NCT04982393

Brief Summary

The purpose of this study is to describe the natural history and progression of patients diagnosed with PH1, and to characterize the long-term real-world safety and effectiveness of lumasiran.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
207

participants targeted

Target at P75+ for all trials

Timeline
28mo left

Started Dec 2021

Longer than P75 for all trials

Geographic Reach
11 countries

30 active sites

Status
active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress65%
Dec 2021Sep 2028

First Submitted

Initial submission to the registry

July 20, 2021

Completed
9 days until next milestone

First Posted

Study publicly available on registry

July 29, 2021

Completed
5 months until next milestone

Study Start

First participant enrolled

December 13, 2021

Completed
6.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 1, 2028

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

September 1, 2028

Last Updated

April 24, 2026

Status Verified

April 1, 2026

Enrollment Period

6.7 years

First QC Date

July 20, 2021

Last Update Submit

April 23, 2026

Conditions

Primary Hyperoxaluria Type 1

Keywords

PH1LumasiranPrimary Hyperoxaluria Type 1

Outcome Measures

Primary Outcomes (1)

  • Incidence of Adverse Events in Lumasiran Treated Patients

    Up to 7 years

Secondary Outcomes (4)

  • Incidence of Selected Events of Interest in PH1 Patients

    Up to 7 years

  • 12-Item Short Form Health Survey Version 2 (SF-12 V2) (Standard Version)

    Up to 7 years

  • Change in Urinary Oxalate Excretion

    Baseline and every 12 months for up to 7 years

  • Change in Plasma Oxalate

    Baseline and every 12 months for up to 7 years

Study Arms (1)

Patients with PH1

Patients with a diagnosis of PH1 will be eligible for the study and will be managed and treated per routine clinical practice.

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

Patients diagnosed with PH1 will be managed and treated per routine clinical practice.

You may qualify if:

  • Documented diagnosis of PH1, per physician's determination

You may not qualify if:

  • Currently enrolled in a clinical trial for any investigational agent

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (30)

Clinical Trial Site

Phoenix, Arizona, 85016, United States

Location

Clinical Trial Site

Washington D.C., District of Columbia, 20010, United States

Location

Clinical Trial Site

Boston, Massachusetts, 02115, United States

Location

Clinical Trial Site

Rochester, Minnesota, 55905, United States

Location

Clinical Trial Site

Cincinnati, Ohio, 45229, United States

Location

Clinical Trial Site

Pittsburgh, Pennsylvania, 15213, United States

Location

Clinical Trial Site

Dallas, Texas, 75390, United States

Location

Clinical Trial Site

Houston, Texas, 77030, United States

Location

Clinical Trial Site

Ghent, Belgium

Location

Clinical Trial Site

Liège, Belgium

Location

Clinical Trial Site

Hamilton, Ontario, Canada

Location

Clinical Trial Site

Toronto, Ontario, Canada

Location

Clinical Trial Site

Laurier, Quebec, Canada

Location

Clinical Trial Site

Bordeaux, France

Location

Clinical Trial Site

Lyon, France

Location

Clinical Trial Site

Paris, France

Location

Clinical Trial Site

Berlin, Germany

Location

Clinical Trial Site

Cologne, Germany

Location

Clinical Trial Site

Hamburg, Germany

Location

Clinical Trial Site

Jerusalem, Israel

Location

Clinical Trial Site

Orbassano, Torino, Italy

Location

Clinical Trial Site

Milan, Italy

Location

Clinical Trial Site

Verona, Italy

Location

Clinical Trial Site

Amsterdam, Netherlands

Location

Clinical Trial Site

Santa Cruz de Tenerife, Canary Islands, Spain

Location

Clinical Trial Site

Barcelona, Spain

Location

Clinical Trial Site

Las Palmas, Spain

Location

Clinical Trial Site

Bern, Switzerland

Location

Clinical Trial Site

London, England, United Kingdom

Location

Clinical Trial Site

London, United Kingdom

Location

MeSH Terms

Conditions

Primary hyperoxaluria type 1

Study Officials

  • Medical Director

    Alnylam Pharmaceuticals

    STUDY DIRECTOR

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

July 20, 2021

First Posted

July 29, 2021

Study Start

December 13, 2021

Primary Completion (Estimated)

September 1, 2028

Study Completion (Estimated)

September 1, 2028

Last Updated

April 24, 2026

Record last verified: 2026-04

Data Sharing

IPD Sharing
Will not share

Locations

FR(3)CH(1)IL(1)US(8)IT(3)DE(3)NL(1)ES(3)GB(2)BE(2)CA(3)