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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT03392896
Other study ID # DCR-PHXC-101
Secondary ID
Status Completed
Phase Phase 1
First received
Last updated
Start date December 6, 2017
Est. completion date November 19, 2019

Study information

Verified date January 2020
Source Dicerna Pharmaceuticals, Inc.
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

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).


Recruitment information / eligibility

Status Completed
Enrollment 43
Est. completion date November 19, 2019
Est. primary completion date November 19, 2019
Accepts healthy volunteers Accepts Healthy Volunteers
Gender All
Age group 6 Years and older
Eligibility 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 Design


Related Conditions & MeSH terms


Intervention

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
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.

Locations

Country Name City State
France Centre d'Investigation Clinique - CIC 1407 - Hospices Civils de Lyon Bron
Germany Universitätsklinikum Bonn-Institut für Klinische Chemie und Klinische Pharmakologie Bonn
Netherlands University of Amsterdam Amsterdam
United Kingdom Birmingham Children's Hospital NHS Trust Birmingham
United Kingdom Queen Elizabeth Hospital Birmingham Birmingham
United Kingdom Clinical Trial Site Wales
United States Boston Children's Hospital Boston Massachusetts

Sponsors (1)

Lead Sponsor Collaborator
Dicerna Pharmaceuticals, Inc.

Countries where clinical trial is conducted

United States,  France,  Germany,  Netherlands,  United Kingdom, 

Outcome

Type Measure Description Time frame Safety issue
Primary Number of patients with Treatment-Related Adverse Events (TEAEs) Part A (SAD in HVs) screening through Day 29; Part B (SAD in PH patients) screening through Day 57
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