Clinical trials: Relationship, Family
Transthyretin-Mediated Amyloidosis, ATTR Amyloidosis
ConTTRibute: A Global Observational Study of Patients With Transthyretin (TTR)-Mediated Amyloidosis (ATTR Amyloidosis)
The purpose of this study is to: Describe epidemiological and clinical characteristics, natural history and real-world clinical management of ATTR amyloidosis patients Characterize the safety and effectiveness of patisiran and…
- Trial name
- ConTTRibute (NCT04561518)
- Where
- 40 sites in United States, Brazil and 10 more
- Volunteers
- 1,500 people
- Sponsor
- Alnylam Pharmaceuticals
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
X-Linked Hypophosphataemia
A UK Multicentre, Health-Related Quality of Life Study for Children and Adolescents With XLH
This study aims to provide Health-related Quality of Life (HRQoL) data from children and adolescents with growing skeletons in the United Kingdom (UK), including those treated with burosumab or alternative XLH treatment, as part…
- Trial name
- NCT04819490
- Where
- 6 sites in United Kingdom
- Volunteers
- 32 people
- Sponsor
- Kyowa Kirin Pharmaceutical Development Ltd
Amyloidosis, Hereditary, Transthyretin Amyloidosis
HELIOS-A: A Study of Vutrisiran (ALN-TTRSC02) in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)
The purpose of this study is to evaluate the efficacy and safety of vutrisiran (ALN-TTRSC02) in participants with hereditary transthyretin amyloidosis (hATTR amyloidosis).
- Trial name
- NCT03759379
- Testing
- Patisiran, Vutrisiran
- Where
- 64 sites in United States, Argentina and 20 more
- Volunteers
- 164 people
- Sponsor
- Alnylam Pharmaceuticals
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
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
Heartfailure
Maximizing CRT Delivery by Using MultipolAr Coronary Sinus Lead FamiLy ACUITY® X4 - RALLY X4 Study
The objective of this study is to collect clinical data on safety and performance of ACUITY X4® leads when used in a standard clinical setting.
- Trial name
- RallyX4 (NCT02066467)
- Testing
- Left Ventricular lead implant: ACUITY X4® Lead Family
- Where
- 83 sites in Austria, Belgium and 16 more
- Volunteers
- 863 people
- Sponsor
- Boston Scientific Corporation
Amyloidosis, Amyloid Cardiomyopathy
Efficacy and Safety of AG10 in Subjects With Transthyretin Amyloid Cardiomyopathy
Phase 3 efficacy and safety study to evaluate acoramidis (AG10) HCl 800 mg administered orally twice a day compared to placebo in subjects with symptomatic Transthyretin Amyloid Cardiomyopathy (ATTR-CM).
- Conditions
- Amyloidosis, Amyloid Cardiomyopathy, Transthyretin Amyloidosis, Cardiomyopathies
- Trial name
- ATTRibute-CM (NCT03860935)
- Testing
- acoramidis
- Where
- 104 sites in United States, Australia and 16 more
- Volunteers
- 632 people
- Sponsor
- Eidos Therapeutics, a BridgeBio company
Anxiety Disorders, Childhood Mental Disorder
Family-Focused CBT Skills App and Standard Self Help Options for Childhood Anxiety
Anxiety disorders are among the most common psychological disorders, with prevalent onset in childhood and adolescence. While cognitive behavioral therapy (CBT) is considered the first-line treatment for pediatric anxiety…
- Conditions
- Anxiety Disorders, Childhood Mental Disorder, Anxiety
- Trial name
- NCT03269370
- Testing
- Anchors Away, Parent Enhanced Anchors Away, Self-Help E-Book
- Where
- United States
- Volunteers
- 110 people, healthy volunteers welcome
- Sponsor
- Kansas City Center for Anxiety Treatment, P.A.
PFIC - Progressive Familial Intrahepatic Cholestasis
A Study to Assess the Genetic Variations in Bile Flow Disorders: Linking Progressive Familial Intrahepatic Cholestasis (PFIC)-Related Genes to Symptoms in Adults With Recurrent Cholestasis in Spain
Progressive Familial Intrahepatic Cholestasis (PFIC) is a group of inherited conditions that affect how bile moves in the liver, which can lead to serious liver problems.
- Trial name
- REGENIC (NCT07191704)
- Where
- 10 sites in Spain
- Volunteers
- 150 people
- Sponsor
- Ipsen
Aging, Mortality
Human Observatory Study
The Human Observatory Study is a prospective observational and ecological surveillance study building a continuously-updating world model for human health, disease, and death at the individual and population level.
- Conditions
- Aging, Mortality, All-cause Mortality, Life Expectancy
- Trial name
- HOS (NCT07646782)
- Testing
- Multi-Domain Ecological and Clinical Data Linkage
- Where
- United States
- Volunteers
- 1,000,000 people, healthy volunteers welcome
- Sponsor
- Longevity Metrics, Inc.
Mitochondrial Diseases
A Long-term Extension of Study RP103-MITO-001 (NCT02023866) to Assess Cysteamine Bitartrate Delayed-release Capsules (RP103) in Children With Inherited Mitochondrial Disease
A long-term extension study to assess the safety, tolerability and efficacy of cysteamine bitartrate delayed-release capsules (RP103) in children with inherited mitochondrial diseases who previously enrolled into study…
- Trial name
- NCT02473445
- Testing
- Cysteamine Bitartrate
- Where
- 5 sites in United States
- Volunteers
- 22 people
- Sponsor
- Amgen
Family Relations, Sibling Relations
SIB-Time Web-application Tool for Typically Developing Siblings
In the United States, over 32.7 million people have special health, developmental, and mental health concerns. Most of these people have typically developing brothers and sisters.
- Trial name
- SIBTime (NCT04633473)
- Testing
- SIB-Time Web-Application Tool
- Where
- United States
- Volunteers
- 43 people, healthy volunteers welcome
- Sponsor
- Oregon Research Behavioral Intervention Strategies, Inc.
Hereditary Transthyretin Amyloidosis
hATTR Polyneuropathy in Russia
A multicenter observational retrosPective Registry of patIents with transthyretin aMyloid polynEuropathy (hATTR-PN) and chRonic idiopathic axonal polyneuropathy (CIAP) in the population of the Russian Federation (PRIMER) There…
- Trial name
- PRIMER (NCT06365593)
- Where
- 3 sites in Russia
- Volunteers
- 100 people
- Sponsor
- AstraZeneca
Amyloid Cardiomyopathy, Transthyretin-Related
Open-Label Safety Study of Acoramidis (AG10) in Symptomatic ATTR Participants
Open-Label Extension and Safety Monitoring Study of Acoramidis (AG10) in Participants with Symptomatic Transthyretin Amyloid Cardiomyopathy Who Completed the Phase 3 ATTRibute-CM Trial (AG10-301)
- Trial name
- AG10 (NCT04988386)
- Testing
- Acoramidis (AG10)
- Where
- 72 sites in United States, Australia and 15 more
- Volunteers
- 389 people
- Sponsor
- Eidos Therapeutics, a BridgeBio company
Psychosocial Deprivation, Work-Related Stress
Family Supportive Supervisor Training and Workplace Assessment Tool
This study will evaluate the effectiveness of FSST 2.0 (family supportive supervisor training plus support for use of family and sick leaves) using a randomized control trial design.
- Conditions
- Psychosocial Deprivation, Work-Related Stress, Life-work Imbalance
- Trial name
- FSST (NCT05080426)
- Testing
- Family Supportive Supervisor Training 2.0
- Where
- United States
- Volunteers
- 1,175 people, healthy volunteers welcome
- Sponsor
- Work Life Help
Familial Mediterranean Fever
A Study to Describe a Group of Spanish Patients With FMF and to Identify Suboptimally Treated Patients With Minimal Disease Activity and Its Impact on Quality of Life
A retrospective, non-interventional, single-center study conducted in Spain, based on the collection of data from the medical histories of patients with Familial Mediterranean fever (FMF) diagnosed during childhood from January…
- Trial name
- NCT06923124
- Where
- United States
- Volunteers
- 44 people
- Sponsor
- Novartis Pharmaceuticals
TTR-mediated Amyloidosis, Amyloidosis, Hereditary
Expanded Access Protocol of Patisiran for Patients With Hereditary ATTR Amyloidosis (hATTR)
The purpose of this study is to provide expanded access of patisiran to patients with hereditary transthyretin-mediated amyloidosis (hATTR).
- Conditions
- TTR-mediated Amyloidosis, Amyloidosis, Hereditary, Amyloid Neuropathies, Familial, Familial Amyloid Polyneuropathies
- Trial name
- NCT02939820
- Testing
- patisiran (ALN-TTR02)
- Sponsor
- Alnylam Pharmaceuticals
Polyneuropathy, Amyloid, Neuropathic Pain
Screening for the Transthyretin-Related Familial Amyloidotic Polyneuropathy (TTR FAP)
An International, multicenter, epidemiological observational study investigating the prevalence of Transthyretin-Related Familial Amyloidotic Polyneuropathy (TTR-FAP) in participants with small fiber polyneuropathy of no obvious…
- Conditions
- Polyneuropathy, Amyloid, Neuropathic Pain, Cardiac Failure, Orthostatic Hypotension
- Trial name
- TRAP2-1 (NCT01705626)
- Where
- 11 sites in Austria, Hungary and 4 more
- Volunteers
- 500 people
- Sponsor
- CENTOGENE GmbH Rostock
Opioid-use Disorder, Opioid-related Disorders
An Observational Study of Environmental and SocioEconomic Factors in Opioid Recovery
The RECOVER Study will examine clinical, environmental and socioeconomic factors in recovery from opioid use disorder (OUD) over a 24-month period following exit from a Phase III clinical program for a buprenorphine…
- Trial name
- RECOVER® (NCT03604861)
- Testing
- Not applicable - no defined intervention
- Where
- 35 sites in United States
- Volunteers
- 534 people
- Sponsor
- Indivior Inc.