Clinical trials: Amyloidosis, Hereditary
Tuberous Sclerosis, Angiofibroma
Phase III Trial of Topical Formulation of Sirolimus to Skin Lesions in Patients With Tuberous Sclerosis Complex (TSC)
The purpose of this trial is to evaluate the efficacy and safety of NPC-12G gel (topical formulation of sirolimus) versus placebo gel to facial angiofibroma and other skin lesions in patients with tuberous sclerosis complex (TSC)
- Conditions
- Tuberous Sclerosis, Angiofibroma, Hypomelanotic Macule, Plaque
- Trial name
- NCT02635789
- Testing
- NPC-12G gel
- Where
- Japan
- Volunteers
- 62 people
- Sponsor
- Nobelpharma
Transthyretin (TTR) Amyloid Cardiomyopathy, Cardiac Amyloidosis
Liver and Coagulation Disorders in Cardiac Transthyretin Amyloidosis
Transthyretin cardiac amyloidosis (ATTR-CA) is a progressive infiltrative cardiomyopathy caused by the deposition of misfolded transthyretin protein within the myocardium.
- Conditions
- Transthyretin (TTR) Amyloid Cardiomyopathy, Cardiac Amyloidosis, Cardiomyopathies, Wild-Type Transthyretin Cardiac Amyloidosis
- Trial name
- LICA2025 (NCT07766135)
- Where
- Italy
- Volunteers
- 70 people
- Sponsor
- University of Messina
Aortic Stenosis, Transthyretin Amyloidosis
Positron Emission Tomography / Magnetic Resonance Imaging in Aortic Stenosis
Aortic stenosis is the most common valve disease requiring surgery in the Western world. It is defined by progressive calcification and fibrosis of the valve leaflets and restricted valve opening.
- Trial name
- PASS (NCT03352089)
- Where
- United Kingdom
- Volunteers
- 22 people, healthy volunteers welcome
- Sponsor
- University of Edinburgh
Amyloidosis Cardiac, Healthy Adults
Intracardiac Flow Assessment in Cardiac Amyloidosis
The primary objective of this study is to define the intracardiac flow imaging biomarkers in cardiac amyloidosis.
- Trial name
- NCT05379101
- Testing
- Cardiac magnetic resonance image (CMR), Transthoracic Echocardiogram (TTE), Six-minute Walk Test
- Where
- United States
- Volunteers
- 81 people, healthy volunteers welcome
- Sponsor
- Mayo Clinic
Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission
High-Dose Chemotherapy With or Without Total-Body Irradiation Followed by Autologous Stem Cell Transplant in Treating Patients With Hematologic Cancer or Solid Tumors
This pilot trial studies different high-dose chemotherapy regimens with or without total-body irradiation (TBI) to compare how well they work when given before autologous stem cell transplant (ASCT) in treating patients with…
- Conditions
- Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q)
- Trial name
- NCT00536601
- Testing
- etoposide, cyclophosphamide, carmustine
- Where
- United States
- Volunteers
- 174 people
- Sponsor
- Roswell Park Cancer Institute
Brain and Central Nervous System Tumors, Chronic Myeloproliferative Disorders
Perceptions of Burden in Patients With Late-Stage Cancer and Their Caregivers
RATIONALE: Gathering information over time about patients' sense of being a burden on their caregiver, and caregivers' sense of burden on themselves, may help doctors learn more about the desire to die in patients with late-stage…
- Conditions
- Brain and Central Nervous System Tumors, Chronic Myeloproliferative Disorders, Depression, Leukemia
- Trial name
- NCT00770419
- Testing
- medical chart review, questionnaire administration, psychosocial assessment and care
- Sponsor
- Rutgers, The State University of New Jersey
Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
A Study to Assess the Long-Term Safety and Efficacy of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Patients With Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
The purpose of this study is to evaluate the safety and tolerability of extended dosing with eplontersen in participants with hereditary transthyretin-mediated amyloid polyneuropathy (hATTR-PN).
- Trial name
- NCT05071300
- Testing
- Eplontersen
- Where
- 33 sites in United States, Argentina and 11 more
- Volunteers
- 151 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Lumbar Spinal Stenosis, Cardiac Amyloidosis
Analysis of Lumbar Spine Stenosis Specimens for Identification of Transthyretin Cardiac Amyloidosis
Primary objective: To identify older adults with transthyretin cardiac amyloidosis (ATTR-CA) early in the course of the illness, at a time when disease modifying therapies are most effective.
- Conditions
- Lumbar Spinal Stenosis, Cardiac Amyloidosis, ATTR Amyloidosis Wild Type, ATTR Gene Mutation
- Trial name
- NCT06034405
- Testing
- Tc99-PYP or Tc99-HDMP Scan
- Where
- 6 sites in United States
- Volunteers
- 1,663 people, healthy volunteers welcome
- Sponsor
- Columbia University
Familial Mediterranean Fever
AGE and CALLY Index in Familial Mediterranean Fever
Familial Mediterranean Fever (FMF) is an autoinflammatory disease characterized by recurrent inflammatory attacks and persistent low-grade inflammation.
- Trial name
- NCT07439341
- Testing
- Skin Autofluorescence Measurement
- Volunteers
- 150 people, healthy volunteers welcome
- Sponsor
- Bursa City Hospital
ATTR Amyloidosis, Cardiopathy
Orthopaedic Specimen Pathology and Early Diagnosis of ATTR Cardiopathy (ATTR-ORTHO)
The goal of this observational study is to learn about the frequency of ATTR amyloid, cardiac involvement and associated features, in 150 patients aged 50 or more years, and operated for an idiopathic carpal tunnel syndrome…
- Conditions
- ATTR Amyloidosis, Cardiopathy, Carpal Tunnel Syndrome, Lumbar Spine Stenosis
- Trial name
- ATTR-ORTHO (NCT06367842)
- Where
- France
- Volunteers
- 150 people
- Sponsor
- Institut Mutualiste Montsouris
Cancer
Natural History Study of Fungal Infections of the Blood in Patients With Cancer or in Patients Who Have Undergone a Stem Cell Transplant
RATIONALE: Gathering information about how often fungal infections of the blood occur in patients with cancer or in patients who have undergone stem cell transplant may help doctors learn more about the disease.
- Trial name
- NCT00445952
- Testing
- clinical observation, management of therapy complications
- Where
- 5 sites in Belgium, France and 3 more
- Volunteers
- 304 people
- Sponsor
- European Organisation for Research and Treatment of Cancer - EORTC
Cancer
Caspofungin Acetate Compared With Amphotericin B Liposomal in Treating Patients With Persistent Fever and Neutropenia Following Cancer Treatment
RATIONALE: Caspofungin acetate or amphotericin B liposomal may be effective in preventing or controlling fever and neutropenia caused by chemotherapy, bone marrow transplantation, or peripheral stem cell transplantation.
- Trial name
- NCT00008359
- Testing
- caspofungin acetate, liposomal amphotericin B
- Where
- United States
- Sponsor
- Memorial Sloan Kettering Cancer Center
Brain and Central Nervous System Tumors, Chronic Myeloproliferative Disorders
Collecting and Storing Blood Samples From Patients With Cancer
RATIONALE: Collecting and storing samples of blood from patients with cancer to test in the laboratory may help the study of cancer in the future.
- Conditions
- Brain and Central Nervous System Tumors, Chronic Myeloproliferative Disorders, Leukemia, Lymphoma
- Trial name
- NCT00900120
- Sponsor
- University of Medicine and Dentistry of New Jersey