Clinical trials: Hereditary Amyloidosis
Cardiac Amyloidosis, Transthyretin Amyloidosis
Retrospective Identification of Scintigraphic Cardiac Amyloidosis (RISCA)
This study aims to determine the positive predictive value of incidental cardiac uptake on bone scintigraphy for diagnosing transthyretin cardiac amyloidosis, in patients who underwent scintigraphy for reasons other than…
- Trial name
- RISCA (NCT06921408)
- Volunteers
- 57 people
- Sponsor
- University Hospital, Lille
Breast Cancer, Leukemia
High-Dose Chemotherapy, Total-Body Irradiation, and Autologous Stem Cell Transplantation or Bone Marrow Transplantation in Treating Patients With Hematologic Cancer or Solid Tumors
RATIONALE: Radiation therapy uses high-energy x-rays to damage cancer cells. Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die.
- Conditions
- Breast Cancer, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm
- Trial name
- NCT00060255
- Testing
- busulfan, carboplatin, carmustine
- Where
- United States
- Volunteers
- 451 people
- Sponsor
- Roswell Park Cancer Institute
Cardiac Amyloidosis
Cardiac Amyloidosis : Diagnostic Using Red Flag Signals
Cardiac amyloidosis is an increasingly contributor of degenerative cardiac diseases. However, its frequency remains underestimated, and diagnosis is often realized at late stages of the disease.
- Trial name
- TEAM Red Flags (NCT04459169)
- Testing
- diagnostic algorithm
- Where
- 3 sites in Guadeloupe, Martinique
- Volunteers
- 424 people
- Sponsor
- University Hospital Center of Martinique
Cardiovascular Diseases, Hypertrophic Cardiomyopathy (HCM)
AI-enabled Screening and Diagnosis of Cardiomyopathies Using Coronary CTA
The goal of this observational and diagnostic study is to develop and validate an artificial intelligence assisted approach for coronary computer tomography angiography-(CCTA)-based screening and diagnosis of cardiomyopathies in…
- Conditions
- Cardiovascular Diseases, Hypertrophic Cardiomyopathy (HCM), Dilated Cardiomyopathy (DCM), Restrictive Cardiomyopathy
- Trial name
- Atlantis (NCT06748261)
- Testing
- CCTAI model
- Volunteers
- 5,000 people, healthy volunteers welcome
- Sponsor
- Shanghai Zhongshan Hospital
Amyloidosis Cardiac, Amyloid Polyneuropathy
Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM) in Patients With Idiopathic Peripheral Neuropathy
The goal of the present study is to determine the occurrence of wild-type and hereditary transthyretin amyloidosis cardiomyopathy among patients with the diagnosis of idiopathic peripheral neuropathy in the setting of a…
- Conditions
- Amyloidosis Cardiac, Amyloid Polyneuropathy, Amyloidosis, Heart Failure
- Trial name
- NCT04985734
- Testing
- Specific diagnostic work-up for ATTR-CM patients
- Where
- Belgium
- Volunteers
- 200 people
- Sponsor
- University Hospital, Antwerp
Amyloidosis, Hereditary
ATTR Expanded Access Program (EAP) by Ionis
The purpose of this program is to provide expanded access to Inotersen for up to 100 Patients with Hereditary Transthyretin Amyloidosis (hTTR).
- Trial name
- NCT03400098
- Testing
- Inotersen
- Sponsor
- Ionis Pharmaceuticals, Inc.
Leukemia, Lymphoma
Umbilical Cord Blood Stem Cell Transplant in Treating Patients With Hematologic Cancer or Other Disease
RATIONALE: Giving low doses of chemotherapy and total-body irradiation before a donor umbilical cord blood stem cell transplant helps stop the growth of cancer or abnormal cells.
- Conditions
- Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes
- Trial name
- NCT00423826
- Testing
- Busulfan, Cytarabine, Fludarabine phosphate
- Where
- United States
- Sponsor
- Barbara Ann Karmanos Cancer Institute
Cardiac Amyloidosis
Screening Algorithm for Early Detection of Occult Cardiac Amyloidosis
Early diagnosis of cardiac amyloidosis (CA) is crucial because of the poor overall survival, high mortality and the need for early therapy including new treatment possibilities for transthyretin amyloidosis.
- Trial name
- EARLY-CA (NCT05693376)
- Where
- Germany
- Volunteers
- 85 people, healthy volunteers welcome
- Sponsor
- University Hospital, Essen
Chronic Myeloproliferative Disorders, Leukemia
Tissue, Blood, and Body Fluid Sample Collection From Patients With Hematologic Cancer
RATIONALE: Collecting and storing samples of tissue, blood, and body fluid from patients with cancer to study in the laboratory may help the study of cancer in the future.
- Conditions
- Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Lymphoproliferative Disorder
- Trial name
- NCT01137643
- Testing
- biologic sample preservation procedure, cytology specimen collection procedure
- Where
- United States
- Volunteers
- 15,000 people
- Sponsor
- UNC Lineberger Comprehensive Cancer Center
Cardiac Amyloidosis
Prevalence and Prognosis of Cardiac Amiloidosis in Turkey
Patients with left ventricular hypertrophy are further examined according to an algorithm to check if they have a cardiac amyloidosis
- Trial name
- PAPCAT (NCT04915235)
- Testing
- Algorithm
- Where
- Turkey (Türkiye)
- Volunteers
- 2,087 people
- Sponsor
- Turkish Society of Cardiology
Transthyretin Amyloidosis, Carpal Tunnel Syndrome
Transthyretin Cardiac Amyloidosis in Patients With Idiopathic Carpal Tunnel Syndrome Referred for Release Surgery
This study will determine the prevalence of transthyretin amyloidosis in pathology of the transverse carpal ligament and cardiac involvement using natriuretic peptides, electrocardiography and echocardiography in patients…
- Trial name
- PRATHYC (NCT04611204)
- Testing
- Descriptive cohort
- Where
- France
- Volunteers
- 6 people
- Sponsor
- University Hospital, Toulouse
Secondary (AA) Amyloidosis, Rheumatoid Arthritis
Study of the Safety and Efficacy of NC-503 in Secondary (AA) Amyloidosis
The main objective of this study is to evaluate the safety and efficacy of NC-503 compared to placebo in patients with secondary (AA) amyloidosis using a composite assessment of clinical improvement/worsening of both renal and…
- Conditions
- Secondary (AA) Amyloidosis, Rheumatoid Arthritis, Nephrotic Syndrome, Familial Mediterranean Syndrome
- Trial name
- NCT00035334
- Testing
- NC-503 (Anti-amyloidotic (AA) Agent)
- Where
- 26 sites in United States, Finland and 10 more
- Volunteers
- 150 people
- Sponsor
- Bellus Health Inc. - a GSK company
Multiple Myeloma, Amyloidosis
Registry for Adults With Plasma Cell Disorders (PCD's)
The primary purpose of this protocol is to create a registry of patients with plasma cell disorders (PCDs), including for example the cancer multiple myeloma (MM), who complete the assessment, previously known as a "geriatric…
- Conditions
- Multiple Myeloma, Amyloidosis, Cryoglobulinemia, Castleman's Disease
- Trial name
- NCT03717844
- Where
- United States
- Volunteers
- 2,000 people
- Sponsor
- UNC Lineberger Comprehensive Cancer Center
Amyloidosis Transthyretin, Athlete Heart
Exercise Effect on Transthyretin Stability
The goal of this study to investigate if exercise predisposes to transthyretin instability. The investigators will evaluate the effect of exercise on transthyretin biochemistry.
- Trial name
- NCT04986826
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- Oregon Health and Science University
Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy
Efficacy and Safety of AG10 in Subjects with Transthyretin Amyloid Polyneurophathy
See updated study design under NCT04882735. Phase 3 efficacy and safety of AG10 compared with placebo in subjects with symptomatic Transthyretin Amyloid Polyneuropathy (ATTR-PN)
- Trial name
- ATTRibute-PN (NCT04418024)
- Testing
- AG10
- Where
- United States
- Sponsor
- Eidos Therapeutics, a BridgeBio company
Cardiac Amyloidosis, Heart Diseases
N-of-1 for Beta-Blockers in Cardiac Amyloidosis
The study team will generate preliminary data on whether patients with cardiac amyloidosis feel better when their beta-blocker is stopped. To achieve this objective, 20 N-of-1 trials (on vs.
- Conditions
- Cardiac Amyloidosis, Heart Diseases, TTR Cardiac Amyloidosis
- Trial name
- NCT05019027
- Testing
- Beta blocker
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- Weill Medical College of Cornell University
Transthyretin Cardiac Amyloidosis
Safety, Efficacy, and Pharmacokinetics of Tafamidis in Patients With Transthyretin-mediated Amyloidosis Post Orthotopic Heart Transplantation
Transthyretin cardiac amyloidosis (ATTR-CA) is a relentlessly progressive disease that can progress to end stage heart failure, at which point recently approved transthyretin production silencing or structure stabilizing…
- Trial name
- NCT05489523
- Testing
- Tafamidis 61 MG
- Where
- 4 sites in United States
- Volunteers
- 25 people
- Sponsor
- University of Texas Southwestern Medical Center
Heart Failure, Amyloid Cardiomyopathy
EOSS-ATTR Study (eHealth Based Operative Support System in ATTR-CM)
Unicenter, proof-of-concept, prospective, randomised, controlled, open-label and blinded end-point adjudication trial to assess the effect on patient-reported outcomes measures (PROMs), patient-reported experience measures…
- Conditions
- Heart Failure, Amyloid Cardiomyopathy, ATTR Amyloidosis Wild Type, ATTR Gene Mutation
- Trial name
- eOSS-ATTR (NCT06499064)
- Testing
- mHealth
- Volunteers
- 50 people
- Sponsor
- Hospital Universitari de Bellvitge
Transthyretin (TTR) Amyloid Cardiomyopathy
Vyndaqel Capsules Special Investigation (ATTR-CM)
Secondary Data Collection : To confirm the safety and effectiveness profiles under the actual medical practice of Vyndaqel in Japan.
- Trial name
- NCT04108091
- Testing
- Treatment for TTR amyloidosis
- Where
- Japan
- Volunteers
- 1,214 people
- Sponsor
- Pfizer
Tuberous Sclerosis, Angiofibroma
Long-term Trial of Topical Sirolimus to Angiofibroma in Patient With Tuberous Sclerosis Complex
The purpose of this trial is to evaluate the safety and efficacy of long-term treatment with NPC-12G gel (0.2% sirolimus gel) to angiofibroma and other skin lesions in patients with tuberous sclerosis complex in the open-label…
- Conditions
- Tuberous Sclerosis, Angiofibroma, Hypomelanotic Macule, Plaque
- Trial name
- NCT02634931
- Testing
- NPC-12G gel
- Where
- Japan
- Volunteers
- 94 people
- Sponsor
- Nobelpharma