Photodynamic Therapy for Basal Cell Carcinoma

Not currently recruiting at 18 trial locations

Overseen By Matthias Lübbert

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: Biofrontera Bioscience GmbH

Must not be taking: Immunosuppressants, Cytotoxic drugs

Disqualifiers: Porphyria, Cardiovascular disease, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

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

Approved in 3 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: up to 6 months

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called photodynamic therapy (PDT) for superficial basal cell carcinoma (BCC). The goal is to evaluate the effectiveness and safety of PDT compared to a placebo. The treatment involves applying a special cream (BF-200 ALA, also known as Ameluz®) and using a special lamp. Individuals with at least one untreated BCC lesion on areas such as the face, neck, or arms, but not in challenging spots like near the ears, may be suitable candidates. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants the opportunity to contribute to a potentially groundbreaking treatment.

Will I have to stop taking my current medications?

The trial protocol does not specify if you must stop taking your current medications, but certain medications are restricted. You cannot start new medications with phototoxic or photoallergic potential, and some systemic treatments like immunosuppressive therapies and corticosteroids must be stopped for a specific period before and during the trial. It's best to discuss your current medications with the trial team to see if any adjustments are needed.

Is there any evidence suggesting that this treatment is likely to be safe for humans?

Research shows that BF-200 ALA (Ameluz®), used in light-based therapy, is generally safe for treating basal cell carcinoma (BCC). Studies have found that most patients respond well to this treatment. In one study, over 93% of patients treated with BF-200 ALA experienced significant improvements.

The treatment is considered safe, with fewer cancer spots returning compared to those who received a placebo. The skin's appearance after treatment also improved with BF-200 ALA.

While some side effects may occur, as with any treatment, current evidence suggests that BF-200 ALA is well-tolerated by most people. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for basal cell carcinoma?

Unlike standard treatments for basal cell carcinoma, which often involve surgical methods or topical medications like imiquimod and fluorouracil, BF-200 ALA (Ameluz®) offers a non-invasive approach with photodynamic therapy (PDT). This treatment utilizes a specially formulated topical application of 5-aminolevulinic acid (5-ALA) that, when activated by light, selectively targets and destroys cancer cells. Researchers are excited about BF-200 ALA because its targeted mechanism may reduce damage to surrounding healthy tissue and potentially offer a quicker recovery time compared to traditional treatments.

What evidence suggests that photodynamic therapy might be an effective treatment for basal cell carcinoma?

Research has shown that BF-200 ALA (Ameluz®) with light therapy effectively treats superficial basal cell carcinoma (BCC). One study found a recurrence rate of 14.1% with BF-200 ALA, significantly lower than the 27.4% with a placebo, demonstrating its effectiveness. Patients reported high satisfaction with their skin's appearance after treatment. Another study confirmed the treatment's safety and effectiveness, performing comparably to another light therapy treatment. These findings suggest that BF-200 ALA is a promising option for those with superficial BCC. Participants in this trial will receive either BF-200 ALA or a placebo vehicle as part of the study's treatment arms. ^{1 2 3 6 7}

Who Is on the Research Team?

David M. Pariser

Principal Investigator

Virginia Clinical Research, Inc.

Are You a Good Fit for This Trial?

Adults with superficial basal cell carcinoma (sBCC) who can sign consent forms, have a lesion size suitable for treatment without skin grafting, and are willing to avoid certain cosmetics and sun exposure. They must not have hypersensitivity to porphyrins or other exclusion criteria like photodermatoses, coagulation defects, recent use of certain drugs, or unstable medical conditions.

Inclusion Criteria

I am willing and able to sign the consent and HIPAA forms for this study.

My skin cancer lesion fits within the treatment light area.

I have at least one new skin lesion likely to be basal cell carcinoma not previously treated or in sensitive areas.

Exclusion Criteria

I have a skin condition that worsens with light exposure.

I have not treated skin tumors near my target lesion with surgery or creams recently.

Any of the systemic treatments listed below, within the designated period prior to PDT and during the clinical observation period. Interferon - 6 weeks. Immuno modulators or immunosuppressive therapies - 12 weeks. Cytotoxic drugs - 6 months. Investigational drugs - 8 weeks. Drugs known to have major organ toxicity - 8 weeks. Corticosteroids (oral or injectable) - 6 weeks. MAL or ALA - 12 weeks. Systemic treatment with NSAIDs is not to be used 7 days prior to and 7 days after PDT. ASA (e.g. Aspirin®) up to 100 mg/ day, ibuprofen up to 200 mg/ day, and acetaminophen (e.g. Tylenol®) is allowed during this period.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

up to 4 weeks

Treatment

Participants receive photodynamic therapy (PDT) with BF-200 ALA or placebo, consisting of up to two PDT cycles with two sessions each, one to two weeks apart.

up to 6 months

Multiple visits for PDT sessions and assessments

Follow-up

Participants are monitored for safety and effectiveness after treatment, with a focus on clinical and histological clearance of lesions.

5 years

What Are the Treatments Tested in This Trial?

Interventions

• BF-200 ALA (Ameluz®)

Trial Overview The trial is testing the effectiveness of Ameluz® with BF-RhodoLED® lamp in treating sBCC compared to a placebo therapy. Participants will undergo up to four PDT sessions within 3.5 months and then have the main target lesion removed for evaluation.

How Is the Trial Designed?

Group I: BF-200 ALA Experimental Treatment 1 Intervention

Topical application of BF-200 ALA containing 7.8% 5-ALA (5-aminolevulinic acid). Photodynamic therapy (PDT)

Group II: Vehicle Placebo Group 1 Intervention

Topical application of vehicle to BF-200 ALA containing no active ingredient. Photodynamic therapy (PDT)

BF-200 ALA (Ameluz®) is already approved in European Union, United Kingdom, United States for the following indications:

Approved in European Union as Ameluz for:

• Actinic keratosis of mild to moderate intensity on the face and scalp
• Actinic keratosis on extremities and trunk/neck

Approved in United Kingdom as Ameluz for:

• Actinic keratosis of mild to moderate intensity on the face and scalp
• Actinic keratosis on extremities and trunk/neck

Approved in United States as Ameluz for:

• Actinic keratosis of mild to moderate severity on the face and scalp

Who Is Running the Clinical Trial?

Biofrontera Bioscience GmbH

Lead Sponsor

Trials

12

Recruited

1,900+

Published Research Related to This Trial

In a study involving 54 patients with 95 non-aggressive basal cell carcinomas (BCCs), photodynamic therapy (PDT) using low-concentration hexyl aminolevulinate (HAL) showed similar histological clearance rates (87.9%) compared to methyl aminolevulinate (MAL) (93.8%) and 5-aminolevulinic acid nanoemulsion (BF-200ALA) (90.9%), indicating HAL is an effective alternative.

All treatment options (HAL, MAL, and BF-200 ALA) demonstrated comparable tolerability in terms of pain, post-treatment reactions, and cosmetic outcomes, suggesting that HAL can be a safe and effective choice for PDT in treating BCCs.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Hexyl aminolevulinate, 5-aminolevulinic acid nanoemulsion and methyl aminolevulinate in photodynamic therapy of non-aggressive basal cell carcinomas: A non-sponsored, randomized, prospective and double-blinded trial. Salmivuori, M., Grönroos, M., Tani, T., et al.[2021]

In a clinical study involving 19 patients with 55 superficial basal cell carcinomas, 85% of the tumors showed a complete response to photodynamic therapy using a novel nanocolloid lotion of 5-aminolevulinic acid (5-ALA) after just one treatment.

The treatment was well tolerated without the need for local anesthetics, causing only slight discomfort during the light irradiation, indicating its safety and convenience for patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Photodynamic therapy of superficial basal cell carcinomas using topical 5-aminolevulinic acid in a nanocolloid lotion. Hürlimann, AF., Hänggi, G., Panizzon, RG.[2018]

In a study involving four patients with seven superficial basal cell carcinomas (sBCCs), treatment with 5-aminolevulinate (ALA) patches resulted in a complete response for all lesions by week 24, indicating strong efficacy.

The ALA patches demonstrated an excellent safety profile, suggesting they could be a promising treatment option for sBCCs, although they are currently FDA and EMA approved only for grade I to II actinic keratosis.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Photodynamic therapy with 5-aminolevulinate patch for the treatment of superficial basal cell carcinomas. De Luca, EV., Tambone, S., Catapano, S., et al.[2021]

The nanoemulsion-based BF-200 ALA gel enhances the skin's absorption of aminolevulinic acid (ALA) for effective photodynamic therapy (PDT) in treating actinic keratosis, with minimal systemic absorption observed in pharmacokinetic trials involving patients with severe cases.

After applying BF-200 ALA, plasma levels of ALA peaked at 2.5 to 3.3 times above baseline within 3 hours and returned to baseline within 10 hours, indicating that only a small fraction of the drug is absorbed systemically, which aligns with its known safety profile.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Clinical Pharmacokinetics and Safety of a 10% Aminolevulinic Acid Hydrochloride Nanoemulsion Gel (BF-200 ALA) in Photodynamic Therapy of Patients Extensively Affected With Actinic Keratosis: Results of 2 Maximal Usage Pharmacokinetic Trials. Novak, B., DuBois, J., Chahrour, O., et al.[2022]

In a pilot study involving 24 patients with 112 superficial basal cell carcinomas (BCC), both 10% ALA and methyl-ALA (mALA) thermogels showed a 60% success rate in treating tumors after the first session.

Despite the expectation that mALA would provide better penetration and therapeutic outcomes, the results indicated no significant difference in efficacy between mALA and ALA treatments, suggesting further research is needed to confirm these findings.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Comparison of aminolevulinic acid (ALA)-thermogel-PDT with methyl-ALA-thermogel-PDT in basal cell carcinoma. Schleier, P., Berndt, A., Kolossa, S., et al.[2022]

Aminolevulinic acid photodynamic therapy (ALA-PDT) is a safe and effective noninvasive treatment for superficial basal cell carcinoma (BCC) and Bowen's disease, offering excellent cosmetic results with minimal scarring.

ALA-PDT is not suitable for aggressive BCC subtypes or invasive squamous cell carcinoma, but it may help prevent new skin cancers in high-risk patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Aminolevulinic acid photodynamic therapy for skin cancers. Blume, JE., Oseroff, AR.[2013]

Citations

1. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/29432644/

A randomized, multinational, noninferiority, phase III trial to ... Treatment of nonaggressive BCC with BF-200 ALA-PDT is highly effective and well tolerated with proven noninferiority to MAL-PDT.

2. clinicaltrials.gov clinicaltrials.gov/study/NCT03573401

Study to Evaluate the Safety and Efficacy of BF-200 ALA ... The study will be conducted as randomized, double blind and vehicle-controlled ( 4:1 ratio of verum (BF-200 ALA; Ameluz®) to vehicle (placebo)) clinical trial ...

3. jaad.org jaad.org/article/S0190-9622(21)00575-2/pdf

Red light photodynamic therapy with BF-200 ALA showed ... One-year overall lesion recurrence rate was 14.1% versus 27.4% (BF-200 ALA vs vehicle; P = .0068). Patients were more satisfied by the cosmetic ...

4. aaem.pl aaem.pl/Efficacy-of-photodynamic-therapy-using-ALAHCl-in-gel-with-a-lipid-nanoemulsion-and,183059,0,2.html

Efficacy of photodynamic therapy using ALAHCl in gel with ... The aim of the study was to assess the efficacy of topical PDT in the treatment of superficial BCC (sBCC) using two different photosensitizers.

5. southcarolinablues.com southcarolinablues.com/web/public/brands/medicalpolicyhb/external-policies/dermatologic-applications-of-photodynamic-therapy/

Dermatologic Applications of Photodynamic Therapy The trial results showed PDT with BF-200 ALA was superior to placebo PDT ... basal cell carcinoma: results of a multicenter randomized prospective trial.

6. researchgate.net researchgate.net/publication/296193574_A_randomized_double-blind_phase_III_multi-centre_study_to_evaluate_the_safety_and_efficacy_of_BF-200_ALA_AmeluzR_versus_placebo_in_the_field-directed_treatment_of_mild_to_moderate_actinic_keratosis_wi

A randomized, double-blind, phase III, multi-centre study to ... Conclusions The FUP data confirmed the high efficacy and safety of PDT with BF-200 ALA. The slightly lower recurrence rates after BF-200 ALA treatment ...

7. onlinelibrary.wiley.com onlinelibrary.wiley.com/doi/10.1111/bjd.14498

A randomized, double‐blind, phase III, multicentre study to ... The cosmetic outcome was improved in the BF-200 ALA group compared with placebo. Conclusions. Field-directed therapy with BF-200 ALA and BF- ...