Clinical trials: Hereditary Neoplastic Syndrome
Neurofibromatosis Type 1
Pharmacokinetics, Safety and Efficacy of the Selumetinib Granule Formulation in Children Aged ≥1 to <7 Years With NF1-related Symptomatic, Inoperable PN
This study is designed to define a dosing regimen and assess the pharmacokinetics(PK) and safety of the granule formulation; the study will also include descriptive analyses of exploratory efficacy endpoints.
- Trial name
- SPRINKLE (NCT05309668)
- Testing
- Selumetinib granule formulation, Selumetinib capsule formulation
- Where
- 16 sites in United States, Germany and 5 more
- Volunteers
- 36 people
- Sponsor
- AstraZeneca
Polycystic Ovary Syndrome
Study of Pharmacodynamics of LIK066 in Overweight and Obese Women With Polycystic Ovary Syndrome
The purpose of the study was to evaluate whether LIK066 can be developed for the treatment of polycystic ovary syndrome (PCOS) in overweight and obese women.
- Trial name
- PCOS (NCT03152591)
- Testing
- LIK066
- Where
- 5 sites in United States, Germany
- Volunteers
- 29 people
- Sponsor
- Novartis Pharmaceuticals
Sturge-Weber Syndrome (SWS), Glaucoma
A Study to Determine the Safety and Tolerability of 2 Concentrations of QLS-101
Safety and tolerability study of 2 concentrations of QLS-101 for adult subjects with Sturge Weber Syndrome (SWS)-related glaucoma due to elevated episcleral venous pressure (EVP).
- Conditions
- Sturge-Weber Syndrome (SWS), Glaucoma, Glaucoma Congenital, Ocular Hypertension
- Trial name
- NCT04947124
- Testing
- QLS-101ophthalmic solution 1%, QLS-101ophthalmic solution 2%
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Qlaris Bio, Inc.
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
Familial Adenomatous Polyposis (FAP)
Phase 3 Trial of eRapa in Patients With Familial Adenomatous Polyposis
The main goal of this clinical trial is to learn if the drug eRapa works to slow down the progression of disease in patients diagnosed with Familial Adenomatous Polyposis (FAP). Researchers will compare eRapa to Placebo.
- Trial name
- SERENTA (NCT06950385)
- Testing
- eRapa (encapsulated rapamycin)
- Where
- 29 sites in United States, Denmark and 5 more
- Volunteers
- 168 people
- Sponsor
- Rapamycin Holdings Inc.
Advanced Nonhaematologic Malignancies
Safety, Efficacy, and Tolerability of BOS172738 in Patients With Advanced Rearranged During Transfection (RET) Gene-Altered Tumors
This study will be conducted to assess the safety and tolerability of BOS172738 when administered to patients with advanced solid tumors with rearranged during transfection (RET) gene alterations and also to establish the maximum…
- Trial name
- NCT03780517
- Testing
- BOS172738
- Where
- 21 sites in United States, Belgium and 5 more
- Volunteers
- 117 people
- Sponsor
- Boston Pharmaceuticals
Carcinoma, Renal Cell
A Phase II Study of GSK1363089 (Formerly XL880) for Papillary Renal-Cell Carcinoma (PRC)
This clinical study is being conducted at multiple sites to determine the best confirmed response rate, safety, and tolerability of GSK1363089 treatment in papillary renal cell carcinoma.
- Trial name
- NCT00726323
- Testing
- foretinib (formerly GSK1363089 or XL880)
- Where
- 12 sites in United States
- Volunteers
- 74 people
- Sponsor
- GlaxoSmithKline
Basal Cell Carcinoma in Basal Cell Nevus Syndrome
Study of ASN-002 to Treat Basal Cell Carcinomas (BCCs) in Individuals With Basal Cell Nevus Syndrome (BCNS)
The primary objective is to confirm the safety of treating multiple BCCs once weekly x 3 weeks in individuals with Basal Cell Nevus Syndrome (BCNS).
- Trial name
- NCT03208296
- Testing
- ASN-002
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- Ascend Biopharmaceuticals Ltd
Acute Myeloid Leukemia (AML)
PKC412, Daunorubicin, and Cytarabine in Treating Patients With Newly Diagnosed Acute Myeloid Leukemia
RATIONALE: PKC412 may stop the growth of cancer cells by blocking the enzymes necessary for their growth. It may also increase the effectiveness of daunorubicin and cytarabine by making cancer cells more sensitive to the drugs.
- Trial name
- NCT00093600
- Testing
- cytarabine, daunorubicin hydrochloride, midostaurin
- Where
- 6 sites in United States, Germany
- Volunteers
- 69 people
- Sponsor
- Novartis 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
Non-squamous Metastatic Non-Small-Cell Lung Carcinoma
NIS to Examine the Effectiveness of TDC in Patients With Metastatic Non-squamous NSCLC and High-risk Genetic Alterations
This prospective, multicenter, non-interventional study (NIS) in Germany aims to collect real-life data of patients with non-squamous (NSQ) metastatic non-small cell lung cancer (mNSCLC) (incl.
- Trial name
- NAUTIC (NCT06494540)
- Where
- 30 sites in Germany
- Volunteers
- 600 people
- Sponsor
- AstraZeneca
Familial Adenomatous Polyposis
A Trial of Low Dose Sulindac Combined With Eflornithine in Patients With Familial Adenomatous Polyposis (FAP)
The purpose of this phase III study is to evaluate the safety and efficacy of the combination of eflornithine and sulindac compared to single agent sulindac or eflornithine in reducing the number of polyps in patients with…
- Trial name
- NCT01245816
- Testing
- Eflornithine plus Sulindac
- Sponsor
- Cancer Prevention Pharmaceuticals, Inc.
Alzheimer Disease
A Study to Assess Safety and Target Engagement of E2814 in Participants With Mild to Moderate Cognitive Impairment Due to Dominantly Inherited Alzheimer's Disease
The primary objective of the study is to assess the safety and tolerability of intravenous (IV) infusions of E2814 in participants with dominantly inherited Alzheimer's disease (DIAD), and to evaluate target engagement (TE) of…
- Trial name
- NCT04971733
- Testing
- E2814
- Where
- 3 sites in United States, United Kingdom
- Volunteers
- 8 people
- Sponsor
- Eisai Inc.
Melanoma, Non-small Cell Lung Cancer
A Study of CDX-1140 (CD40) as Monotherapy or in Combination in Patients With Advanced Malignancies
This is a study to determine the maximum tolerated dose (MTD) for CDX-1140 (CD40 antibody), either alone or in combination with CDX-301 (FLT3L), pembrolizumab, or chemotherapy and to further evaluate its tolerability and efficacy…
- Conditions
- Melanoma, Non-small Cell Lung Cancer, Breast Cancer, Gastric Cancer
- Trial name
- NCT03329950
- Testing
- CDX-1140, CDX-301, pembrolizumab
- Where
- 11 sites in United States
- Volunteers
- 132 people
- Sponsor
- Celldex Therapeutics
Microcystic Lymphatic Malformation
SELVA: A Phase 3 Study Evaluating QTORIN 3.9% Rapamycin Anhydrous Gel in the Treatment of Microcystic Lymphatic Malformations
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…
- Trial name
- SELVA (NCT06239480)
- Testing
- QTORIN 3.9% Rapamycin Anhydrous Gel
- Where
- 15 sites in United States
- Volunteers
- 51 people
- Sponsor
- Palvella Therapeutics, Inc.
Neurofibromatosis Type 2
Efficacy and Safety of REC-2282 in Patients With Progressive Neurofibromatosis Type 2 (NF2) Mutated Meningiomas
This is a two-staged, Phase 2/3, randomized, multi-center study to investigate the efficacy and safety of REC-2282 in participants with progressive NF2 mutated meningiomas.
- Trial name
- POPLAR-NF2 (NCT05130866)
- Testing
- REC-2282
- Where
- 15 sites in United States, United Kingdom
- Volunteers
- 25 people
- Sponsor
- Recursion Pharmaceuticals Inc.
Acute Myeloid Leukemia Refractory, Acute Myeloid Leukemia, in Relapse
AB8939 in Patients With Relapsed/Refractory Acute Myeloid Leukemia
The primary objective is to define the safety and tolerability of AB8939 in patients with AML by determining the dose-limiting toxicities, the maximum tolerated dose, and the recommended dose for dose expansion study.
- Conditions
- Acute Myeloid Leukemia Refractory, Acute Myeloid Leukemia, in Relapse, Myelodysplastic Syndrome Acute Myeloid Leukemia
- Trial name
- NCT05211570
- Testing
- AB8939, Venetoclax
- Where
- 10 sites in United States, France and 2 more
- Volunteers
- 78 people
- Sponsor
- AB Science
Age-Related Macular Degeneration, Allergies
Tissue Repository Providing Annotated Biospecimens for Approved Investigator-directed Biomedical Research Initiatives
To collect, preserve, and/or distribute annotated biospecimens and associated medical data to institutionally approved, investigator-directed biomedical research to discover and develop new treatments, diagnostics, and…
- Conditions
- Age-Related Macular Degeneration, Allergies, Alpha-Gal Syndrome, Alzheimer Disease
- Trial name
- NCT05635266
- Testing
- Specimen sample
- Where
- United States
- Volunteers
- 20,000 people, healthy volunteers welcome
- Sponsor
- Sanguine Biosciences
Leukemia
Cytarabine With or Without VNP40101M in Treating Patients With Relapsed Acute Myeloid Leukemia
RATIONALE: Drugs used in chemotherapy, such as cytarabine and VNP40101M, work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing.
- Trial name
- NCT00112554
- Testing
- cytarabine, laromustine
- Where
- 62 sites in United States, Belgium and 8 more
- Volunteers
- 420 people
- Sponsor
- Vion Pharmaceuticals
Ichthyosis, Pruritus
Study Determining Safety and Efficacy of Avena Sativa (Oat) Skincare Products for Treating Skin Dryness and Itching in Cancer Patients
This study will test the safety and efficacy of three topical agents containing oat kernel flour to determine how well they relieve skin dryness and itching related to cancer therapies.
- Trial name
- NCT03796052
- Testing
- Avena Sativa Skincare Regimen
- Where
- United States
- Volunteers
- 53 people
- Sponsor
- Johnson & Johnson Consumer Inc. (J&JCI)