Clinical trials: Genetic Cancer Syndromes
Neurofibromatosis Type 1, Reading Disabilities
Neurobiology and Treatment of Reading Disability in NF-1
The goal of this trial is to determine if children with neurofibromatosis type 1 who have reading disabilities respond the same way-both behaviorally and neurobiologically-to specialized treatment programs as children with…
- Trial name
- NCT00624234
- Testing
- Tutoring Program I, Tutoring Program II
- Where
- United States
- Volunteers
- 184 people, healthy volunteers welcome
- Sponsor
- Vanderbilt University
Neurofibromatosis 2, Vestibular Schwannoma
Trial of Selumetinib in Patients With Neurofibromatosis Type II Related Tumors
In this research study the researchers want to learn more about the effects (both good and bad) the study drug selumetinib has on participants with neurofibromatosis type II (NF2) related tumor.
- Conditions
- Neurofibromatosis 2, Vestibular Schwannoma, Meningioma, Ependymoma
- Trial name
- SEL-TH-1601 (NCT03095248)
- Testing
- Selumetinib
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Advanced Malignant Solid Neoplasm, Ann Arbor Stage III Childhood Non-Hodgkin Lymphoma
Vemurafenib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With BRAF V600 Mutations (A Pediatric MATCH Treatment Trial)
This phase II Pediatric MATCH trial studies how well vemurafenib works in treating patients with solid tumors, non-Hodgkin lymphoma, or histiocytic disorders with BRAF V600 mutations that have spread to other places in the body…
- Conditions
- Advanced Malignant Solid Neoplasm, Ann Arbor Stage III Childhood Non-Hodgkin Lymphoma, Ann Arbor Stage IV Childhood Non-Hodgkin Lymphoma, Ependymoma
- Trial name
- NCT03220035
- Testing
- Laboratory Biomarker Analysis, Vemurafenib
- Where
- 116 sites in United States, Puerto Rico
- Volunteers
- 4 people
- Sponsor
- National Cancer Institute (NCI)
Tuberous Sclerosis
Adjunctive Ganaxolone Treatment (Part A) in TSC Followed by Long-term Treatment (Part B)
To assess preliminary safety and efficacy of ganaxolone as adjunctive therapy for the treatment of primary seizure types in patients with genetically- or clinically-confirmed TSC-related epilepsy through the end of the 12 week…
- Trial name
- TSC (NCT04285346)
- Testing
- Ganaxolone
- Where
- 7 sites in United States
- Volunteers
- 23 people
- Sponsor
- Marinus Pharmaceuticals
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
Adenomatous Polyposis Coli
Sirolimus and Familial Adenomatous Polyposis (FAP)
The aim of the study is to investigate the effect of sirolimus on the progression of intestinal adenomas in patients with FAP and to assess the safety of this treatment.
- Trial name
- NCT03095703
- Testing
- Sirolimus
- Where
- Netherlands
- Volunteers
- 4 people
- Sponsor
- Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
Childhood Acute Lymphoblastic Leukemia in Remission, Childhood Acute Myeloid Leukemia in Remission
Supersaturated Calcium Phosphate Rinse in Preventing Oral Mucositis in Young Patients Undergoing Autologous or Donor Stem Cell Transplant
This randomized phase III trial is studying how well Caphosol rinse works in preventing mucositis in young patients undergoing autologous or donor stem cell transplant.
- Conditions
- Childhood Acute Lymphoblastic Leukemia in Remission, Childhood Acute Myeloid Leukemia in Remission, Childhood Chronic Myelogenous Leukemia, Childhood Myelodysplastic Syndromes
- Trial name
- NCT01305200
- Testing
- supersaturated calcium phosphate rinse, questionnaire administration, quality-of-life assessment
- Where
- 35 sites in United States, Australia and 1 more
- Volunteers
- 226 people
- Sponsor
- Children's Oncology Group
Angiofibromas, Tuberous Sclerosis
Topical Rapamycin to Erase Angiofibromas in TSC
The study is a multi-center prospective, randomized, double-blind, placebo-controlled evaluation of the safety and efficacy of a topically applied formulation of rapamycin to cutaneous angiofibromas in subjects with Tuberous…
- Trial name
- Treatment (NCT01526356)
- Testing
- Rapamycin
- Where
- 10 sites in United States, Australia
- Volunteers
- 179 people
- Sponsor
- The University of Texas Health Science Center, Houston
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
Skin Carcinoma
Topical Tazarotene in Treating Patients With Basal Cell Skin Cancer and Basal Cell Nevus Syndrome on the Chest and Back
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
- NCT00783965
- Testing
- tazarotene
- Where
- 3 sites in United States
- Volunteers
- 34 people
- Sponsor
- UCSF Benioff Children's Hospital Oakland
Prostate Cancer
Use of β-hydroxy-β-methylbutyrate to Counteract Muscle Loss in Men With Prostate Cancer on Androgen Ablation
This is a study of HMB plus amino acids in older men with prostate cancer starting androgen deprivation therapy (ADT). The investigators hypothesize that the use of this nutritional supplementation will decrease the loss of…
- Trial name
- NCT01607879
- Testing
- Standard of care ADT + (HMB + arginine + glutamine), Standard of care ADT
- Where
- United States
- Volunteers
- 48 people
- Sponsor
- Medical College of Wisconsin
Zollinger-Ellison Syndrome, Multiple Endocrine Neoplasia
Treatment of Zollinger-Ellison Syndrome With Prevacid
The purpose of this study is to study the safety and efficacy of high dose Prevacid in the long-term treatment of patients who secrete abnormally large amounts of gastric acid.
- Trial name
- NCT00204373
- Testing
- Lansoprazole (Prevacid)
- Volunteers
- 72 people
- Sponsor
- Charles Mel Wilcox, MD
Advanced Malignant Solid Neoplasm, Recurrent Childhood Ependymoma
Palbociclib in Treating Patients With Relapsed or Refractory Rb Positive Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With Activating Alterations in Cell Cycle Genes (A Pediatric MATCH Treatment Trial)
This phase II Pediatric MATCH trial studies how well palbociclib works in treating patients with Rb positive solid tumors, non-Hodgkin lymphoma, or histiocytic disorders with activating alterations (mutations) in cell cycle genes…
- Conditions
- Advanced Malignant Solid Neoplasm, Recurrent Childhood Ependymoma, Recurrent Ewing Sarcoma, Recurrent Glioma
- Trial name
- NCT03526250
- Testing
- Laboratory Biomarker Analysis, Palbociclib, Pharmacological Study
- Where
- 117 sites in United States, Puerto Rico
- Volunteers
- 23 people
- Sponsor
- National Cancer Institute (NCI)
Healthy Subject
HPV Vaccine Intervention for Young Sexual Minority Men
This phase III trial studies how well the Outsmart HPV intervention works in increasing HPV vaccine initiation and completion among young individuals who report having a history of same-sex partners, being sexually attracted to…
- Trial name
- NCT04032106
- Testing
- Best Practice, Informational Intervention, Questionnaire Administration
- Where
- 2 sites in United States
- Volunteers
- 1,227 people, healthy volunteers welcome
- Sponsor
- Ohio State University Comprehensive Cancer Center
Multiple Osteochondroma, Ollier Disease
Ready to Sail: Evaluating Sailing's Feasibility as Ergotherapy
Background Individuals with rare skeletal disorders frequently experience psychological distress, social isolation, unmet needs, and significant challenges due to limited treatment options.
- Conditions
- Multiple Osteochondroma, Ollier Disease, Osteogenesis Imperfecta
- Trial name
- PaS (NCT06397443)
- Testing
- 5-day sailing activity
- Where
- Italy
- Volunteers
- 8 people
- Sponsor
- Istituto Ortopedico Rizzoli
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.
Neurofibromatosis Type 1, Sarcoma
Combination Chemotherapy in Treating Patients With Stage III or Stage IV Malignant Peripheral Nerve Sheath Tumors
RATIONALE: Drugs used in chemotherapy, such as doxorubicin, ifosfamide, and etoposide, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing.
- Trial name
- NCT00304083
- Testing
- filgrastim, doxorubicin hydrochloride, etoposide
- Where
- 17 sites in United States
- Volunteers
- 48 people
- Sponsor
- Sarcoma Alliance for Research through Collaboration
Hyperparathyroidism, Hypercalcemia
Studies of Elevated Parathyroid Activity
Observational Phase: Patients whose parathyroid activity is elevated above normal are referred to as having hyperparathyroidism.
- Conditions
- Hyperparathyroidism, Hypercalcemia, Parathyroid Neoplasm, Multiple Endocrine Neoplasia
- Trial name
- NCT00001277
- Testing
- 68Ga-Dotatate, 18F-DOPA
- Where
- United States
- Volunteers
- 1,553 people
- Sponsor
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Epilepsy, Dravet Syndrome
A Phase 2, Multicenter, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy, Safety, and Tolerability of TAK-935 (OV935) as an Adjunctive Therapy in Pediatric Participants With Developmental and/or Epileptic Encephalopathies
The purpose of this study is to investigate the effect on the frequency of all seizures (convulsive and drop) in participants treated with TAK-935 compared to placebo.
- Conditions
- Epilepsy, Dravet Syndrome, Lennox-Gastaut Syndrome
- Trial name
- ELEKTRA (NCT03650452)
- Testing
- TAK-935
- Where
- 44 sites in United States, Australia and 6 more
- Volunteers
- 141 people
- Sponsor
- Takeda
Fanconi Anemia, Squamous Cell Carcinoma
Quercetin Chemoprevention for Squamous Cell Carcinoma in Patients With Fanconi Anemia
Fanconi anemia (FA) is an autosomal recessive disease characterized by progressive bone marrow failure, variable congenital abnormalities and a predisposition to malignancy, particularly acute myeloid leukemia (AML) and squamous…
- Trial name
- NCT03476330
- Testing
- Quercetin (dietary supplement)
- Where
- United States
- Volunteers
- 48 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati