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A Study of DCR-PH1 in Patients With Primary Hyperoxaluria Type 1 (PH1)

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. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02795325
Recruitment Status : Terminated
First Posted : June 10, 2016
Last Update Posted : October 3, 2018
Sponsor:
Information provided by (Responsible Party):
Dicerna Pharmaceuticals, Inc.

Brief Summary:
A phase 1 study of DCR-PH1 in patients with primary hyperoxaluria type 1 (PH1) to determine the safety, tolerability, pharmacokinetic (PK) and pharmacodynamics (PD) effects of DCR-PH1 administered via-intravenous infusion (IV)

Condition or disease Intervention/treatment Phase
Primary Hyperoxaluria Type 1 Drug: DCR-PH1 Other: Placebo Phase 1

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 41 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 1 Study of DCR-PH1 in Patients With Primary Hyperoxaluria Type 1 (PH1)
Actual Study Start Date : May 13, 2016
Actual Primary Completion Date : October 14, 2016
Actual Study Completion Date : October 14, 2016

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: PH Patients Drug: DCR-PH1
IV infusion of DCR-PH1

Experimental: Healthy Volunteers Drug: DCR-PH1
IV infusion of DCR-PH1

Other: Placebo



Primary Outcome Measures :
  1. The safety of DCR-PH1 evaluated by the proportion of subjects that experience adverse events (AEs) [ Time Frame: Through Day 29 ]

Secondary Outcome Measures :
  1. Profile of pharmacokinetics (PK) of DCR-PH1 - Cmax [ Time Frame: Through Day 29 ]
  2. Profile of pharmacokinetics (PK) of DCR-PH1 - tmax [ Time Frame: Through Day 29 ]
  3. Profile of pharmacokinetics (PK) of DCR-PH1 - AUC [ Time Frame: Through Day 29 ]
  4. Profile of pharmacokinetics (PK) of DCR-PH1 - t½ [ Time Frame: Through Day 29 ]
  5. The effect of DCR-PH1 on plasma glycolate levels [ Time Frame: Through Day 29 ]
  6. The effect of DCR-PH1 on urine glycolate levels [ Time Frame: Through Day 29 ]
  7. The effect of DCR-PH1 on plasma oxalate levels [ Time Frame: Through Day 29 ]
  8. The effect of DCR-PH1 on urine oxalate levels [ Time Frame: Through Day 29 ]


Information from the National Library of Medicine

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Ages Eligible for Study:   12 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Male or female, at least 12 years of age
  • Diagnosis of PH1, confirmed by genotyping
  • 24-hour urine oxalate excretion as defined in the protocol
  • eGFR ≥ 40 mL/min normalized to 1.73 m2 BSA
  • Written informed consent for adults (≥18 years old, or per local regulatory requirement); written informed assent for adolescents (12 to <18 years old, or per local regulatory requirement)

Exclusion Criteria:

  • Prior renal and/or hepatic transplantation
  • Participation in any clinical study involving administration of any investigational drug within the 30 days before enrollment
  • Pregnancy or lactation at the time of screening or enrollment
  • Women of child-bearing potential must have a negative pregnancy test, cannot be breastfeeding and must be willing to use contraception
  • Patients with a known history of human immunodeficiency virus (HIV) or active infection with hepatitis B virus or hepatitis C virus
  • Moderate to severe liver impairment
  • Liver function test abnormalities: alanine transaminases (ALT) and/or aspartate transaminases (AST) > 2 times upper limit of normal (ULN)
  • History of severe reaction to a liposomal product or a known hypersensitivity to lipid products.
  • Unable to collect required study samples or follow study procedures
  • No clinically significant health concerns

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): NCT02795325


Locations
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Germany
Bonn, Germany
Netherlands
Amsterdam, Netherlands
Sponsors and Collaborators
Dicerna Pharmaceuticals, Inc.
Additional Information:
Publications:
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Responsible Party: Dicerna Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT02795325    
Other Study ID Numbers: DCR-PH1-101
First Posted: June 10, 2016    Key Record Dates
Last Update Posted: October 3, 2018
Last Verified: December 2017
Keywords provided by Dicerna Pharmaceuticals, Inc.:
Primary Hyperoxaluria Type 1
PH1
Calcium Oxalate Stones
Kidney Stones
Liver Enzyme Deficiency
Genetic Diseases
Inborn Kidney Diseases
Metabolic Diseases
Inborn Errors
Urological Diseases
Carbohydrate Metabolism
Hyperoxaluria
AGT
RNAi
siRNA
DCR-PH1
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