Amyloidosis, HereditaryNCT03400098
ATTR Expanded Access Program (EAP) by Ionis
A study testing Inotersen in people with Amyloidosis, Hereditary, run by Ionis Pharmaceuticals, Inc..
Trial summary
The purpose of this program is to provide expanded access to Inotersen for up to 100 Patients with Hereditary Transthyretin Amyloidosis (hTTR). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Amyloidosis, Hereditary
- What is being tested?
- Inotersen
- 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 at least 18 years of age with a diagnosis of hATTR
- Symptoms consistent with polyneuropathy
- Meet Polyneuropathy Disability (PND) Stage I-III requirements
Who cannot join
- Known Primary Amyloidosis, Leptomeningeal Amyloidosis or Monoclonal Gammopathy of Undetermined Significance or Multiple Myeloma
- Have inadequate cardiac function
- Have low platelet counts
- Have inadequate renal function
About this study
Official title: Expanded Access Program for Inotersen (ISIS 420915) in Patients With Hereditary Transthyretin Amyloidosis (hATTR)
The purpose of this program is to provide expanded access to Inotersen for up to 100 Patients with Hereditary Transthyretin Amyloidosis (hTTR).
Read the detailed description
The Program is intended to provided expanded access to Inotersen for eligible patients with hATTR who have limited or no available treatment options.
- Conditions
- Amyloidosis, Hereditary
- Being tested
- Inotersen
- Medical subject headings
- Amyloidosis, Familial
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
InotersenDrug
Inotersen administered by subcutaneous (SC) injections in the abdomen, thigh, or upper arm
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Jan 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Amyloidosis, HereditaryStudies of the same condition, open and under way.
- More trials run by Ionis Pharmaceuticals, Inc.The 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
- Ionis Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03400098
- Sponsor's protocol ID
- ISIS 420915-CS5
- Status last verified
- Aug 2019
- First submitted
- 8 Jan 2018
- Last updated
- 5 Aug 2019
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Aug 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Expanded Access Program for Inotersen (ISIS 420915) in Patients With Hereditary Transthyretin Amyloidosis (hATTR)
- ClinicalTrials.gov ID
- NCT03400098
- Acronym / sponsor ID
- ISIS 420915-CS5
- Conditions
- Amyloidosis, Hereditary
- Study type
- Expanded access
- Recruitment status
- Approved For Marketing
- Lead sponsor
- Ionis Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- First posted
- 17 Jan 2018 (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.
The purpose of this program is to provide expanded access to Inotersen for up to 100 Patients with Hereditary Transthyretin Amyloidosis (hTTR).
Detailed description
The Program is intended to provided expanded access to Inotersen for eligible patients with hATTR who have limited or no available treatment options.
Study design
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Male or female at least 18 years of age with a diagnosis of hATTR
- Symptoms consistent with polyneuropathy
- Meet Polyneuropathy Disability (PND) Stage I-III requirements
Key exclusion criteria (4 listed)
- Known Primary Amyloidosis, Leptomeningeal Amyloidosis or Monoclonal Gammopathy of Undetermined Significance or Multiple Myeloma
- Have inadequate cardiac function
- Have low platelet counts
- Have inadequate renal function
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 |
|---|---|---|
| InotersenInotersen administered by subcutaneous (SC) injections in the abdomen, thigh, or upper arm | 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 5 Aug 2019.
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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.
Questions about this trial
What is this clinical trial studying?
A study testing Inotersen in people with Amyloidosis, Hereditary, run by Ionis Pharmaceuticals, Inc..
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 3 inclusion and 4 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 5 Aug 2019. 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?
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 5 Aug 2019.