Study of DCR-PHXC-101 in Normal Healthy Volunteers and Patients With Primary Hyperoxaluria

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1

Total Enrollment

43 participants

Study Classification

INTERVENTIONAL

Study Start Date

2017-12-06

Study Completion Date

2019-11-19

Brief Summary

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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).

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Detailed Description

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Conditions

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Primary Hyperoxaluria

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Two-arm (active and placebo), single-blind, SAD period (Group A, HVs) followed by open-label, SAD period (Group B, PH1 and PH2 patients).

Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Participants Investigators

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.

Study Groups

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Group A Active (DCR-PHXC)

HVs, single ascending doses of DCR-PHXC.

Group Type EXPERIMENTAL

DCR-PHXC

Intervention Type DRUG

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.

Group A Placebo

HVs, normal saline 0.9% injection to match active doses.

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

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.

Group B Active (DCR-PHXC)

PH1 and PH2 patients, open label, single ascending doses of DCR-PHXC.

Group Type EXPERIMENTAL

DCR-PHXC

Intervention Type DRUG

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.

Interventions

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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.

Intervention Type 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.

Intervention Type DRUG

Eligibility Criteria

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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.

* 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.

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.

* 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.

Minimum Eligible Age

6 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

Yes