Primary HyperoxaluriaNCT04125472
Expanded Access Protocol to Provide Lumasiran to Patients With Primary Hyperoxaluria Type 1
A study testing Lumasiran in people with Primary Hyperoxaluria, run by Alnylam Pharmaceuticals.
Trial summary
The purpose of this study is to provide expanded access to lumasiran for adults and pediatric patients with Primary Hyperoxaluria Type 1 (PH1), Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Primary Hyperoxaluria
- What is being tested?
- Lumasiran
- 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 Any age
- All sexes
- Child, Adult, Older adult
Who may be able to join
- Documented diagnosis of Primary Hyperoxaluria Type 1
Who cannot join
- Clinically significant health concerns (with the exception of PH1)
- Received an investigational agent within 30 days before the first dose of lumasiran or are in follow-up of another clinical study
- Previously or currently participating in lumasiran clinical study
- History of liver transplant
About this study
The purpose of this study is to provide expanded access to lumasiran for adults and pediatric patients with Primary Hyperoxaluria Type 1 (PH1),
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 link provided in More Information.
- Conditions
- Primary Hyperoxaluria
- Being tested
- Lumasiran
- Keywords
- Hyperoxaluria, Primary Hyperoxaluria, Kidney Diseases, Urologic Diseases, Metabolism, Inborn Errors, Genetic Diseases, Inborn, Metabolic Diseases, RNA Therapeutic, siRNA
- Medical subject headings
- Hyperoxaluria, Primary, Hyperoxaluria, Kidney Diseases, Urologic Diseases, Metabolism, Inborn Errors, Genetic Diseases, Inborn, Metabolic Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LumasiranDrug
Also called ALN-GO1
Lumasiran administered as a subcutaneous (SC) injection
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Oct 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Primary HyperoxaluriaStudies 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
- NCT04125472
- Sponsor's protocol ID
- ALN-GO1-006
- Status last verified
- Feb 2024
- First submitted
- 10 Oct 2019
- Last updated
- 16 Feb 2024
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Feb 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Expanded Access Protocol to Provide Lumasiran to Patients With Primary Hyperoxaluria Type 1
- ClinicalTrials.gov ID
- NCT04125472
- Acronym / sponsor ID
- ALN-GO1-006
- Conditions
- Primary Hyperoxaluria
- Keywords
- Hyperoxaluria, Primary Hyperoxaluria, Kidney Diseases, Urologic Diseases, Metabolism, Inborn Errors, Genetic Diseases, Inborn, Metabolic Diseases, RNA Therapeutic
- Study type
- Expanded access
- Recruitment status
- Approved For Marketing
- Lead sponsor
- Alnylam PharmaceuticalsIndustry (pharma, biotech, devices)
- First posted
- 14 Oct 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.
The purpose of this study is to provide expanded access to lumasiran for adults and pediatric patients with Primary Hyperoxaluria Type 1 (PH1),
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 link provided in More Information.
Study design
Who may qualify
- Sex
- All
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (1 listed)
- Documented diagnosis of Primary Hyperoxaluria Type 1
Key exclusion criteria (4 listed)
- Clinically significant health concerns (with the exception of PH1)
- Received an investigational agent within 30 days before the first dose of lumasiran or are in follow-up of another clinical study
- Previously or currently participating in lumasiran clinical study
- History of liver 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 |
|---|---|---|
| LumasiranAlso: ALN-GO1Lumasiran administered as a subcutaneous (SC) injection | 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 16 Feb 2024.
- Information on the Early Access Program for LumasiranLink 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 Lumasiran in people with Primary Hyperoxaluria, 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 1 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 16 Feb 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?
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 16 Feb 2024.