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Poster display session: Breast cancer - early stage, locally advanced & metastatic, CNS tumours, Developmental therapeutics, Genitourinary tumours - prostate & non-prostate, Palliative care, Psycho-oncology, Public health policy, Sarcoma, Supportive care

5748 - A phase I safety study of topical calcitriol (BPM31543) for the prevention of chemotherapy-induced alopecia (CIA): Final study results

Date

22 Oct 2018

Session

Poster display session: Breast cancer - early stage, locally advanced & metastatic, CNS tumours, Developmental therapeutics, Genitourinary tumours - prostate & non-prostate, Palliative care, Psycho-oncology, Public health policy, Sarcoma, Supportive care

Topics

Bioethical Principles and GCP

Tumour Site

Presenters

Mario Lacouture

Citation

Annals of Oncology (2018) 29 (suppl_8): viii603-viii640. 10.1093/annonc/mdy300

Authors

M. Lacouture1, H. Dion2, S. Ravipaty3, V.R. Akmaev4, R. Sarangarajan3, J.J. Jimenez5, N.R. Narain6, B. Berman7, S. Goldfarb8

Author affiliations

  • 1 Dermatology, Memorial Sloan Kettering Cancer Center, 10022 - New York/US
  • 2 Clinical Operations, BERG, LLC, 01701 - Framingham/US
  • 3 Biosystems, BERG, LLC, 01701 - Framingham/US
  • 4 Analytics, BERG, LLC, 01701 - Framingham/US
  • 5 Dermatology And Cutaneous Surgery, University of Miami Miller School of Medicine, 33136 - Miami/US
  • 6 Biosystems, Analytics, & Precision Medicine, BERG, LLC, 01701 - Framingham/US
  • 7 Dermatology, University of Miami Miller School of Medicine, 33136 - Miami/US
  • 8 Oncology, Hematology And Oncology, Memorial Sloan Kettering Cancer Center, 10022 - New York/US

Resources

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Abstract 5748

Background

Chemotherapy-induced alopecia (CIA) negatively effects psychosocial health and quality of life. Currently there are no FDA approved pharmacologic agents available to prevent CIA. Topical calcitriol, a vitamin D3 analog, has been shown in murine studies to reduce CIA, likely due to arrest of cell cycle in healthy hair follicles, and reduction in the sensitivity of follicular epithelium to chemotherapy.

Methods

A 3 + 3 dose-escalation Phase 1 study with 3-6 patients at 5, 10, 20, 40, 60, 80 μg/mL was examined in 23 patients (pts) with breast cancer, gynecologic cancer or sarcomas receiving a taxane-based chemotherapy regimen. Pts applied 1mL of topical calcitriol (BPM31543) to the scalp BID, ≥ 5 days prior to chemotherapy for 3 months or treatment completed. The maximum tolerated dose (MTD) was determined by dose escalation in stepwise increments of the prior dose, in the absence of dose-limiting toxicity (DLT) during the first 28 days of application. Adverse event (AE) monitoring, pharmacokinetics, blinded photographic assessments and patient self-assessment were assessed.

Results

22 treated pts experienced 1 treatment emergent adverse event (TEAE). The most frequently experienced TEAEs were fatigue (47.8%), nausea (39.1%), peripheral sensory neuropathy (30.4%), maculopapular rash and elevated vitamin D (26.1%). Elevated vitamin D and rash were possibly or probably related to treatment, while fatigue, nausea, neuropathy were likely due to chemotherapy. The majority of AEs were mild to moderate in severity. 13 TEAEs were considered Grade 3, but were not considered treatment related by the investigators. Hair loss <50% from baseline was observed in 8 pts at Week 7. At Week 15, 2 pts had <50% hair loss maintained. There were no detectable effects of topical BPM31543 on serum levels of calcitriol.

Conclusions

BPM31543 applied topically twice daily to the scalp in patients receiving taxane-based chemotherapy is safe and well-tolerated with no apparent differences in safety between doses. Here, no DLT was observed at up to 80 µg/mL and MTD was not reached. Some efficacy was detected at each dose. These data are encouraging and support further investigation in Phase 2/3 trials.

Clinical trial identification

NCT01588522.

Legal entity responsible for the study

BERG, LLC.

Funding

BERG, LLC.

Editorial Acknowledgement

Khampaseuth Thapa, BERG, LLC.

Disclosure

M. Lacouture: Research sponsorship: BERG, LLC, Veloce Pharma. H. Dion, S. Ravipaty, V.R. Akmaev: Employee and stock options: BERG, LLC. R. Sarangarajan: Co-founder, employee and stock options: BERG, LLC. J.J. Jimenez, S. Goldfarb: Research sponsorship: BERG, LLC. N.R. Narain: Co-founder, President, CEO, stock options: BERG, LLC. B. Berman: Research sponsorship; BERG, LLC., Leo, Biofrontera, Tigercat; Stock or other ownership: Dermira, Sonoma, Celumigen.

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