TTR-mediated AmyloidosisNCT02939820
Expanded Access Protocol of Patisiran for Patients With Hereditary ATTR Amyloidosis (hATTR)
A study testing patisiran (ALN-TTR02) in people with TTR-mediated Amyloidosis and Amyloidosis, Hereditary, run by Alnylam Pharmaceuticals.
Trial summary
The purpose of this study is to provide expanded access of patisiran to patients with hereditary transthyretin-mediated amyloidosis (hATTR). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- TTR-mediated Amyloidosis, Amyloidosis, Hereditary, Amyloid Neuropathies, Familial
- What is being tested?
- patisiran (ALN-TTR02)
- Type of treatment
- Injection or infusion
- How many people
- Not given
- Where
- No sites listed yet
- Study dates
- Start not givento not given
- Phase
- Expanded access
- 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
- Adult, Older adult
Who may be able to join
- Male or female greater than or equal to 18 years of age
- Have a diagnosis of hATTR
- Meet Karnofsky performance status and Polyneuropathy Disability (PND) score requirements
- Have adequate complete blood counts, liver function tests and coagulation tests
Who cannot join
- Participated in an interventional hATTR amyloidosis clinical trial involving RNA interference (RNAi) therapeutics within the last 12 months
- Are currently eligible to participate in or currently enrolled in an ongoing interventional hATTR amyloidosis clinical trial
- Have inadequate cardiac function
- Known primary amyloidosis (AL amyloidosis) or leptomeningeal amyloidosis
- Have known serious comorbidities or considered unfit for the program by the investigator
- Prior or planned liver or heart transplantation
About this study
Official title: Expanded Access Protocol of Patisiran for Patients With Hereditary Transthyretin-Mediated Amyloidosis (hATTR Amyloidosis) With Polyneuropathy
The purpose of this study is to provide expanded access of patisiran to patients with hereditary transthyretin-mediated amyloidosis (hATTR).
Read the detailed description
Choosing to participate in an expanded access program is an important personal decision. Talk with your doctor and family members or friends about deciding to join a research study. To learn more about this study, please have your doctor contact the study research staff using the Contacts provided. For general information, see the links provided in More Information.
- Conditions
- TTR-mediated Amyloidosis, Amyloidosis, Hereditary, Amyloid Neuropathies, Familial, Familial Amyloid Polyneuropathies, Amyloid Neuropathies, Amyloidosis, Hereditary, Transthyretin-Related
- Being tested
- patisiran (ALN-TTR02)
- Keywords
- RNAi therapeutic, FAP, Familial Amyloid Polyneuropathies, TTR, Transthyretin, Amyloidosis
- Medical subject headings
- Amyloidosis, Familial, Amyloid Neuropathies, Familial, Amyloid Neuropathies, Amyloidosis, Hereditary, Transthyretin-Related, Amyloidosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
patisiran (ALN-TTR02)Drug
patisiran (ALN-TTR02) administered by intravenous (IV) infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Oct 2016 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for TTR-mediated 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
- Expanded access
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
- NCT02939820
- Sponsor's protocol ID
- ALN-TTR02-007
- Status last verified
- May 2024
- First submitted
- 18 Oct 2016
- Last updated
- 20 May 2024
- Results posted
- No
Publications
Miah KM, Hyde SC, Gill DR. Emerging gene therapies for cystic fibrosis. Expert Rev Respir Med. 2019 Aug;13(8):709-725. doi: 10.1080/17476348.2019.1634547. Epub 2019 Jun 27.
PubMed 31215818
Study at a glance
From the registry record, last updated by the sponsor on 20 May 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Expanded Access Protocol of Patisiran for Patients With Hereditary Transthyretin-Mediated Amyloidosis (hATTR Amyloidosis) With Polyneuropathy
- ClinicalTrials.gov ID
- NCT02939820
- Acronym / sponsor ID
- ALN-TTR02-007
- Conditions
- TTR-mediated Amyloidosis, Amyloidosis, Hereditary, Amyloid Neuropathies, Familial, Familial Amyloid Polyneuropathies, Amyloid Neuropathies, Amyloidosis, Hereditary, Transthyretin-Related
- Keywords
- RNAi therapeutic, FAP, Familial Amyloid Polyneuropathies, TTR, Transthyretin, Amyloidosis
- Study type
- Expanded access
- Recruitment status
- Approved For Marketing
- Lead sponsor
- Alnylam PharmaceuticalsIndustry (pharma, biotech, devices)
- First posted
- 20 Oct 2016 (estimated)
- 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.
The purpose of this study is to provide expanded access of patisiran to patients with hereditary transthyretin-mediated amyloidosis (hATTR).
Detailed description
Choosing to participate in an expanded access program is an important personal decision. Talk with your doctor and family members or friends about deciding to join a research study. To learn more about this study, please have your doctor contact the study research staff using the Contacts provided. For general information, see the links provided in More Information.
Study design
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Male or female greater than or equal to 18 years of age
- Have a diagnosis of hATTR
- Meet Karnofsky performance status and Polyneuropathy Disability (PND) score requirements
- Have adequate complete blood counts, liver function tests and coagulation tests
Key exclusion criteria (6 listed)
- Participated in an interventional hATTR amyloidosis clinical trial involving RNA interference (RNAi) therapeutics within the last 12 months
- Are currently eligible to participate in or currently enrolled in an ongoing interventional hATTR amyloidosis clinical trial
- Have inadequate cardiac function
- Known primary amyloidosis (AL amyloidosis) or leptomeningeal amyloidosis
- Have known serious comorbidities or considered unfit for the program by the investigator
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 |
|---|---|---|
| patisiran (ALN-TTR02)patisiran (ALN-TTR02) administered by intravenous (IV) infusion | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 May 2024.
- Derived from this study: PubMed 31215818Miah KM, Hyde SC, Gill DR. Emerging gene therapies for cystic fibrosis. Expert Rev Respir Med. 2019 Aug;13(8):709-725. doi: 10.1080/17476348.2019.1634547. Epub 2019 Jun 27.
- https://ghr.nlm.nih.gov/condition/transthyretin-amyloidosisLink provided by the sponsor.
- https://www.nlm.nih.gov/medlineplus/amyloidosis.htmlLink 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.
Medical Director study director
Alnylam Pharmaceuticals
Questions about this trial
What is this clinical trial studying?
A study testing patisiran (ALN-TTR02) in people with TTR-mediated Amyloidosis and Amyloidosis, Hereditary, run by Alnylam Pharmaceuticals.
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 6 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: status not confirmed, as of the sponsor's last update on 20 May 2024. Status can change between updates, so confirm with the study contact before making plans.
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?
Results are not available in the source record.
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.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02939820, which also lists 1 linked publication.
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 20 May 2024.