Phase 1/2 pilot study of methotrexate-laurocapram topical gel for the treatment of patients with early-stage mycosis fungoides.
OBJECTIVE
To assess the safety and tolerability of a topical gel formulation combining methotrexate and laurocapram and to obtain preliminary information on the therapeutic potential of methotrexate-laurocapram in patients with early-stage mycosis fungoides (stage IA or IB).


METHODS
An open-label, phase 1/2 pilot study.


METHODS
Two academic referral centers.


METHODS
Ten patients 18 years or older with histologically confirmed stage IA or IB mycosis fungoides. Intervention The gel formulation of methotrexate-laurocapram was applied to the total body surface, excluding genital, perianal areas, nipples, face, and skin under the breasts, on an every-other-day basis for 24 consecutive weeks.


METHODS
The safety of methotrexate-laurocapram was assessed in this study by reviewing adverse events and laboratory data. Efficacy outcomes included changes in lesion condition and severity assessments, reduction in area of sample lesions, and the investigator's global evaluation.


RESULTS
Adverse events consisted of skin reactions of mild severity. No clinically significant laboratory abnormalities were observed. Based on the investigator's global evaluation at the end of the treatment phase (week 24), 7 (78%) of 9 patients demonstrated a slight-to-moderate response to treatment with methotrexate-laurocapram. Statistical significance (P =.049) was reached for induration and pruritus, a trend (P =.10) was observed for erythema, and no change was found for scaling (P =.37).


CONCLUSIONS
These findings indicate that the topical administration of methotrexate-laurocapram is safe and in general well tolerated. This treatment may represent a new therapeutic potential for patients with mycosis fungoides.
Demierre
Marie-France
MF
Skin Oncology Program, Department of Dermatology, Boston Medical Center, Boston University School of Medicine, 720 Harrison Avenue, Doctors Office Building 801A, Boston, MA 02118, USA. mariefrance.demierre@bmc.org
Vachon
Luc
L
Ho
Vincent
V
Sutton
Lynda
L
Cato
Allen
A
Leyland-Jones
Brian
B
D000287:Administration, Topical; D000328:Adult; D000368:Aged; D001381:Azepines / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D003879:Dermatologic Agents / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D004338:Drug Combinations; D005260:Female; D005500:Follow-Up Studies; D005782:Gels; D006801:Humans; D008297:Male; D008727:Methotrexate / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D008875:Middle Aged; D009182:Mycosis Fungoides / Q000188:drug therapy* / Q000473:pathology; D009367:Neoplasm Staging; D010865:Pilot Projects; D012878:Skin Neoplasms / Q000188:drug therapy* / Q000473:pathology; D013997:Time Factors; D016896:Treatment Outcome
D001381:Azepines; D003879:Dermatologic Agents; D004338:Drug Combinations; D005782:Gels; C035229:laurocapram; D008727:Methotrexate