Symptoms and General Pathology

All Conditions

Neoplasms

Nervous System Diseases

Diseases and Abnormalities at or Before Birth

Skin and Connective Tissue Diseases

Rare Diseases

Sclerosis

Tuberous Sclerosis

Angiofibroma

Hamartoma

Neoplasms, Multiple Primary

Syndrome

Neoplastic Syndromes, Hereditary

Congenital Abnormalities

Malformations of Cortical Development

Nervous System Malformations

Neurocutaneous Syndromes

Heredodegenerative Disorders, Nervous System

Neurodegenerative Diseases

Genetic Diseases, Inborn

Neoplasms, Vascular Tissue

Neoplasms by Histologic Type

Tuberous Sclerosis Complex

Anti-Infective Agents

Antineoplastic Agents

All Drugs and Chemicals

Sirolimus

Placebo gel is matched ingredient with NPC-12G gel

NPC-12G gel is administered topically twice a day for 12 weeks

NPC-12G gel

NPC-12G gel placebo is administered topically twice a day for 12 weeks

Placebo gel

Mari Wataya-Kaneda, MD, PhD

The purpose of this trial is to evaluate the efficacy and safety of NPC-12G gel (topical formulation of sirolimus) versus placebo gel to facial angiofibroma and other skin lesions in patients with tuberous sclerosis complex (TSC)

Tuberous Sclerosis Complex (TSC) is an autosomal dominant hereditary disease that causes benign tumors on the almost whole body (including skin, brain, kidney, lung and heart), behavior disorder as autism, mental retardation and neurologic symptom as epilepsy. Angiofibroma is TSC-specific facial skin lesions, and hamartoma caused by increase of the component of skin connective tissues and blood vessels. Other skin lesions due to TSC are white macule(hypomelanotic macule), plaque, shagreen patch and ungual fibromas. Current therapeutic methods for angiofibroma are laser or surgical treatments, but there are problems as many relapses, deficiency of evidence, change of pigment, scar and risk of infection.

This will be a multicenter, double-blind, randomized, placebo-controlled parallel group trial. The trial has three phases; the screening phase, double-blinded treatment phase, and post-treatment phase. The screening phase comprises a screening visit where subject's initial eligibility will be evaluated. During double-blinded treatment phase, patients who meet all entry criteria for the trial will be randomized into two groups, and they will apply 0.2% NPC-12G gel or placebo gel topically twice a day for 12 weeks.

Phase III Trial of Topical Formulation of Sirolimus to Skin Lesions in Patients With Tuberous Sclerosis Complex (TSC)

A Double-blind, Randomized, Placebo-controlled Phase III Trial to Investigate the Efficacy and Safety of NPC-12G Gel (Topical Formulation of Sirolimus) to Angiofibroma and Other Skin Lesions in Patients With Tuberous Sclerosis Complex

Improvements comparing with baseline is assessed using photograph by the central photo-judgement committee

Improvements comparing with baseline is assessed by the investigator

Improvement comparing with baseline is assessed by the central photo-judgement committee and the investigator

DLQI for subjects 16 years old and greater, or CDLQI for children of less than 16 years old is assessed by patients

Serious adverse events during the study period

Laboratory findings during the study period

Blood concentration of Sirolimus is assessed by drug monitoring

Nobelpharma

Temsirolimus

MTOR Inhibitors

Anti-Bacterial Agents

Antibiotics, Antitubercular

Clotrimazole

Miconazole

Antifungal Agents

Immunosuppressive Agents

Immunologic Factors

Protein Kinase Inhibitors

Enzyme Inhibitors

Study cream is applied nightly to the affected areas on the face.

Placebo

Study cream is applied nightly to the affected areas on the face. Low Dose

Rapamycin

Study cream is applied nightly to the affected areas on the face. High Dose

Mary Kay Koenig, MD

Hope Northrup, MD

The study is a multi-center prospective, randomized, double-blind, placebo-controlled evaluation of the safety and efficacy of a topically applied formulation of rapamycin to cutaneous angiofibromas in subjects with Tuberous Sclerosis Complex (TSC). Subjects will apply either the topical vehicle containing rapamycin or the topical vehicle alone nightly to their angiofibromas for six months.

The primary goal of this study is to evaluate the efficacy of the topical medication for reduction of cutaneous angiofibromas in patients with TSC. The secondary goal of this study is to confirm the safety of the topical medication.

Topical Rapamycin to Erase Angiofibromas in TSC

Phase II Study of Topical Rapamycin to Erase Angiofibromas in TSC-Multicenter Evaluation of a Novel Therapy

Dermatologic sensitivity includes pain, pruritis, or erythema at the application site.

Blood levels checked to confirm the lack of systemic rapamycin.

Subject's lesion/s will be digitally photographed before initial application (visit 1), at each study visit (visits 2 through 6), and immediately prior to study termination (visit 7). Following completion of the study, all photographs will be evaluated by two independent dermatologists blinded to both the treatment arm and the stage of treatment. The dermatologists will assess each photograph's appearance using the facial Angiofibroma Grading Scale (AGS), which assesses the forehead, nose, cheeks, and chin for erythema, average lesion size, lesion density, and percent involvement, as well as presence of any pedunculated angiofibromas. A total score is reported with a range of 0 to 202, with higher scores representing a worse outcome.

The Dermatology life Quality Index (DLQI) is a ten-question questionnaire used to measure the impact of skin disease on the quality of life of an affected person. It is designed for people aged 16 years and above. Each question is scored from 0 to 3, giving a possible total score range from 0 (meaning no impact of skin disease on quality of life) to 30 (meaning maximum impact on quality of life).

The Children's Dermatology life Quality Index (DLQI) is validated from the age of four years to 16 years. It is a ten-question questionnaire used to measure the impact of skin disease on the quality of life of an affected person. Each question is scored from 0 to 3, giving a possible total score range from 0 (meaning no impact of skin disease on quality of life) to 30 (meaning maximum impact on quality of life).

The Family Dermatology life Quality Index (FLQI) is designed for adult (more than 16 years of age) family members or partners of patients (of any age) with any skin disease. It is a ten-question questionnaire used to measure the impact of skin disease on the quality of life of an affected person. Each question is scored from 0 to 3, giving a possible total score range from 0 (meaning no impact of skin disease on quality of life) to 30 (meaning maximum impact on quality of life).

The University of Texas Health Science Center, Houston

Associate Professor

Serious adverse events were determined to be unrelated to study drug.

Cream only

Placebo: Study cream is applied nightly to the affected areas on the face.

0.1% Rapamycin cream

Rapamycin: Study cream is applied nightly to the affected areas on the face. Low Dose

0.1 % Rapamycin

1% Rapamycin cream

Rapamycin: Study cream is applied nightly to the affected areas on the face. High Dose

1% Rapamycin

Total

Age, Continuous

Sex: Female, Male

This data was not collected for 12 in the 1% rapamycin arm, 17 in the 0.1% rapamycin arm, and 17 in the placebo arm.

Change From Baseline in the Facial Angiofibroma Grading Scale (AGS) Score

This data was not collected for 4 in the 1% rapamycin arm, 5 in the 0.1% rapamycin arm, and 6 in the placebo arm.

Photo Readers' Rating (Better, Same, or Worse) of Paired Baseline and End-of-trial (EOT) Photographs for Each Patient

This data was not collected for 29 in the 1% rapamycin arm, 34 in the 0.1% rapamycin arm, and 35 in the placebo arm.

Change in Quality of Life (QOL) Questionnaire Scores as Assessed by the Dermatology Life Quality Index (DLQI)

This data was not collected for 46 in the 1% rapamycin arm, 57 in the 0.1% rapamycin arm, and 48 in the placebo arm.

Change in Quality of Life (QOL) Questionnaire Scores as Assessed by the Children's Dermatology Life Quality Index (CDLQI)

This data was not collected for 41 in the 1% rapamycin arm, 35 in the 0.1% rapamycin arm, and 38 in the placebo arm.

Change in Quality of Life (QOL) Questionnaire Scores as Assessed by the Family Dermatology Life Quality Index (FDLQI)

Application site pain

Application site pruritis

Application site erythema

Number of Events of Dermatologic Sensitivity at the Site of Application

Number of Participants With Systemic Uptake of Topically Applied Rapamycin

Overall Study

NPC-12G gel is administered topically twice a day for 52 weeks or longer

Mari Wataya-Kaneda,, MD, PhD

The purpose of this trial is to evaluate the safety and efficacy of long-term treatment with NPC-12G gel (0.2% sirolimus gel) to angiofibroma and other skin lesions in patients with tuberous sclerosis complex in the open-label trial.

Tuberous Sclerosis Complex (TSC) is an autosomal dominant hereditary disease that causes benign tumors on the almost whole body (including skin, brain, kidney, lung and heart), behavior disorder as autism, mental retardation and neurologic symptom as epilepsy. Angiofibroma is a TSC-specific facial skin lesion, and hamartoma caused by increase of the component of skin connective tissues and blood vessels. Other skin lesions due to TSC are white macule (hypomelanotic macule), plaque, shagreen patch and ungual fibromas. Current therapeutic methods for angiofibroma are laser and surgical treatments, but there are problems as many relapses, deficiency of evidence, change of pigment, scar and risk of infection.

This is a multicenter and open-label trial. The trial consists of two phase. In the first trial phase for 52 weeks, the efficacy as well as the safety is evaluated. For the second trial phase the trial will be continued until the date of approval of NDA for NPC-12G. The safety is evaluated during the second trial phase, but not the efficacy. Patients who meet all entry criteria for the trial apply 0.2% NPC-12G gel twice a day. Patients will visit at 4 to 5-week intervals for the first 6 months of the first trial phase, and then 3 months intervals thereafter.

Long-term Trial of Topical Sirolimus to Angiofibroma in Patient With Tuberous Sclerosis Complex

A Long-term, Single-arm, Open-label Trial of NPC-12G (Topical Formulation of Sirolimus) to Angiofibroma and Other Skin Lesions in Patients With Tuberous Sclerosis Complex

The first discontinuation in each patient due to adverse events is assessed.Completion of week 26 and 52 are cut-off points for interim-analyses by Kaplan-Meier method

The number of discontinuation/ resume due to adverse events are evaluated. Completion of week 26 and 52 are cut-off points for interim-analyses

The incidence of adverse events are evaluated. Completion of week 26 and 52 are cut-off points for interim-analyses

The incidence of serious adverse events are evaluated. Completion of week 26 and 52 are cut-off points for interim-analyses

The incidence of significant adverse events such as local irritation are evaluated. Completion of week 26 and 52 are cut-off points for interim-analyses

Completion of week 26 and 52 are cut-off points for interim-analyses

Whole blood level of sirolimus are measured any day time at baseline and every 3 months visit

Improvements comparing with baseline is assessed using photograph by the investigator and the central photo-judgement committee. Completion of week 26 is a cut-off point for interim-analysis.

Completion of week 26 is a cut-off point for interim-analysis.

DLQI for subjects 16 years old and greater, or CDLQI for children of less than 16 years old is assessed by patients. Completion of week 26 is a cut-off point for interim-analysis.

Patient's satisfaction is assessed by patient. Completion of week 26 is a cut-off point for interim-analysis.

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Phase III Trial of Topical Formulation of Sirolimus to Skin Lesions in Patients With Tuberous Sclerosis Complex (TSC)	NCT02635789	Tuberous Sclero... Angiofibroma Hypomelanotic M... Plaque	NPC-12G gel Placebo gel	3 Years -	Nobelpharma
Topical Rapamycin to Erase Angiofibromas in TSC	NCT01526356	Angiofibromas Tuberous Sclero...	Placebo Rapamycin Rapamycin	-	The University of Texas Health Science Center, Houston
Long-term Trial of Topical Sirolimus to Angiofibroma in Patient With Tuberous Sclerosis Complex	NCT02634931	Tuberous Sclero... Angiofibroma Hypomelanotic M... Plaque	NPC-12G gel	3 Years -	Nobelpharma
Phase III Trial of Topical Formulation of Sirolimus to Skin Lesions in Patients With Tuberous Sclerosis Complex (TSC)	NCT02635789	Tuberous Sclero... Angiofibroma Hypomelanotic M... Plaque	NPC-12G gel Placebo gel	3 Years -	Nobelpharma
Phase III Trial of Topical Formulation of Sirolimus to Skin Lesions in Patients With Tuberous Sclerosis Complex (TSC)	NCT02635789	Tuberous Sclero... Angiofibroma Hypomelanotic M... Plaque	NPC-12G gel Placebo gel	3 Years -	Nobelpharma

Spots Global Cancer Trial Database for angiofibroma

Every month we try and update this database with for angiofibroma cancer trials from around the world to help patients and their families find trials that might be right for them.
We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.