Clinical trials: Genetic Cancer Syndromes
Tuberous Sclerosis Complex, Focal Cortical Dysplasia
Astroscape: A Study of Radiprodil on Safety, Tolerability, Pharmacokinetics, and Effect on Seizures and Behavioral Symptoms in Patients With TSC or FCD Type II
Study RAD-GRIN-201 is a phase 1B/2A trial to assess safety, tolerability, pharmacokinetics (PK), and potential efficacy of radiprodil in participants with Tuberous Sclerosis Complex (TSC) or Focal Cortical Dysplasia (FCD) type…
- Trial name
- NCT06392009
- Testing
- Radiprodil
- Where
- 18 sites in Australia, Belgium and 6 more
- Volunteers
- 30 people
- Sponsor
- GRIN Therapeutics, Inc.
Renal Angiomyolipoma
A Phase IV Study of Safety and Efficacy of Everolimus in Taiwanese Patients With Tuberous Sclerosis Complex Who Have Renal Angiomyolipoma (TSC-AML)
The purpose of this prospective study is to assess the safety and efficacy of everolimus in Taiwanese patients with renal angiomyolipoma (AML) associated with tuberous sclerosis complex (TSC) .
- Trial name
- NCT05252585
- Testing
- Everolimus
- Where
- 4 sites in Taiwan
- Volunteers
- 4 people
- Sponsor
- Novartis Pharmaceuticals
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.
Type I Neurofibromatosis
A Clinical Trial to Evaluate the Tolerability and Pharmacokinetics of TQ-B3234 in Patients With Type I Neurofibromatosis
This study is a Phase I clinical trial to evaluate the tolerability and pharmacokinetics of TQ-B3234 capsules in Chinese subjects associated with neurofibromatosis type I (neurofibroma and peripheral malignant neurilemmoma).
- Trial name
- NCT05107037
- Testing
- TQ-B3234 capsule
- Where
- China
- Volunteers
- 120 people
- Sponsor
- Chia Tai Tianqing Pharmaceutical Group Co., Ltd.
Developmental and Epileptic Encephalopathy, Dravet Syndrome
Open-label, Long-term Safety Study of LP352 in Subjects With Developmental and Epileptic Encephalopathy
The objective of this study is to assess the long-term safety, tolerability, and efficacy of adjunctive therapy of LP352 in subjects with developmental and epileptic encephalopathies who completed participation in Study LP352-201.
- Conditions
- Developmental and Epileptic Encephalopathy, Dravet Syndrome, Lennox-Gastaut Syndrome
- Trial name
- NCT05626634
- Testing
- LP352, bexicaserin
- Where
- 28 sites in United States, Australia
- Volunteers
- 41 people
- Sponsor
- Longboard Pharmaceuticals
Tuberous Sclerosis Complex, Seizures
A Randomized Controlled 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 blinded phase only will be described in this record. Participants will receive 1 of 2 doses of GWP42003-P or matching placebo.
- Trial name
- NCT02544763
- Testing
- GWP42003-P
- Where
- 44 sites in United States, Australia and 4 more
- Volunteers
- 224 people
- Sponsor
- Jazz 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
Myelodysplastic Syndromes (MDS), Beta-thalassemia
A Study to Evaluate Long-term Safety in Participants Who Have Participated in Other Luspatercept (ACE-536) Clinical Trials
A Phase 3b, open-label, single-arm, rollover study to evaluate the long-term safety of luspatercept, to the following participants: Participants receiving luspatercept on a parent protocol at the time of their transition to the…
- Conditions
- Myelodysplastic Syndromes (MDS), Beta-thalassemia, Myeloproliferative Neoplasm(MPN)-Associated Myelofibrosis
- Trial name
- NCT04064060
- Testing
- Luspatercept
- Where
- 143 sites in United States, Australia and 20 more
- Volunteers
- 665 people
- Sponsor
- Celgene
Sleep Disorders
Efficacy and Safety of Circadin® in the Treatment of Sleep Disturbances in Children With Neurodevelopment Disabilities
The purpose of this study is to establish the efficacy and safety of Circadin in children with neurodevelopmental disorders and to determine the dose, this randomized, placebo-controlled study is planned to evaluate the efficacy…
- Trial name
- NCT01906866
- Testing
- Circadin 2/5/10 mg
- Where
- 28 sites in United States, Finland and 3 more
- Volunteers
- 125 people
- Sponsor
- Neurim Pharmaceuticals Ltd.
Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child
Safety Study of Gene Modified Donor T-cells Following TCRαβ+ 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 Syndrome
- Trial name
- NCT02065869
- Testing
- BPX-501 T cells, Rimiducid
- Where
- 4 sites in Italy, United Kingdom
- Volunteers
- 187 people
- Sponsor
- Bellicum Pharmaceuticals
Irritable Bowel Syndrome
A Phase 1, Randomized, Placebo-Controlled, Ascending Cohort, Dose Escalation Study in Normal Healthy Volunteers
GSK3179106 is a potent and relatively selective inhibitor of RET kinase which has been designed to be a safe and effective therapy for irritable bowel syndrome (IBS) patients .
- Trial name
- NCT02727283
- Testing
- GSK3179106
- Where
- Australia
- Volunteers
- 16 people, healthy volunteers welcome
- Sponsor
- GlaxoSmithKline
Neurofibromatosis and Malignant Peripheral Nerve Sheath Tumors
A Clinical Trial to Evaluate the Safety and Efficacy of AL2846 Capsules in Chinese Patients With Type I Neurofibromatosis
AL2846 is a multi-target receptor tyrosine kinase inhibitor. The purpose of this study is to evaluate the safety and efficacy of AL2846 capsules in Chinese patients with type I neurofibromatosis (NF1) (neurofibromas and malignant…
- Trial name
- NCT05011019
- Testing
- AL2846 capsules
- Where
- China
- Volunteers
- 22 people
- Sponsor
- Chia Tai Tianqing Pharmaceutical Group Co., Ltd.
Male Contraception
Open Label, Dose Escalation, Repeat Dose Study Evaluating YCT-529 in Healthy Males
This is a Phase 1b/2a open-label, dose escalation 3 part-study, 28-day, 90-day or 180 day repeat dose study of YCT-529 in healthy males who have decided to have a vasectomy and are waiting for the procedure and for men who have…
- Trial name
- NCT06542237
- Testing
- YCT-529
- Where
- New Zealand
- Volunteers
- 88 people, healthy volunteers welcome
- Sponsor
- YourChoice Therapeutics, Inc.
Advanced Solid Tumors or Non-Hodgkin's Lymphoma (NHL)
A Phase 1/2 Trial of SRA737 in Subjects With Advanced Cancer
The purpose of this clinical study is to establish the safety profile, determine the maximum tolerated dose (MTD) and recommend a Phase 2 dose and schedule of SRA737; and to evaluate the efficacy of SRA737 in…
- Trial name
- NCT02797964
- Testing
- SRA737
- Where
- 15 sites in United Kingdom
- Volunteers
- 107 people
- Sponsor
- Sierra Oncology LLC - a GSK company
Cancer, Colorectal Cancer
FOG-001 in Locally Advanced or Metastatic Solid Tumors
The goal of this clinical trial is to determine if FOG-001 is safe and effective in participants with locally advanced or metastatic solid tumors or in participants with familial adenomatous polyposis (FAP).
- Conditions
- Cancer, Colorectal Cancer, Solid Tumor, Locally Advanced Solid Tumor
- Trial name
- NCT05919264
- Testing
- FOG-001, mFOLFOX-6, Nivolumab
- Where
- 33 sites in United States, Australia
- Volunteers
- 619 people
- Sponsor
- Parabilis Medicines, Inc.
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.
Neurofibromatosis Type 1
PASS of Paediatric Patients Initiating Selumetinib
Neurofibromatosis type 1 (NF1) is a rare, autosomal dominant genetic disorder that is caused by germline mutations in the NF1 tumour suppressor gene, which encodes the tumour suppressor protein neurofibromin 1.
- Trial name
- NCT05388370
- Where
- 46 sites in Austria, France and 8 more
- Volunteers
- 124 people
- Sponsor
- AstraZeneca
Neurofibromatosis 1, Neurofibroma Plexiform
A Study of Selumetinib in Chinese Paediatric and Adult Subjects With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas (PN)
A Phase 1 Open Label Study to Assess the Safety, Tolerability, Pharmacokinetics and Clinical Efficacy of Selumetinib, a Selective Mitogen Activated Protein Kinase Kinase (MEK) 1 Inhibitor, in Chinese Paediatric and Adult Subjects…
- Trial name
- NCT04590235
- Testing
- Selumetinib
- Where
- 2 sites in China
- Volunteers
- 32 people
- Sponsor
- AstraZeneca
Breast Cancer, Hereditary Breast/Ovarian Cancer (brca1, brca2)
Outcomes After Perforator Flap Reconstruction for Breast Reconstruction and/or Lymphedema Treatment
The goal of this prospective, observational study is to investigate the clinical, psychosocial, and patient satisfaction outcomes of patients who undergo perforator flap reconstruction for breast reconstruction and/or…
- Conditions
- Breast Cancer, Hereditary Breast/Ovarian Cancer (brca1, brca2), Acquired Lymphedema, Congenital Lymphedema
- Trial name
- NCT01273909
- Testing
- Perforator Flap Breast Reconstruction, Vascularized Lymph Node Transfer
- Where
- 3 sites in United States
- Volunteers
- 1,000 people
- Sponsor
- The National Institute of Lymphology
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