- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT03392896
Study of DCR-PHXC-101 in Normal Healthy Volunteers and Patients With Primary Hyperoxaluria
January 17, 2020 updated by: Dicerna Pharmaceuticals, Inc.
A Placebo-Controlled, Single-Blind, Single-Center Phase 1 Study in Normal Healthy Volunteers and Open-Label Multi-Center Study in Patients With Primary Hyperoxaluria to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single Ascending Doses of DCR-PHXC Solution for Injection (Subcutaneous Use)
This is a double-blind, placebo-controlled, dose escalation trial of DCR-PHXC in Healthy Volunteers (HVs) and patients with Primary Hyperoxaluria (PH).
Once safety has been established in HV, PH patients with a confirmed diagnosis of PH1 and PH2 will be enrolled across multiple dosing cohorts.
The study design will allow enrollment of PH patient cohorts at a given dose level once safety has been demonstrated in HV at that dose level.
The study will be conducted in two parts: Part A: Single ascending dose (SAD) in HV; Part B: SAD in patients with PH1 and PH2 (lagging Part A by 1 dose level cohort).
Study Overview
Status
Completed
Conditions
Intervention / Treatment
Study Type
Interventional
Enrollment (Actual)
43
Phase
- Phase 1
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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Bron, France, 69677
- Centre d'Investigation Clinique - CIC 1407 - Hospices Civils de Lyon
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Bonn, Germany, 53127
- Universitätsklinikum Bonn-Institut für Klinische Chemie und Klinische Pharmakologie
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Amsterdam, Netherlands, 1012 WX
- University of Amsterdam
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Birmingham, United Kingdom, B15 2GW
- Queen Elizabeth Hospital Birmingham
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Birmingham, United Kingdom, B4 6NH
- Birmingham Children's Hospital NHS Trust
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Wales, United Kingdom, CF484DR
- Clinical Trial Site
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Massachusetts
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Boston, Massachusetts, United States, 02115
- Boston Children's Hospital
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
6 years and older (Child, Adult, Older Adult)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
Group A (HVs) Major Inclusion Criteria:
- Willing and able to provide informed consent and comply with study requirements.
- Male or female subjects between 18 and 55 years of age, inclusive.
- Subject must have a body mass index (BMI) 19.0 to 32 kg/m2, inclusive.
- Non-smokers, at least 1-month tobacco free, and willing to remain tobacco free through end of study (EOS).
- Women of child bearing potential must have a negative pregnancy test, cannot be breastfeeding, and must be willing to use contraception.
Group A (HVs) Major Exclusion Criteria:
- Presence of any medical condition, including but not limited to: Severe intercurrent illness, known causes of active liver disease.
- Routine or chronic use of more than 3 grams of acetaminophen (Tylenol) daily.
- History of kidney stones.
- Use of any investigational agent within 90 days before the first dose of study medication.
- History of donation of more than 450 mL of blood within 90 days prior to dosing in the clinical research center or planned donation less than 30 days after receiving Investigational Medicinal Product (IMP).
- Plasma or platelet donation within 7 days of dosing and through EOS.
- History of reactions to an oligonucleotide-based therapy.
- Males with female partners who are planning to attempt to become pregnant during this study or within 90 days after last dosing of IMP.
- Plasma or platelet donation within 7 days of dosing and through EOS.
Group B (PH1 and PH2 patients) Major Inclusion Criteria:
- Willing and able to provide informed consent and comply with study requirements.
- Male or female, at least 6 years of age.
- Minimum body weight of 25 kg.
- Genetic confirmation of PH1 and PH2 disease.
- Meet the 24 hour urine oxalate excretion requirements.
- Estimated glomerular filtration rate (eGFR) ≥30 mL/min/1.73 m2.
- If taking Vitamin B6 (pyridoxine), must have been on stable regimen for at least 4 weeks.
Group B (PH1 and PH2 patients) Major Exclusion Criteria:
- Prior renal and/or hepatic transplantation.
- Currently receiving dialysis.
- Participation in any clinical study where they received an investigational agent within 4 months before enrollment.
- Presence of any medical condition, including but not limited to: Severe intercurrent illness, known causes of active liver disease.
- Liver function test (LFT) abnormalities.
- History of reactions to an oligonucleotide-based therapy.
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Parallel Assignment
- Masking: Double
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
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Experimental: Group A Active (DCR-PHXC)
HVs, single ascending doses of DCR-PHXC.
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DCR-PHXC is a novel, potent, and long-acting small interference ribonucleic acid (siRNA) molecule conjugated to N-acteylgalactosamine (GalNAc) that is designed to decrease liver oxalate production.
DCR-PHXC is delivered via subcutaneous (SC) injection.
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Placebo Comparator: Group A Placebo
HVs, normal saline 0.9% injection to match active doses.
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Single SC administration of placebo, which will be a sterile, preservative-free normal saline 0.9% solution for SC injection, which is of similar osmolality to the DCR-PHXC formulation.
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Experimental: Group B Active (DCR-PHXC)
PH1 and PH2 patients, open label, single ascending doses of DCR-PHXC.
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DCR-PHXC is a novel, potent, and long-acting small interference ribonucleic acid (siRNA) molecule conjugated to N-acteylgalactosamine (GalNAc) that is designed to decrease liver oxalate production.
DCR-PHXC is delivered via subcutaneous (SC) injection.
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
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Number of patients with Treatment-Related Adverse Events (TEAEs)
Time Frame: Part A (SAD in HVs) screening through Day 29; Part B (SAD in PH patients) screening through Day 57
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Part A (SAD in HVs) screening through Day 29; Part B (SAD in PH patients) screening through Day 57
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
December 6, 2017
Primary Completion (Actual)
November 19, 2019
Study Completion (Actual)
November 19, 2019
Study Registration Dates
First Submitted
December 21, 2017
First Submitted That Met QC Criteria
January 2, 2018
First Posted (Actual)
January 8, 2018
Study Record Updates
Last Update Posted (Actual)
January 22, 2020
Last Update Submitted That Met QC Criteria
January 17, 2020
Last Verified
January 1, 2020
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- DCR-PHXC-101
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
No
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Yes
Studies a U.S. FDA-regulated device product
No
product manufactured in and exported from the U.S.
Yes
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Primary Hyperoxaluria
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Dicerna Pharmaceuticals, Inc.CompletedKidney Diseases | Urologic Diseases | Genetic Disease | Primary Hyperoxaluria Type 1 (PH1) | Primary Hyperoxaluria Type 2 (PH2)Poland, United Kingdom, New Zealand, United States, Australia, Canada, France, Germany, Israel, Italy, Japan, Lebanon, Netherlands, Romania, Spain
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Dicerna Pharmaceuticals, Inc., a Novo Nordisk companyEnrolling by invitationKidney Diseases | Urologic Diseases | Genetic Disease | Primary Hyperoxaluria Type 1 (PH1) | Primary Hyperoxaluria Type 2 (PH2) | Primary Hyperoxaluria Type 3 (PH3)United States, France, Germany, Japan, Lebanon, Spain, United Kingdom, Australia, Canada, Italy, Netherlands, Norway, Turkey
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Alnylam PharmaceuticalsCompletedPrimary Hyperoxaluria Type 1 (PH1)France, United Kingdom, Netherlands, Israel, Germany
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Dicerna Pharmaceuticals, Inc., a Novo Nordisk companyRecruitingPrimary Hyperoxaluria Type 3 | Primary Hyperoxaluria Type 2 | Primary Hyperoxaluria Type 1 | Primary HyperoxaluriaUnited States, Canada, Lebanon, Turkey, United Kingdom, Germany, Italy, Japan, Poland, Spain, United Arab Emirates
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VA New York Harbor Healthcare SystemMayo Clinic; New York UniversityUnknownOxalate, Primary Hyperoxaluria, MicrobiomeUnited States
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Dicerna Pharmaceuticals, Inc.CompletedPrimary Hyperoxaluria Type 3United States, Germany, Netherlands, United Kingdom
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University of CologneCompletedPrimary Hyperoxaluria Type IGermany
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Alnylam PharmaceuticalsCompletedPrimary Hyperoxaluria Type 1 (PH1)United States, France, United Kingdom, Switzerland, Netherlands, Israel, Germany, United Arab Emirates
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Dicerna Pharmaceuticals, Inc., a Novo Nordisk companyAvailablePrimary Hyperoxaluria Type 1 (PH1)
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Alnylam PharmaceuticalsActive, not recruitingPrimary Hyperoxaluria Type 1 (PH1) | Primary HyperoxaluriaUnited States, France, United Kingdom, Israel, Germany
Clinical Trials on DCR-PHXC
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Dicerna Pharmaceuticals, Inc., a Novo Nordisk companyRecruitingEnd Stage Renal Disease | Primary Hyperoxaluria Type 2 | Primary Hyperoxaluria Type 1Germany, Spain, United States, United Kingdom, Italy, United Arab Emirates, Lebanon, Morocco, France, Romania
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Dicerna Pharmaceuticals, Inc., a Novo Nordisk companyRecruitingPrimary Hyperoxaluria Type 3 | Primary Hyperoxaluria Type 2 | Primary Hyperoxaluria Type 1 | Primary HyperoxaluriaUnited States, Canada, Lebanon, Turkey, United Kingdom, Germany, Italy, Japan, Poland, Spain, United Arab Emirates
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Dicerna Pharmaceuticals, Inc.CompletedPrimary Hyperoxaluria Type 3United States, Germany, Netherlands, United Kingdom
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Dicerna Pharmaceuticals, Inc., a Novo Nordisk companyEnrolling by invitationKidney Diseases | Urologic Diseases | Genetic Disease | Primary Hyperoxaluria Type 1 (PH1) | Primary Hyperoxaluria Type 2 (PH2) | Primary Hyperoxaluria Type 3 (PH3)United States, France, Germany, Japan, Lebanon, Spain, United Kingdom, Australia, Canada, Italy, Netherlands, Norway, Turkey
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Dicerna Pharmaceuticals, Inc.CompletedKidney Diseases | Urologic Diseases | Genetic Disease | Primary Hyperoxaluria Type 1 (PH1) | Primary Hyperoxaluria Type 2 (PH2)Poland, United Kingdom, New Zealand, United States, Australia, Canada, France, Germany, Israel, Italy, Japan, Lebanon, Netherlands, Romania, Spain
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Tehran University of Medical SciencesUnknownPrimary Acquired NasolacrimalIran, Islamic Republic of
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Dicerna Pharmaceuticals, Inc.ParexelCompleted
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Dicerna Pharmaceuticals, Inc.CompletedHepatitis B, ChronicKorea, Republic of, New Zealand, Australia, Thailand, Hong Kong
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Dicerna Pharmaceuticals, Inc., a Novo Nordisk companyCompleted
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Dicerna Pharmaceuticals, Inc.TerminatedHepatocellular CarcinomaKorea, Republic of, United States, Singapore