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Natural History of Patients With PH3 and a History of Stone Events (PHYOX-OBX)

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: NCT04542590
Recruitment Status : Recruiting
First Posted : September 9, 2020
Last Update Posted : November 18, 2021
Sponsor:
Information provided by (Responsible Party):
Dicerna Pharmaceuticals, Inc., a Novo Nordisk company

Study Description
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Brief Summary:
This is a natural history study of adults, adolescents, and children (starting at birth) with genetically confirmed primary hyperoxaluria type 3 (PH3) who have a history of stone events during the last 3 years and/or the presence of pre existing stones detected by renal ultrasound at screening.

Condition or disease
Primary Hyperoxaluria Type 3

Detailed Description:

The relationship between the level of Uox and the incidence of kidney stones and/or nephrocalcinosis in patients with PH3 has not been established. The goal of this study is to record 24-hour Uox levels and the incidence of new stone formation and/or the degree of nephrocalcinosis in patients with PH3 over time.

Potential participants are screened over an up-to-7 week period according to the eligibility criteria and will then be followed every 6 months over an up-to-2-year period to determine the annualized new stone formation rate (number of new stones being formed per year) and the change in the degree of nephrocalcinosis (if applicable).

New stone formation is defined as occurrence of any of the following:

  • Spontaneous stone passage in the absence of pre-existing stones
  • Stone passage occurring without change in the number of pre-existing stones detected by renal ultrasound
  • Appearance of new stones on renal ultrasound or 100% or more growth of a pre-existing stone (estimated area)
  • Surgical removal of newly formed stones Note: Spontaneous passage or surgical removal of pre-existing stones does not meet the criteria for new stone formation.

This is a non-interventional study that will last up to 2 years.

Study Design
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Study Type : Observational [Patient Registry]
Estimated Enrollment : 30 participants
Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration: 2 Years
Official Title: A Natural History Study of Patients With Genetically Confirmed Primary Hyperoxaluria Type 3 and, as Applicable Per Age, a History of Stone Events
Actual Study Start Date : September 9, 2021
Estimated Primary Completion Date : February 28, 2024
Estimated Study Completion Date : February 28, 2024

Resource links provided by the National Library of Medicine


Groups and Cohorts
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Outcome Measures
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Primary Outcome Measures :
  1. Collect stone formation data in PH3 patients [ Time Frame: Assess participants' stone formation rates over the course of 2 years ]
    Collect data on the rate of new stone formation in PH3 patients of at least 2 years of age

  2. Assess relationship between urine oxalate (Uox) levels and stone formations in PH3 patients [ Time Frame: Measure participants' Uox levels over the course of 2 years ]
    Explore the potential relationship between Uox levels and new stone formation in patients (≥ 2 years of age) with genetically confirmed PH3 and relatively intact renal function

  3. Collect data on the degree of nephrocalcinosis in PH3 patients [ Time Frame: Assess the change in nephrocalcinosis grade over the course of 2 years ]
    Collect data on the degree of nephrocalcinosis in PH3 patients of at least 2 years of age


Eligibility Criteria
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Ages Eligible for Study:   2 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
This is a natural history study in adults, adolescents, and children (starting from birth) with genetically confirmed PH3 who have a history of stone events during the last 3 years and/or the presence of preexisting stones detected by renal ultrasounds at Screening, and an eGFR > 30 mL/min/1.73 m2 or serum creatinine below the 97th percentile for patients younger than 12 months old.
Criteria

Key inclusion criteria

  • Genetically confirmed PH3
  • For participants at least 2 years of age, history of stone events (defined as presence of calcifications in the urinary tract and/or kidney, their relative location, and the number and size of stones) during the last 3 years and/or presence of pre-existing stones detected by renal ultrasound at Screening

  • Uox ≥ 0.7 mmol/24 hours (adjusted per 1.73 m2 BSA in participants < 18 years of age) OR if not able to collect 24-hour urine, average spot Uox to creatinine ratio at Screening above the 95th percentile for age:

    • > 220 mmol/mol in participants < 6 months
    • > 170 mmol/mol in participants from 6 months to < 12 months
    • > 130 mmol/mol in participants 12 months to < 2 years
    • > 100 mmol/mol in participants from 2 to < 3 years and
    • > 80 mmol/mol in participants from 3 to 5 years
  • eGFR at Screening ≥ 30 mL/min or for infants aged less than 12 months, serum creatinine below the 97th percentile of a healthy population

Key Exclusion Criteria:

  • Prior or planned liver transplant within study period
  • Currently receiving dialysis or anticipating dialysis during study period
  • Unwillingness to comply with study procedures
Contacts and Locations
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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): NCT04542590


Contacts
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Contact: Medical Info 617-621-8097 medicalinfo@dicerna.com

Locations
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United States, Massachusetts
Clinical Trial Site Recruiting
Boston, Massachusetts, United States, 02115
United States, Minnesota
Clinical Trial Site Not yet recruiting
Rochester, Minnesota, United States, 55905
United States, New York
Clinical Trial Site Recruiting
New York, New York, United States, 10016
United States, North Carolina
Clinical Trial Site Recruiting
Durham, North Carolina, United States, 27710
Canada, Ontario
Clinical Trial Site Recruiting
Toronto, Ontario, Canada, M5G 1X8
France
Clinical Trial Site Recruiting
Paris, France, 75019
Germany
Clinical Trial Site Not yet recruiting
Bonn, Germany, 53127
Clinical Trial Site Recruiting
Heidelberg, Germany, 69120
Lebanon
Clinical Trial Site Recruiting
Beirut, Lebanon
Poland
Clinical Trial Site Recruiting
Warsaw, Poland, 04-141
United Kingdom
Clinical Trial Site Not yet recruiting
London, United Kingdom, NW3 2QG
Clinical Trial Site Not yet recruiting
London, United Kingdom, WC1N 3JH
Clinical Trial Site Not yet recruiting
Manchester, United Kingdom, M13 9WL
Sponsors and Collaborators
Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
Investigators
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Study Director: Alexandra Haagensen, MD, MBA Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
More Information
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Responsible Party: Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
ClinicalTrials.gov Identifier: NCT04542590    
Other Study ID Numbers: DCR-PHXC-502
First Posted: September 9, 2020    Key Record Dates
Last Update Posted: November 18, 2021
Last Verified: November 2021

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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 Dicerna Pharmaceuticals, Inc., a Novo Nordisk company:
primary hyperoxaluria
PH3
observational
kidney stones
nephrocalcinosis
Additional relevant MeSH terms:
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Hyperoxaluria, Primary
Hyperoxaluria
Kidney Diseases
Urologic Diseases
Female Urogenital Diseases
Female Urogenital Diseases and Pregnancy Complications
 Urogenital Diseases
Male Urogenital Diseases
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases