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| Name | Class |
|---|---|
| FDA Office of Orphan Products Development | FED |
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SELVA: A Multicenter, Phase 3 Baseline-Controlled Study Evaluating the Safety and Efficacy of QTORIN 3.9% Rapamycin Anhydrous Gel in the Treatment of Microcystic Lymphatic Malformations
The main purpose of this study is to assess the change in microcystic lymphatic malformations IGA after 24 weeks of treatment with QTORIN 3.9% Rapamycin Anhydrous Gel in approximately 40 participants with microcystic lymphatic malformations. Efficacy will be evaluated at 24 weeks and patients have the option of continuing on treatment for >24 weeks.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Active | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| QTORIN 3.9% Rapamycin Anhydrous Gel | Drug | QTORIN 3.9% Rapamycin Anhydrous Gel |
|
| Measure | Description | Time Frame |
|---|---|---|
| Overall microcystic lymphatic malformations Investigator Global Assessment (mLM-IGA) | A multi-point scale with higher values indicating improvement. | At Week 24 |
| Measure | Description | Time Frame |
|---|---|---|
| Change in the blinded clinician microcystic lymphatic malformations Multicomponent Severity Scale (mLM-MCSS) | A multi-point scale with lower values indicating improvement. | At Week 24 |
| Change in the live clinician microcystic lymphatic malformations Multicomponent Severity Scale (mLM-MCSS) |
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Key Inclusion Criteria:
Key Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Joyce Teng | Stanford University | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Children's Hospital of Orange County | Irvine | California | 92612 | United States | ||
| Stanford University |
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A multi-point scale with lower values indicating improvement. |
| Change from Baseline to Week 24 |
| Overall patient global impression of change (PGI-C) | A multi-point scale with lower values indicating improvement. | Change from Baseline to Week 24 |
| Change in Overall Clinician Global Impression of Severity (CGI-S) | A multi-point scale with lower values indicating improvement. | Change from Baseline to Week 24 |
| Change in Overall Patient Global Impression of Severity (PGI-S) | A multi-point scale with lower values indicating improvement. | Change from Baseline to Week 24 |
| Incidence and severity of adverse events | From Baseline to Week 24 |
| Palo Alto |
| California |
| 94305 |
| United States |
| Children's Healthcare of Atlanta | Atlanta | Georgia | 30342 | United States |
| Minnesota Clinical Study Center | New Brighton | Minnesota | 55112 | United States |
| Mayo Clinic | Rochester | Minnesota | 55905 | United States |
| Vascular Birthmark Institute | New York | New York | 10016 | United States |
| University of North Carolina | Chapel Hill | North Carolina | 27516 | United States |
| Cincinnati Children's Hospital | Cincinnati | Ohio | 45229 | United States |
| Cleveland Clinic | Cleveland | Ohio | 44195 | United States |
| Penn State Hershey Medical Center | Hershey | Pennsylvania | 17033 | United States |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | United States |
| Vanderbilt Children's Hospital | Nashville | Tennessee | 37232 | United States |
| University of Texas, Dell Children's | Austin | Texas | 78723 | United States |
| Texas Children's Hospital | Houston | Texas | 77030 | United States |
| University of Utah Health | Salt Lake City | Utah | 84113 | United States |
| ID | Term |
|---|---|
| D008202 | Lymphangioma |
| D044148 | Lymphatic Abnormalities |
| D054079 | Vascular Malformations |
| D000013 | Congenital Abnormalities |
| ID | Term |
|---|---|
| D018190 | Neoplasm, Lymphatic Tissue |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
| D008206 | Lymphatic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D018376 | Cardiovascular Abnormalities |
| D002318 | Cardiovascular Diseases |
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