Clinical trials: Epidermal Nevus Syndrome
Epidermal Nevi, Nevus Sebaceus
12-week Study of NFX-179 Gel in Subjects With Epidermal Nevi
This study of NFX-179 is an open-label study, evaluating safety, tolerability, pharmacodynamic activity, and the clinical effect in subjects with Epidermal Nevus(ENS). NFX-179 is formulated as a gel for topical administration.
- Trial name
- NCT05195762
- Testing
- NFX-179 Gel 1.50%
- Where
- United States
- Sponsor
- Albert Chiou
Hypophosphatemia, Hypophosphatemic Rickets
Open Label Trial Assessing Safety and Efficacy of Burosumab (KRN23), in a Patient With ENS and Hypophosphatemic Rickets
A 52 week, open label trial to assess the safety and efficacy of KRN23, an investigational antibody to FGF23, in a single pediatric patient with Epidermal Nevus Syndrome(ENS) and associated hypophosphatemic rickets A 26 weeks…
- Conditions
- Hypophosphatemia, Hypophosphatemic Rickets, Pain, Chronic
- Trial name
- NCT03581591
- Testing
- Burosumab
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Redwood Dermatology Sciences
Vascular Anomaly, Generalized Lymphatic Anomaly
Identification of Biomarkers for Patients with Vascular Anomalies
The study will use blood (serum and plasma) and tissue obtained from participants undergoing prescribed surgical resection of vascular anomalies of interest proposed in this study.
- Conditions
- Vascular Anomaly, Generalized Lymphatic Anomaly, Kaposiform Hemangioendothelioma, Kaposiform Lymphangiomatosis
- Trial name
- NCT03001180
- Where
- 2 sites in United States
- Volunteers
- 1,000 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
PIK3CA-Related Overgrowth Spectrum (PROS), Lymphatic Malformations
A Phase 2 Study of Mutant-selective PI3Kα Inhibitor, RLY-2608, in Adults and Children With PIK3CA Related Overgrowth Spectrum and Malformations Driven by PIK3CA Mutation (The ReInspire Study)
This is a 3-part Phase 2 randomized study evaluating the safety and efficacy of the mutant-selective PI3Kα inhibitor, zovegalisib (RLY-2608), in adults and children with PIK3CA Related Overgrowth Spectrum (PROS) and malformations…
- Conditions
- PIK3CA-Related Overgrowth Spectrum (PROS), Lymphatic Malformations, Vascular Malformations, PIK3CA Mutation
- Trial name
- NCT06789913
- Testing
- RLY-2608
- Where
- 40 sites in United States, Australia and 7 more
- Volunteers
- 277 people
- Sponsor
- Relay Therapeutics, Inc.
Advanced Solid Tumor
A Clinical Study to Test if an Investigational Treatment Called BNT326 is Safe and Potentially Beneficial When Used Alone or in Combination With Other Investigational Treatments Such as BNT327, for People With Advanced Malignant Tumors
This study will evaluate the safety, efficacy, optimal dose, and pharmacokinetics (PK) of BNT326 as monotherapy (Part 1) and as combination treatment with immunotherapeutic agents (Part 2) in participants with histologically or…
- Trial name
- NCT07070232
- Testing
- BNT326, Pumitamig, Itraconazole
- Where
- 77 sites in United States, Australia and 7 more
- Volunteers
- 1,438 people
- Sponsor
- BioNTech SE
Tumor-Induced Osteomalacia or Epidermal Nevus Syndrome
A Study of KRN23 in Subjects With Tumor-Induced Osteomalacia or Epidermal Nevus Syndrome
Before switching to the post-marketing study: To evaluate the efficacy and safety of KRN23 after its 144-week once every 4 weeks (Q4W) repeated SC administration to Japanese and Korean patients with TIO or ENS by a multicenter…
- Trial name
- NCT02722798
- Testing
- KRN23
- Where
- 3 sites in Japan, South Korea
- Volunteers
- 14 people
- Sponsor
- Kyowa Kirin Co., Ltd.
Tumor Induced Osteomalacia (TIO), Epidermal Nevus Syndrome (ENS)
Study of Burosumab (KRN23) in Adults With Tumor-Induced Osteomalacia (TIO) or Epidermal Nevus Syndrome (ENS)
The primary objectives of this study are to evaluate the effect of burosumab treatment on: Increasing serum phosphorus levels in adults with TIO or ENS-associated osteomalacia Improvement in TIO/ENS-associated osteomalacia as…
- Trial name
- NCT02304367
- Testing
- Burosumab
- Where
- 7 sites in United States
- Volunteers
- 17 people
- Sponsor
- Kyowa Kirin, Inc.
Epidermal Nevi
A Pilot Study to Examine the Efficacy and Tolerability of Sirolimus on Epidermal Nevi Lesions in Patients 6 Years Old and Above.
We hypothesize that topical sirolimus could suppress the mTOR activation of linear verrucous epidermal nevi, particularly when the variants are involving genes upstream of mTOR and perhaps even those of the RAS pathway through…
- Trial name
- NCT07813533
- Testing
- sirolimus topical gel 0.2%
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- Northwestern University
Lymphatic Malformation, Generalized Lymphatic Anomaly (GLA)
Lymphatic Anomalies Registry for the Assessment of Outcome Data
Lymphatic anomalies are a rare subset of vascular anomalies that are poorly understood. the understanding of the natural history, long-term outcomes, risk factors for morbidity and mortality, and the relative benefit of medical…
- Conditions
- Lymphatic Malformation, Generalized Lymphatic Anomaly (GLA), Central Conducting Lymphatic Anomaly, CLOVES Syndrome
- Trial name
- NCT02399527
- Where
- United States
- Volunteers
- 1,000 people
- Sponsor
- Boston Children's Hospital
Cutaneous Skeletal Hypophosphatemia Syndrome (CSHS), Epidermal Nevus Syndrome
Burosumab for CSHS
Burosumab (also known as the drug, Crysvita®) is a fully human immunoglobulin G1 (IgG1) monoclonal antibody (mAb) that binds to and inhibits the activity of fibroblast growth factor 23 (FGF23), leading to an increase in serum…
- Trial name
- NCT03993821
- Testing
- Burosumab
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Laura Tosi
Epidermal Nevus Syndrome
A Trial to Assess the Safety and Efficacy of KRN23 in Epidermal Nevus Syndrome (ENS)
KRN23 is a fully human immunoglobulin monoclonal antibody (mAb) that binds to and inhibits the activity of fibroblast growth factor 23 (FGF23), leading to an increase in serum phosphorus levels.
- Trial name
- ENS (NCT04320316)
- Testing
- Crysvita (burosumab-twza) Treatment
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- University of Alabama at Birmingham
Venous Malformations, Lymphatic Malformations
A Study to Investigate Efficacy and Safety of KP-001 Compared With Placebo in Patients Aged ≥2 Years With Common VM, Common LM, or KTS/CLOVES Syndrome
This is a phase 3, double-blind, randomized, placebo-controlled, parallel group, adaptive, multicenter study planned to be conducted at multiple sites in North America, Canada, Taiwan and South Korea.
- Conditions
- Venous Malformations, Lymphatic Malformations, Klippel-Trenaunay Syndrome, CLOVES Syndrome
- Trial name
- S-KY (NCT07285005)
- Testing
- KP-001
- Where
- 5 sites in United States, South Korea and 1 more
- Volunteers
- 150 people
- Sponsor
- Kaken Pharmaceutical
Chronic Venous Insufficiency, CVI, Venous Malformations
Safety of the Sonablate HIFU System for the Ablation of Incompetent Veins of the Periphery
The goal of this clinical trial is to learn if the Sonablate High Intensity Focused Ultrasound device can be safely used to treat patients with Chronic Venous Incompetence (CVI) including those with venous malformations of the…
- Conditions
- Chronic Venous Insufficiency, CVI, Venous Malformations, Venous Leg Ulcers, Klippel-Trenaunay Syndrome
- Trial name
- HIFIVE (NCT06642051)
- Testing
- High Intensity focused ultrasound
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Sonablate
PIK3CA-related Overgrowth Spectrum, CLOVES Syndrome
National Evaluation of Patients With PIK3CA-Related Overgrowth Spectrum (PROS)
Overgrowth syndromes are rare genetic disorders defined by tissue hypertrophy that can be either localized or generalized, affecting both latitudinal and longitudinal growth.
- Conditions
- PIK3CA-related Overgrowth Spectrum, CLOVES Syndrome, Klippel Trenaunay Syndrome, Megalencephaly
- Trial name
- COSY (NCT05563831)
- Testing
- national registry
- Where
- France
- Volunteers
- 28 people
- Sponsor
- Institut National de la Santé Et de la Recherche Médicale, France