Expanded Access Protocol to Provide Lumasiran to Patients... | NCT04125472

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

NCT04125472

Sponsor

Alnylam Pharmaceuticals

Status

Approved for marketing

Last Update Posted

Feb 16, 2024Actual

Enrollment

Not provided

Phase

Not provided

Conditions

Primary Hyperoxaluria

Interventions

Lumasiran

Countries

Not provided

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Feb 2024

Overall Recruitment Status or Expanded Access Status

Approved for marketing

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Not provided

Primary Completion Date

Not provided

Completion Date

Not provided

First Submitted Date

Oct 10, 2019

First Submission Date that Met QC Criteria

Oct 10, 2019

First Posted Date

Oct 14, 2019Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Feb 15, 2024

Last Update Posted Date

Feb 16, 2024Actual

Description Module

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),

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.

Design Module

Study Type

Expanded Access

Number of References to an Expanded Access Study

Expanded Access Types

Individual PatientsTreatment IND/Protocol

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Not provided

Interventional Study Design

No data available

No data is available for this block.

Biospecimen

No data available

No data is available for this block.

Enrollment

Not provided

Name	Type	Description	Arm Group Labels	Other Names
Lumasiran	Drug	Lumasiran administered as a subcutaneous (SC) injection		ALN-GO1

Eligibility Module

Eligibility 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

Accepts Healthy Volunteers

Not provided

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

Not provided

Maximum Age

Not provided

Standard Ages

ChildAdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Name	Affiliation	Role
Medical Director	Alnylam Pharmaceuticals	Study Director

Label	URL
Information on the Early Access Program for Lumasiran	View source

Condition Browse Module

MeSH Terms

ID	Term
D006960	Hyperoxaluria, Primary
D006959	Hyperoxaluria
D007674	Kidney Diseases
D014570	Urologic Diseases
D008661	Metabolism, Inborn Errors
D030342	Genetic Diseases, Inborn
D008659	Metabolic Diseases

Ancestor Terms

ID	Term
D052776	Female Urogenital Diseases
D005261	Female Urogenital Diseases and Pregnancy Complications
D000091642	Urogenital Diseases
D052801	Male Urogenital Diseases

Browse Leaves

Not provided

Browse Branches

Not provided