Status:
COMPLETED
Study of DCR-PHXC-101 in Normal Healthy Volunteers and Patients With Primary Hyperoxaluria
Lead Sponsor:
Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
Conditions:
Primary Hyperoxaluria
Eligibility:
All Genders
6+ years
Phase:
PHASE1
Brief 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 patie...
Eligibility Criteria
Inclusion
- Group A (HVs) Major
- 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
- 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
Key Trial Info
Start Date :
December 6 2017
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
November 19 2019
Estimated Enrollment :
43 Patients enrolled
Trial Details
Trial ID
NCT03392896
Start Date
December 6 2017
End Date
November 19 2019
Last Update
September 19 2024
Active Locations (7)
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1
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
2
Centre d'Investigation Clinique - CIC 1407 - Hospices Civils de Lyon
Bron, France, 69677
3
Universitätsklinikum Bonn-Institut für Klinische Chemie und Klinische Pharmakologie
Bonn, Germany, 53127
4
University of Amsterdam
Amsterdam, Netherlands, 1012 WX