A Study to Learn About Tafamidis in Patients With Transthyretin Amyloid Cardiomyopathy (ATTR-CM)...
Transthyretin Amyloid CardiomyopathyThe purpose of this study is to learn about the safety of Tafamidis for the treatment of Transthyretin amyloid cardiomyopathy (ATTR-CM) in India. ATTR-CM is a condition that affects people's hearts. Transthyretin is a protein that is made in the liver. In some people this protein stops working and forms clumps called as amyloid. Transthyretin amyloid builds up in heart and stops the heart from pumping properly. This study is seeking for participants who are: confirmed with ATTR-CM. given Tafamidis capsules to be taken by mouth. The safety of Tafamidis capsules will be checked based on side effects. These side effects can happen within 6 months after taking Tafamidis. A side effect is something (expected or unexpected) that you feel was caused by a medicine or treatment you take. The study doctor will collect side effect information and put the information on patient's case form. Follow-up of the patient's will be performed via clinic re-visit or over a call. It is not a rule for the participants to visit the clinic in this study. This study will help to see if Tafamidis is safe.
The Study of an Investigational Drug, Revusiran (ALN-TTRSC), for the Treatment of Transthyretin...
Transthyretin (TTR)-Mediated AmyloidosisFamilial Amyloidotic Polyneuropathy (FAP)2 moreThe purpose of this study was to evaluate the safety and effectiveness of revusiran (ALN-TTRSC) in adults with transthyretin-mediated amyloidosis (ATTR), whose disease has continued to worsen after liver transplantation. Dosing has been discontinued; patients are being followed-up for safety.
The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin...
AmyloidosisThe purpose of this study is to evaluate the safety and efficacy of long-term dosing with ALN-TTR02 (patisiran) in patients with transthyretin (TTR) mediated amyloidosis (ATTR).
Vinorelbine and Gemcitabine in Myeloma
Multiple MyelomaAmyloidosisThe purpose of this study is to determine whether the efficacy of gemcitabine is comparable with the efficacy of the standard chemotherapy with vinorelbine for mobilization of autologous stem cells in myeloma patients
A Randomised, Open-Label, Cross Over Group, Single-Centre Controlled Study To Evaluate The Clinical...
Hemodialysis-Associated AmyloidosisDialysis Amyloidosis1 moreThis is an open label, cross-over group, single-centre randomised controlled trial comparing Medium Cut Off Dialyser with High Flux Dialyser in patients with end-stage renal disease on chronic haemodialysis.
Patisiran in Patients With Hereditary Transthyretin-mediated Amyloidosis (hATTR Amyloidosis) Disease...
AmyloidosisFamilial1 moreThe 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 transplant.
A Study of Doxycycline and Tauroursodeoxycholic Acid (Doxy/TUDCA) Plus Standard Supportive Therapy...
TTR Cardiac AmyloidosisCardiac amyloidosis caused by transthyretin either mutated (in ATTRm amyloidosis) or wildtype (in ATTRwt, formerly senile, amyloidosis) is a rare disease but is diagnosed with increasing frequency thanks to the availability of non-invasive scintigraphy-based means. Cardiac ATTR amyloidosis is characterized by progressive heart failure with a median survival of less than 4 years, and there is no standard treatment for this disease. It was proved that the marketed antibiotic doxycycline (Doxy) disrupts amyloid fibrils in vitro and in animal models synergistically with tauroursodeoxycholic acid (TUDCA). Based on these pre-clinical data, a clinical trial of Doxy/TUDCA in ATTR Amyloidosis (NCT01171859) was conducted. Treatment was well tolerated and was able to prevent progression of cardiac dysfunction.
The Study of ALN-TTR02 (Patisiran) for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis...
TTR-mediated AmyloidosisThe purpose of this study is to evaluate the safety and tolerability of long-term dosing with ALN-TTR02 (patisiran) in patients with transthyretin (TTR) mediated amyloidosis (ATTR).
Safety and Efficacy Study of Doxycycline/UrsoDeoxyCholicAcid on Disease Progression in ATTR Amyloidosis...
Transthyretin AmyloidosisCardiomyopathyThe primary objective for this study is to evaluate the efficacy of doxycycline + ursodeoxycholic acid (UDCA) on disease progression in Transthyretin Amyloidosis (ATTR) subjects with cardiomyopathy with or without neuropathy.
Doxycycline and TUDCA in Patients With Transthyretin Amyloid Cardiomyopathy
Amyloidosis; Heart (Manifestation)Senile Cardiac AmyloidosisThe objective of the study is to determine whether the combination of the bile acid TUDCA, and doxycycline will slow the progression of familial and senile amyloidosis.