Clinical trials: FAP
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
Hereditary Transthyretin-mediated (ATTRv) Amyloidosis, Polyneuropathy
A Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of ATTRv Amyloidosis With a V122I or T60A Mutation
To evaluate the effectiveness of patisiran in patients with ATTRv amyloidosis with polyneuropathy who have a V122I or T60A mutation.
- Trial name
- NCT04201418
- Testing
- Patisiran
- Where
- 27 sites in United States
- Volunteers
- 67 people
- Sponsor
- Alnylam Pharmaceuticals
Transthyretin Gene Mutations, Transthyretin Amyloidosis
Transthyretin Amyloidosis Outcome Survey (THAOS)
THAOS is a global, multi-center, longitudinal observational survey open to all patients with transthyretin amyloidosis (ATTR), including ATTR-PN (polyneuropathy), ATTR-CM (cardiomyopathy) and wild-type ATTR-CM.
- Trial name
- THAOS (NCT00628745)
- Testing
- None. Observational Study.
- Where
- 113 sites in United States, Argentina and 23 more
- Volunteers
- 6,718 people
- Sponsor
- Pfizer
Hereditary Transthyretin-Mediated Amyloidosis With Polyneuropathy, hATTR-PN
TRITON-PN: A Study to Evaluate the Efficacy and Safety of Nucresiran in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy
The purpose of this study is to: Determine the efficacy of nucresiran in patients with hATTR-PN by evaluating the effect on neurologic impairment, quality of life, nutritional status, disability, and gait speed Demonstrate…
- Trial name
- TRITON-PN (NCT07223203)
- Testing
- Nucresiran, Vutrisiran
- Where
- 61 sites in United States, Argentina and 20 more
- Volunteers
- 125 people
- Sponsor
- Alnylam Pharmaceuticals
Familial Adenomatous Polyposis (FAP)
A Safety Study of ST316 in Participants With Familial Adenomatous Polyposis (FAP).
This is a Phase 1b, open- label, dose-optimization study evaluating ST316 in adult participants with familial adenomatous polyposis (FAP) who have undergone colectomy and have recurrent polyps.
- Trial name
- FAP (NCT07729371)
- Testing
- ST316 IV
- Volunteers
- 40 people
- Sponsor
- Sapience Therapeutics
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
Solid Tumor Malignancy
Safety Trial of 68Ga/177Lu-NRT6020 in FAP-Positive Advanced Solid Tumor Patients
Stage 1 (Safety study for 68Ga-NRT6020 Injection): The goal of this clinical trial stage is to evaluate the safety of 68Ga-NRT6020 Injection in participants with advanced solid tumors.
- Trial name
- NCT06911489
- Testing
- 68Ga-NRT6020 Injection, 177Lu-NRT6020 Injection (stage 2), 177Lu-NRT6020 Injection (stage 3)
- Where
- 2 sites in China
- Volunteers
- 132 people
- Sponsor
- Chengdu New Radiomedicine Technology Co. LTD.
Salivary Gland Tumor, Urothelial Carcinoma
A Study Evaluating the Safety, Pharmacokinetics and Early Efficacy of AVA6000 in Solid Tumours
This is a first-in-human (FIH), Phase 1 open-label, multicentre dose escalation study investigating AVA6000 monotherapy administered intravenously in patients with locally advanced (unresectable) or metastatic solid tumours that…
- Conditions
- Salivary Gland Tumor, Urothelial Carcinoma, Ovarian Carcinoma, Breast Cancer
- Trial name
- NCT04969835
- Testing
- AVA6000
- Where
- 9 sites in United States, United Kingdom
- Volunteers
- 158 people
- Sponsor
- Avacta Life Sciences Ltd
Amyloidosis, Familial, Transthyretin Amyloidosis
Patisiran in Patients With Hereditary Transthyretin-mediated Amyloidosis (hATTR Amyloidosis) Disease Progression Post-Liver Transplant
The purpose of this study is to evaluate the efficacy, safety and pharmacokinetics of patisiran in participants with hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) with disease progression after liver…
- Trial name
- NCT03862807
- Testing
- Patisiran
- Where
- 9 sites in France, Germany and 5 more
- Volunteers
- 24 people
- Sponsor
- Alnylam Pharmaceuticals
Transthyretin Familial Amyloidosis Polyneuropathy (TTR-FAP), Transthyretin Cardiomyopathy (TTR-CM)
Burden of Disease Study In Patients With Transthyretin Familial Amyloidosis Polyneuropathy (TTR-FAP) orTransthyretin Cardiomyopathy (TTR-CM) And Caregivers
This study is an online (web-based) or paper-based survey for patients with transthyretin familial amyloidosis polyneuropathy (TTR-FAP) and caregivers.
- Conditions
- Transthyretin Familial Amyloidosis Polyneuropathy (TTR-FAP), Transthyretin Cardiomyopathy (TTR-CM), Familial Amyloid Cardiomyopathy, Senile Systemic Amyloidosis (SSA)
- Trial name
- NCT01604122
- Testing
- No drug
- Volunteers
- 1 people
- Sponsor
- Pfizer
Familial Adenomatous Polyposis
A Trial of Low Dose Sulindac Combined With Eflornithine in Patients With Familial Adenomatous Polyposis (FAP)
The purpose of this phase III study is to evaluate the safety and efficacy of the combination of eflornithine and sulindac compared to single agent sulindac or eflornithine in reducing the number of polyps in patients with…
- Trial name
- NCT01245816
- Testing
- Eflornithine plus Sulindac
- Sponsor
- Cancer Prevention Pharmaceuticals, Inc.
Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
HELIOS-B: A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy
This study will evaluate the efficacy and safety of vutrisiran 25 mg administered subcutaneously (SC) once every 3 months (q3M) compared to placebo in participants with ATTR amyloidosis with cardiomyopathy.
- Trial name
- NCT04153149
- Testing
- Vutrisiran, Sterile Normal Saline (0.9% NaCl)
- Where
- 119 sites in United States, Argentina and 30 more
- Volunteers
- 655 people
- Sponsor
- Alnylam Pharmaceuticals
FAP, Familial Amyloid Polyneuropathy
Efficacy and Safety of Inotersen in Familial Amyloid Polyneuropathy
The purpose of this study is to evaluate the efficacy and safety of inotersen given for 65 weeks in participants with Familial Amyloid Polyneuropathy (FAP).
- Conditions
- FAP, Familial Amyloid Polyneuropathy, TTR, Transthyretin
- Trial name
- NCT01737398
- Testing
- Inotersen
- Where
- 24 sites in United States, Argentina and 8 more
- Volunteers
- 173 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Familial Amyloid Polyneuropathy
Safety and Efficacy Study of Fx-1006A in Patients With Familial Amyloidosis
This study will examine whether Fx-1006A is effective in halting the progression of Familial Amyloid Polyneuropathy (FAP). Deposition of TTR amyloid is associated with a variety of human diseases.
- Trial name
- NCT00409175
- Testing
- Fx-1006A
- Where
- 10 sites in United States, Argentina and 7 more
- Volunteers
- 128 people
- Sponsor
- Pfizer
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
Advanced/Metastatic Head and Neck, Oesophageal and Cervical Cancers
Basket Study to Evaluate the Therapeutic Activity of Simlukafusp Alfa as a Combination Therapy in Participants With Advanced and/or Metastatic Solid Tumors
This is an open-label, multicenter, basket trial Phase II study to evaluate the antitumor activity of simlukafusp alfa in combination with atezolizumab in participants with advanced and/or metastatic solid tumors.
- Trial name
- NCT03386721
- Testing
- simlukafusp alfa, Atezolizumab (MPDL3280A), an Engineered Anti-PD-L1 Antibody, Gemcitabine
- Where
- 44 sites in United States, Belgium and 13 more
- Volunteers
- 256 people
- Sponsor
- Hoffmann-La Roche
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
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
Liver Fibrosis, Hepatic Cirrhosis
The Phase 1, Open-label, PET Trial Designed to Investigate the Effect of AZD2389 on FAP Occupancy in the Liver in Participants With Advanced Liver Fibrosis.
This is a phase 1, open-label, PET trial. The study is designed to investigate the effect of AZD2389 on FAP occupancy in the liver in participants with advanced liver fibrosis.
- Trial name
- PECHORA (NCT07069725)
- Testing
- AZD2389, PET scan and radioligand
- Where
- Sweden
- Volunteers
- 8 people
- Sponsor
- AstraZeneca
Metastatic Colorectal Cancer
Study To Evaluate Safety, Pharmacokinetics, Pharmacodynamics, And Preliminary Anti-Tumor Activity Of RO7122290 In Combination With Cibisatamab With Obinutuzumab Pre-Treatment
This is an open-label, multicenter, Phase Ib study to determine the maximum tolerated dose (MTD) and/or the recommended Phase 2 dose (RP2D) in the weekly (QW) and/or every 3 weeks (Q3W) regimens, safety, tolerability, PK…
- Trial name
- NCT04826003
- Testing
- RO7122290, Cibisatamab, Obinutuzumab
- Where
- 13 sites in Denmark, Netherlands and 3 more
- Volunteers
- 54 people
- Sponsor
- Hoffmann-La Roche