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

2019-10-18 11:21:40 | BioPortfolio

Summary

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

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

Conditions

Primary Hyperoxaluria

Intervention

Lumasiran

Status

Available

Source

Alnylam Pharmaceuticals

Results (where available)

View Results

Links

Published on BioPortfolio: 2019-10-18T11:21:40-0400

Clinical Trials [35 Associated Clinical Trials listed on BioPortfolio]

A Study of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1

The purpose of this study is to evaluate the efficacy, safety, pharmacokinetics (PK), and pharmacodynamics (PD) of lumasiran in infants and young children with confirmed primary hyperoxalu...

A Study to Evaluate Lumasiran in Patients With Advanced Primary Hyperoxaluria Type 1

The purpose of this study is to evaluate the efficacy, safety, pharmacokinetics (PK), and pharmacodynamics (PD) of lumasiran in patients with Advanced Primary Hyperoxaluria Type 1 (PH1).

A Study to Evaluate Lumasiran in Children and Adults With Primary Hyperoxaluria Type 1

The purpose of this study is to evaluate the efficacy and safety of Lumasiran in Children and Adults with Primary Hyperoxaluria Type 1 (PH1).

A Study of DCR-PH1 in Patients With Primary Hyperoxaluria Type 1 (PH1)

A phase 1 study of DCR-PH1 in patients with primary hyperoxaluria type 1 (PH1) to determine the safety, tolerability, pharmacokinetic (PK) and pharmacodynamics (PD) effects of DCR-PH1 admi...

Primary Hyperoxaluria Mutation Genotyping

This study will help us determine whether certain genetic mutations, more than others, are a cause of more severe disease in Primary Hyperoxaluria.

PubMed Articles [5850 Associated PubMed Articles listed on BioPortfolio]

Primary Hyperoxaluria- Imaging of Renal Oxalosis.

Primary Hyperoxaluria is a rare autosomal recessive hereditary disorder due to deficient alanine-glyoxylate aminotransferase (AGT) enzyme with defective glyoxylate metabolism leading to excessive oxal...

The ocular phenotype in primary hyperoxaluria type 1.

To investigate ophthalmic features in a large group of patients with primary hyperoxaluria type 1 (PH1), and to determine the relation between ocular involvement and systemic disease severity.

Mutation Hot Spot Region in the HOGA1 Gene Associated with Primary Hyperoxaluria Type 3 in the Chinese Population.

Primary hyperoxaluria type 3 (PH3) is a rare autosomal recessive disorder that affects glyoxylate metabolism. PH3 is caused by defects in 4-hydroxy-2-oxoglutarate aldolase, which is encoded by the HOG...

Regulation of human 4-hydroxy-2-oxoglutarate aldolase by pyruvate and α-ketoglutarate. Implications for Primary Hyperoxaluria Type-3.

4-hydroxy-2-oxoglutarate aldolase (HOGA1) is a mitochondrial enzyme that plays a gatekeeper role in hydroxyproline metabolism. Its loss of function in humans causes primary hyperoxaluria type 3, a rar...

Are two operations better than one? The debate over combined versus sequential liver-kidney transplantation from a single live donor in the treatment of primary hyperoxaluria 1.

Medical and Biotech [MESH] Definitions

A genetic disorder characterized by excretion of large amounts of OXALATES in urine; NEPHROLITHIASIS; NEPHROCALCINOSIS; early onset of RENAL FAILURE; and often a generalized deposit of CALCIUM OXALATE. There are subtypes classified by the enzyme defects in glyoxylate metabolism.

Excretion of an excessive amount of OXALATES in the urine.

The primary responsibility of one nurse for the planning, evaluation, and care of a patient throughout the course of illness, convalescence, and recovery.

Techniques or methods of patient care used by nurses as primary careproviders.

The primary responsibility of one nurse for the planning, evaluation, and care of a patient throughout the course of illness, convalescence, and recovery.

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