Clinical trials: Genetic Cancer Syndromes
Von Hippel Lindau Disease
MSD Belzutifan PAS
This is a prospective observational cohort study evaluating the effectiveness and safety of belzutifan treatment in routine clinical practice.
- Trial name
- NCT06554730
- Testing
- Belzutifan
- Sponsor
- Merck Sharp & Dohme LLC
Vasomotor Symptoms as a Sex Hormone-dependent Disorder in Women and Men, Hot Flashes
A Study to Learn How Safe the Study Treatment Elinzanetant is and How it Moves Into, Through and Out of the Body When Given as Single Increasing Doses That Are Higher Than Normally Used Compared to Placebo and Moxifloxacin in Healthy Participants
Researchers are looking for a better way to treat vasomotor symptoms, a condition of having hot flashes. The condition is caused by hormonal changes and occurs primarily in women, but can also affect men.
- Conditions
- Vasomotor Symptoms as a Sex Hormone-dependent Disorder in Women and Men, Hot Flashes, Healthy Volunteers
- Trial name
- NCT05481528
- Testing
- Moxifloxacin, Elinzanetant (BAY3427080)
- Where
- Germany
- Volunteers
- 51 people, healthy volunteers welcome
- Sponsor
- Bayer
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
Focal Cortical Dysplasia, Tuberous Sclerosis Complex (TSC)
Safety, Tolerability, and Pharmacokinetics of SVG103 (Paxalisib) in Focal Cortical Dysplasia Type II (FCD-II), Tuberous Sclerosis Complex (TSC) or Hemimegalencephaly (HME)
This is a multinational, open-label, single-arm trial of adjunctive SVG103 (paxalisib) treatment in adults with FCD-II, TSC, and HME.
- Conditions
- Focal Cortical Dysplasia, Tuberous Sclerosis Complex (TSC), Hemimegalencephaly
- Trial name
- NCT07287202
- Testing
- SVG103
- Where
- 2 sites in Australia
- Volunteers
- 12 people
- Sponsor
- Sovargen
Neurofibromatosis Type 1
Prevalence, Clinical Characteristics, Progression, and Management of Neurofibromatosis Type 1 in Egypt (NF1-Egy)
Neurofibromatosis type 1 (NF1), a genetic disorder, results from NF1 gene mutations with nearly complete penetrance (1). NF1 is considered common as a rare disease; it has a birth incidence of approximately one every 3000 and a…
- Trial name
- PRECIOUS Egypt (NCT07221331)
- Where
- 9 sites in Egypt
- Volunteers
- 200 people
- Sponsor
- AstraZeneca
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
Solid Tumor
A Study of LY3200882 in Participants With Solid Tumors
The main purpose of this study is to evaluate the safety of the study drug known as LY3200882 in participants with solid tumors.
- Trial name
- NCT02937272
- Testing
- LY3200882, LY3300054, Gemcitabine
- Where
- 18 sites in United States, Australia and 6 more
- Volunteers
- 223 people
- Sponsor
- Eli Lilly and Company
Advanced Stage Solid Tumors
First in Human Dose Escalation Study of Vactosertib (TEW-7197) in Subjects With Advanced Stage Solid Tumors
The phase I dose escalation study will investigate the safety, tolerability, and pharmacokinetics of the TGF-β pathway inhibitor TEW 7197 in subjects with advanced, refractory solid tumors.
- Trial name
- NCT02160106
- Testing
- TEW-7197
- Where
- 3 sites in United States
- Volunteers
- 35 people
- Sponsor
- MedPacto, Inc.
Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child
Study of Gene Modified Donor T-cells Following TCR Alpha Beta Positive Depleted Stem Cell Transplant
This study will evaluate pediatric patients with malignant or non-malignant blood cell disorders who are having a blood stem cell transplant depleted of T cell receptor (TCR) alfa and beta cells that comes from a partially…
- Conditions
- Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child, Lymphoma, Non-Hodgkin, Myelodysplastic Syndromes
- Trial name
- NCT03301168
- Testing
- BPX-501 T cells, Rimiducid
- Where
- 10 sites in United States
- Volunteers
- 120 people
- Sponsor
- Bellicum Pharmaceuticals
Tuberous Sclerosis Complex, Seizures
An Open-label Extension Trial of Cannabidiol (GWP42003-P, CBD) for Seizures in Tuberous Sclerosis Complex (GWPCARE6)
This trial consists of 2 parts: a double-blinded phase and an open-label extension phase. The open-label extension phase only will be described in this record. All participants will receive the same dose of GWP42003-P.
- Trial name
- NCT02544750
- Testing
- GWP42003-P
- Where
- United States
- Volunteers
- 199 people
- Sponsor
- Jazz 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
Advanced Breast Cancer
Study to Assess the Safety of Alpelisib Plus Fulvestrant, in Men and Post-menopausal Women With HR-positive, HER2-negative, Advanced Breast Cancer (aBC) With PIK3CA Mutation, Whose Disease Progressed on or After Endocrine Treatment
The purpose of this study was to determine the safety of alpelisib plus fulvestrant in men and post-menopausal women with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative, advanced or…
- Trial name
- ALPINIST (NCT05631795)
- Testing
- Alpelisib, Fulvestrant
- Where
- 12 sites in India
- Volunteers
- 40 people
- Sponsor
- Novartis Pharmaceuticals
Basal Cell Nevus Syndrome
Extension Study of Patidegib Topical Gel, 2% in Subjects With Gorlin Syndrome (Basal Cell Nevus Syndrome)
This is a multicenter, open label extension study evaluating the safety of Patidegib Topical Gel, 2%, applied topically twice daily to the face of adult subjects with Gorlin syndrome.
- Trial name
- NCT04308395
- Testing
- Patidegib Topical Gel, 2%
- Where
- 37 sites in United States, Belgium and 7 more
- Volunteers
- 108 people
- Sponsor
- Sol-Gel Technologies, Ltd.
RET-altered Non Small Cell Lung Cancer, Medullary Thyroid Cancer
Phase 1/2 Study of the Highly-selective RET Inhibitor, Pralsetinib (BLU-667), in Participants With Thyroid Cancer, Non-Small Cell Lung Cancer, and Other Advanced Solid Tumors
This is a Phase 1/2, open-label, first-in-human (FIH) study designed to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary antineoplastic activity of pralsetinib (BLU-667)…
- Conditions
- RET-altered Non Small Cell Lung Cancer, Medullary Thyroid Cancer, RET-altered Papillary Thyroid Cancer, RET-altered Colon Cancer
- Trial name
- ARROW (NCT03037385)
- Testing
- pralsetinib (BLU-667)
- Where
- 71 sites in United States, Belgium and 11 more
- Volunteers
- 590 people
- Sponsor
- Hoffmann-La Roche
Nevus, Dysplasia; Epithelial
Skin Fluorescent Imaging (SFI) System in Patients With Nevi
The Sponsor has developed the Skin Fluorescent Imaging (SFI) system, an in vivo imaging modality, for the purpose of providing physicians with insight into the biological changes occurring during tissue remodeling in a nevus.
- Trial name
- NCT04368247
- Testing
- Skin Fluorescent Imaging
- Where
- 4 sites in United States
- Volunteers
- 300 people
- Sponsor
- Orlucent, Inc
Neurofibromatosis 2
PTC299 for Treatment of Neurofibromatosis Type 2
Formation of new blood vessels (angiogenesis) is important for tumor growth in neurofibromatosis type 2 (NF2). It is known that tumors make a protein called vascular endothelial growth factor (VEGF) and there are higher levels of…
- Trial name
- NCT00911248
- Testing
- PTC299
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- PTC Therapeutics
Familial Adenomatous Polyposis Coli, FAP
Chemoprevention Trial in Familial Adenomatous Polyposis (FAP) Coli Using EPA
This purpose of this study is to investigate whether the number and size of rectal polyps can be reduced in patients with Familial Adenomatous Polyposis (FAP) by using a highly-purified form of a naturally occurring substance…
- Trial name
- NCT00510692
- Testing
- Eicosapentanoic Acid (EPA), Endoscopy, Biopsies taken
- Where
- United Kingdom
- Volunteers
- 58 people
- Sponsor
- S.L.A. Pharma AG
Biliary Tract Cancer, Cholangiocarcinoma
M7824 Monotherapy in Locally Advanced or Metastatic Second Line (2L) Biliary Tract Cancer (Cholangiocarcinoma and Gallbladder Cancer)
The study to evaluate M7824 monotherapy in participants with advanced or metastatic biliary tract cancer (BTC) who failed or were intolerant to first-line (1L) chemotherapy.
- Conditions
- Biliary Tract Cancer, Cholangiocarcinoma, Gallbladder Cancer
- Trial name
- NCT03833661
- Testing
- M7824
- Where
- 33 sites in United States, China and 7 more
- Volunteers
- 159 people
- Sponsor
- EMD Serono Research & Development Institute, Inc.
Neurofibromatosis Type 1, Plexiform Neurofibromas
US Selumetinib Registry
The purpose of this study is to understand treatment patterns and assess long-term effectiveness and safety outcomes associated with selumetinib treatment as well as to explore clinical and non-clinical factors affecting those…
- Trial name
- NCT05683678
- Where
- 10 sites in United States
- Volunteers
- 37 people
- Sponsor
- Alexion Pharmaceuticals, Inc.