A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate Efficacy and Safety of OSD-001 in patients with Neurofibromatosis type 1

A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate Efficacy and Safety of OSD-001 in patients with Neurofibromatosis type 1 - A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate Efficacy and Safety of OSD-001 in patients with Neurofibromatosis type 1

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

JPRN

Registry ID

JPRN-UMIN000021030

Enrollment

Registered

2016-02-22

Start date

2016-03-16

Completion date

Unknown

Last updated

2026-06-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Neurofibromatosis type 1

Interventions

0.2% Sirolimus gel twice daily 24 weeks topical application 0.4% Sirolimus gel twice daily 24 weeks topical application Placebo gel twice daily 24 weeks topical application

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1) Patients diagnosed as neurofibromatosis type 1 based on the clinical diagnostic criteria in guideline of Japanese Dermatological Association 2) Patients who have the skin lesions of 450 cm2 or less to be evaluable 3) Patients whose skin lesions increase within 6 months . 4) Patients who is 16 years old or elder and younger than 70 years old

Exclusion criteria

Exclusion criteria: 1) Patients who received radiotherapy or neutron capture therapy on skin lesions within 12 months prior to enrollment. 2) Patients who have received surgical therapy on skin lesions within 6 months prior to enrollment. 3) Patients who treated with sirolimus, everolimus or temsirolimus (mTOR inhibitors), within 6 months prior to enrollment. 4) Patients who received topical treatment of tacrolimus within 3 months prior to enrollment. 5) Patients who have severe complications such as cardiac disease, liver disease, pulmonary disease, hematological disorder . 6) Patients who have previously experienced alcoholic sensitivity or allergy to sirolimus. 7) Patients who are pregnant or lactating. 8) Patients who have entered another clinical trial within 6 months prior to enrollment.

Design outcomes

Primary

Measure	Time frame
Changes from baseline in the size of cutaneous tumor (CT scan) after 24 week treatment	—

Secondary

Measure	Time frame
1. Changes from baseline in the size of cutaneous tumor (measured by ruler) after 24 week treatment 2. Improvements of cutaneous lesions after 12 or 24 week treatment 3. Histopathological evaluation of cutaneous tumor after 24 week treatment 4. Evaluation of Dermatology Life Quality Index (DLQI) after 12 or 24 week treatment	—

Countries

Japan

Contacts

Public ContactMari Wataya-Kaneda

Graduate School of Medicine, Osaka University Department of Dermatology

mkaneda@derma.med.osaka-u.ac.jp06-6879-5111

Outcome results

None listed

Source: JPRN (via WHO ICTRP) · Data processed: Jul 3, 2026