Hereditary Transthyretin-mediated (ATTRv) AmyloidosisNCT04201418
A Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of ATTRv Amyloidosis With a V122I or T60A Mutation
An observational study of Hereditary Transthyretin-mediated (ATTRv) Amyloidosis and Polyneuropathy, run by Alnylam Pharmaceuticals at 27 sites in United States.
Trial summary
To evaluate the effectiveness of patisiran in patients with ATTRv amyloidosis with polyneuropathy who have a V122I or T60A mutation. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Hereditary Transthyretin-mediated (ATTRv) Amyloidosis, Polyneuropathy
- What is being tested?
- Patisiran
- Type of treatment
- Injection or infusion
- How many people
- 67 participants
- Where
- 27 sitesUnited States
- Study dates
- 18 Dec 2019to 24 May 2022
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Participants with ATTRv amyloidosis with polyneuropathy who have a V122I or T60A mutation
Who may be able to join
- Diagnosed with ATTRv amyloidosis with polyneuropathy, with a documented V122I or T60A mutation
- PND score of I-IIIB at baseline.
- Exposure to commercial patisiran in one of the 3 cohorts:
- Prospective Cohort: Naive to patisiran treatment at the time of enrollment with intention to initiate treatment with patisiran.
- Mixed cohort: Currently on commercial patisiran therapy for less than 12 months at study enrollment.
- Retrospective cohort: Exposed to commercial patisiran treatment for at least 12 months prior to study enrollment, regardless of current treatment status at enrollment.
Who cannot join
- New York Heart Association (NYHA) heart failure classification ≥3
- Karnofsky Performance Status (KPS) <60%
- Unstable congestive heart failure (CHF)
- Known primary amyloidosis (AL) or leptomeningeal amyloidosis
- Prior major organ transplant
- Previously received patisiran
- Previous treatment with a TTR silencing therapy
Trial locations and contacts
27 sites in 1 country.
27 sites in 1 country
United States27 sites
Clinical Trial Site
Allentown, Pennsylvania, 18103
Clinical Trial Site
Aurora, Colorado, 80045
Clinical Trial Site
Austin, Texas, 78756
Clinical Trial Site
Bethlehem, Pennsylvania, 18015
Clinical Trial Site
Boston, Massachusetts, 02111
Clinical Trial Site
Braselton, Georgia, 30517
Clinical Trial Site
Chapel Hill, North Carolina, 27599
Clinical Trial Site
Charlotte, North Carolina, 28207
Clinical Trial Site
Chicago, Illinois, 60611
Clinical Trial Site
Chicago, Illinois, 60612
Clinical Trial Site
Columbus, Ohio, 43210
Clinical Trial Site
Detroit, Michigan, 48202
Clinical Trial Site
Farmington, Connecticut, 06030
Clinical Trial Site
Germantown, Tennessee, 38138
Clinical Trial Site
Jackson, Mississippi, 39216
Clinical Trial Site
Jacksonville, Florida, 32224
Clinical Trial Site
Kansas City, Missouri, 64111
Clinical Trial Site
Lancaster, Pennsylvania, 17602
Clinical Trial Site
Los Angeles, California, 90095
Clinical Trial Site
Macon, Georgia, 31201
Clinical Trial Site
Milwaukee, Wisconsin, 53226
Clinical Trial Site
Omaha, Nebraska, 68198
Clinical Trial Site
Pittsburgh, Pennsylvania, 15212
Clinical Trial Site
Pittsburgh, Pennsylvania, 15232
Clinical Trial Site
San Antonio, Texas, 78229
Clinical Trial Site
San Diego, California, 92103
Clinical Trial Site
Tampa, Florida, 33609
About this study
Official title: A Phase 4 Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of Hereditary Transthyretin-Mediated (ATTRv) Amyloidosis With a V122I or T60A Mutation
To evaluate the effectiveness of patisiran in patients with ATTRv amyloidosis with polyneuropathy who have a V122I or T60A mutation.
- Being tested
- Patisiran
- Keywords
- Valine to isoleucine substitution at position 122, V122I, Threonine to alanine substitution at position 60, T60A, Familial Amyloid Polyneuropathies, ATTR, Transthyretin amyloidosis, Transthyretin, Amyloidosis, TTR-mediated Amyloidosis, RNAi therapeutic, FAP, Polyneuropathies, Amyloid neuropathies, Amyloid neuropathies, familial, Amyloidosis, familial, Peripheral nervous system diseases, Nervous system diseases, TTR, Neuromuscular diseases
- Medical subject headings
- Amyloidosis, Polyneuropathies, Amyloid Neuropathies, Familial, Amyloidosis, Hereditary, Transthyretin-Related, Amyloid Neuropathies, Amyloidosis, Familial, Peripheral Nervous System Diseases, Nervous System Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PatisiranDrug
Also called ONPATTRO, ALN-TTR02
Patisiran-lipid complex injection, for intravenous use
| Study group | Role | What they receive |
|---|---|---|
| Patisiran Prospective Cohort | Patients who are naive to patisiran at study enrollment with the intention to initiate commercial patisiran therapy. Receives: Drug: Patisiran | |
| Patisiran Mixed Cohort | Patients who are currently on commercial patisiran therapy for less than 12 months at study enrollment. Receives: Drug: Patisiran | |
| Patisiran Retrospective Cohort | Patients who have been on commercial patisiran therapy for at least 12 months prior to study enrollment, regardless of current treatment status at enrollment. Receives: Drug: Patisiran |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of Participants with Stable or Improved Polyneuropathy Disability (PND) Score at 12 Months Relative to Baseline
PND Scores: Stage 0=No symptoms, Stage 1=Sensory disturbances but preserved walking capability, Stage 2=Impaired walking capacity, but ability to walk without a stick or crutches, Stage 3A/B=Walking with the help of 1 or 2 sticks or crutches, Stage 4=confined to wheel chair or bedridden.
Measured: Baseline, Month 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Dec 2019
- Study start18 Dec 2019
- Primary completion24 May 2022
- Study completion24 May 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hereditary Transthyretin-mediated (ATTRv) AmyloidosisStudies of the same condition, open and under way.
- More trials run by Alnylam PharmaceuticalsThe 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
- Prospective
- Enrolment
- 67 participants (actual)
Sponsor and investigators
- Lead sponsor
- Alnylam Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Alnylam Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04201418
- Sponsor's protocol ID
- ALN-TTR02-012
- Status last verified
- Jun 2022
- First submitted
- 13 Dec 2019
- Last updated
- 6 Jun 2022
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Jun 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 4 Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of Hereditary Transthyretin-Mediated (ATTRv) Amyloidosis With a V122I or T60A Mutation
- ClinicalTrials.gov ID
- NCT04201418
- Acronym / sponsor ID
- ALN-TTR02-012
- Keywords
- Valine to isoleucine substitution at position 122, V122I, Threonine to alanine substitution at position 60, T60A, Familial Amyloid Polyneuropathies, ATTR, Transthyretin amyloidosis, Transthyretin
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 67 participants (actual)
- Lead sponsor
- Alnylam PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 18 Dec 2019 (actual)
- Primary completion
- 24 May 2022 (actual)
- Study completion
- 24 May 2022 (actual)
- First posted
- 17 Dec 2019 (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.
To evaluate the effectiveness of patisiran in patients with ATTRv amyloidosis with polyneuropathy who have a V122I or T60A mutation.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Patisiran Prospective Cohort | – | Drug: PatisiranPatients who are naive to patisiran at study enrollment with the intention to initiate commercial patisiran therapy. |
| Patisiran Mixed Cohort | – | Drug: PatisiranPatients who are currently on commercial patisiran therapy for less than 12 months at study enrollment. |
| Patisiran Retrospective Cohort | – | Drug: PatisiranPatients who have been on commercial patisiran therapy for at least 12 months prior to study enrollment, regardless of current treatment status at enrollment. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Participants with ATTRv amyloidosis with polyneuropathy who have a V122I or T60A mutation
Key inclusion criteria (6 listed)
- Diagnosed with ATTRv amyloidosis with polyneuropathy, with a documented V122I or T60A mutation
- PND score of I-IIIB at baseline.
- Exposure to commercial patisiran in one of the 3 cohorts:
Key exclusion criteria (7 listed)
- New York Heart Association (NYHA) heart failure classification ≥3
- Karnofsky Performance Status (KPS) <60%
- Unstable congestive heart failure (CHF)
- Known primary amyloidosis (AL) or leptomeningeal amyloidosis
- Prior major organ transplant
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PatisiranAlso: ONPATTRO, ALN-TTR02Patisiran-lipid complex injection, for intravenous use | Drug | Patisiran Mixed Cohort, Patisiran Prospective Cohort, Patisiran Retrospective Cohort |
Primary outcome
Percentage of Participants with Stable or Improved Polyneuropathy Disability (PND) Score at 12 Months Relative to Baseline
Time frame: Baseline, Month 12
PND Scores: Stage 0=No symptoms, Stage 1=Sensory disturbances but preserved walking capability, Stage 2=Impaired walking capacity, but ability to walk without a stick or crutches, Stage 3A/B=Walking with the help of 1 or 2 sticks or crutches, Stage 4=confined to wheel chair or bedridden.
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 6 Jun 2022.
- Individual participant dataSharing planned: No
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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.
Medical Director study director
Alnylam Pharmaceuticals
27 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-mediated (ATTRv) Amyloidosis and Polyneuropathy, run by Alnylam Pharmaceuticals at 27 sites in United States.
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 6 inclusion and 7 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 6 Jun 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 27 sites in United States. 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?
Percentage of Participants with Stable or Improved Polyneuropathy Disability (PND) Score at 12 Months Relative to Baseline (time frame: Baseline, Month 12). 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 6 Jun 2022.