Clinical trials: FAP
Transthyretin Amyloidosis, Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy
Biomarker for Transthyretin-Related Familial Amyloidotic Polyneuropathy (BioTRAP)
International, multicenter, observational, longitudinal study to identify biomarker/s for the development of a new MS-based biomarker for the early and sensitive diagnosis of Transthyretin-Related Familial Amyloidotic…
- Conditions
- Transthyretin Amyloidosis, Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy, Transthyretin Amyloid Cardiopathy
- Trial name
- BioTRAP (NCT02713880)
- Where
- 2 sites in Germany, India
- Sponsor
- CENTOGENE GmbH Rostock
Adenomatous Polyposis Coli
Trial In Pediatric Patients With Familial Adenomatous Polyposis (FAP)
To test whether celecoxib can be used to prevent colon polyp formation in children with familial adenomatous polyposis (FAP).
- Trial name
- CHIP (NCT00585312)
- Testing
- Celecoxib
- Where
- 42 sites in United States, Belgium and 12 more
- Volunteers
- 106 people
- Sponsor
- Pfizer's Upjohn has merged with Mylan to form Viatris Inc.
Abdominal Pain
Efficacy of Lactobacillus Reuteri Protectis on Functional Abdominal Pain (FAP) in Children 8-14 Years Old
The present clinical trial will examine the use of L. reuteri Protectis in children with functional abdominal pain. The aim of the study is to evaluate the use of L.
- Trial name
- LactoFAP (NCT01498666)
- Testing
- L. reuteri protectis
- Where
- 2 sites in Italy
- Volunteers
- 9 people
- Sponsor
- BioGaia AB
Adenomatous Polyposis Coli
Post-Trial Access for Guselkumab in Participants With Familial Adenomatous Polyposis
The purpose of this post-trial access (PTA) program is to provide guselkumab to participants with Familial Adenomatous Polyposis (FAP) who are experiencing clinical benefit after completing 48 weeks of treatment in Study…
- Trial name
- NCT04948398
- Testing
- Guselkumab
- Sponsor
- Janssen Research & Development, LLC
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
Replacement Therapy in Preterm Infants
Efficacy and Safety of Bile Salt Stimulated Lipase (BSSL) as Replacement Therapy in Pasteurized Breast Milk for Preterm Infants
The purpose of this study is to evaluate the efficacy in preterm infants following treatment with BSSL when administered in pasteurized breast milk.
- Trial name
- NCT00659243
- Testing
- rhBSSL
- Where
- 8 sites in France, Italy
- Volunteers
- 32 people
- Sponsor
- Swedish Orphan Biovitrum
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
Familial Adenomatous Polyposis (FAP)
Observational Familial Adenomatous Polyposis Registry Study In Patients Receiving Celecoxib Compared to Control Patients
This is a registry-based observational study assessing clinical outcomes in FAP patients receiving celecoxib compared with historical/concurrent registry patients who have not received celecoxib.
- Trial name
- NCT00151476
- Testing
- Celecoxib, Routine Medical Care
- Where
- 4 sites in United States, Canada and 2 more
- Volunteers
- 68 people
- Sponsor
- Pfizer
Replacement Therapy in Preterm Infants
Efficacy and Safety of Bile Salt Stimulated Lipase (BSSL) as Replacement Therapy in Infant Formula for Preterm Infants
The purpose of this study is to evaluate the efficacy in preterm infants following treatment with BSSL when administered in infant formula.
- Trial name
- NCT00658905
- Testing
- rhBSSL
- Where
- 6 sites in Italy
- Volunteers
- 33 people
- Sponsor
- Swedish Orphan Biovitrum
Familial Adenomatous Polyposis (FAP)
Expanded Access to 0.5 mg eRapa for Familial Adenomatous Polyposis
This expanded access program provides 0.5 mg eRapa (encapsulated rapamycin) to patients with familial adenomatous polyposis (FAP) who have no satisfactory alternative treatment options and are not eligible to participate in a…
- Trial name
- eRapa-FAP (NCT07689942)
- Testing
- eRapa (encapsulated rapamycin)
- Sponsor
- Biodexa Pharmaceuticals
Hereditary Transthyretin-mediated Amyloidosis With Polyneropathy, Pregnancy
TEGSEDI Pregnancy Surveillance Program
This is a worldwide safety surveillance study of pregnancy outcomes in women with hATTR-PN who may have been exposed or were not exposed to TEGSEDI prior to or during the pregnancy and of pediatric outcomes up to 1 year of age.
- Trial name
- NCT04270058
- Testing
- inotersen
- Volunteers
- 20 people
- Sponsor
- Akcea Therapeutics
Transthyretin Amyloidosis
The Eplontersen Pregnancy and Lactation Outcomes Study
The overall objective of this DPSS is to describe the occurrence of pregnancy and maternal complications, adverse effects on the developing fetus and neonate, and adverse effects on the infant associated with exposure to…
- Trial name
- EPPRO (NCT07205666)
- Where
- Germany
- Volunteers
- 10 people
- Sponsor
- AstraZeneca