Clinical trial · Observational
Monitoring of Early Disease Progression in Hereditary Transthyretin Amyloidosis
NCT03431896CI-TRIAL-00105372MED-hATTRcompletedClinicalTrials.gov clinicaltrialsProvenance
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
This study measures circulating, misfolded ATTR oligomers in asymptomatic ATTRm amyloidosis genetic carriers longitudinally over five years.
Conditions
Conditions (7)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| AL Amyloidosis | AL Amyloidosis | ONTOLOGY_EXACT | 0.98 |
| Amyloid | — | UNRESOLVED | — |
| Amyloid Cardiomyopathy | — | UNRESOLVED | — |
| Amyloid Neuropathies, Familial | — | UNRESOLVED | — |
| Amyloidosis | Amyloidosis | ONTOLOGY_EXACT | 0.98 |
| Amyloid - Primary | — | UNRESOLVED | — |
| Transthyretin Amyloidosis | — | UNRESOLVED | — |
Interventions
Interventions (0)
Data not yet available
No intervention recorded.
Design
Arms and outcomes
Arms (1)
- label
- Primary
- description
- 1.) To evaluate the relative amount of misfolded ATTR oligomers in asymptomatic ATTR amyloid genetic carriers and correlate their levels with clinical symptoms and outcomes. 1. Determine if misfolded ATTR oligomers are elevated compared to healthy control data obtained by Scripps during probe development 2. Describe the levels longitudinally 3. Determine if treatment with ATTR-specific medications (examples: diflunisal, doxycycline, ursodiol, tauroursodeoxycholic acid (TUDCA), green tea extract, curcumin, tafamidis, inotersen, patisiran) lead to reduction in the probe levels in those with elevated levels at baseline
Primary outcomes (1)
- measure
- Average % change in oligomers in patients with new onset TTR amyloid symptoms
- timeFrame
- Annually over 5 years
- description
- Change (%) for oligomer level at the time of TTR amyloid symptoms compared to baseline
Secondary outcomes (1)
- measure
- % change of oligomer levels relative to baseline level in patients with ATTR specific medication changes
- timeFrame
Eligibility
Eligibility (as posted)
- Sex
- All
Show eligibility criteria text
Inclusion Criteria: * Patients with known hereditary ATTR amyloidosis genetic mutations as identified by genetic testing. Exclusion Criteria: * Patients with ATTR amyloidosis identified as wild-type.
References
Publications (0)
Data not yet available
No reference posted for this study.