Topical Rapamycin and Calcitriol for Angiofibroma of Tuberous Sclerosis

NCT03140449 | Completed Phase 3

• 1 other identifier: 201306009MINB
• Study Type: interventional
• Target: 52
• Locations: 0 countries
• Sites: N/A

Brief Summary

The aim of the study is to determine the effect and safety of topical rapamycin or calcitriol and their combination for the treatment of TSC-associated facial angiofibroma. Methods: A total of 52 TSC patients including 20 male and 32 female subjects were recruited, and 50 of them completed the period 1 study. In period 1, topical rapamycin (0.1%) or calcitriol (3 mcg/g) single-agent therapy versus their combination were applied twice a day by a left-right randomized, split-face comparison for 12 weeks. The primary end point was the reduction of facial angiofibroma severity index (FASI) for the grade of erythema, papule size, elevation and extension of the lesions at week 12. In period 2, the patients entered an open-label study and were reassigned to use the more effective ointment on both cheeks for another 12 weeks (week 13-24). A follow-up FASI analysis for recurrence after drug discontinuance for 12 weeks was also performed (week 36). The secondary end point was the reduction of Visual Analysis Score (VAS) evaluated by the subjects themselves at week 12.

Conditions

Facial Angiofibroma

Keywords

Tuberous sclerosis complex; facial angiofibroma; rapamycin; calcitriol

Outcome Measures

Primary Outcomes (1)

• facial angiofibroma severity index (FASI)

  the reduction of facial angiofibroma severity index (FASI) for the grade of erythema, papule size, elevation and extension of the lesions at week 12

  at week 12

Study Arms (3)

Rapamycin

EXPERIMENTAL

Rapamycin(0.1%)

Drug: Rapamycin

Calcitriol

EXPERIMENTAL

Calcitriol(3mcg/g)

Drug: Calcitriol

Rapamycin-calcitriol combination

EXPERIMENTAL

Rapamycin(0.1%) with Calcitriol(3mcg/g)

Drug: Rapamycin-calcitriol combination

Interventions

Rapamycin DRUG

topical rapamycin (0.1%) or calcitriol (3 mcg/g) single-agent therapy versus their combination were applied twice a day by a left-right randomized, split-face comparison for 12 weeks. In period 2, the patients entered an open-label study and were reassigned to use the more effective ointment on both cheeks for another 12 weeks (week 13-24).

Also known as: Sirolimus

Rapamycin

Calcitriol DRUG

topical rapamycin (0.1%) or calcitriol (3 mcg/g) single-agent therapy versus their combination were applied twice a day by a left-right randomized, split-face comparison for 12 weeks. In period 2, the patients entered an open-label study and were reassigned to use the more effective ointment on both cheeks for another 12 weeks (week 13-24).

Also known as: Rocaltrol

Calcitriol

Rapamycin-calcitriol combination DRUG

topical rapamycin (0.1%) or calcitriol (3 mcg/g) single-agent therapy versus their combination were applied twice a day by a left-right randomized, split-face comparison for 12 weeks. In period 2, the patients entered an open-label study and were reassigned to use the more effective ointment on both cheeks for another 12 weeks (week 13-24).

Also known as: Sirolimus-Rocaltrol combination

Rapamycin-calcitriol combination

Eligibility Criteria

• Age: 7 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Subjects must have been diagnosed or highly suspected as having TSC.
• Subjects must be aged 7 to 70 years at Screening, and can be either sex.
• Subjects must have symmetric facial angiofibromas.

You may not qualify if:

• Pregnancy or with a plan to be pregnant.
• Subjects who cannot comply the treatment protocol.
• Subjects with kidney or liver/ biliary dysfunction.
• Subjects with hypercalcaemia and patients known to suffer from abnormal calcium metabolism.
• Subjects on systemic treatment of calcium deficiency.
• Subjects known to be hypersensitive to rapamycin or calcitriol.

Sponsors & Collaborators

• National Taiwan University Hospital: lead

MeSH Terms

Conditions

Tuberous Sclerosis

Interventions

Sirolimus; Calcitriol

Condition Hierarchy (Ancestors)

Hamartoma; Neoplasms; Neoplasms, Multiple Primary; Neoplastic Syndromes, Hereditary; Malformations of Cortical Development, Group I; Malformations of Cortical Development; Nervous System Malformations; Nervous System Diseases; Neurocutaneous Syndromes; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn

Intervention Hierarchy (Ancestors)

Macrolides; Lactones; Organic Chemicals; Dihydroxycholecalciferols; Hydroxycholecalciferols; Cholecalciferol; Cholestenes; Cholestanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds; Sterols; Vitamin D; Secosteroids; Membrane Lipids; Lipids

Study Officials

• YI-HUA LIAO, A.P.

  A.P.

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 26, 2017
• First Posted: May 4, 2017
• Study Start: September 5, 2013
• Primary Completion: November 7, 2016
• Study Completion: April 25, 2017
• Last Updated: May 4, 2017

Record last verified: 2013-09