Patient Journey Mapping and validation of the AmyloScan® in a self-reported version. Online data collection to record the quality of life and functionality of people with ATTR amyloidosis (wild-type and hereditary ATTR amyloidosis) using comparison groups (CIDP and confirmed neuropathies of other origins).

Status

Active, not recruiting

Phases

Unknown

Study type

Observational

Source

DRKS

Registry ID

DRKS00034972

Enrollment

575

Registered

2024-09-12

Start date

2024-11-11

Completion date

Unknown

Last updated

2026-01-12

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

E85.1 E85.0 G60-G64 G61.8

Interventions

Group 1: 120 patients with hereditary transthyretin amyloidosis (hATTR) are to be surveyed online. Patients answer the online questionnaire once. The online questionnaire contains the following valida

Eligibility

Sex/Gender

All

Age

18 Years to No maximum

Inclusion criteria

Inclusion criteria: - Diagnosis of one of the following diseases with typical symptoms: => hATTR amyloidosis (symptomatic amyloidosis, genetically confirmed) => wtATTR amyloidosis (biopsy evidence of amyloidosis, exclusion of a genetic form) => CIDP (chronic inflammatory demyelinating polyneuropathy) (electrophysiologically and chemically confirmed) => Polyneuropathies of other genes (electrophysiologically confirmed, exclusion of the above diagnoses) -proficiency in the German language at a level sufficient for giving informed consent and completing the survey

Exclusion criteria

Exclusion criteria: - <18 years - None of the above diagnoses - Exclusively genetically proven ATTR amyloidosis without typical symptoms - Polyneuropathies of unknown origin - missing consent

Design outcomes

Primary

Measure	Time frame
The aim is to validate the sensitivity and specificity of the AmyloScan®-SR in a digital version without sensory tests. For this purpose, the sensitivity and specificity of possible limit values are determined. The central quality feature is the area under the receiver operating curve (ROC-AUC) and its confidence interval. This general approach is repeated for various sub-questions: 1. primary outcome measure: detection of ATTR (hATTR+wtATTR) versus neuropathy of other genesis (CIDP+other), lower confidence interval >0.5	—

Measure

Time frame

The aim is to validate the sensitivity and specificity of the AmyloScan®-SR in a digital version without sensory tests. For this purpose, the sensitivity and specificity of possible limit values are determined. The central quality feature is the area under the receiver operating curve (ROC-AUC) and its confidence interval. This general approach is repeated for various sub-questions: 1. primary outcome measure: detection of ATTR (hATTR+wtATTR) versus neuropathy of other genesis (CIDP+other), lower confidence interval >0.5

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Secondary

Measure	Time frame
2. secondary outcome 1: detection of ATTR (hATTR+wtATTR) versus CIDP, lower confidence interval >0.5 3. secondary outcome measure 2: detection of hATTR versus neuropathy of other origin (CIDP+other), lower confidence interval >0.5 4. secondary outcome 3: detection of wtATTR versus neuropathy of other etiology (CIDP+other), lower confidence interval >0.5 5. secondary outcome 4: detection of hATTR versus CIDP, lower confidence interval >0.5 6. secondary target 5: detection of wtATTR versus CIDP, lower confidence interval >0.5	—

Measure

Time frame

2. secondary outcome 1: detection of ATTR (hATTR+wtATTR) versus CIDP, lower confidence interval >0.5 3. secondary outcome measure 2: detection of hATTR versus neuropathy of other origin (CIDP+other), lower confidence interval >0.5 4. secondary outcome 3: detection of wtATTR versus neuropathy of other etiology (CIDP+other), lower confidence interval >0.5 5. secondary outcome 4: detection of hATTR versus CIDP, lower confidence interval >0.5 6. secondary target 5: detection of wtATTR versus CIDP, lower confidence interval >0.5

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Countries

Germany

Contacts

Public ContactReiner Lehmann

DontBePatient Intelligence GmbH

reiner.lehmann@dontbepatient.com+49 40 228683981

Outcome results

None listed

Source: DRKS (via WHO ICTRP) · Data processed: Feb 4, 2026