Safety and Efficacy Study for the Treatment of Non-Aggressive Basal Cell Carcinoma With Photodynamic Therapy

NCT02144077 | Completed Phase 3 Results

• 2 other identifiers: ALA-BCC-CT0082013-003241-42
• Study Type: interventional
• Target: 281
• Locations: 1 country
• Sites: 1

Brief 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).

Conditions

Basal Cell Carcinoma (BCC)

Keywords

Photodynamic Therapy; PDT; non-aggressive BCC

Outcome Measures

Primary Outcomes (1)

• 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.

  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

  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

  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

  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)

  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)

  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).

Other Outcomes (2)

• Patient Recurrence Rate (Overall, Cumulative)

  6, 12, 24, 36 and 60 months post-PDT

• Lesion Recurrence Rate (Cumulative)

  6, 12, 24, 36 and 60 months post-PDT

Study Arms (2)

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.

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.

Drug: methyl-aminolevulinate

Interventions

BF-200 ALA DRUG

Topical treatment for photodynamic therapy combining drug application and subsequent illumination with a narrow spectrum light source (after 3 h of drug incubation)

Also known as: Ameluz

BF-200 ALA

methyl-aminolevulinate DRUG

Topical treatment for photodynamic therapy combining drug application and subsequent illumination with a narrow spectrum light source (after 3 h of drug incubation)

Also known as: Metvix / Metvixia

methyl-aminolevulinate

Eligibility Criteria

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

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Biofrontera Bioscience GmbH: lead
• Accovion GmbH: collaborator

Study Sites (1)

Klinikum Vest GmbH

Recklinghausen, Westfalen-Lippe, 45657, Germany

Location

Related Publications (1)

• 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.

  PMID: 29432644 RESULT

Related Links

• US National Library of Medicine National Institutes of Health

MeSH Terms

Conditions

Carcinoma, Basal Cell

Interventions

BF-200 ALA; Aminolevulinic Acid; methyl 5-aminolevulinate

Condition Hierarchy (Ancestors)

Carcinoma; Neoplasms, Glandular and Epithelial; Neoplasms by Histologic Type; Neoplasms; Neoplasms, Basal Cell

Intervention Hierarchy (Ancestors)

Levulinic Acids; Keto Acids; Carboxylic Acids; Organic Chemicals; Amino Acids; Amino Acids, Peptides, and Proteins

Results Point of Contact

• Title: Clinical Trial Department
• Organization: Biofrontera Bioscience GmbH

ameluz@biofrontera.com

+49 214 876 32 66

Study Officials

• Rolf M. Szeimies, Prof. Dr.

  Klinik fuer Dermatologie und Allergologie (Klinikum Vest - Knappschaftskrankenhaus), Recklinghausen, Germany

  PRINCIPAL INVESTIGATOR

• Colin Morton, Dr.

  Dermatology Department, Stirling Community Hospital, NHS Forth Valley, United Kingdom

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 20, 2014
• First Posted: May 21, 2014
• Study Start: January 28, 2014
• Primary Completion: November 17, 2015
• Study Completion: September 9, 2020
• Last Updated: November 3, 2022
• Results First Posted: March 11, 2019

Record last verified: 2022-10

Locations

DE (1)