Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04125472
Expanded Access Status : Approved for marketing
First Posted : October 14, 2019
Last Update Posted : May 14, 2021
Sponsor:
Information provided by (Responsible Party):
Alnylam Pharmaceuticals

Brief Summary:
The purpose of this study is to provide expanded access to lumasiran for adults and pediatric patients with Primary Hyperoxaluria Type 1 (PH1),

Condition or disease Intervention/treatment
Primary Hyperoxaluria Drug: Lumasiran

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.

Layout table for study information
Study Type : Expanded Access
Expanded Access Type : Individual Patients, Treatment IND/Protocol
  See clinical trials of the intervention/treatment in this expanded access record.
Official Title: Expanded Access Protocol to Provide Lumasiran to Patients With Primary Hyperoxaluria Type 1



Intervention Details:
  • Drug: Lumasiran
    Lumasiran administered as a subcutaneous (SC) injection
    Other Name: ALN-GO1

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Criteria

Inclusion Criteria:

  • Documented diagnosis of Primary Hyperoxaluria Type 1

Exclusion Criteria:

  • 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04125472


Locations
Layout table for location information
Belgium
Program Site
Brussels, Belgium
Program Site
Liège, Belgium
Italy
Program Site
Florence, Italy
Program Site
Milan, Italy
Program Site
Padua, Italy
Program Site
Pesaro, Italy
Program Site
Turin, Italy
Netherlands
Program Site
Amsterdam, Netherlands
Spain
Program Site
Cartagena, Spain
Program Site
Las Palmas, Spain
Program Site
Santa Cruz De Tenerife, Spain
Sponsors and Collaborators
Alnylam Pharmaceuticals
Investigators
Layout table for investigator information
Study Director: Medical Director Alnylam Pharmaceuticals
Additional Information:
Layout table for additonal information
Responsible Party: Alnylam Pharmaceuticals
ClinicalTrials.gov Identifier: NCT04125472    
Other Study ID Numbers: ALN-GO1-006
First Posted: October 14, 2019    Key Record Dates
Last Update Posted: May 14, 2021
Last Verified: May 2021
Keywords provided by Alnylam Pharmaceuticals:
Hyperoxaluria
Primary Hyperoxaluria
Kidney Diseases
Urologic Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases
RNA Therapeutic
siRNA
Additional relevant MeSH terms:
Layout table for MeSH terms
Hyperoxaluria, Primary
Hyperoxaluria
Kidney Diseases
Urologic Diseases
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases