Study of DCR-PHXC-101 in Normal Healthy Volunteers and Patients With Primary Hyperoxaluria (NCT03392896) | Clinical Trial Compass
CompletedPhase 1
Study of DCR-PHXC-101 in Normal Healthy Volunteers and Patients With Primary Hyperoxaluria
United States, France, Germany43 participantsStarted 2017-12-06
Plain-language 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).
Who can participate
Age range
6 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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:
*…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Number of patients with Treatment-Related Adverse Events (TEAEs)
Timeframe: Part A (SAD in HVs) screening through Day 29; Part B (SAD in PH patients) screening through Day 57
Trial details
NCT IDNCT03392896
SponsorDicerna Pharmaceuticals, Inc., a Novo Nordisk company