Clinical trials: Hereditary Amyloidosis
Cancer
Fentanyl Sublingual Spray in Treating Patients With Breakthrough Cancer Pain
This is a phase III, randomized, double-blind, placebo-controlled, multicenter study of the clinical response to fentanyl sublingual spray as a treatment for breakthrough cancer pain.
- Trial name
- NCT00538850
- Testing
- Fentanyl sublingual spray
- Where
- United States
- Volunteers
- 130 people
- Sponsor
- INSYS Therapeutics Inc
Transthyretin Amyloidosis, ATTR-CM
Non-interventional Study of Patients With Transthyretin (ATTR) Amyloidosis
The MaesTTRo study aims to enroll a global cohort of patients with transthyretin (ATTR) amyloidosis to longitudinally observe the natural course of the disease and describe real-world treatment patterns and outcomes.
- Conditions
- Transthyretin Amyloidosis, ATTR-CM, ATTRv-PN, ATTR
- Trial name
- MaesTTRo (NCT06465810)
- Testing
- Treatment of transthyretin (ATTR) amyloidosis in observational study setting
- Where
- 89 sites in United States, Canada and 4 more
- Volunteers
- 1,850 people
- Sponsor
- AstraZeneca
FAP, Familial Amyloid Polyneuropathy
Extension Study Assessing Long Term Safety and Efficacy of IONIS-TTR Rx in Familial Amyloid Polyneuropathy (FAP)
This study evaluates the safety and tolerability of extended dosing with IONIS-TTR Rx in patients with Familial Amyloid Polyneuropathy.
- Conditions
- FAP, Familial Amyloid Polyneuropathy, TTR, Transthyretin
- Trial name
- NCT02175004
- Testing
- Inotersen
- Where
- 22 sites in United States, Argentina and 7 more
- Volunteers
- 135 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Amyloid Cardiomyopathy, Transthyretin-Related
Screening for Cardiac Amyloidosis With Nuclear Imaging for Minority Populations
In this study, the investigators recruited a cohort of elderly Black and Hispanic patients with heart failure to define the number of patients who have cardiac amyloidosis by utilizing highly sensitive heart imaging and blood…
- Trial name
- SCAN-MP (NCT03812172)
- Testing
- 99mTc-PYP or 99m Tc-HDP
- Where
- 4 sites in United States
- Volunteers
- 646 people
- Sponsor
- Mathew S. Maurer, MD
Cardiac Amyloidosis
Cardiac Amyloidosis Discovery Trial
This is a single center, diagnostic clinical trial in which the investigators aim to prospectively validate a deep learning model that identifies patients with features suggestive of cardiac amyloidosis, including transthyretin…
- Trial name
- NCT06469372
- Testing
- Cardiac amyloidosis deep learning model
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Pierre Elias
Amyloidosis
Safety and Effect of Doxycycline in Patients With Amyloidosis
The tetracycline antibiotic doxycycline disrupts A beta amyloid fibrils (AB) in Alzheimer's disease, transthyretin (ATTR) amyloid fibrils in familial amyloidotic polyneuropathy, and immunoglobulin light chain (AL) amyloid fibrils…
- Trial name
- NCT01677286
- Testing
- Doxycycline 100 mg po bid x 12 months
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Boston University
Transthyretin-mediated Amyloidosis (ATTR Amyloidosis)
A Safety and Tolerability Study of an Investigational Drug, ALN-TTRSC02, in Healthy Subjects
The purpose of this study is to determine the safety, tolerability, pharmacokinetics and pharmacodynamics of ALN-TTRSC02 in healthy subjects.
- Trial name
- NCT02797847
- Testing
- ALN-TTRSC02, Sterile Normal Saline (0.9% NaCl)
- Where
- United Kingdom
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- Alnylam Pharmaceuticals
Hematopoietic/Lymphoid Cancer, Accelerated Phase Chronic Myelogenous Leukemia
Infection Prophylaxis and Management in Treating Cytomegalovirus (CMV) Infection in Patients With Hematologic Malignancies Previously Treated With Donor Stem Cell Transplant
RATIONALE: Infection prophylaxis and management may help prevent cytomegalovirus (CMV) infection caused by a stem cell transplant.
- Conditions
- Hematopoietic/Lymphoid Cancer, Accelerated Phase Chronic Myelogenous Leukemia, Acute Undifferentiated Leukemia, Adult Acute Lymphoblastic Leukemia in Remission
- Trial name
- NCT01199562
- Testing
- infection prophylaxis and management, laboratory biomarker analysis, flow cytometry
- Where
- United States
- Volunteers
- 153 people
- Sponsor
- City of Hope Medical Center
Cardiac Amyloidosis
Cardiac Uptake of 18F Florbetapir in Patients Undergoing Chemotherapy
This is a pilot study designed to assess for relative change in cardiac 18F Florbetapir uptake in patients with AL cardiac amyloidosis after appropriate chemotherapy.
- Trial name
- NCT03333551
- Testing
- F18 Florbetapir (amyvid) cardiac PET/CT imaging
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Medical College of Wisconsin
Chronic Myeloproliferative Disorders, Fever, Sweats, and Hot Flashes
Moxifloxacin Compared With Ciprofloxacin/Amoxicillin in Treating Fever and Neutropenia in Patients With Cancer
RATIONALE: Antibiotics such as amoxicillin, ciprofloxacin, and moxifloxacin may be effective in preventing or controlling fever and neutropenia in patients with cancer.
- Conditions
- Chronic Myeloproliferative Disorders, Fever, Sweats, and Hot Flashes, Infection, Leukemia
- Trial name
- NCT00062231
- Testing
- amoxicillin-clavulanate potassium, ciprofloxacin, moxifloxacin hydrochloride
- Where
- 23 sites in Belgium, France and 6 more
- Volunteers
- 351 people
- Sponsor
- European Organisation for Research and Treatment of Cancer - EORTC
Autonomic Nervous System Disease, Amyloid Neuropathies
Autonomic Evaluation of Patients With Hereditary Amyloidotic Cardiomyopathy: Hereditary Amyloidotic Heart Disease
Transthyretin amyloidosis exhibits a variety of possible phenotypes, the hereditary neurological form being the most commonly found and studied (familial amyloidotic polyneuropathy or FAP), which can present from oligosymptomatic…
- Trial name
- NCT05087953
- Testing
- Head-Up Tilt table test, Heart Rate Variability
- Where
- 3 sites in Brazil
- Volunteers
- 60 people
- Sponsor
- University of Sao Paulo General Hospital
Familial Amyloid Polyneuropathy, Familial Amyloidosis
The Effect of Diflunisal on Familial Amyloidosis
The purpose of this study is to determine if diflunisal can prevent progressive lower leg nerve damage in patients with familial amyloidosis polyneuropathy.
- Trial name
- NCT00294671
- Testing
- diflunisal
- Where
- 8 sites in United States, Italy and 3 more
- Volunteers
- 130 people
- Sponsor
- Boston University
Chronic Spontaneous Urticaria (CSU), Hidradenitis Suppurativa (HS)
SKIN Disease Profiling by an Exploratory, pRospective, Biomarker Study in dermatoloGY Practice (SKINERGY)
The goal of this observational study is to comprehensively profile six immune-mediated inflammatory diseases, including atopic dermatitis (AD), plaque psoriasis (PSO), hidradenitis suppurativa (HS), cutaneous T-cell lymphoma…
- Conditions
- Chronic Spontaneous Urticaria (CSU), Hidradenitis Suppurativa (HS), Psoriasis (PsO), Atopic Dermatitis (AD)
- Trial name
- SKINERGY (NCT07021495)
- Where
- 8 sites in Netherlands
- Volunteers
- 840 people, healthy volunteers welcome
- Sponsor
- Leiden University Medical Center
Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy
Efficacy and Safety of Acoramidis (AG10) in Subjects with Transthyretin Amyloid Polyneurophathy (ATTRibute-PN)
Phase 3 efficacy and safety of acoramidis in subjects with symptomatic Transthyretin Amyloid Polyneuropathy (ATTR-PN)
- Trial name
- NCT04882735
- Testing
- Acoramidis
- Sponsor
- Eidos Therapeutics, a BridgeBio company
Brain and Central Nervous System Tumors, Chronic Myeloproliferative Disorders
3'-Deoxy-3'-[18F] Fluorothymidine PET Imaging in Patients With Cancer
RATIONALE: Diagnostic procedures, such as 3'-deoxy-3'-[18F] fluorothymidine (FLT) PET imaging, may help find and diagnose cancer.
- Conditions
- Brain and Central Nervous System Tumors, Chronic Myeloproliferative Disorders, Leukemia, Lymphoma
- Trial name
- NCT00935090
- Testing
- 3'-deoxy-3'-[18F]fluorothymidine
- Where
- United States
- Sponsor
- Barbara Ann Karmanos Cancer Institute
Hereditary Corneal Dystrophy, Corneal Abnormality
Safety and Efficacy Study of GEB-101 Injection in Subjects With TGFBI-related Corneal Dystrophy
The goal of this clinical trial is to evaluate the safety and efficacy of GEB-101 injection in subjects with TGFBI-related Corneal Dystrophy
- Conditions
- Hereditary Corneal Dystrophy, Corneal Abnormality, Corneal Dystrophies, Corneal Dystrophies, Hereditary
- Trial name
- CLARITY (NCT07750678)
- Testing
- GEB-101 Injection
- Where
- 2 sites in United States, China
- Volunteers
- 30 people
- Sponsor
- GenEditBio Limited
Amyloid Cardiomyopathy, Congestive Heart Failure
Longitudinal Changes in Left and Right Ventricular Global Strain After Chemotherapy in Cardiac Light Chain Amyloidosis
The purpose of this study to assess the longitudinal changes in left and right ventricular global strain after chemotherapeutic strategies in cardiac light chain amyloidosis.
- Conditions
- Amyloid Cardiomyopathy, Congestive Heart Failure, Chemotherapy Effect
- Trial name
- NCT05452850
- Testing
- Left and Right Ventricular Global Strain Imaging
- Where
- Turkey (Türkiye)
- Volunteers
- 30 people
- Sponsor
- Istanbul University - Cerrahpasa
Transthyretin Amyloid Cardiomyopathy (ATTR-CM)
A Study to Learn About the Use of Acoramidis in Patients With a Heart Condition Called Transthyretin Amyloid Cardiomyopathy (ATTR-CM) in a Real-world Setting
Transthyretin Amyloid Cardiomyopathy (ATTR-CM) is a serious and life-threatening condition where a protein called transthyretin (TTR) misfolds and builds up as amyloid fibrils in the heart muscle.
- Trial name
- ACO-REAL (NCT07235462)
- Testing
- Acoramidis (356 mg film-coated tablets)
- Where
- 17 sites in Austria, Belgium and 15 more
- Volunteers
- 2,000 people
- Sponsor
- Bayer
Familial Amyloid Neuropathy, Transthyretin Amyloidosis
Assessment of the Prevalence of TTR Amyloid Neuropathy in a Population of Patients With Neuropathy of Unknown Aetiology
Familial amyloid neuropathy due to transthyretin gene mutations (TTR-FAP) is a rare autosomal dominant inherited disease resulting in the abnormal multi-system deposition of amyloid proteins.
- Trial name
- PRE-TRANS (NCT03190577)
- Testing
- blood sample
- Where
- 12 sites in France
- Volunteers
- 400 people
- Sponsor
- Nantes University Hospital
Alzheimer's Disease, Dementia
DIAN-TU Amyloid Removal Trial (ART) in Dominantly Inherited Alzheimer's Disease
This is an open label study to treat dominantly inherited Alzheimer's disease (DIAD) mutation carrier participants from the DIAN-TU-001 gantenerumab Open Label Extension (OLE) period with lecanemab to determine the effects of…
- Conditions
- Alzheimer's Disease, Dementia, Alzheimer's Disease, Familial
- Trial name
- DIAN-TU (NCT06384573)
- Testing
- lecanemab
- Where
- 6 sites in United States, Australia and 1 more
- Volunteers
- 40 people
- Sponsor
- Washington University School of Medicine