platelet-derived growth factor (PDGF) plays a crucial role in wound healing and bone repair, leading to the FDA approval of recombinant PDGF-BB (rhPDGF-BB) for treating chronic diabetic foot ulcers and periodontal bone defects.

Recent clinical trials suggest that rhPDGF-BB, when combined with beta tricalcium phosphate, may effectively replace autogenous bone grafts in hindfoot and ankle arthrodesis, highlighting its potential in bone regeneration.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The evolution of rhPDGF-BB in musculoskeletal repair and its role in foot and ankle fusion surgery. DiGiovanni, CW., Petricek, JM.[2018]

Recombinant human platelet-derived growth factor (rhPDGF-BB) combined with bone graft materials can effectively regenerate periodontal tissue by promoting cellular migration and proliferation, leading to improved healing outcomes.

The use of rhPDGF-BB not only accelerates soft tissue healing at surgical sites but also allows for quicker prosthetic care and earlier resumption of plaque control by patients, enhancing overall recovery.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Using recombinant platelet-derived growth factor to facilitate wound healing. Rosen, PS.[2018]

In a study involving 12 patients with 14 refractory chronic ulcers, 64% of the ulcers healed after treatment with topical rhPDGF gel 0.01%, with an average healing time of 26 weeks.

The treatment was found to be effective and well-tolerated, although 22% of the healed ulcers reopened during a 15-month follow-up, indicating the need for ongoing monitoring after healing.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Recombinant human platelet-derived growth factor for refractory nondiabetic ulcers: a retrospective series. Harrison-Balestra, C., Eaglstein, WH., Falabela, AF., et al.[2022]

In a 36-month study involving 135 participants with localized severe periodontal defects, the use of recombinant human platelet-derived growth factor (rhPDGF-BB) significantly improved clinical outcomes, with 87% of patients in the 0.3 mg/mL group achieving a composite success benchmark compared to only 53.8% in the control group.

The study demonstrated that rhPDGF-BB treatment leads to long-term stability in clinical attachment levels and bone growth, indicating its effectiveness in promoting periodontal regeneration over an extended period.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Platelet-derived growth factor promotes periodontal regeneration in localized osseous defects: 36-month extension results from a randomized, controlled, double-masked clinical trial. Nevins, M., Kao, RT., McGuire, MK., et al.[2022]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06632418

Evaluating an RhPDGF-BB-enhanced Collagen Plug for ... The average success rate for healing and remission of complex perianal fistulas, idiopathic or Crohn's-related, is approximately 50%.

2. journals.lww.com journals.lww.com/plasreconsurg/abstract/9900/_pure_recombinant_platelet_derived_growth_factor.2779.aspx

Pure Recombinant Platelet-Derived Growth Factor in Tissue... Pure recombinant platelet-derived growth factor BB (PDGF-BB) enhances healing in multiple tissues and is the most thoroughly studied growth ...

3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC7961612/

Recombinant Human Platelet–Derived Growth Factor Use of rhPDGF was shown to be beneficial when combined with allografts, xenografts, and alloplasts (the latter tricalcium phosphate [β-TCP]) for the treatment ...

4. synapse.patsnap.com synapse.patsnap.com/drug/d52d39255eac4e189d4ca13becd52661

Recombinant Human Platelet-derived Growth Factor B ... Results: An 8‐month cone beam computed tomography assessment showed a 6.2 mm vertical bone gain and complete implant coverage. Conclusion: This approach ...

5. digitalcommons.library.uab.edu digitalcommons.library.uab.edu/cgi/viewcontent.cgi?article=1565&context=etd-collection

A Comparison of Growth Factor Release Between Platelet ... Because physical properties were not affected in this study, the addition of. rhPDGF-BB to the bone graft prior to making sticky bone with β-TCP and PRF, may.

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC3324841/

Safety of recombinant human platelet-derived growth factor ... The safety data presented provide evidence that recombinant human platelet-derived growth factor-BB is a safe therapeutic when used in combination products ...

7. lynchregen.com lynchregen.com/wp-content/uploads/2025/09/RPT-001v1.0-A-Review-of-the-Safety-of-Exogenously-Applied-rhPDGF-vFinal.pdf?fbclid=PARlRTSAMtIsBleHRuA2FlbQIxMQABp6pLgHxdqMEsxeNEPS6PCt81y8w1Y942lun5IPYoLbARtrXDqsWfRw8lD-8w_aem_AQ5_jRdBeYLDqY0PBx6e2A

A Review of the Safety of Exogenously Applied ... ABSTRACT: • Recombinant human platelet-derived growth factor-BB (rhPDGF-BB) has been extensively studied for safety and efficacy in tissue repair and ...

8. clinconnect.io clinconnect.io/trials/NCT06632418

Evaluating an RhPDGF-BB-enhanced Collagen Plug for The average success rate for healing and remission of complex perianal fistulas, idiopathic or Crohn's-related, is approximately 50%.