Clinical trials: Multiple Primary Neoplasm
Bloom Syndrome
Biological Significance of the Bloom's Syndrome Protein
Since 1960, persons with the very rare disorder Bloom's syndrome (BS) have been followed clinically, documenting clinical matters as obtained from their doctors.
- Trial name
- NCT00021437
- Where
- United States
- Sponsor
- National Center for Research Resources (NCRR)
Tuberous Sclerosis, STXBP1 Encephalopathy With Epilepsy
Gait in Rare Diseases
The aim of this pilot study is to explore whether the knowledge and experience gained during the T-GaiD project (Treatment of Gait Disorders in Dravet Syndrome - NCT03857451) can be transferred to other populations with similar…
- Trial name
- GAGA (NCT05161494)
- Where
- Belgium
- Volunteers
- 41 people
- Sponsor
- Universiteit Antwerpen
Parkinson Disease, Progressive Supranuclear Palsy
Biomarkers in Parkinsonian Syndromes
This is a prospective observational study to identify biomarkers in parkinson syndromes. Patients with parkinsonian syndromes at the early stages of disease will be recruited and will be followed up until their established…
- Conditions
- Parkinson Disease, Progressive Supranuclear Palsy, Multiple System Atrophy, Atypical Parkinsonism
- Trial name
- PROATYP (NCT06501469)
- Where
- Greece
- Volunteers
- 200 people
- Sponsor
- Non-profit organization for scientific research in Parkinson's disease and related disorders
Life Threatening Diseases
Phase II Study of Imatinib Mesylate in Patients With Life Threatening Malignant Rare Diseases
Exploratory study to examine the effect(s) of Imatinib mesylate treatment on life threatening rare diseases with known associations to one or more Imatinib mesylate -sensitive tyrosine kinases, and to identify the contribution of…
- Trial name
- NCT00154388
- Testing
- Imatinib mesylate
- Volunteers
- 185 people
- Sponsor
- Novartis Pharmaceuticals
Refractory Cancer, CNS Tumors
A Study of Avutometinib for People With Solid Tumor Cancers
The purpose of this study is to find out whether avutometinib is a safe treatment for advanced or recurrent solid tumor cancers in children and young adults.
- Conditions
- Refractory Cancer, CNS Tumors, CNS Tumor, Adult, CNS Tumor, Childhood
- Trial name
- NCT06104488
- Testing
- Avutometinib
- Where
- 2 sites in United States
- Volunteers
- 3 people
- Sponsor
- Memorial Sloan Kettering Cancer Center
Facial Angiofibroma
Tuberous Sclerosis Complex: Facial Angiofibroma Skin Cream
The objective of this study is to evaluate the safety and efficacy of a 0.1% formulation of rapamune cream in children with Tuberous Sclerosis Complex (TSC), ages 3 years and older, who have facial angiofibromas that would…
- Trial name
- NCT01853423
- Testing
- Rapamune
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- Le Bonheur Children's Hospital
Childhood Obesity, Pediatric Obesity
Management of Childhood Obesity
The significant increase in the prevalence of obesity can also be attributed to various social changes, in which the environment (political, economic, social, cultural), and not only the individual and his choices, takes a…
- Trial name
- NCT05966259
- Testing
- Intervention Group- Intensive Multilevel Intervention, Control Group
- Where
- Brazil
- Volunteers
- 78 people, healthy volunteers welcome
- Sponsor
- Adaliene Versiani M. Ferreira
Growth Disorders
Treatment of Children and Adolescents With Growth Failure Associated With Primary IGF-1 Deficiency
This is an extension study to Tercica study MS301 (NCT00125164) and is intended to collect long term safety and efficacy data on the continued use of recombinant human insulin-like growth factor-1 (rh IGF-1) in children and…
- Trial name
- NCT00330668
- Testing
- rh IGF-1 (mecasermin)
- Where
- France
- Volunteers
- 114 people
- Sponsor
- Ipsen
Antifungal Prophylaxis of Invasive Fungal Infections
Voriconazole Versus Itraconazole In Primary Prophylaxis Of Invasive Fungal Infection (IFI) In Subjects With Allogeneic Hematopoietic Stem Cell Transplants (HSCT)
Study is to compare antifungal prophylaxis of Voriconazole and Itraconazole in subjects who have had a Stem Cell Transplant. The success of the end point will be measured using evidence of Infection, drug compliance and survival.
- Trial name
- IMPROVIT (NCT00289991)
- Testing
- Itraconazole, Vfend - voriconazole
- Where
- 49 sites in Canada, Czechia and 10 more
- Volunteers
- 489 people
- Sponsor
- Pfizer
Developmental and Epileptic Encephalopathy, Dravet Syndrome
Study to Investigate LP352 in Subjects With Developmental and Epileptic Encephalopathies
The objective of this study is to assess the safety, tolerability, efficacy, and pharmacokinetics of adjunctive therapy of LP352 in adults and adolescents with developmental and epileptic encephalopathies.
- Conditions
- Developmental and Epileptic Encephalopathy, Dravet Syndrome, Lennox Gastaut Syndrome
- Trial name
- PACIFIC (NCT05364021)
- Testing
- LP352
- Where
- 34 sites in United States, Australia
- Volunteers
- 52 people
- Sponsor
- Longboard Pharmaceuticals
CD55 - Cluster of Differentiation Antigen 55 Deficiency, Primary Intestinal Lymphangiectasis
Transcriptome and Metabolic Analyses of CHAPLE Disease
CHAPLE syndrome (complement hyperactivation, angiopathic thrombosis, protein losing enteropathy) is a newly discovered genetic disorder, which is caused by deleterious mutations in the CD55 gene.
- Conditions
- CD55 - Cluster of Differentiation Antigen 55 Deficiency, Primary Intestinal Lymphangiectasis, Protein-Losing Enteropathies, Complement Regulatory Factor Defect
- Trial name
- CHAPLEOMIC (NCT03950804)
- Testing
- Eculizumab Injection
- Where
- Turkey (Türkiye)
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Marmara University
Helicobacter Pylori Infection
Esomeprazole/Vonoprazan Combined With High-dose Amoxicillin Dual Therapy for Primary Eradication of Helicobacter Pylori Infection
This study intends to collect the clinical data of patients with Helicobacter pylori infection, and to observe and count the high-dose double therapy and the traditional quadruple / triple therapy ( i.e., esomeprazole +…
- Trial name
- NCT06250634
- Testing
- esomeprazole + amoxicillin, esomeprazole + amoxicillin + clarithromycin + colloidal bismuth tartrate agent, Vonoprazan + amoxicillin
- Where
- China
- Volunteers
- 806 people
- Sponsor
- The Third Xiangya Hospital of Central South University
Chronic Myeloproliferative Disorders, Leukemia
Bone Marrow Transplantation in Treating Patients With Multiple Myeloma, Chronic Phase Chronic Myelogenous Leukemia, or Agnogenic Myeloid Metaplasia
RATIONALE: Bone marrow transplantation may be able to replace immune cells that were destroyed by the chemotherapy or radiation therapy that was used to kill cancer cells.
- Conditions
- Chronic Myeloproliferative Disorders, Leukemia, Multiple Myeloma and Plasma Cell Neoplasm
- Trial name
- NCT00004181
- Testing
- busulfan, cyclophosphamide, allogeneic bone marrow transplantation
- Where
- United States
- Sponsor
- Northwestern University
IgA Nephropathy
The Chinese Registry of Prognostic Study of IgA Nephropathy (CRPIGA)
1. Establish an IgAN cohort collaboration group and expert committee to carry out registration research. 2. Construct IgAN structured data set standards, formulate structured data collection templates of diagnosis and treatment …
- Trial name
- NCT04858724
- Where
- China
- Volunteers
- 2,000 people
- Sponsor
- Ruijin Hospital
Primary Ciliary Dyskinesia
Utility of PCD Diagnostics to Improve Clinical Care
This cross-sectional and longitudinal observational study is to gather data on the utility of tests that are used to make a diagnosis of primary ciliary dyskinesia (PCD).
- Trial name
- PCD (NCT05889013)
- Testing
- Nasal Nitric Oxide testing
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Connecticut Children's Medical Center
Coronavirus Infection (COVID-19), Pulmonary Arterial Hypertension
Pharmacokinetics, Pharmacodynamics, and Safety Profile of Understudied Drugs Administered to Children Per Standard of Care (POPS)
The study investigators are interested in learning more about how drugs, that are given to children by their health care provider, act in the bodies of children and young adults in hopes to find the most safe and effective dose…
- Conditions
- Coronavirus Infection (COVID-19), Pulmonary Arterial Hypertension, Urinary Tract Infections in Children, Hypertension
- Trial name
- POPS or POP02 (NCT04278404)
- Testing
- The POP02 study is collecting bodily fluid samples (i.e., whole blood, effluent samples) of children prescribed the following drugs of interest per standard of care:
- Where
- 52 sites in United States, Canada
- Volunteers
- 5,000 people, healthy volunteers welcome
- Sponsor
- Duke University
Dental Plaque, Dental Prophylaxis
Effectiveness of Customized Handle Toothbrush
People with special needs suffer from many difficulties, including mental, physical or motor, which increase the difficulty of obtaining good oral health.
- Conditions
- Dental Plaque, Dental Prophylaxis, Dental Care
- Trial name
- NCT04845633
- Testing
- Evaluation of the Effectiveness of Conventional Toothbrush in Dental Plaque Control in Healthy Children, Evaluation of the Effectiveness of Customized Handle Toothbrush in Dental Plaque Control in Healthy Children, Evaluation of the Effectiveness of Conventional Toothbrush in Dental Plaque Control in Children with Down Syndrome
- Where
- Syria
- Volunteers
- 48 people
- Sponsor
- Damascus University
Hypomelanotic Macules, Facial Angiofibroma
Biomarkers for Tuberous Sclerosis Complex (BioTuScCom)
International, multicenter, observational, longitudinal study to identify biomarker/s for Tuberous Sclerosis Complex and to explore the clinical robustness, specificity, and long´-term variability of these biomarker/s
- Conditions
- Hypomelanotic Macules, Facial Angiofibroma, Shagreen Patches, Ungual Fibromas
- Trial name
- TuScCom (NCT02654340)
- Where
- 8 sites in Albania, Egypt and 6 more
- Volunteers
- 20 people
- Sponsor
- CENTOGENE GmbH Rostock
Cancer
Liposomal Amphotericin B in Treating Granulocytopenia and Persistent Unexplained Fever in Cancer Patients
RATIONALE: Liposomal amphotericin B may be effective in controlling fever and granulocytopenia. It is not yet known which regimen of liposomal amphotericin B is more effective in treating cancer patients who have these…
- Trial name
- NCT00003938
- Testing
- liposomal amphotericin B
- Where
- 24 sites in Belgium, Czechia and 13 more
- Volunteers
- 115 people
- Sponsor
- European Organisation for Research and Treatment of Cancer - EORTC
Meningioma, Metastatic Well Differentiated Neuroendocrine Neoplasm
68Ga-DOTA-TOC PET/CT in Imaging Participants With Neuroendocrine Tumors
This trial studies how well gallium Ga 68-edotreotide (68Ga-DOTA-TOC) positron emission tomography (PET)/computer tomography (CT) works in imaging participants with neuroendocrine tumors.
- Conditions
- Meningioma, Metastatic Well Differentiated Neuroendocrine Neoplasm, Multiple Endocrine Neoplasia Type 1, Neuroendocrine Neoplasm
- Trial name
- NCT03001349
- Testing
- Computed Tomography, Gallium Ga 68-Edotreotide, Positron Emission Tomography
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- M.D. Anderson Cancer Center