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Trial record 1 of 1 for:    NCT04125472
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Expanded Access Protocol to Provide Lumasiran to Patients With Primary Hyperoxaluria Type 1

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ClinicalTrials.gov Identifier: NCT04125472
Expanded Access Status : Available
First Posted : October 14, 2019
Last Update Posted : October 17, 2019
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.

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Study Type : Expanded Access
Expanded Access Type : 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.


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Ages Eligible for Study:   6 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Criteria

Inclusion Criteria:

  • Documented diagnosis of Primary Hyperoxaluria Type1
  • Meet the urine oxalate excretion requirements

Exclusion Criteria:

  • Clinically significant health concerns (with the exception of PH1)
  • An estimated GFR of ≤ 45 mL/min/1.73m2 at screening
  • Clinically significant abnormal laboratory results at screening
  • 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 renal or liver transplant
  • Women who are pregnant, planning a pregnancy, or breast-feeding or those of child bearing potential and not willing to use contraception

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


Contacts
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Contact: Alnylam EAP Hotline (617) 715-0200 eap@alnylam.com

Sponsors and Collaborators
Alnylam Pharmaceuticals
Investigators
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Study Director: Karen Franscello Alnylam Pharmaceuticals

Additional Information:
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Responsible Party: Alnylam Pharmaceuticals
ClinicalTrials.gov Identifier: NCT04125472     History of Changes
Other Study ID Numbers: ALN-GO1-006
First Posted: October 14, 2019    Key Record Dates
Last Update Posted: October 17, 2019
Last Verified: October 2019
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:
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Hyperoxaluria, Primary
Hyperoxaluria
Kidney Diseases
Urologic Diseases
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases