Hereditary Transthyretin AmyloidosisPRIMERNCT06365593
hATTR Polyneuropathy in Russia
An observational study of Hereditary Transthyretin Amyloidosis, run by AstraZeneca at 3 sites in Russia.
Trial summary
A multicenter observational retrosPective Registry of patIents with transthyretin aMyloid polynEuropathy (hATTR-PN) and chRonic idiopathic axonal polyneuropathy (CIAP) in the population of the Russian Federation (PRIMER) There are no comprehensive epidemiological data on patients with hereditary ATTR-PN (hATTR-PN) and… Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Hereditary Transthyretin Amyloidosis
- How many people
- 100 participants
- Where
- 3 sitesRussia
- Study dates
- 29 Dec 2023to 30 May 2024
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: This non-interventional multicenter observational study will include 50 adult patients with hATTR-PN and 50 adult patients with CIAP in 8 clinical sites in the Russian Federation. The diagnosis should be established in patients' source medical documentation.
Who may be able to join
- Patients with hATTR-PN or CIAP ;
- The diagnosis of hATTR-PN or CIAP was established between the 1st January 2017 and the 1st March 2024;
- Presence of at least 1 month between the date of hATTR-PN or CIAP diagnosis and the date of inclusion;
- Age ≥ 18 years at the time of hATTR-PN or CIAP diagnosis.
Who cannot join
- The participation in any clinical trial with investigational product from the moment of diagnosis of ATTR PN or CIAP until the end of retrospective follow-up period.
Trial locations and contacts
3 sites in 1 country.
Russia3 sites
Research Site
Kazan'
Research Site
Moscow
Research Site
Saint Petersburg, 194354
About this study
Official title: A Multicenter Observational retrosPective Registry of patIents With Transthyretin aMyloid polynEuropathy (hATTR-PN) and chRonic Idiopathic Axonal Polyneuropathy (CIAP) in the Population of the Russian Federation
A multicenter observational retrosPective Registry of patIents with transthyretin aMyloid polynEuropathy (hATTR-PN) and chRonic idiopathic axonal polyneuropathy (CIAP) in the population of the Russian Federation (PRIMER) There are no comprehensive epidemiological data on patients with hereditary ATTR-PN (hATTR-PN) and CIAP in the Russian Federation. Therefore, there is a need to conduct a large-scale observational study in the Russian population to obtain information on clinical, electrophysiological and demographic characteristics of patients with hATTR-PN and CIAP. Obtaining the study data will help to identify the patients with axonal polyneuropathy, who deserve TTR gene sequencing, and therefore to allow early treatment and potentially modify disease progression in patients.
Read the detailed description
Since the increasing number of treatments available to help slow the progression of neuropathy it is critical to timely identify and diagnose the ATTR-PN. Early identification and intervention are also crucial to improve patient outcomes because newly available treatments have been shown to have maximum therapeutic benefit when started in the early stages of the disease.
Identification of ATTRv amyloidosis with PN can be challenging, particularly in non-endemic regions, and a high level of suspicion is required to diagnose patients as early as possible.
Previously proposed suspicion index of ATTRv amyloidosis was based on disease's red flags, that is, on the presence of a progressive polyneuropathy in addition to at least one red flag symptom suggestive of multi-systemic involvement. However, sometimes the demonstration of a progressive neuropathy requires follow-up evaluations, risking wasting time. Moreover, some red flags (e.g., cardiomyopathy or vitreous opacities) need specialist evaluations that could be often lacking during the first neurological evaluation.
Patients can present with heterogeneous symptoms and variable levels of disease severity, which often leads to a misdiagnosis. Early and accurate diagnosis may also be confounded by a lack of family history and the presence of various phenotypes common to multiple disease conditions such as GI disorders. In fact, CIAP still represents a common misdiagnosis for ATTRv patients.
Study is planned to determine the principal differences between the hATTR-PN and CIAP patients and valorize them in a compound score which can help clinician through a specific cut-off to recognize patients deserving TTR genetic analysis. The application of the compound score in patients with sensory-motor neuropathy may have a major role, representing a screening tool to avoiding wasting time and therefore shortening the time to reach a correct diagnosis.
Previously reported compound score included presence of muscle weakness and CTS history as clinical parameters and amplitude of Sensory action potential (SAP), Compound muscular action potential (CMAP) of the median and ulnar nerves, CMAP of the tibial nerve as electrophysiological parameters. Electrophysiological findings in this study showed that ATTRv patients, although they had the same disease duration of CIAP patients, had a greater reduction of amplitude of potentials in all nerves with a more frequently absence of potential at lower limbs and reduction at upper limbs.
At the same time, the landscape of mutations and phenotypes of ATTR amyloidosis are very different between countries, which does not allow extrapolating the results from Italian study, and there are no comprehensive epidemiological data on patients with hATTR-PN and CIAP in the Russian Federation.
Therefore, there is a need to conduct a large-scale observational study in the Russian population to obtain information on clinical, electrophysiological and demographic characteristics of patients with hATTR-PN and CIAP. Obtaining the study data will help to identify the patients with axonal polyneuropathy, who deserve TTR gene sequencing, and therefore to allow early treatment and potentially modify disease progression in patients.
- Conditions
- Hereditary Transthyretin Amyloidosis
- Keywords
- hATTR, CIAP, hATTR-PN, ATTR-PN, ATTRv, ATTR, polyneuropathy, transthyretin amyloid polyneuropathy, chronic idiopathic axonal polyneuropathy, hereditary transthyretin amyloidosis
- Medical subject headings
- Amyloidosis, Hereditary, Transthyretin-Related, Polyneuropathies, Amyloid Neuropathies, Familial
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Patients with hATTR-PN | 50 adult patients with hATTR-PN | |
| Patients with CIAP | 50 adult patients with CIAP |
What is being measured
The primary outcome is the main question the study is designed to answer.
In order to describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIAP in the Russian Federation, Mean age at the hATTR-PN or CIAP diagnosis information will be collected
Measured: up to 5 months
In order to describe the baseline demographic characteristics of patients with hATTR-PN and CIAP in the Russian Federation, information on Proportion of women and men will be collected
Measured: up to 5 months
In order to describe the baseline demographic characteristics of patients with hATTR-PN and CIAP in the Russian Federation, information on Proportion of patients with each race and ethnicity will be collected
Measured: up to 5 months
to describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIA, information on Proportion of patients from each federal district of the Russian Federation will be collected
Measured: up to 5 months
in order to describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIAP in the Russian Federation, tha following Anthropometric characteristics will be collected:
Mean height (in m and cm), body weight (in kg), BMI (kg/m2)
Measured: up to 5 months
In order to describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIAP in the Russian Federation, Proportion of patients with underweight will be collected
BMI <18,5 kg/m2
Measured: up to 5 months
in order to describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIAP in the Russian Federation, information on Proportion of patients with negative lifestyle factors will be collected:
- Proportion of patients with a history of smoking;
- Proportion of patients with a history of alcohol abuse
Measured: up to 5 months
in order to describe the baseline demographic characteristics of patients with hATTR-PN and CIAP in the Russian Federation, information on Proportion of patients with positive family history in close relatives will be collected
Measured: up to 5 months
to describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIAP, information on Mean (or median) time from the first symptoms of neuropathy to the diagnosis of PN will be collected
Measured: up to 5 months
to describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIAP, Proportion of patients with different firstly suspected etiology of PN will be calculated
- Diabetic;
- Alcoholic;
- Toxic (toxic agents other than alcohol, e.g. drugs or chemotherapy);
- Other Hereditary;
- Deficiency (due to vitamin B12, thiamine or pyridoxine deficiency);
- Immunological (due to rheumatological, paraneoplastic or celiac disease);
- Haematological (paraproteinemic syndromes);
- Infective;
- Idiopathic;
- Other;
Measured: up to 5 months
To describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIAP, information on Mean (or median) time from first PN manifestation/diagnosis to the hATTR-PN or CIAP diagnosis will be collected
Measured: up to 5 months
To describe the baseline electrophysiological and clinical characteristics of patients, Proportion of patients with chronic sensory or sensorimotor polyneuropathy and progressive chronic polyneuropathy among patients with CIAP will be calculated
Measured: up to 5 months
To describe the baseline clinical characteristics of patients with hATTR-PN and CIAP, Proportion of patients with each result of TTR gene testing will be calculated
(positive, negative, not performed); the proportion of patients with each identified mutation in TTR gene in case of positive result
Measured: up to 5 months
To describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIAP in the Russian Federation, Proportion of patients with each clinical manifestation will be calculated:
- Sensory symptoms;
- Motor symptoms;
- CTS (proportion of patients with any CTS in total, as well as proportion of patients with uni- and bilateral CTS);
- Biceps tendon rupture;
- Autonomic symptoms;
- Spinal channel stenosis of the lumbar region;
- GI symptoms;
- Ocular manifestations;
- Cardiac manifestations;
- Renal manifestations;
- CNS involvement.
Measured: up to 5 months
To describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIAP in the Russian Federation, Proportion of patients with each PND score will be calculated
Measured: up to 5 months
To describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIAP in the Russian Federation, Proportion of patients with each score by each parameter of neurological examination will be calculated
Measured: up to 5 months
To describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIAP in the Russian Federation, information on Mean point by MRS will be calculated
Measured: up to 5 months
To describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIAP in the Russian Federation, information on Mean point by INCAT scale (total score, arm disability, leg disability) will be collected
Measured: up to 5 months
To describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIAP in the Russian Federation, information on Mean left ventricular ejection fraction (LVEF) will be collected
Measured: up to 5 months
To describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIAP in the Russian Federation, information Mean wall thickness of the left ventricular (LV) will be collected
Measured: up to 5 months
To describe the baseline electrophysiological characteristics of patients with hATTR-PN and CIAP, information Mean variables based on the results of electrophysiological investigation, including the following (on both extremities) will be collected
Compound muscular action potential (CMAP) (mV) of the median, ulnar, tibial and peroneal nerves; (b) (Distal motor latency) DML (ms) of the median, ulnar, tibial and peroneal nerves; (c) (Motor nerve conduction velocity) MNCV (m/s) of the median, ulnar, tibial and peroneal nerves; (d) Sensory action potential (SAP) (μV) of the median, ulnar, peroneal superficial and sural nerves; (e) Sensory nerve conduction velocity (SNCV) (m/s) of the median, ulnar, peroneal superficial and sural nerves
Measured: up to 5 months
To describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIAP in the Russian Federation, Proportion of patients with diagnosis of HFpEF will be calculated
Measured: up to 5 months
To describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIAP in the Russian Federation, information on Mean NT-proBNP level will be collected
Measured: up to 5 months
To describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIAP in the Russian Federation, information Mean estimated glomerular filtration rate (eGFR) will be collected
based on CKD-EPI (Chronic Kidney Disease Epidemiology Collaboration) formula
Measured: up to 5 months
To describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIAP in the Russian Federation, information on Mean urine albumin-to-creatinine ratio (UACR) in a spot urine sample will be collected
Measured: up to 5 months
To describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIAP in the Russian Federation, Proportion of patients with a positive paraprotein blood test result will be calculated
Measured: up to 5 months
To describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIAP in the Russian Federation, information on Mean blood pressure (BP) (systolic and diastolic) and Mean heart rate (HR)
Measured: up to 5 months
To describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIAP in the Russian Federation, Proportion of patients with each concomitant disease will be calculated
Measured: up to 5 months
To describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIAP in the Russian Federation, Proportion of patients underwent surgery for CTS to the moment of diagnosis will be calculated
Measured: up to 5 months
Secondary outcomes (1)
To arrange a score to select patients with axonal neuropathy who deserve screening for TTR mutation.
In order to achieve secondary objective all above baseline demographic, electrophysiological and clinical characteristics will be compared between groups (hATTR-PN and CIAP arm). The score will be arranged based on the parameters by which a significant difference will be determined based on the results of the comparison.
Measured: Up to 5 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Apr 2024
- Study start29 Dec 2023
- Primary completion30 May 2024
- Study completion30 May 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hereditary Transthyretin AmyloidosisStudies of the same condition, open and under way.
- More trials run by AstraZenecaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 100 participants (actual)
Sponsor and investigators
- Lead sponsor
- AstraZeneca
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06365593
- Sponsor's protocol ID
- D8450R00005
- Acronym
- PRIMER
- Status last verified
- May 2025
- First submitted
- 1 Mar 2024
- Last updated
- 25 May 2025
- Results posted
- No
- FDA-regulated drug
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 May 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter Observational retrosPective Registry of patIents With Transthyretin aMyloid polynEuropathy (hATTR-PN) and chRonic Idiopathic Axonal Polyneuropathy (CIAP) in the Population of the Russian Federation
- ClinicalTrials.gov ID
- NCT06365593
- Acronym / sponsor ID
- PRIMER / D8450R00005
- Conditions
- Hereditary Transthyretin Amyloidosis
- Keywords
- hATTR, CIAP, hATTR-PN, ATTR-PN, ATTRv, ATTR, polyneuropathy, transthyretin amyloid polyneuropathy
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 100 participants (actual)
- Lead sponsor
- AstraZenecaIndustry (pharma, biotech, devices)
- Study start
- 29 Dec 2023 (actual)
- Primary completion
- 30 May 2024 (actual)
- Study completion
- 30 May 2024 (actual)
- First posted
- 15 Apr 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
A multicenter observational retrosPective Registry of patIents with transthyretin aMyloid polynEuropathy (hATTR-PN) and chRonic idiopathic axonal polyneuropathy (CIAP) in the population of the Russian Federation (PRIMER) There are no comprehensive epidemiological data on patients with hereditary ATTR-PN (hATTR-PN) and CIAP in the Russian Federation. Therefore, there is a need to conduct a large-scale observational study in the Russian population to obtain information on clinical, electrophysiological and demographic characteristics of patients with hATTR-PN and CIAP. Obtaining the study data will help to identify the patients with axonal polyneuropathy, who deserve TTR gene sequencing, and therefore to allow early treatment and potentially modify disease progression in patients.
Detailed description
Since the increasing number of treatments available to help slow the progression of neuropathy it is critical to timely identify and diagnose the ATTR-PN. Early identification and intervention are also crucial to improve patient outcomes because newly available treatments have been shown to have maximum therapeutic benefit when started in the early stages of the disease.
Identification of ATTRv amyloidosis with PN can be challenging, particularly in non-endemic regions, and a high level of suspicion is required to diagnose patients as early as possible.
Previously proposed suspicion index of ATTRv amyloidosis was based on disease's red flags, that is, on the presence of a progressive polyneuropathy in addition to at least one red flag symptom suggestive of multi-systemic involvement. However, sometimes the demonstration of a progressive neuropathy requires follow-up evaluations, risking wasting time. Moreover, some red flags (e.g., cardiomyopathy or vitreous opacities) need specialist evaluations that could be often lacking during the first neurological evaluation.
Patients can present with heterogeneous symptoms and variable levels of disease severity, which often leads to a misdiagnosis. Early and accurate diagnosis may also be confounded by a lack of family history and the presence of various phenotypes common to multiple disease conditions such as GI disorders. In fact, CIAP still represents a common misdiagnosis for ATTRv patients.
Study is planned to determine the principal differences between the hATTR-PN and CIAP patients and valorize them in a compound score which can help clinician through a specific cut-off to recognize patients deserving TTR genetic analysis. The application of the compound score in patients with sensory-motor neuropathy may have a major role, representing a screening tool to avoiding wasting time and therefore shortening the time to reach a correct diagnosis.
Previously reported compound score included presence of muscle weakness and CTS history as clinical parameters and amplitude of Sensory action potential (SAP), Compound muscular action potential (CMAP) of the median and ulnar nerves, CMAP of the tibial nerve as electrophysiological parameters. Electrophysiological findings in this study showed that ATTRv patients, although they had the same disease duration of CIAP patients, had a greater reduction of amplitude of potentials in all nerves with a more frequently absence of potential at lower limbs and reduction at upper limbs.
At the same time, the landscape of mutations and phenotypes of ATTR amyloidosis are very different between countries, which does not allow extrapolating the results from Italian study, and there are no comprehensive epidemiological data on patients with hATTR-PN and CIAP in the Russian Federation.
Therefore, there is a need to conduct a large-scale observational study in the Russian population to obtain information on clinical, electrophysiological and demographic characteristics of patients with hATTR-PN and CIAP. Obtaining the study data will help to identify the patients with axonal polyneuropathy, who deserve TTR gene sequencing, and therefore to allow early treatment and potentially modify disease progression in patients.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Patients with hATTR-PN | – | –50 adult patients with hATTR-PN |
| Patients with CIAP | – | –50 adult patients with CIAP |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- This non-interventional multicenter observational study will include 50 adult patients with hATTR-PN and 50 adult patients with CIAP in 8 clinical sites in the Russian Federation. The diagnosis should be established in patients' source medical documentation.
Key inclusion criteria (4 listed)
- Patients with hATTR-PN or CIAP ;
- The diagnosis of hATTR-PN or CIAP was established between the 1st January 2017 and the 1st March 2024;
- Presence of at least 1 month between the date of hATTR-PN or CIAP diagnosis and the date of inclusion;
- Age ≥ 18 years at the time of hATTR-PN or CIAP diagnosis.
Key exclusion criteria (1 listed)
- The participation in any clinical trial with investigational product from the moment of diagnosis of ATTR PN or CIAP until the end of retrospective follow-up period.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
In order to describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIAP in the Russian Federation, Mean age at the hATTR-PN or CIAP diagnosis information will be collected
Time frame: up to 5 months
In order to describe the baseline demographic characteristics of patients with hATTR-PN and CIAP in the Russian Federation, information on Proportion of women and men will be collected
Time frame: up to 5 months
In order to describe the baseline demographic characteristics of patients with hATTR-PN and CIAP in the Russian Federation, information on Proportion of patients with each race and ethnicity will be collected
Time frame: up to 5 months
to describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIA, information on Proportion of patients from each federal district of the Russian Federation will be collected
Time frame: up to 5 months
in order to describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIAP in the Russian Federation, tha following Anthropometric characteristics will be collected:
Time frame: up to 5 months
Mean height (in m and cm), body weight (in kg), BMI (kg/m2)
In order to describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIAP in the Russian Federation, Proportion of patients with underweight will be collected
Time frame: up to 5 months
BMI <18,5 kg/m2
in order to describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIAP in the Russian Federation, information on Proportion of patients with negative lifestyle factors will be collected:
Time frame: up to 5 months
1. Proportion of patients with a history of smoking; 2. Proportion of patients with a history of alcohol abuse
in order to describe the baseline demographic characteristics of patients with hATTR-PN and CIAP in the Russian Federation, information on Proportion of patients with positive family history in close relatives will be collected
Time frame: up to 5 months
to describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIAP, information on Mean (or median) time from the first symptoms of neuropathy to the diagnosis of PN will be collected
Time frame: up to 5 months
to describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIAP, Proportion of patients with different firstly suspected etiology of PN will be calculated
Time frame: up to 5 months
1. Diabetic; 2. Alcoholic; 3. Toxic (toxic agents other than alcohol, e.g. drugs or chemotherapy); 4. Other Hereditary; 5. Deficiency (due to vitamin B12, thiamine or pyridoxine deficiency); 6. Immunological (due to rheumatological, paraneoplastic or celiac disease); 7. Haematological (paraproteinemic syndromes); 8. Infective; 9. Idiopathic; 10. Other;
To describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIAP, information on Mean (or median) time from first PN manifestation/diagnosis to the hATTR-PN or CIAP diagnosis will be collected
Time frame: up to 5 months
To describe the baseline electrophysiological and clinical characteristics of patients, Proportion of patients with chronic sensory or sensorimotor polyneuropathy and progressive chronic polyneuropathy among patients with CIAP will be calculated
Time frame: up to 5 months
To describe the baseline clinical characteristics of patients with hATTR-PN and CIAP, Proportion of patients with each result of TTR gene testing will be calculated
Time frame: up to 5 months
(positive, negative, not performed); the proportion of patients with each identified mutation in TTR gene in case of positive result
To describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIAP in the Russian Federation, Proportion of patients with each clinical manifestation will be calculated:
Time frame: up to 5 months
1. Sensory symptoms; 2. Motor symptoms; 3. CTS (proportion of patients with any CTS in total, as well as proportion of patients with uni- and bilateral CTS); 4. Biceps tendon rupture; 5. Autonomic symptoms; 6. Spinal channel stenosis of the lumbar region; 7. GI symptoms; 8. Ocular manifestations; 9. Cardiac manifestations; 10. Renal manifestations; 11. CNS involvement.
To describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIAP in the Russian Federation, Proportion of patients with each PND score will be calculated
Time frame: up to 5 months
To describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIAP in the Russian Federation, Proportion of patients with each score by each parameter of neurological examination will be calculated
Time frame: up to 5 months
To describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIAP in the Russian Federation, information on Mean point by MRS will be calculated
Time frame: up to 5 months
To describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIAP in the Russian Federation, information on Mean point by INCAT scale (total score, arm disability, leg disability) will be collected
Time frame: up to 5 months
To describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIAP in the Russian Federation, information on Mean left ventricular ejection fraction (LVEF) will be collected
Time frame: up to 5 months
To describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIAP in the Russian Federation, information Mean wall thickness of the left ventricular (LV) will be collected
Time frame: up to 5 months
To describe the baseline electrophysiological characteristics of patients with hATTR-PN and CIAP, information Mean variables based on the results of electrophysiological investigation, including the following (on both extremities) will be collected
Time frame: up to 5 months
Compound muscular action potential (CMAP) (mV) of the median, ulnar, tibial and peroneal nerves; (b) (Distal motor latency) DML (ms) of the median, ulnar, tibial and peroneal nerves; (c) (Motor nerve conduction velocity) MNCV (m/s) of the median, ulnar, tibial and peroneal nerves; (d) Sensory action potential (SAP) (μV) of the median, ulnar, peroneal superficial and sural nerves; (e) Sensory nerve conduction velocity (SNCV) (m/s) of the median, ulnar, peroneal superficial and sural nerves
To describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIAP in the Russian Federation, Proportion of patients with diagnosis of HFpEF will be calculated
Time frame: up to 5 months
To describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIAP in the Russian Federation, information on Mean NT-proBNP level will be collected
Time frame: up to 5 months
To describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIAP in the Russian Federation, information Mean estimated glomerular filtration rate (eGFR) will be collected
Time frame: up to 5 months
based on CKD-EPI (Chronic Kidney Disease Epidemiology Collaboration) formula
To describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIAP in the Russian Federation, information on Mean urine albumin-to-creatinine ratio (UACR) in a spot urine sample will be collected
Time frame: up to 5 months
To describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIAP in the Russian Federation, Proportion of patients with a positive paraprotein blood test result will be calculated
Time frame: up to 5 months
To describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIAP in the Russian Federation, information on Mean blood pressure (BP) (systolic and diastolic) and Mean heart rate (HR)
Time frame: up to 5 months
To describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIAP in the Russian Federation, Proportion of patients with each concomitant disease will be calculated
Time frame: up to 5 months
To describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIAP in the Russian Federation, Proportion of patients underwent surgery for CTS to the moment of diagnosis will be calculated
Time frame: up to 5 months
1 secondary outcome
To arrange a score to select patients with axonal neuropathy who deserve screening for TTR mutation.
Time frame: Up to 5 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 May 2025.
- D8450R00005 Clinical Study Report (CSR) Synopsis\_RedactedLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hereditary Transthyretin Amyloidosis, run by AstraZeneca at 3 sites in Russia.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 25 May 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in Russia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
In order to describe the baseline electrophysiological, clinical and demographic characteristics of patients with hATTR-PN and CIAP in the Russian Federation, Mean age at the hATTR-PN or CIAP diagnosis information will be collected (time frame: up to 5 months). The study lists 29 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 May 2025.