Clinical trials: Gorlin Syndrome
PTCH1 or SMO Activated Solid and Hematologic Tumors
LDE225 for Patients With PTCH1 or SMO Mutated Tumors
The purpose of this signal seeking study is to determine whether treatment with LDE225 demonstrates sufficient efficacy in hedgehog pathway-mutated solid tumors and/or hematologic malignancies to warrant further study
- Trial name
- SIGNATURE (NCT02002689)
- Testing
- LDE225
- Where
- 10 sites in United States
- Volunteers
- 10 people
- Sponsor
- Novartis Pharmaceuticals
Desmoplastic/Nodular Medulloblastoma, Medulloblastoma
Combination Chemotherapy in Treating Younger Patients With Newly Diagnosed, Non-metastatic Desmoplastic Medulloblastoma
This phase II trial studies how well combination chemotherapy works in treating younger patients with newly diagnosed, non-metastatic desmoplastic medulloblastoma.
- Conditions
- Desmoplastic/Nodular Medulloblastoma, Medulloblastoma, Medulloblastoma With Extensive Nodularity, Nevoid Basal Cell Carcinoma Syndrome
- Trial name
- NCT02017964
- Testing
- Carboplatin, Cognitive Assessment, Cyclophosphamide
- Where
- 173 sites in United States, Australia and 2 more
- Volunteers
- 26 people
- Sponsor
- Children's Oncology Group
Basal Cell Carcinoma, Locally Advanced Basal Cell Carcinoma
Oral Hedgehog Inhibitors in the Treatment of Basal Cell Carcinoma in the Netherlands: a Prospective Registration Study
Background: Oral hedgehog inhibitors vismodegib and sonidegib have been used for the treatment of locally advanced (laBCC), metastatic basal cell carcinoma (mBCC) and in basal cell nevus syndrome (BCNS) patients.
- Conditions
- Basal Cell Carcinoma, Locally Advanced Basal Cell Carcinoma, Metastatic Basal Cell Carcinoma, Gorlin Syndrome
- Trial name
- NCT05463757
- Testing
- Vismodegib, Sonidegib
- Where
- 8 sites in Netherlands
- Volunteers
- 80 people
- Sponsor
- Maastricht University Medical Center
Basal Cell Carcinoma
Efficacy, Safety and Tolerability of Topically Applied LDE225 Cream (Hedgehog Pathway Inhibitor) in Adult Patients With Nevoid Basal Cell Carcinoma Syndrome (NBCCS)
This 22 week study will assess the efficacy, safety, and tolerability of LDE225 versus vehicle when applied topically to basal cell carcinoma (BCC) in patients with NBCCS. Patients will treat multiple BCCs for up to 12 weeks.
- Trial name
- NCT03070691
- Testing
- LDE225B, Vehicle
- Where
- 35 sites in Belgium, Canada and 8 more
- Sponsor
- Novartis Pharmaceuticals
Basal Cell Carcinoma, Keratosis Palmaris et Plantaris
The Evaluation of Oral Acitretin in the Treatment of Psoriasis, Cutaneous Disorders of Keratinization, Multiple Basal Cell Carcinomas and Other Retinoid Responsive Diseases
This is a continuing study which evaluates the long-term safety and efficacy of oral acitretin in an open manner in the treatment of psoriasis, cutaneous disorders of keratinization, multiple basal cell carcinomas and other…
- Conditions
- Basal Cell Carcinoma, Keratosis Palmaris et Plantaris, Psoriasis
- Trial name
- NCT00005660
- Where
- United States
- Volunteers
- 130 people
- Sponsor
- National Cancer Institute (NCI)
Gorlin Syndrome
Efficacy and Safety of Patidegib Gel 2% for Preventing Basal Cell Carcinomas on the Face of Adults With Gorlin Syndrome
The aim of this clinical study is to find out how well Patidegib Gel 2% works in preventing new basal cell carcinomas (BCCs) developing on the face of adults with Gorlin syndrome, and how safe Patidegib Gel 2% is to use.
- Trial name
- NCT06050122
- Testing
- Patidegib Topical Gel, Patidegib Topical Gel with no active patidegib
- Where
- 40 sites in United States, France and 5 more
- Volunteers
- 113 people
- Sponsor
- Sol-Gel Technologies, Ltd.
Melanoma (Skin), Non-melanomatous Skin Cancer
Isotretinoin in Preventing Skin Cancer
RATIONALE: Chemoprevention therapy is the use of certain drugs to try to prevent the development or recurrence of cancer. Isotretinoin may be effective in preventing the development or recurrence of skin cancer.
- Trial name
- NCT00025012
- Testing
- isotretinoin
- Where
- United States
- Sponsor
- National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
Carcinoma, Basal Cell, Recurrent Skin Cancer
Pilot Study of Sonidegib and Buparlisib in Treating Patients With Advanced or Metastatic Basal Cell Carcinoma
This pilot trial studies how well sonidegib and buparlisib work in treating patients with basal cell carcinoma that has spread to other places in the body.
- Conditions
- Carcinoma, Basal Cell, Recurrent Skin Cancer, Skin Neoplasms, Basal Cell Nevus Syndrome
- Trial name
- NCT02303041
- Testing
- Buparlisib, Sonidegib
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Anne Chang
Basal Cell Nevus Syndrome
Nivolumab With Vismodegib in Patients With Basal Cell Nevus Syndrome
This is a single-arm, phase II study to assess the efficacy of combined SMO and PD-1 inhibition with Vismodegib (SMO inhibitor) and Nivolumab (anti-PD-1 antibody) in BCNS patients (target enrollment of 22 patients), with a…
- Trial name
- NCT03767439
- Testing
- Vismodegib, Nivolumab, Ipilimumab
- Sponsor
- Columbia University
Neoplastic Syndrome
Topical Tazarotene in Treating Patients With Basal Cell Skin Cancer and Basal Cell Nevus Syndrome on the Face
RATIONALE: Drugs used in chemotherapy, such as tazarotene, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing.
- Trial name
- NCT00489086
- Testing
- tazarotene
- Where
- 2 sites in United States
- Volunteers
- 36 people
- Sponsor
- UCSF Benioff Children's Hospital Oakland
Treatment for Basal Cell Carcinomas (BCCs) in Gorlin Syndrome Patients
A Trial to Evaluate the Safety, Local Tolerability, Pharmacokinetics and Pharmacodynamics of LDE225 on Skin Basal Cell Carcinomas in Gorlin Syndrome Patients
Part I was a double-blind, randomized, vehicle-controlled Proof of Concept (PoC) study to evaluate the safety, local tolerability, pharmacokinetics and pharmacodynamics of multiple topical administrations of LDE225 (a specific…
- Trial name
- NCT00961896
- Testing
- Vehicle, LDE225 0.25%, LDE225 0.75%
- Where
- 3 sites in Austria, Switzerland
- Volunteers
- 18 people
- Sponsor
- Novartis Pharmaceuticals
Basal Cell Carcinoma (BCC)
Blue-Light Photodynamic Therapy and Sonidegib for Multiple Basal Cell Carcinomas
This research study is testing combination Blue-light photodynamic therapy and Sonidegib as a possible treatment for people with multiple basal cell carcinoma lesions.
- Trial name
- NCT06623201
- Testing
- aminolevulinic acid HCL (ALA), BLU-U device model 4170E
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Nathalie Zeitouni
Basal Cell Nevus Syndrome
Study of BMS-833923 in Two Specific Patients With Basal Cell Nevus Syndrome
This is an extension study of Protocol CA194002 to allow 2 specific participants with basal cell nevus syndrome in the CA194002 study at Princess Margaret Cancer Centre who are still benefitting from the study drug BMS-833923 to…
- Trial name
- NCT02100371
- Testing
- BMS-833923
- Where
- Canada
- Volunteers
- 2 people
- Sponsor
- University Health Network, Toronto
BCCs in Gorlin Syndrome Patients
CODY: A Study Evaluating The Safety And Efficacy Of QTORIN 3.9% Sirolimus Topical Gel For The Prevention Of Basal Cell Carcinomas (BCCs) In Patients With Gorlin Syndrome
A Phase 2b study looks at the safety and efficacy of a treatment that is being investigated for people with certain diseases. This study will be conducted at multiple centers in the United States where participants with Gorlin…
- Trial name
- NCT04893486
- Testing
- PTX-022, Vehicle comparator
- Where
- 16 sites in United States, United Kingdom
- Volunteers
- 73 people
- Sponsor
- Palvella Therapeutics, Inc.
Basal Cell Carcinomas
Clinical Trial of Patidegib Gel 2%, 4%, and Vehicle Applied Once or Twice Daily to Decrease the GLI1 Biomarker in Sporadic Nodular Basal Cell Carcinomas
This is a double-blind, dose escalating, randomized, vehicle-controlled study designed to compare the efficacy and safety of patidegib gel 2% and 4% applied once or twice daily in comparison with that of vehicle in patients with…
- Trial name
- BCC (NCT02828111)
- Testing
- Patidegib, Vehicle gel
- Where
- United States
- Volunteers
- 36 people
- Sponsor
- PellePharm, Inc.
Quality of Life, Ichthyosis
Qualitative Study in Patients With Genodermatoses and Healthcare Professionals on Reproductive Counselling
The goal of this observational study is to understand the perspectives and needs of patients with genodermatoses and their partners who wish to have children, regarding their decision-making process and their consideration of…
- Conditions
- Quality of Life, Ichthyosis, Palmoplantar Keratoses, Epidermolysis Bullosa
- Trial name
- NCT06330350
- Testing
- Qualitative interview
- Where
- Netherlands
- Volunteers
- 25 people
- Sponsor
- Maastricht University Medical Center
Basal Cell Carcinoma in Basal Cell Nevus Syndrome
Open-label Trial of SUBA™-Itraconazole (SUBA-Cap) in Subjects With Basal Cell Carcinoma Nevus Syndrome (BCCNS)
The study will assess the safety and efficacy of SUBA-Cap in subjects with Basal Cell Carcinoma Nevus Syndrome.
- Trial name
- NCT02354261
- Testing
- SUBA-Itraconazole
- Where
- 5 sites in United States
- Volunteers
- 38 people
- Sponsor
- HedgePath Pharmaceuticals, Inc.
Ichthyosis, Palmoplantar Keratoses
Reproductive Options in Inherited Skin Diseases
The goal of this observational study is to learn about the indications for prenatal diagnostics and preimplantation genetic testing for patients/couples affected by an inherited skin disease, and evaluate the clinical outcomes of…
- Conditions
- Ichthyosis, Palmoplantar Keratoses, Epidermolysis Bullosa, Ectodermal Dysplasia
- Trial name
- REPRO-ISD (NCT06330324)
- Where
- Netherlands
- Volunteers
- 650 people
- Sponsor
- Maastricht University Medical Center
Acute Leukemia, Adenomatous Polyposis
Familial Investigations of Childhood Cancer Predisposition
NOTE: This is a research study and is not meant to be a substitute for clinical genetic testing. Families may never receive results from the study or may receive results many years from the time they enroll.
- Conditions
- Acute Leukemia, Adenomatous Polyposis, Adrenocortical Carcinoma, AML
- Trial name
- SJFAMILY (NCT03050268)
- Where
- United States
- Volunteers
- 1,500 people
- Sponsor
- St. Jude Children's Research Hospital
Basal Cell Carcinoma
A Study of Two Vismodegib Regimens in Participants With Multiple Basal Cell Carcinomas
This randomized, double-blind, regimen-controlled, phase II, multicenter study will assess the efficacy and safety of two different vismodegib regimens in participants with multiple basal cell carcinoma.
- Trial name
- NCT01815840
- Testing
- Vismodegib
- Where
- 58 sites in United States, Austria and 8 more
- Volunteers
- 229 people
- Sponsor
- Hoffmann-La Roche