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A Study to Evaluate DCR-PHXC in Children and Adults With Primary Hyperoxaluria Type 1 and Primary Hyperoxaluria Type 2 (PHYOX2)

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ClinicalTrials.gov Identifier: NCT03847909
Recruitment Status : Recruiting
First Posted : February 20, 2019
Last Update Posted : November 18, 2019
Sponsor:
Information provided by (Responsible Party):
Dicerna Pharmaceuticals, Inc.

Brief Summary:
The purpose of this study is to evaluate the efficacy and safety of DCR-PHXC in Children and Adults with Primary Hyperoxaluria Type 1 (PH1) and Primary Hyperoxaluria Type 2 (PH2)

Condition or disease Intervention/treatment Phase
Primary Hyperoxaluria Type 1 (PH1) Primary Hyperoxaluria Type 2 (PH2) Kidney Diseases Urologic Diseases Genetic Disease Drug: DCR-PHXC Drug: Sterile Normal Saline (0.9% NaCl) Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 36 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 2 Placebo-Controlled, Double-Blind, Multicenter Study to Evaluate the Efficacy, Safety, and Tolerability of DCR-PHXC Solution for Injection (Subcutaneous Use) in Patients With Primary Hyperoxaluria
Actual Study Start Date : October 28, 2019
Estimated Primary Completion Date : June 2020
Estimated Study Completion Date : January 2021


Arm Intervention/treatment
Experimental: DCR-PHXC
Intervention, drug, DCR-PHXC
Drug: DCR-PHXC
Multiple fixed doses of DCR-PHXC by subcutaneous (SC) injection

Placebo Comparator: Placebo - Sterile Normal Saline (0.9% NaCl)
Placebo, sterile normal saline (0.9% NaCl) for subcutaneous (SC) injection
Drug: Sterile Normal Saline (0.9% NaCl)
Sterile Normal Saline (0.9% NaCl) for subcutaneous (SC) injection, administered at same injection volume as DCR-PHXC, to serve as placebo




Primary Outcome Measures :
  1. Time-weighted standardized (TWS) area under the curve (AUC) of percent change from baseline in 24-hour urinary oxalate excretion between Day 90 and Day 180 [ Time Frame: 3 months (Last 3 months of the 6 month treatment period) ]
    Four measurements of percent change from baseline in 24-hour urinary oxalate are combined to determine a single AUC value


Secondary Outcome Measures :
  1. TWS AUC of percent change from baseline in 24-hour urinary oxalate-to-creatinine ratio (value/upper limit of normal [ULN]) between Day 90 and Day 180 [ Time Frame: 3 months (Last 3 months of the 6 month treatment period) ]
    Four measurements of percent change from baseline in 24-hour urinary oxalate-to-creatinine ratio are combined to determine a single AUC value

  2. Proportion of participants with a 24-hour Uox level < 0.46 mmol/24 hours or ≥ 0.46 - < 0.60 mmol/24 hours at at least two consecutive study visits commencing at Day 90 and ending at Day 180 [ Time Frame: At least 2 months starting at Day 90 and through Day 180 ]
  3. Proportion of participants with 24-hour Uox levels in each of 4 quartile ranges (< 1.1 mmol, 1.1 to < 1.6 mmol, 1.6 to ≤ 2.4 mmol, and > 2.4 mmol/24 hours from baseline to Day 180 [ Time Frame: 7 months ]
  4. TWS AUC of percent change from baseline in 24-hour urinary oxalate-to-creatinine ratio (value/upper limit of normal [ULN]) between baseline and Day 180 [ Time Frame: 7 months ]
  5. TWS AUC of percent change from baseline in plasma oxalate [ Time Frame: 3 months (Last 3 months of the 6 month treatment period) ]
    Four measurements of percent change from baseline in plasma oxalate are combined to determine a single AUC value

  6. Frequency of Adverse events (AE) from baseline to study completion [ Time Frame: 7 months ]
  7. Frequency of serious adverse events (SAE) from baseline to study completion [ Time Frame: 7 months ]


Information from the National Library of Medicine

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Ages Eligible for Study:   6 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Key Inclusion Criteria:

  • Willing to provide written informed consent or assent
  • Confirmation of PH1 and PH2 disease
  • Must meet the 24 hour urine oxalate excretion requirements

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  • Estimated GFR at screening ≥ 30 mL/min normalized to 1.73 m2 BSA

Key Exclusion Criteria:

  • Renal or hepatic transplantation (prior or planned within the study period)
  • Currently on dialysis or anticipated requirement for dialysis during the study period
  • Documented evidence of clinical manifestations of systemic oxalosis
  • Use of an RNA interference (RNAi) drug within the last 6 months
  • Participation in any clinical study in which you received an investigational medicinal product (IMP) within 4 months before Screening
  • Liver function test (LFT) abnormalities: Alanine aminotransferase (ALT) and/or aspartate aminotransferase (AST) >1.5 times upper limit of normal (ULN) for age and gender
  • Unwillingness to comply with study procedures

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): NCT03847909


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 Recruiting
Minneapolis, Minnesota, United States, 55905
United States, Pennsylvania
Clinical Trial Site Recruiting
Pittsburgh, Pennsylvania, United States, 15224
Australia
Clinical Trial Site Recruiting
Parkville, Australia, 3052
Canada
Clinical Trial Site Recruiting
Hamilton, Canada
France
Clinical Trial Site Recruiting
Bron, France
Clinical Trial Site Recruiting
Paris, France, 75019
Netherlands
Clinical Trial Site Recruiting
Amsterdam, Netherlands, 1105 AZ
New Zealand
Clinical Trial Site Recruiting
Auckland, New Zealand
Poland
Clinical Trial Site Recruiting
Białystok, Poland
United Kingdom
Clinical Trial Site Recruiting
Birmingham, United Kingdom
Clinical Trial Site Recruiting
London, United Kingdom
Sponsors and Collaborators
Dicerna Pharmaceuticals, Inc.

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Responsible Party: Dicerna Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT03847909     History of Changes
Other Study ID Numbers: DCR-PHXC-201
First Posted: February 20, 2019    Key Record Dates
Last Update Posted: November 18, 2019
Last Verified: September 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Dicerna Pharmaceuticals, Inc.:
PH1
PH2
Primary Hyperoxaluria
RNAi
RNAi therapeutic
GalNAc
LDHA gene
LDH
siRNA
Additional relevant MeSH terms:
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Kidney Diseases
Hyperoxaluria, Primary
Urologic Diseases
Genetic Diseases, Inborn
Hyperoxaluria
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Metabolic Diseases