Clinical trials: Hereditary Cancer Syndrome
Neurofibromatosis Type II, NF2
Study to Determine Optimal Dose, Evaluate the Efficacy and Safety of PRG-N-01 in Patients With Neurofibromatosis Type II
The goal of this clinical trial is to learn if Trineumin(Code name:PRG-N-01) works to treat Neurofibromatosis Type II(NF2) in adults. It will also learn about the safety and tolerability and toxicity of PRG-N-01.
- Trial name
- NCT07131722
- Testing
- Trineumin
- Volunteers
- 25 people
- Sponsor
- PRG Science & Technology Co., Ltd.
Leukemia, Myelodysplastic Syndromes
VNP40101M in Treating Patients With Acute Myelogenous Leukemia or High-Risk Myelodysplasia
RATIONALE: Drugs used in chemotherapy, such as VNP40101M and hydroxyurea, work in different ways to stop cancer cells from dividing so they stop growing or die.
- Conditions
- Leukemia, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms
- Trial name
- NCT00083187
- Testing
- hydroxyurea, laromustine
- Where
- 5 sites in United States, France and 1 more
- Volunteers
- 230 people
- Sponsor
- Vion Pharmaceuticals
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 Nevus Syndrome, Skin Neoplasm
A Study of the Efficacy and Safety of ASN-002 in Adult Patients With Low-risk Nodular Basal Cell Carcinoma
The study evaluates whether ASN-002 is safe and effective in the treatment of nodular basal cell carcinoma (nBCC) in patients aged 18 years or over.
- Conditions
- Basal Cell Nevus Syndrome, Skin Neoplasm, Nodular Basal Cell Carcinoma of Skin
- Trial name
- ASN-002-001 (NCT02550678)
- Testing
- ASN-002, 5-FU
- Where
- 4 sites in Australia
- Volunteers
- 16 people
- Sponsor
- Ascend Biopharmaceuticals Ltd
Neurofibromatosis Type 1
Phase I Study to Assess the Effect of Food on the PK and Gastrointestinal Tolerability of Selumetinib in Adolescent Children With Neurofibromatosis Type 1 Related Plexiform Neurofibromas
This study in adolescent participants with NF1 who have inoperable PN is designed to evaluate the effect of a low fat meal on steady state selumetinib exposure; to assess the effect on GI tolerability when selumetinib is dosed…
- Trial name
- NCT05101148
- Testing
- Selumetinib
- Where
- 9 sites in United States, Poland and 2 more
- Volunteers
- 24 people
- Sponsor
- AstraZeneca
Lower Urinary Tract Symptoms, Bladder Outlet Obstruction
A Study of Mirabegron (YM178) in Men With Lower Urinary Tract Symptoms (LUTS) and Bladder Outlet Obstruction (BOO)
This study examined the safety, tolerability, and efficacy of mirabegron (YM178) compared to placebo.
- Trial name
- NCT00410514
- Testing
- Mirabegron
- Where
- 27 sites in United States, Canada
- Volunteers
- 200 people
- Sponsor
- Astellas Pharma Inc
Tuberous Sclerosis Complex
Clinical Study of NPC-12Y Gel in Patients With Skin Lesions Associated With TSC
The purpose of this study is to investigate the efficacy and safety of NPC-12Y gel compared with placebo for skin lesions associated with tuberous sclerosis.
- Trial name
- NCT05495425
- Testing
- NPC-12Y gel
- Where
- 5 sites in Japan
- Volunteers
- 43 people
- Sponsor
- Nobelpharma
Secondary (AA) Amyloidosis, Rheumatoid Arthritis
Study of the Safety and Efficacy of NC-503 in Secondary (AA) Amyloidosis
The main objective of this study is to evaluate the safety and efficacy of NC-503 compared to placebo in patients with secondary (AA) amyloidosis using a composite assessment of clinical improvement/worsening of both renal and…
- Conditions
- Secondary (AA) Amyloidosis, Rheumatoid Arthritis, Nephrotic Syndrome, Familial Mediterranean Syndrome
- Trial name
- NCT00035334
- Testing
- NC-503 (Anti-amyloidotic (AA) Agent)
- Where
- 26 sites in United States, Finland and 10 more
- Volunteers
- 150 people
- Sponsor
- Bellus Health Inc. - a GSK company
Vasomotor Symptoms as a Sex Hormone-dependent Disorder in Women and Men
A Study to Learn How Different Levels of Decreased Liver Function Influence Blood Levels of Elinzanetant Compared to Normal Liver Function in Male and Female Participants
Researchers are looking for a better way to treat people who have symptoms caused by hormonal changes, like those that happen in women during menopause. These symptoms can include vasomotor symptoms.
- Trial name
- NCT04903821
- Testing
- BAY3427080
- Where
- 2 sites in Germany
- Volunteers
- 33 people, healthy volunteers welcome
- Sponsor
- Bayer
Metastatic Head and Neck Squamous Cell Carcinoma, Recurrent Head and Neck Squamous Cell Carcinoma
FORTIFI-HN01: A Study of Ficerafusp Alfa (BCA101) or Placebo in Combination With Pembrolizumab in First-Line PD-L1-pos, R or M HNSCC
Ficerafusp alfa is directed against two targets, Epidermal Growth Factor Receptor (EGFR) and Transforming Growth Factor beta (TGF-β).
- Trial name
- FORTIFI-HN01 (NCT06788990)
- Testing
- Ficerafusp alfa, Pembrolizumab (KEYTRUDA®)
- Where
- 231 sites in United States, Argentina and 26 more
- Volunteers
- 650 people
- Sponsor
- Bicara Therapeutics
Neurogenic Detrusor Overactivity
Open-label, Long-term Safety, Efficacy, and Pharmacokinetics Study of Vibegron in Pediatric Subjects 2 Years to < 18 Years of Age With NDO and on CIC
The purpose of this study is to evaluate the safety, efficacy, and PK of Vibegron in pediatric participants with NDO who are regularly using CIC
- Trial name
- KANGUROO (NCT05491525)
- Testing
- Vibegron
- Where
- 38 sites in United States, Belgium and 14 more
- Volunteers
- 71 people
- Sponsor
- Urovant Sciences GmbH
Familial Adenomatous Polyposis
Evaluate REC-4881 in Participants With Familial Adenomatous Polyposis (FAP)
This is a multicenter, two-part trial in participants with FAP.
- Trial name
- TUPELO (NCT05552755)
- Testing
- REC-4881
- Where
- 17 sites in United States
- Volunteers
- 67 people
- Sponsor
- Recursion Pharmaceuticals Inc.
Breast Cancer, Advanced Solid Tumors
A Phase I/II Study of BEZ235 in Patients With Advanced Solid Malignancies Enriched by Patients With Advanced Breast Cancer
This is a first-in-human, phase I/Ib clinical research study with BEZ235, an inhibitor of phosphatidylinositol 3'-kinase (PI3K).
- Conditions
- Breast Cancer, Advanced Solid Tumors, Cowden Syndrome
- Trial name
- NCT00620594
- Testing
- BEZ235
- Where
- 15 sites in United States, Germany and 3 more
- Volunteers
- 183 people
- Sponsor
- Novartis Pharmaceuticals
Tuberous Sclerosis, Angiofibroma
Long-term Trial of Topical Sirolimus to Angiofibroma in Patient With Tuberous Sclerosis Complex
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…
- Conditions
- Tuberous Sclerosis, Angiofibroma, Hypomelanotic Macule, Plaque
- Trial name
- NCT02634931
- Testing
- NPC-12G gel
- Where
- Japan
- Volunteers
- 94 people
- Sponsor
- Nobelpharma
Multiple Myeloma
WT1 Vaccine Treatment of Patients With Multiple Myeloma After Autologous Stem Cell Transplantation
The purpose of this trial is to assess the immune response after vaccination with a peptide vaccine called galinpepimut-S (or GPS) in a type of blood cancer called multiple myeloma.
- Trial name
- NCT01827137
- Testing
- Galinpepimut-S, GM-CSF, Montanide
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Sellas Life Sciences Group
Adenomatous Polyposis Coli
A Study of JNJ-64251330 in Participants With Familial Adenomatous Polyposis
The purpose of this study is to determine the effect of JNJ-64251330 in participants with Familial Adenomatous Polyposis (FAP) on colorectal polyp burden (sum of the polyp diameters).
- Trial name
- NCT05014360
- Testing
- JNJ-64251330
- Where
- 16 sites in United States, France and 5 more
- Volunteers
- 42 people
- Sponsor
- Janssen Research & Development, LLC
Cryopyrin-Associated Periodic Syndromes
Kineret CAPS Post Authorisation Study
A non-interventional, post authorization safety study to evaluate the safety of Kineret in the treatment of Cryopyrin Associated Periodic Syndromes (CAPS) in routine clinical care with regard to serious infections, malignancies…
- Trial name
- NCT02326376
- Testing
- anakinra (Kineret)
- Where
- 2 sites in Netherlands, United Kingdom
- Volunteers
- 12 people
- Sponsor
- Swedish Orphan Biovitrum
Neurofibromatosis Type 1, Plexiform Neurofibromas
Selumetinib in Chinese Paediatric With Post-operative NF1-PNs, PhaseⅡ, Double-Blinded, Placebo-Controlled Study
This is a phase II, multicenter, randomised, parallel, double-blind, placebo-controlled study assessing the efficacy and safety of the MEKi selumetinib compared with placebo in Chinese paediatric participants with post-operative…
- Conditions
- Neurofibromatosis Type 1, Plexiform Neurofibromas, Post-operative
- Trial name
- PROTECT (NCT05825365)
- Testing
- Selumetinib
- Sponsor
- AstraZeneca
Cancer
Fentanyl Sublingual Spray in Treating Patients With Breakthrough Cancer Pain
This is a phase III, randomized, double-blind, placebo-controlled, multicenter study of the clinical response to fentanyl sublingual spray as a treatment for breakthrough cancer pain.
- Trial name
- NCT00538850
- Testing
- Fentanyl sublingual spray
- Where
- United States
- Volunteers
- 130 people
- Sponsor
- INSYS Therapeutics Inc
Hereditary Cancer
Impact of a Process Intervention on Screening and Testing Outcomes for Common Hereditary Cancer Syndromes
A prospective, non-interventional study to evaluate the impact of a process engineering intervention on screening and testing outcomes for common hereditary cancer syndromes in community-based OB/GYN settings.
- Trial name
- NCT03081455
- Testing
- Diagnostic Test
- Where
- 2 sites in United States
- Volunteers
- 145 people, healthy volunteers welcome
- Sponsor
- Myriad Genetic Laboratories, Inc.