Tafamidis meglumine soft capsules for adults with transthyretin amyloid polyneuropathy

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a phase 4 interventional study giving tafamidis meglumine soft capsules to adults with biopsy-confirmed transthyretin amyloid deposits and a disease-associated TTR mutation who have stage 1 peripheral neuropathy. Eligible participants are 18–80 years old with a Karnofsky Performance Status ≥50 and no other causes of amyloidosis. The protocol excludes recent use of tafamidis or other ATTR-specific therapies and limits NSAID exposure. Participants will be followed for measures of efficacy and safety per the study protocol at the enrolling site.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* Male or female participants between the ages of 18 and 80 years.
* Participant has amyloid deposits confirmed by biopsy (Biopsy must have been performed within 5 years prior to enrollment).
* Participant must have a TTR mutation that is associated with ATTR-PN (TTR mutation test must have been performed within 5 years prior to enrollment).
* Participant has peripheral neuropathy at screening.
* Participant has a Karnofsky Performance Status Score ≥50.
* Stages of disease according to symptom severity-stage 1.

Exclusion Criteria:

* Participant has other causes of amyloidosis, such as light chain amyloidosis, AA amyloidosis.
* Participant has used tafamidis within 2 months prior to enrollment.
* Participant has used diflunisal, patisiran, inotersen, or other agents for familial amyloidosis within 30 days prior to enrollment or plan to use them during the study period.
* Participant has used non-protocol NSAIDs more than 4 times within 30 days prior to enrollment or plan to use them more than 4 times per month during the study period.
* Participant has used doxycycline,tauroursodeoxycholate within 14 days prior to enrollment or plan to use them during the study period.
* Participant has sensory motor neuropathy caused by other causes, such as chronic inflammatory demyelinating polyradiculopathy, chronic idiopathic axonal neuropathy, diabetic neuropathy, chronic alcoholic neuropathy, paraneoplastic neuropathy,Guillain-Barre syndrome, vitamin B12 deficiency.
* Participant has received liver or any other organ except cornea transplantation.

Where it's running

• Qilu Hospital of Shandong University
  Jinan, China Recruiting

Study contacts

• Study coordinator: chuanzhu yan, Doctor
• Email: zzl2002@medmail.com
• Phone: 18560082288

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Acoramidis in adults with transthyretin amyloid cardiomyopathy in France

  This is an observational study to see how acoramidis is used in everyday care for adults with ATTR‑CM in France and how it affects their quality of life and daily function.

  Recruiting Bayer · Many Locations · NCT07791160

• Acoramidis for transthyretin amyloid cardiomyopathy in routine care

  See how acoramidis works for adults with transthyretin amyloid cardiomyopathy (wild-type or variant) when given as part of regular clinical care.

  Recruiting at 17 sites Bayer · Multiple Locations and 16 other locations · NCT07235462

• Coramitug (NNC6019-0001) for heart failure from transthyretin amyloid cardiomyopathy

  This study tests whether coramitug (NNC6019-0001) can lower the risk of heart-related death and illness in adults with transthyretin amyloid cardiomyopathy (ATTR-CM) while they continue their usual heart medicines.

  Recruiting at 237 sites Phase 3 Novo Nordisk A/S · Phoenix, Arizona and 298 other locations · NCT07207811

• Registry for studying Transthyretin Amyloid Cardiomyopathy in Thailand

  This study is collecting information from people with Transthyretin Amyloid Cardiomyopathy in Thailand to better understand the disease and improve early detection and treatment options.

  Recruiting Mahidol University · Bangkok and 7 other locations · NCT06338696

• Ocular Imaging to help detect ATTR-CM in adults with heart failure

  This multicenter pilot study tests whether eye imaging can help distinguish people with transthyretin amyloid cardiomyopathy from other heart failure patients.

  Recruiting AstraZeneca · Palo Alto, California and 19 other locations · NCT07690436

• Coramitug PET/CT imaging to track and affect heart amyloid in ATTR-CM

  This study tests whether a radioactive version of the antibody coramitug can be detected in and displaced from heart amyloid in people aged 60 and older with transthyretin amyloid cardiomyopathy, and whether the drug is safe and well tolerated.

  Recruiting Phase 1 Novo Nordisk A/S · Groningen · NCT07448623

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.