Trial · NCT05023889

Spectrum of Peripheral and Autonomic Neuropathies in Patients With aTTRwt Amyloidosis and Response to Patisiran Therapy

NCT05023889, Early phase 1, Completed, Austin Neuromuscular Center

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Spectrum of Peripheral and Autonomic Neuropathies in Patients With aTTRwt Amyloidosis and Response to Patisiran Therapy. Trials, record NCT05023889. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT05023889

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Spectrum of Peripheral and Autonomic Neuropathies in Patients With aTTRwt Amyloidosis and Response to Patisiran Therapy
NCT number: NCT05023889
Status: Completed
Phases: Early phase 1
Enrollment: 10 participants, actual
Lead sponsor: Austin Neuromuscular Center
Conditions: Polyneuropathies, Wild Type ATTR Amyloidosis, Wild-Type Transthyretin-Related (ATTR)Amyloidosis, Wild-Type Transthyretin Cardiac Amyloidosis, Transthyretin Amyloidosis
Interventions: patisiran
First posted: 2021-08-27
Last updated: 2026-05-22

Source ClinicalTrials.gov

Registered outcomes14

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Change in Neurological Impairment Score	baseline to 24 months	to assess the severity of functional impairment of motor and sensory nerves.NIS is a measure of motor strength, comprised of cranial nerve and both upper and lower limb motor assessments. The minimum and maximum values are 0 and 192, respectively. A higher score indicates a worse outcome.
Primary	Norfolk QOL-DN	baseline to 24 months	Change in Norfolk Quality of Life Questionnaire (Norfolk QOL-DN).The change from baseline in Norfolk QoL-DN at 24 months. The Norfolk QoL-DN questionnaire is a standardized 35-item patient-reported outcomes measure of diabetic neuropathy - small fiber, large fiber, and autonomic nerve function. The minimum and maximum values are -4 and 136, respectively. A higher score indicates a worse outcome.
Primary	COMPASS 31score	baseline to 24 months	Composite Autonomic Symptom Score (COMPASS) 31a self-assessment instrument for patient reported autonomic symptoms such as dizziness, constipation, diarrhea, nausea/vomiting, and incontinence. The minimum and maximum values are 0 and 100, respectively. A higher score indicates a worse outcome
Secondary	PND Polyneuropathy disability (PND) Score	baseline to 24 months	To compare and identify changes. Stage 0: no impairment Stage I: sensory disturbances but preserved walking capability Stage II: impaired walking capability but ability to walk without a stick or crutches Stage III a: walking only with the help of one stick or crutch Stage III b: walking with the help of two sticks or crutches. V: confined to wheelchair or bedridden A higher score indicates a worse outcome.
Secondary	Karnofsky, performance status score	baseline to 24 months	Karnofsky performance score is 11 level score which ranges between 0 (death) to 100 (no evidence of disease). Higher score means higher ability to perform daily tasks.
Secondary	EuroQOL	baseline to 24 months	EuroQOL,- A measurement tool that assesses generic quality of life in mobility, self-care, usual activities, pain/discomfort, anxiety/depression The minimum and maximum values are 0 and 100, respectively. A higher score indicates a worse outcome.
Secondary	EMG -Electromyography	baseline to 24 months	(EMG) is a diagnostic test that measures how the muscles and nerves work to evaluate peripheral Neuropathies pattern and progression
Secondary	Tilt Table Test	24 months	Evaluate and compare the response of blood pressure and heart rate changes in posture and position.
Secondary	Optional exploratory nerve and muscle biopsy	during screening visit ( 28 day window)	to identify amyloid deposits in skeletal muscle and peripheral nerve
Other	Cardiac MRI	start of study, and study end at 2 years	to identify cardiac abnormalities or changes and comparison

1 to 10 of 14Source ClinicalTrials.gov

See all 14 registered outcomes

Outcomes0

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

None in this release.

Results by group0

Each measurement the trial posted, group by group, in the words and numbers it posted.

None in this release.

Participant flow0

How many started, completed or left each group, period by period.

None in this release.

Adverse events0

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

None in this release.

Adverse event totals0

The totals the trial posted for each group.

None in this release.

Papers0

The papers the trial lists on ClinicalTrials.gov.

None in this release.

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons0

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

None in this release.

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/trials/v1/NCT05023889

GET https://conductscience.org/api/trials/v1/NCT05023889/registered

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