Clinical trials: Families
Orphan Cholestatic Liver Diseases, Primary Biliary Cirrhosis
A Safety and Pharmakokinetic Study of A4250 Alone or in Combination With A3384
The primary objectives of the study are to evaluate the safety, tolerability and pharmacokinetics of A4250 after single or multiple oral doses in healthy subjects.
- Conditions
- Orphan Cholestatic Liver Diseases, Primary Biliary Cirrhosis, Progressive Familial Intrahepatic Cholestasis, Alagille Syndrome
- Trial name
- NCT02963077
- Testing
- A4250, CRC (A3384), Questran
- Volunteers
- 94 people, healthy volunteers welcome
- Sponsor
- Albireo
X-linked Hypophosphatemia
Study of KRN23 (Burosumab), a Recombinant Fully Human Monoclonal Antibody Against Fibroblast Growth Factor 23 (FGF23), in Pediatric Subjects With X-linked Hypophosphatemia (XLH)
The objectives of the study are to: Identify a dose and dosing regimen of burosumab, based on safety and pharmacodynamic (PD) effect, in pediatric XLH participants Establish the safety profile of burosumab for the treatment of…
- Trial name
- NCT02163577
- Testing
- burosumab
- Where
- 9 sites in United States, France and 2 more
- Volunteers
- 52 people
- Sponsor
- Kyowa Kirin, Inc.
Lysosomal Acid Lipase Deficiency
A Study to Identify and Characterize LAL-D Patients in High-risk Populations
The objective of this study is to determine the frequency of Lysosomal Acid Lipase Deficiency (LAL D) by lysosomal acid lipase (LAL) enzyme activity assay in patients who are considered to be at risk.
- Trial name
- NCT02345421
- Where
- 15 sites in United States
- Volunteers
- 640 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Autosomal Dominant Hypocalcemia
ADH1 and ADH2 Disease Monitoring Study (DMS)
A global, multi-center, Disease Monitoring Study (DMS) in participants with Autosomal Dominant Hypocalcemia Type 1 (ADH1) or Autosomal Dominant Hypocalcemia Type 2 (ADH2) designed to characterize ADH1 and ADH2 disease…
- Trial name
- CLARIFY (NCT05227287)
- Where
- 27 sites in United States, Australia and 11 more
- Volunteers
- 95 people
- Sponsor
- Calcilytix Therapeutics, Inc., a BridgeBio company
Familial Adenomatous Polyposis
Trial of eRapa to Prevent Progression in Familial Adenomatous Polyposis Patients Under Active Surveillance
Patients with Familial Adenomatous Polyposis (FAP) who are undergoing endoscopic surveillance will be given Encapsulated Rapamycin (eRapa) at one of three escalating doses/schedules for 12 months with the aim of reducing polyp…
- Trial name
- NCT04230499
- Testing
- Encapsulated Rapamycin (eRapa)
- Where
- 4 sites in United States
- Volunteers
- 30 people
- Sponsor
- Rapamycin Holdings Inc.
Coronary Artery Disease
Clinical Study Comparing Single Long BioMimeTM Morph Coronary Stent System vs. Two Overlapping Xience Family Coronary Stent Systems in the Treatment of Patients With Long de Novo Lesions.
A prospective, active control, open-label, multinational, randomized clinical trial comparing single long BioMime™ Morph Coronary Stent System vs.
- Trial name
- Morph RCT-1 (NCT03721614)
- Testing
- BioMime™ Morph - Sirolimus Eluting Coronary Stent System, Xience family Everolimus Coronary Stent Systems
- Volunteers
- 200 people
- Sponsor
- Meril Life Sciences Pvt. Ltd.
Parent-Child Relations
The Effective Parenting Program (EPP)
The Phase I objective is to determine the feasibility of the EPP program by examining user responses to the first three units of EPP's nine interactive training modules.
- Trial name
- NCT05135507
- Testing
- An integrated parenting intervention for families of children with developmental disabilities
- Sponsor
- Double S Instructonal Systems
Hypertriglyceridemia, Familial Hypercholesterolemia
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of IONIS ANGPTL3-LRx in Healthy Volunteers With Elevated Triglycerides and Participants With Familial Hypercholesterolemia
The purpose is to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of IONIS ANGPTL3-LRx (ISIS 703802) given to healthy volunteer subjects with elevated triglycerides and subjects with familial…
- Trial name
- NCT02709850
- Testing
- IONIS ANGPTL3-LRx
- Where
- Canada
- Volunteers
- 48 people, healthy volunteers welcome
- Sponsor
- Ionis Pharmaceuticals, Inc.
Language Development
STEM (Science, Technology, Engineering & Math) Familia Talk
After development of the Talk Stem Familia (TSF) smart-speaker application prototype, the study will evaluate the prototype's efficacy in a within-subjects pre-post design study.
- Trial name
- NCT03766906
- Testing
- Talk STEM Familia app
- Where
- United States
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- Oregon Research Behavioral Intervention Strategies, Inc.
Off Pump Coronary Artery Bypass Surgery
Getinge's Beating Heart Product Family Post-Market Clinical Follow-up (PMCF) Study
This observational study aims to collect data on the performance and safety of Getinge's Beating Heart products used during open-heart coronary artery bypass graft surgery.
- Trial name
- NCT07286890
- Testing
- Vacuum Stabilizer System, Vacuum Positioner System, Blower/Mister
- Where
- 7 sites in United States, Germany and 1 more
- Volunteers
- 200 people
- Sponsor
- Maquet Cardiovascular
Familial Mediterranean Fever, Still Disease
A Study on the Use of Canakinumab Among Familial Mediterranean Fever and Still's Disease Patients
This study aims to assess and characterize the treatment patterns, and long-term clinical outcomes and demographic characteristics of patients diagnosed with Familial Mediterranean fever (FMF) and Still's disease (including…
- Conditions
- Familial Mediterranean Fever, Still Disease, Systemic Juvenile Idiopathic Arthritis, Adult-Onset Still Disease
- Trial name
- NCT07517250
- Where
- Switzerland
- Volunteers
- 160 people
- Sponsor
- Novartis Pharmaceuticals
Fredrickson Type IIa & Type IIb Dyslipidaemia
IIIb 6 Week Open Label Multicentre Rosuvastatin & Simvastatin
The purpose of this study is to assess the effects on the kidney of rosuvastatin and simvastatin in subjects with Fredrickson Type IIa \& Type IIb Dyslipidaemia
- Trial name
- NCT00654446
- Testing
- Rosuvastatin, Simvastatin
- Volunteers
- 442 people
- Sponsor
- AstraZeneca
Attention Deficit Disorder With Hyperactivity
The Impact of Osmotic Release Oral Delivery System Methylphenidate (OROS MPH) Upon Family of Children and Adolescents With Attention Deficit Hyperactivity Disorder (ADHD)
The purpose of this study is to evaluate whether familial relationships and psychological status of participants or caregivers as well as Attention Deficit Hyperactivity Disorder (ADHD) symptoms of participants can be improved by…
- Trial name
- NCT00758160
- Testing
- OROS Methylphenidate
- Volunteers
- 296 people
- Sponsor
- Johnson & Johnson Taiwan Ltd
Alzheimer Disease
Virtual Patient Behavioral Response Training for Family Caregivers
Our study is designed to help caregivers of someone with Alzheimer's Disease and related dementia to understand and deal with the difficult behaviors of a person with dementia.
- Trial name
- NCT04489303
- Testing
- Caring Response App, Traditional Educational/Resources Program
- Where
- United States
- Volunteers
- 156 people, healthy volunteers welcome
- Sponsor
- Photozig, Inc.
Homozygous Familial Hypercholesterolemia HoFH
Safety and Tolerability of Repatha® (Evolocumab) in Indian Participants With Homozygous Familial Hypercholesterolemia
To describe the safety and tolerability of evolocumab in participants with homozygous familial hypercholesterolemia (HoFH) in India. All participants will receive evolocumab over an 8-week period.
- Trial name
- RAMAN (NCT03403374)
- Testing
- evolocumab
- Where
- 12 sites in India
- Volunteers
- 30 people
- Sponsor
- Amgen
Fertility Disorders
Quantum Natural Family Planning Pilot
•The Pilot study will involve current Marquette Method users who own and use a ClearBlue Fertility monitor and who will be asked to simultaneously use the Mira monitor for the sake of comparing the two devices to monitor the…
- Trial name
- QNFP (NCT03908697)
- Testing
- Mira monitor comparison to ClearBlue monitor
- Where
- Canada
- Volunteers
- 26 people, healthy volunteers welcome
- Sponsor
- Quanovate Tech Inc.