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BONAPH1DE, A Prospective Observational Study of Patients With Primary Hyperoxaluria Type 1 (PH1) (BONAPH1DE)

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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04982393
Recruitment Status : Recruiting
First Posted : July 29, 2021
Last Update Posted : November 9, 2022
Sponsor:
Information provided by (Responsible Party):
Alnylam Pharmaceuticals

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.

Condition or disease
Primary Hyperoxaluria Type 1

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Study Type : Observational
Estimated Enrollment : 200 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: BONAPH1DE, A Prospective Observational Study of Patients With Primary Hyperoxaluria Type 1 (PH1)
Actual Study Start Date : December 13, 2021
Estimated Primary Completion Date : September 2028
Estimated Study Completion Date : September 2028

Resource links provided by the National Library of Medicine


Group/Cohort
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.



Primary Outcome Measures :
  1. Incidence of Adverse Events in Lumasiran Treated Patients [ Time Frame: Up to 7 years ]

Secondary Outcome Measures :
  1. Incidence of Selected Events of Interest in PH1 Patients [ Time Frame: Up to 7 years ]
    Selected events of interest are defined as hepatic events, kidney stones, acute kidney injury events, nephrocalcinosis, chromic kidney disease, kidney failure, and any cardiac, bone, skin, eye, hematological, or neuropathic manifestations due to oxalosis.

  2. 12-Item Short Form Health Survey Version 2 (SF-12 V2) (Standard Version) [ Time Frame: Up to 7 years ]
    SF-12 V2 is a 12-question measure capturing global quality of life and overall health status and evaluates the following 8 domains: physical functioning, role physical, bodily pain, general health, vitality, social functioning, role emotional, and mental health.

  3. Change in Urinary Oxalate Excretion [ Time Frame: Baseline and every 12 months for up to 7 years ]
  4. Change in Plasma Oxalate [ Time Frame: Baseline and every 12 months for up to 7 years ]


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:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Patients diagnosed with PH1 will be managed and treated per routine clinical practice.
Criteria

Inclusion Criteria:

  • Documented diagnosis of PH1, per physician's determination

Exclusion Criteria:

  • Currently enrolled in a clinical trial for any investigational agent

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): NCT04982393


Contacts
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Contact: Alnylam Clinical Trial Information Line 1-877-ALNYLAM clinicaltrials@alnylam.com
Contact: Alnylam Clinical Trial Information Line 1-877-256-9526 clinicaltrials@alnylam.com

Locations
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United States, Arizona
Clinical Trial Site Recruiting
Phoenix, Arizona, United States, 85016
United States, District of Columbia
Clinical Trial Site Recruiting
Washington, District of Columbia, United States, 20010
United States, Massachusetts
Clinical Trial Site Recruiting
Boston, Massachusetts, United States, 02115
United States, Minnesota
Clinical Trial Site Recruiting
Rochester, Minnesota, United States, 55905
United States, Ohio
Clinical Trial Site Recruiting
Cincinnati, Ohio, United States, 45229
United States, Pennsylvania
Clinical Trial Site Recruiting
Pittsburgh, Pennsylvania, United States, 15213
United States, Texas
Clinical Trial Site Recruiting
Dallas, Texas, United States, 75390
Clinical Trial Site Recruiting
Houston, Texas, United States, 77030
Belgium
Clinical Trial Site Recruiting
Liège, Belgium
France
Clinical Trial Site Recruiting
Bordeaux, France
Clinical Trial Site Recruiting
Lyon, France
Clinical Trial Site Recruiting
Paris, France
Israel
Clinical Trial Site Recruiting
Jerusalem, Israel
Sponsors and Collaborators
Alnylam Pharmaceuticals
Investigators
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Study Director: Medical Director Alnylam Pharmaceuticals
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Responsible Party: Alnylam Pharmaceuticals
ClinicalTrials.gov Identifier: NCT04982393    
Other Study ID Numbers: ALN-GO1-007
First Posted: July 29, 2021    Key Record Dates
Last Update Posted: November 9, 2022
Last Verified: November 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Alnylam Pharmaceuticals:
PH1
Lumasiran
Primary Hyperoxaluria Type 1
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