ClinicalTrials.gg
Approved for marketingNCT04125472Updated Feb 16, 2024

Expanded Access Protocol to Provide Lumasiran to Patients With Primary Hyperoxaluria Type 1

An expanded access record providing Lumasiran in Primary Hyperoxaluria, sponsored by Alnylam Pharmaceuticals. Approved for marketing. Per ClinicalTrials.gov, last updated 2024-02-16.

Sponsored by Alnylam Pharmaceuticals · Expanded access

Study type
Expanded access
Access type
Individual patients · Treatment IND/protocol
Sex
All
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Study 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 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.

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Conditions studied

  • Primary Hyperoxaluria

Keywords

  • Hyperoxaluria
  • Primary Hyperoxaluria
  • Kidney Diseases
  • Urologic Diseases
  • Metabolism, Inborn Errors
  • Genetic Diseases, Inborn
  • Metabolic Diseases
  • RNA Therapeutic
  • siRNA
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In context

Hyperoxaluria, Primary

42 studies on the registry are indexed under Hyperoxaluria, Primary; 11 are open to participants now.

Browse Hyperoxaluria, Primary studies →

Lead sponsor

Alnylam Pharmaceuticals is the lead sponsor of 84 studies on the registry; 19 are open to participants now.

Of its 18 completed or terminated interventional studies of FDA-regulated products, 15 (83%) have results posted.

Counted across the registry records on this site, refreshed daily.

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Who can participate

Ages eligible
Child (0–17), Adult (18–64), Older adult (65+)
Sexes eligible
All

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

Inclusion criteria

  • Documented diagnosis of Primary Hyperoxaluria Type 1

Exclusion criteria

Exclusion Criteria:

  • health concerns (with the exception of PH1)
  • Received an 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
Terms in these criteria, in plain language(2)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

Clinically significant

Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.

Investigational drug or device

A drug or device being studied in a clinical trial.

It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.

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Access details

Study type
Expanded access
Access type
Individual patients · Treatment IND/protocol

Available treatment

  • Lumasiran administered as a subcutaneous (SC) injection

    Also known as: ALN-GO1

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Where to request access

No study locations are listed for this record.

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References and documents

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Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Feb 16, 2024, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
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Registry details

Key details

Study ID
NCT04125472
Responsible party
Sponsor
First posted
Oct 14, 2019
Last update
Feb 16, 2024

Study contacts

Medical Director
study director · Alnylam Pharmaceuticals
View the source record on ClinicalTrials.gov ↗

Requesting access

Expanded access is arranged between your doctor and the company. Ask your care team to contact the provider listed on this record.

No contact was published for this record. The registry link below has the sponsor’s details.

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Discussion

Questions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.

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