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| ID | Type | Description | Link |
|---|---|---|---|
| Department of Defense USAMRMC | Other Grant/Funding Number | W81XWH-11-1-0240 |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Placebo | Placebo Comparator | Cream only |
|
| 0.1 % Rapamycin | Active Comparator | 0.1% Rapamycin cream |
|
| 1% Rapamycin | Active Comparator | 1% Rapamycin cream |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Placebo | Drug | Study cream is applied nightly to the affected areas on the face. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in the Facial Angiofibroma Grading Scale (AGS) Score | 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. | baseline, 6 months |
| Measure | Description | Time Frame |
|---|---|---|
| Photo Readers' Rating (Better, Same, or Worse) of Paired Baseline and End-of-trial (EOT) Photographs for Each Patient | baseline, 6 months | |
| Change in Quality of Life (QOL) Questionnaire Scores as Assessed by the Dermatology Life Quality Index (DLQI) | 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). |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Events of Dermatologic Sensitivity at the Site of Application | Dermatologic sensitivity includes pain, pruritis, or erythema at the application site. | 6 months |
| Number of Participants With Systemic Uptake of Topically Applied Rapamycin |
Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Mary Kay Koenig, MD | The University of Texas Medical School at Houston | Principal Investigator |
| Hope Northrup, MD | The University of Texas Medical School at Houston | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Alabama at Birmingham | Birmingham | Alabama | 35294 | United States | ||
| UCLA Mattel Children's Hospital |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 29800048 | Derived | Koenig MK, Bell CS, Hebert AA, Roberson J, Samuels JA, Slopis JM, Tate P, Northrup H; TREATMENT Trial Collaborators. Efficacy and Safety of Topical Rapamycin in Patients With Facial Angiofibromas Secondary to Tuberous Sclerosis Complex: The TREATMENT Randomized Clinical Trial. JAMA Dermatol. 2018 Jul 1;154(7):773-780. doi: 10.1001/jamadermatol.2018.0464. |
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| ID | Title | Description |
|---|---|---|
| FG000 | 1% Rapamycin | 1% Rapamycin cream Rapamycin: Study cream is applied nightly to the affected areas on the face. High Dose |
| FG001 | 0.1 % Rapamycin | 0.1% Rapamycin cream Rapamycin: Study cream is applied nightly to the affected areas on the face. Low Dose |
| FG002 | Placebo | Cream only Placebo: Study cream is applied nightly to the affected areas on the face. |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | 1% Rapamycin | 1% Rapamycin cream Rapamycin: Study cream is applied nightly to the affected areas on the face. High Dose |
| BG001 | 0.1 % Rapamycin | 0.1% Rapamycin cream Rapamycin: Study cream is applied nightly to the affected areas on the face. Low Dose |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Change From Baseline in the Facial Angiofibroma Grading Scale (AGS) Score | 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. | 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. | Posted | Mean | Standard Deviation | score on a scale | baseline, 6 months |
|
6 months
Serious adverse events were determined to be unrelated to study drug.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Placebo | Cream only Placebo: Study cream is applied nightly to the affected areas on the face. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| cholecystectomy | Hepatobiliary disorders | MedDRA | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Application site pain | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Mary Kay Koenig, MD | The University of Texas Health Science Center at Houston | 713-500-7142 | mary.k.koenig@uth.tmc.edu |
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| ID | Term |
|---|---|
| D018322 | Angiofibroma |
| D014402 | Tuberous Sclerosis |
| ID | Term |
|---|---|
| D009383 | Neoplasms, Vascular Tissue |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
| D006222 | Hamartoma |
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| ID | Term |
|---|---|
| D020123 | Sirolimus |
| ID | Term |
|---|---|
| D018942 | Macrolides |
| D007783 | Lactones |
| D009930 | Organic Chemicals |
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| Rapamycin | Drug | Study cream is applied nightly to the affected areas on the face. Low Dose |
|
| Rapamycin | Drug | Study cream is applied nightly to the affected areas on the face. High Dose |
|
| baseline, 6 months |
| Change in Quality of Life (QOL) Questionnaire Scores as Assessed by the Children's Dermatology Life Quality Index (CDLQI) | 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). | baseline, 6 months |
| Change in Quality of Life (QOL) Questionnaire Scores as Assessed by the Family Dermatology Life Quality Index (FDLQI) | 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). | baseline, 6 months |
Blood levels checked to confirm the lack of systemic rapamycin.
| 6 months |
| Los Angeles |
| California |
| 90095 |
| United States |
| Jack & Julia Center for TSC at Oakland Children's Hospital & Research Center | Oakland | California | 94609 | United States |
| Kennedy Krieger Institute | Baltimore | Maryland | 21205 | United States |
| Herscot Center for Adults and Children with TSC Massachusetts General Hospital | Boston | Massachusetts | 02114 | United States |
| Clinic Without Walls | Saint Paul | Minnesota | 55102-2697 | United States |
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | 45229 | United States |
| Texas Scottish Rite Hospital | Dallas | Texas | 75219 | United States |
| The University of Texas Medical School at Houston | Houston | Texas | 77030 | United States |
| Sydney Children's Hospital | Sydney | New South Wales | Australia |
| BG002 | Placebo | Cream only Placebo: Study cream is applied nightly to the affected areas on the face. |
| BG003 | Total | Total of all reporting groups |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| 1% Rapamycin |
1% Rapamycin cream Rapamycin: Study cream is applied nightly to the affected areas on the face. High Dose |
| OG001 | 0.1 % Rapamycin | 0.1% Rapamycin cream Rapamycin: Study cream is applied nightly to the affected areas on the face. Low Dose |
| OG002 | Placebo | Cream only Placebo: Study cream is applied nightly to the affected areas on the face. |
|
|
| Secondary | 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 4 in the 1% rapamycin arm, 5 in the 0.1% rapamycin arm, and 6 in the placebo arm. | Posted | Count of Participants | Participants | baseline, 6 months |
|
|
|
| Secondary | Change in Quality of Life (QOL) Questionnaire Scores as Assessed by the Dermatology Life Quality Index (DLQI) | 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). | 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. | Posted | Mean | Standard Deviation | score on a scale | baseline, 6 months |
|
|
|
| Secondary | Change in Quality of Life (QOL) Questionnaire Scores as Assessed by the Children's Dermatology Life Quality Index (CDLQI) | 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). | 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. | Posted | Mean | Standard Deviation | score on a scale | baseline, 6 months |
|
|
|
| Secondary | Change in Quality of Life (QOL) Questionnaire Scores as Assessed by the Family Dermatology Life Quality Index (FDLQI) | 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). | 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. | Posted | Mean | Standard Deviation | score on a scale | baseline, 6 months |
|
|
|
| Other Pre-specified | Number of Events of Dermatologic Sensitivity at the Site of Application | Dermatologic sensitivity includes pain, pruritis, or erythema at the application site. | Posted | Number | number of events | 6 months |
|
|
|
| Other Pre-specified | Number of Participants With Systemic Uptake of Topically Applied Rapamycin | Blood levels checked to confirm the lack of systemic rapamycin. | Posted | Count of Participants | Participants | 6 months |
|
|
|
| 0 |
| 59 |
| 1 |
| 59 |
| 10 |
| 59 |
| EG001 | 0.1 % Rapamycin | 0.1% Rapamycin cream Rapamycin: Study cream is applied nightly to the affected areas on the face. Low Dose | 0 | 63 | 1 | 63 | 18 | 63 |
| EG002 | 1% Rapamycin | 1% Rapamycin cream Rapamycin: Study cream is applied nightly to the affected areas on the face. High Dose | 0 | 57 | 2 | 57 | 22 | 57 |
| Subependymal Giant Cell Astrocytoma (SEGA) resection | Nervous system disorders | MedDRA | Systematic Assessment |
|
| Chest pain | General disorders | MedDRA | Systematic Assessment |
|
| Application site pruritus | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
|
| Application site acne | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
|
| Cutaneous eruption | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
|
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
|
| Application site erythema | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
|
| Sunburn | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
|
| Application site irritation | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
|
| Acne | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
|
| Nasopharyngitis | Infections and infestations | MedDRA | Systematic Assessment |
|
| Nasal congestion | Infections and infestations | MedDRA | Systematic Assessment |
|
| Application site papules | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
|
| Sinusitis | Infections and infestations | MedDRA | Systematic Assessment |
|
| Application site paraesthesia | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
|
| Nausea | Gastrointestinal disorders | MedDRA | Systematic Assessment |
|
| Mouth ulceration | General disorders | MedDRA | Systematic Assessment |
|
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
|
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| D009378 |
| Neoplasms, Multiple Primary |
| D009386 | Neoplastic Syndromes, Hereditary |
| D065703 | Malformations of Cortical Development, Group I |
| D054220 | Malformations of Cortical Development |
| D009421 | Nervous System Malformations |
| D009422 | Nervous System Diseases |
| D020752 | Neurocutaneous Syndromes |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D019636 | Neurodegenerative Diseases |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D030342 | Genetic Diseases, Inborn |
| worse |
|
|
| Application site erythema |
|