Study of DCR-PHXC-101 in Normal Healthy Volunteers and Patients With Primary Hyperoxaluria
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ClinicalTrials.gov Identifier: NCT03392896 |
Recruitment Status :
Completed
First Posted : January 8, 2018
Last Update Posted : January 22, 2020
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Condition or disease | Intervention/treatment | Phase |
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Primary Hyperoxaluria | Drug: DCR-PHXC Drug: Placebo | Phase 1 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 43 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Intervention Model Description: | Two-arm (active and placebo), single-blind, SAD period (Group A, HVs) followed by open-label, SAD period (Group B, PH1 and PH2 patients). |
Masking: | Double (Participant, Investigator) |
Masking Description: | SAD period in HV is single-blind (unblinded clinical site staff member who is not a member of study team administers dose). SAD period in Group B (PH1 and PH2 patients) is open-label. |
Primary Purpose: | Treatment |
Official Title: | 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) |
Actual Study Start Date : | December 6, 2017 |
Actual Primary Completion Date : | November 19, 2019 |
Actual Study Completion Date : | November 19, 2019 |

Arm | Intervention/treatment |
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Experimental: Group A Active (DCR-PHXC)
HVs, single ascending doses of DCR-PHXC.
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Drug: DCR-PHXC
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. |
Placebo Comparator: Group A Placebo
HVs, normal saline 0.9% injection to match active doses.
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Drug: Placebo
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. |
Experimental: Group B Active (DCR-PHXC)
PH1 and PH2 patients, open label, single ascending doses of DCR-PHXC.
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Drug: DCR-PHXC
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. |
- 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 ]

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.
Ages Eligible for Study: | 6 Years and older (Child, Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | Yes |
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.

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): NCT03392896
United States, Massachusetts | |
Boston Children's Hospital | |
Boston, Massachusetts, United States, 02115 | |
France | |
Centre d'Investigation Clinique - CIC 1407 - Hospices Civils de Lyon | |
Bron, France, 69677 | |
Germany | |
Universitätsklinikum Bonn-Institut für Klinische Chemie und Klinische Pharmakologie | |
Bonn, Germany, 53127 | |
Netherlands | |
University of Amsterdam | |
Amsterdam, Netherlands, 1012 WX | |
United Kingdom | |
Queen Elizabeth Hospital Birmingham | |
Birmingham, United Kingdom, B15 2GW | |
Birmingham Children's Hospital NHS Trust | |
Birmingham, United Kingdom, B4 6NH | |
Clinical Trial Site | |
Wales, United Kingdom, CF484DR |
Responsible Party: | Dicerna Pharmaceuticals, Inc. |
ClinicalTrials.gov Identifier: | NCT03392896 |
Other Study ID Numbers: |
DCR-PHXC-101 |
First Posted: | January 8, 2018 Key Record Dates |
Last Update Posted: | January 22, 2020 |
Last Verified: | January 2020 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | No |
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 |
Hyperoxaluria, Primary Hyperoxaluria Kidney Diseases Urologic Diseases |
Carbohydrate Metabolism, Inborn Errors Metabolism, Inborn Errors Genetic Diseases, Inborn Metabolic Diseases |