Basal Cell Carcinoma (BCC)Phase 3NCT02144077
Safety and Efficacy Study for the Treatment of Non-Aggressive Basal Cell Carcinoma With Photodynamic Therapy
A Phase 3 study testing BF-200 ALA and methyl-aminolevulinate in people with Basal Cell Carcinoma (BCC), run by Biofrontera Bioscience GmbH at 1 site in Germany.
Trial summary
The aim of this study is to test the effectiveness and safety of the medicine Ameluz® (5-aminolevulinic acid) in comparison to methyl-aminolevulinate (MAL), used with photodynamic therapy (PDT), to treat thin, non-aggressive BCC (basal cell carcinoma). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Basal Cell Carcinoma (BCC)
- What is being tested?
- BF-200 ALA and methyl-aminolevulinate
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 281 participants
- Where
- 1 siteGermany
- Study dates
- 28 Jan 2014to 9 Sep 2020
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Main Inclusion Criteria:
- Willing and able to sign informed consent form; obtained in writing before starting any study procedures
- Presence of 1-3 thin (≤2 mm thickness), clinically non-aggressive, primary BCC lesions (primary superficial, nodular, or mixed superficial/nodular) in the face/forehead, bald scalp, extremities and/or neck/trunk. Confirmation of non-aggressiveness and thickness of BCC through biopsies taken at screening for at least one lesion. Lesions non-eligible according to biopsy should timely be removed by surgery or cryotherapy
- Diameters of lesions should range between ≥0.5cm and ≤2cm; total maximal treated area is 10cm² (including 0.5-1.0cm margin surrounding each lesion)
- Target BCC lesions must be discrete and quantifiable and have to be located within 1-2 treatment areas
- Free of significant physical abnormalities (eg tattoos, dermatoses) in potential treatment area that may cause difficulty with examination or final evaluation
- Accept to abstain from extensive sunbathing and use of solarium during observer blind part. Patients with sunburn within treatment areas cannot be included until fully recovered
- Healthy patients and patients with clinically stable medical conditions, including, but not limited to controlled hypertension, diabetes mellitus type II, hypercholesterolemia, and osteoarthritis, will be permitted to be included in study if their medication is not prohibited by protocol
- Women of childbearing potential are permitted to participate in study only if they have a negative serum pregnancy test at screening and willingness to use a highly effective method of contraception during observer blind part
Main Exclusion Criteria:
- History of hypersensitivity to 5-ALA or any ingredient of BF-200 ALA, MAL or any ingredient of Metvix®, including arachis oil, or to peanut or soya
- Hypersensitivity to porphyrins
- Current treatment with immunosuppression therapy
- Presence of porphyria
- Presence of BCC lesions on embryonic fusion planes (H-zone)
- Presence of more than 3 BCCs
- Presence of malignant or benign tumors of the skin other than non-aggressive BCC within the treatment area (eg malignant melanoma, squamous cell carcinoma (SCC), aggressive BCC clinically diagnosed at screening) within the last 12 weeks
- Gorlin Syndrome or Xeroderma pigmentosum
- Presence of photodermatoses
- Treatment of lesions (actinic keratosis (AK), BCC, SCC, Bowens disease, melanoma) ≤12 weeks prior to first PDT, except physical treatments (eg cryosurgery, excision surgery) that will not be allowed ≤6 weeks prior to first PDT (Visit 2). Lesion(s) that seemed eligible clinically which could not be confirmed by biopsy, and which are located ≥10cm to an eligible lesion should timely be removed physically only
- Presence of inherited or acquired coagulation defect
- Start of intake of medication with hypericin or systemically-acting drugs with phototoxic or photoallergic potential within 8 weeks prior to screening
- Clinically relevant cardiovascular, hepatic, renal, neurologic, endocrine, or other major systemic disease making implementation of protocol or interpretation of study results difficult
- Evidence of clinically significant (CS), unstable medical conditions, eg:
- Metastatic tumor or tumor with high probability of metastasis
- Cardiovascular disease (New York Heart Association [NYHA] class III, IV)
- Immunosuppressive condition
- Hematologic, hepatic, renal, neurologic, or endocrine condition
- Collagen-vascular condition
- Gastrointestinal condition
- Topical treatment with 5-ALA or MAL outside treatment area during the observer blind part
- Any topical treatment including diclofenac and immunomodulatory agents (eg imiquimod, ingenol mebutate) 12 weeks prior to first PDT session and during observer blind part
- Any physical treatment during the observer blind part within treated target areas with exception of lesion(s) determined non-eligible by biopsy
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Randomized, Observer Blind, Multinational Phase III Study to Evaluate the Safety and Efficacy of BF-200 ALA (Ameluz®) in Comparison to Metvix® in the Treatment of Non-aggressive Basal Cell Carcinoma (BCC) With Photodynamic Therapy (PDT)
The aim of this study is to test the effectiveness and safety of the medicine Ameluz® (5-aminolevulinic acid) in comparison to methyl-aminolevulinate (MAL), used with photodynamic therapy (PDT), to treat thin, non-aggressive BCC (basal cell carcinoma).
Read the detailed description
The treatment comprises of up to 2 PDT cycles, each with two PDT sessions one week apart.
If 12 weeks after the the second PDT all lesions are completely cleared the patient will enter the follow-up phase. In case of remaining lesions the patient will receive a second PDT cycle starting on the same day.
- Conditions
- Basal Cell Carcinoma (BCC)
- Being tested
- BF-200 ALA, methyl-aminolevulinate
- Keywords
- Photodynamic Therapy, PDT, non-aggressive BCC
- Medical subject headings
- Carcinoma, Basal Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BF-200 ALADrug
Also called Ameluz
Topical treatment for photodynamic therapy combining drug application and subsequent illumination with a narrow spectrum light source (after 3 h of drug incubation)
methyl-aminolevulinateDrug
Also called Metvix / Metvixia
Topical treatment for photodynamic therapy combining drug application and subsequent illumination with a narrow spectrum light source (after 3 h of drug incubation)
| Study group | Role | What they receive |
|---|---|---|
| BF-200 ALA | Active comparator | Topical application of BF-200 ALA gel containing 78 mg/g 5-aminolevulinic acid. Application of a 1 mm thick layer covering each lesion and 0.5 to 1 cm of surrounding margin. Receives: Drug: BF-200 ALA |
| methyl-aminolevulinate | Active comparator | Topical application of Metvix creme containing 160 mg/g methyl-aminolevulinate. Application of a 1 mm thick layer covering each lesion and 0.5 to 1 cm of surrounding margin. Receives: Drug: methyl-aminolevulinate |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall Patient Complete Response Rate Assessed 12 Weeks After the Last PDT
Overall patient complete response rate assessed 12 weeks after the last PDT. The indicated values give the percentage of overall complete responders. An overall complete responder is defined as a patient in whom all treated lesions were cleared. The PP set is the primary analysis set for the analyses of the primary endpoint.
Measured: 12 weeks after the last PDT (please note: 2 PDT cycles, each cycle consisting of 2 PDTs (= maximum of 4 PDTs per patient) was possible).
Secondary outcomes (5)
Lesion Complete Response Assessed 12 Weeks After the Last PDT
Lesion complete response (completely cleared individual lesions) assessed 12 weeks after the last PDT. The indicated values give percentage of overall completely cleared individual lesions. The PP set is the primary analysis set for the analysis of the secondary endpoint.
Measured: 12 weeks after the last PDT (please note: 2 PDT cycles, each cycle consisting of 2 PDTs (= maximum of 4 PDTs per patient) was possible).
Reduction of Lesion Area 12 Weeks After the Last PDT Compared to Baseline
Reduction of total lesion area (summation of sizes of all treated lesions) per patient, assessed 12 weeks after the last PDT. The PP set is the primary analysis set for the analysis of the secondary endpoint.
Please note that the high SD for BF-200 ALA is due to a patient who had increased lesion area fom 63 mm² at baseline to 225 mm² 12 weeks after PDT. This lesion area included a lesion that was later confirmed to be benign skin condition (lentigo solaris).
Measured: 12 weeks after the last PDT (please note: 2 PDT cycles, each cycle consisting of 2 PDTs (= maximum of 4 PDTs per patient) was possible).
Patient Complete Response 12 Weeks After PDT-2
Patient complete response (complete clearance of all treated lesions) assessed 12 weeks after PDT-2 (first PDT cycle). The PP set is the primary analysis set for the analysis of the secondary endpoint.
Measured: 12 weeks after PDT-2 (=PDT cycle 1; please note: in this study 2 PDT cycles, each cycle consisting of 2 PDTs (= maximum of 4 PDTs per patient) was possible).
Cosmetic Outcome 12 Weeks After Last PDT (Including Patients With a Sum Score of 0 at Baseline)
Overall cosmetic outcome 12 weeks after last PDT is calculated as difference between 12 weeks after PDT sum score and baseline sum score of all skin quality assessments. Each of the below skin quality characteristics are assessed on a 4-point scale from 0 (none) to 3 (severe) by the investigator at baseline and 12 weeks after last PDT:
- Skin surface
- Hyperpigmentation
- Hypopigmentation
- Mottled or irregular pigmentation
- Degree of scarring
- Atrophy
Cosmetic outcome categories are:
- Very good: 12 weeks sum score improved by at least 2 points compared to baseline
- Good: 12 weeks sum score improved by 1 point compared to baseline
- Satisfactory: 12 weeks sum score identical to the one at baseline
- Unsatisfactory: 12 weeks sum score worsened by 1 point compared to baseline
- Impaired: 12 weeks sum score worsened by at least 2 points compared to baseline
Measured: 12 weeks after the last PDT (please note: 2 PDT cycles, each cycle consisting of 2 PDTs (= maximum of 4 PDTs per patient) was possible).
Cosmetic Outcome 12 Weeks After the Last PDT (Including Patients With a Baseline Sum Score >1)
Overall cosmetic outcome 12 weeks after last PDT is calculated as difference between 12 weeks after PDT sum score and baseline sum score of all skin quality assessments. Each of the below skin quality characteristics are assessed on a 4-point scale from 0 (none) to 3 (severe) by the investigator at baseline and 12 weeks after last PDT:
- Skin surface
- Hyperpigmentation
- Hypopigmentation
- Mottled or irregular pigmentation
- Degree of scarring
- Atrophy
Cosmetic outcome categories are:
- Very good: 12 weeks sum score improved by at least 2 points compared to baseline
- Good: 12 weeks sum score improved by 1 point compared to baseline
- Satisfactory: 12 weeks sum score identical to the one at baseline
- Unsatisfactory: 12 weeks sum score worsened by 1 point compared to baseline
- Impaired: 12 weeks sum score worsened by at least 2 points compared to baseline
Measured: 12 weeks after the last PDT (please note: 2 PDT cycles, each cycle consisting of 2 PDTs (= maximum of 4 PDTs per patient) was possible).
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 May 2014 (estimated)
- Study start28 Jan 2014
- Primary completion17 Nov 2015
- Study completion9 Sep 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Basal Cell Carcinoma (BCC)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Investigator, Outcomes assessor
- Enrolment
- 281 participants (actual)
Sponsor and investigators
- Lead sponsor
- Biofrontera Bioscience GmbH
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Accovion GmbH
- Responsible party
- Sponsor
- Investigators
- Rolf M. Szeimies, Prof. Dr., Principal investigator, Klinik fuer Dermatologie und Allergologie (Klinikum Vest - Knappschaftskrankenhaus), Recklinghausen, GermanyColin Morton, Dr., Principal investigator, Dermatology Department, Stirling Community Hospital, NHS Forth Valley, United Kingdom
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02144077
- Sponsor's protocol ID
- ALA-BCC-CT008
- Other IDs
- 2013-003241-42
- Status last verified
- Oct 2022
- First submitted
- 20 Feb 2014
- Last updated
- 3 Nov 2022
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Publications
Morton CA, Dominicus R, Radny P, Dirschka T, Hauschild A, Reinhold U, Aschoff R, Ulrich M, Keohane S, Ekanayake-Bohlig S, Ibbotson S, Ostendorf R, Berking C, Grone D, Schulze HJ, Ockenfels HM, Jasnoch V, Kurzen H, Sebastian M, Stege H, Staubach P, Gupta G, Hubinger F, Ziabreva I, Schmitz B, Gertzmann A, Lubbert H, Szeimies RM. A randomized, multinational, noninferiority, phase III trial to evaluate the safety and efficacy of BF-200 aminolaevulinic acid gel vs. methyl aminolaevulinate cream in the treatment of nonaggressive basal cell carcinoma with photodynamic therapy. Br J Dermatol. 2018 Aug;179(2):309-319. doi: 10.1111/bjd.16441. Epub 2018 May 16.
PubMed 29432644
Study at a glance
From the registry record, last updated by the sponsor on 3 Nov 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Observer Blind, Multinational Phase III Study to Evaluate the Safety and Efficacy of BF-200 ALA (Ameluz®) in Comparison to Metvix® in the Treatment of Non-aggressive Basal Cell Carcinoma (BCC) With Photodynamic Therapy (PDT)
- ClinicalTrials.gov ID
- NCT02144077
- Acronym / sponsor ID
- ALA-BCC-CT008
- Conditions
- Basal Cell Carcinoma (BCC)
- Keywords
- Photodynamic Therapy, PDT, non-aggressive BCC
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 281 participants (actual)
- Lead sponsor
- Biofrontera Bioscience GmbHIndustry (pharma, biotech, devices)
- Collaborators
- Accovion GmbH
- Study start
- 28 Jan 2014 (actual)
- Primary completion
- 17 Nov 2015 (actual)
- Study completion
- 9 Sep 2020 (actual)
- First posted
- 21 May 2014 (estimated)
- Results
- Posted 11 Mar 2019
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The aim of this study is to test the effectiveness and safety of the medicine Ameluz® (5-aminolevulinic acid) in comparison to methyl-aminolevulinate (MAL), used with photodynamic therapy (PDT), to treat thin, non-aggressive BCC (basal cell carcinoma).
Detailed description
The treatment comprises of up to 2 PDT cycles, each with two PDT sessions one week apart.
If 12 weeks after the the second PDT all lesions are completely cleared the patient will enter the follow-up phase. In case of remaining lesions the patient will receive a second PDT cycle starting on the same day.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| BF-200 ALA | Active comparator | Drug: BF-200 ALATopical application of BF-200 ALA gel containing 78 mg/g 5-aminolevulinic acid. Application of a 1 mm thick layer covering each lesion and 0.5 to 1 cm of surrounding margin. |
| methyl-aminolevulinate | Active comparator | Drug: methyl-aminolevulinateTopical application of Metvix creme containing 160 mg/g methyl-aminolevulinate. Application of a 1 mm thick layer covering each lesion and 0.5 to 1 cm of surrounding margin. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| BF-200 ALAAlso: AmeluzTopical treatment for photodynamic therapy combining drug application and subsequent illumination with a narrow spectrum light source (after 3 h of drug incubation) | Drug | BF-200 ALA |
| methyl-aminolevulinateAlso: Metvix / MetvixiaTopical treatment for photodynamic therapy combining drug application and subsequent illumination with a narrow spectrum light source (after 3 h of drug incubation) | Drug | methyl-aminolevulinate |
Primary outcome
Overall Patient Complete Response Rate Assessed 12 Weeks After the Last PDT
Time frame: 12 weeks after the last PDT (please note: 2 PDT cycles, each cycle consisting of 2 PDTs (= maximum of 4 PDTs per patient) was possible).
Overall patient complete response rate assessed 12 weeks after the last PDT. The indicated values give the percentage of overall complete responders. An overall complete responder is defined as a patient in whom all treated lesions were cleared. The PP set is the primary analysis set for the analyses of the primary endpoint.
5 secondary outcomes
Lesion Complete Response Assessed 12 Weeks After the Last PDT
Time frame: 12 weeks after the last PDT (please note: 2 PDT cycles, each cycle consisting of 2 PDTs (= maximum of 4 PDTs per patient) was possible).
Reduction of Lesion Area 12 Weeks After the Last PDT Compared to Baseline
Time frame: 12 weeks after the last PDT (please note: 2 PDT cycles, each cycle consisting of 2 PDTs (= maximum of 4 PDTs per patient) was possible).
Patient Complete Response 12 Weeks After PDT-2
Time frame: 12 weeks after PDT-2 (=PDT cycle 1; please note: in this study 2 PDT cycles, each cycle consisting of 2 PDTs (= maximum of 4 PDTs per patient) was possible).
Cosmetic Outcome 12 Weeks After Last PDT (Including Patients With a Sum Score of 0 at Baseline)
Time frame: 12 weeks after the last PDT (please note: 2 PDT cycles, each cycle consisting of 2 PDTs (= maximum of 4 PDTs per patient) was possible).
Cosmetic Outcome 12 Weeks After the Last PDT (Including Patients With a Baseline Sum Score >1)
Time frame: 12 weeks after the last PDT (please note: 2 PDT cycles, each cycle consisting of 2 PDTs (= maximum of 4 PDTs per patient) was possible).
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Nov 2022.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 29432644Morton CA, Dominicus R, Radny P, Dirschka T, Hauschild A, Reinhold U, Aschoff R, Ulrich M, Keohane S, Ekanayake-Bohlig S, Ibbotson S, Ostendorf R, Berking C, Grone D, Schulze HJ, Ockenfels HM, Jasnoch V, Kurzen H, Sebastian M, Stege H, Staubach P, Gupta G, Hubinger F, Ziabreva I, Schmitz B, Gertzmann A, Lubbert H, Szeimies RM. A randomized, multinational, noninferiority, phase III trial to evaluate the safety and efficacy of BF-200 aminolaevulinic acid gel vs. methyl aminolaevulinate cream in the treatment of nonaggressive basal cell carcinoma with photodynamic therapy. Br J Dermatol. 2018 Aug;179(2):309-319. doi: 10.1111/bjd.16441. Epub 2018 May 16.
- US National Library of Medicine National Institutes of HealthLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Rolf M. Szeimies, Prof. Dr. principal investigator
Klinik fuer Dermatologie und Allergologie (Klinikum Vest - Knappschaftskrankenhaus), Recklinghausen, Germany
Colin Morton, Dr. principal investigator
Dermatology Department, Stirling Community Hospital, NHS Forth Valley, United Kingdom
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing BF-200 ALA and methyl-aminolevulinate in people with Basal Cell Carcinoma (BCC), run by Biofrontera Bioscience GmbH at 1 site in Germany.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 3 Nov 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Germany. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 11 Mar 2019). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall Patient Complete Response Rate Assessed 12 Weeks After the Last PDT (time frame: 12 weeks after the last PDT (please note: 2 PDT cycles, each cycle consisting of 2 PDTs (= maximum of 4 PDTs per patient) was possible).). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02144077, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Nov 2022.