Primary HyperoxaluriaPhase 1NCT03392896
Study of DCR-PHXC-101 in Normal Healthy Volunteers and Patients With Primary Hyperoxaluria
A Phase 1 study testing DCR-PHXC in people with Primary Hyperoxaluria, run by Dicerna Pharmaceuticals, Inc., a Novo Nordisk company at 7 sites in 5 countries.
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. Read more about this study
- Who is it for?
- Ages 6 years and olderHealthy volunteers can take part
- Conditions the study is for
- Primary Hyperoxaluria
- What is being tested?
- DCR-PHXC
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 43 participants
- Where
- 7 sitesin 5 countries
- Study dates
- 6 Dec 2017to 19 Nov 2019
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 years and older
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
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.
Trial locations and contacts
7 sites in 5 countries.
7 sites in 5 countries
United Kingdom3 sites
Queen Elizabeth Hospital Birmingham
Birmingham, B15 2GW
Birmingham Children's Hospital NHS Trust
Birmingham, B4 6NH
Clinical Trial Site
Wales, CF484DR
United States1 site
Boston Children's Hospital
Boston, Massachusetts, 02115
France1 site
Centre d'Investigation Clinique - CIC 1407 - Hospices Civils de Lyon
Bron, 69677
Germany1 site
Universitätsklinikum Bonn-Institut für Klinische Chemie und Klinische Pharmakologie
Bonn, 53127
About this study
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)
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).
- Conditions
- Primary Hyperoxaluria
- Being tested
- DCR-PHXC, Placebo
- Medical subject headings
- Hyperoxaluria, Primary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DCR-PHXCDrug
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.
PlaceboDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| Group A Active (DCR-PHXC) | Experimental | HVs, single ascending doses of DCR-PHXC. Receives: Drug: DCR-PHXC |
| Group A Placebo | Placebo comparator | HVs, normal saline 0.9% injection to match active doses. Receives: Drug: Placebo |
| Group B Active (DCR-PHXC) | Experimental | PH1 and PH2 patients, open label, single ascending doses of DCR-PHXC. Receives: Drug: DCR-PHXC |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of patients with Treatment-Related Adverse Events (TEAEs)
Measured: Part A (SAD in HVs) screening through Day 29; Part B (SAD in PH patients) screening through Day 57
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Jan 2018
- Study start6 Dec 2017
- Primary completion19 Nov 2019
- Study completion19 Nov 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Primary HyperoxaluriaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- 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 blind
- Who is masked
- Participant, Investigator
- Enrolment
- 43 participants (actual)
Sponsor and investigators
- Lead sponsor
- Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03392896
- Sponsor's protocol ID
- DCR-PHXC-101
- Status last verified
- Sep 2024
- First submitted
- 21 Dec 2017
- Last updated
- 19 Sep 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Zhang S, Gamallo P, Rawson V. Population Pharmacokinetic and Pharmacodynamic Modelling and Simulation for Nedosiran Clinical Development and Dose Guidance in Pediatric Patients with Primary Hyperoxaluria Type 1. Clin Pharmacokinet. 2025 Sep;64(9):1395-1411. doi: 10.1007/s40262-025-01540-1. Epub 2025 Jul 2.
PubMed 40601241
Study at a glance
From the registry record, last updated by the sponsor on 19 Sep 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT03392896
- Acronym / sponsor ID
- DCR-PHXC-101
- Conditions
- Primary Hyperoxaluria
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 43 participants (actual)
- Lead sponsor
- Dicerna Pharmaceuticals, Inc., a Novo Nordisk companyIndustry (pharma, biotech, devices)
- Study start
- 6 Dec 2017 (actual)
- Primary completion
- 19 Nov 2019 (actual)
- Study completion
- 19 Nov 2019 (actual)
- First posted
- 8 Jan 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 3
- 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).
| Arm | Type | Receives |
|---|---|---|
| Group A Active (DCR-PHXC) | Experimental | Drug: DCR-PHXCHVs, single ascending doses of DCR-PHXC. |
| Group A Placebo | Placebo comparator | Drug: PlaceboHVs, normal saline 0.9% injection to match active doses. |
| Group B Active (DCR-PHXC) | Experimental | Drug: DCR-PHXCPH1 and PH2 patients, open label, single ascending doses of DCR-PHXC. |
Who may qualify
- Ages
- 6 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| DCR-PHXCDCR-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. | Drug | Group A Active (DCR-PHXC), Group B Active (DCR-PHXC) |
| PlaceboSingle 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. | Drug | Group A Placebo |
Primary outcome
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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Sep 2024.
- Derived from this study: PubMed 40601241Zhang S, Gamallo P, Rawson V. Population Pharmacokinetic and Pharmacodynamic Modelling and Simulation for Nedosiran Clinical Development and Dose Guidance in Pediatric Patients with Primary Hyperoxaluria Type 1. Clin Pharmacokinet. 2025 Sep;64(9):1395-1411. doi: 10.1007/s40262-025-01540-1. Epub 2025 Jul 2.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
7 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing DCR-PHXC in people with Primary Hyperoxaluria, run by Dicerna Pharmaceuticals, Inc., a Novo Nordisk company at 7 sites in 5 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 19 Sep 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in United Kingdom, United States, France, Germany, Netherlands. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03392896, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Sep 2024.