Trial · NCT04042402
Long Term Extension Study in Patients With Primary Hyperoxaluria
NCT04042402, Phase 3, Active not recruiting, Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Long Term Extension Study in Patients With Primary Hyperoxaluria. Trials, record NCT04042402. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04042402
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Long Term Extension Study in Patients With Primary Hyperoxaluria
- NCT number
- NCT04042402
- Status
- Active not recruiting
- Phases
- Phase 3
- Enrollment
- 75 participants, estimated
- Lead sponsor
- Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
- Conditions
- Primary Hyperoxaluria Type 1 (PH1), Primary Hyperoxaluria Type 2 (PH2), Kidney Diseases, Urologic Diseases, Genetic Disease, Primary Hyperoxaluria Type 3 (PH3)
- Interventions
- DCR-PHXC
- First posted
- 2019-08-02
- Last updated
- 2026-10-08
Source ClinicalTrials.gov
Registered outcomes25
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | The annual rate of decline in eGFR in participants with PH1 | Annual change from baseline | To evaluate the effect of DCR PHXC on estimated glomerular filtration rate (eGFR) in participants with PH1 |
| Secondary | The incidence and severity of treatment-emergent adverse events (TEAE) and SAEs associated with abnormal 12 lead electrocardiogram (ECG) readings | TEAEs and SAEs are evaluated monthly for 6 years | To evaluate the safety and tolerability of DCR PHXC when administered monthly to patients with primary hyperoxaluria (PH) via change from baseline and abnormal ECG findings. Standard 12-lead ECGs will be performed in the supine position after the subject has rested comfortably for 10 minutes. The parameters assessed will be rhythm, ventricular rate, PR interval, QRS duration, QT interval, and corrected QT interval (QTcF, Fridericia correction). The Investigator or designee is responsible for reviewing the ECG(s) to assess whether the results are within normal limits and to determine the clinical significance of the results. Standardized ECG acquisition equipment will be provided to all clinical trial sites at the start of the trial, to ensure parity across all sites. |
| Secondary | The incidence and severity of treatment-emergent adverse events (TEAE) and SAEs associated with abnormal physical examination findings | TEAEs and SAEs are evaluated monthly for 6 years | To evaluate the safety and tolerability of DCR PHXC when administered monthly to patients with primary hyperoxaluria (PH) via change from baseline and incidence of abnormal physical exam findings. A full physical examination will include a complete review of body systems: eyes, ears, nose, and throat, chest/respiratory, heart/cardiovascular, gastrointestinal/liver, musculoskeletal/extremities, dermatological/skin, thyroid/neck, lymph nodes, and neurological. A full physical exam is done at Screening, Day 180 and if a participant ends the study early. A brief physical examination will minimally include chest/respiratory, heart/cardiovascular, dermatological/skin, and gastrointestinal/liver. A brief physical examination will be performed at the Investigator's discretion at all other visits. |
| Secondary | The incidence and severity of treatment-emergent adverse events (TEAE) and SAEs associated with abnormal vital signs | TEAEs and SAEs are evaluated monthly for 6 years | To evaluate the safety and tolerability of DCR PHXC when administered monthly to patients with primary hyperoxaluria (PH) via the change from baseline and incidence of abnormal vital signs. Vital signs include blood pressure, pulse/heart rate, oral body temperature, and respiratory rate. Parameters will be measured in the supine position, using an automated instrument or manually, after the participant has rested comfortably for 10 minutes. In the pediatric population, an age-appropriate cuff size should be used for blood pressure measurements. Temperature will be obtained in degrees Celsius (°C), pulse rate will be counted for a full minute and recorded in beats per minute, and respirations will be counted for a full minute and recorded in breaths per minute. |
| Secondary | The incidence and severity of treatment-emergent adverse events (TEAE) and SAEs related to abnormal clinical laboratory tests (hematology, chemistry, coagulation parameters, and urinalysis) | TEAEs and SAEs are evaluated monthly for 6 years | To evaluate the safety and tolerability of DCR PHXC when administered monthly to patients with primary hyperoxaluria (PH) via the change from baseline and incidence of abnormal clinical laboratory tests. |
| Secondary | To identify the proportion of participants with normalized or near-normalized 24 hour urinary oxalate (Uox) | 24 hour urine collections (if applicable) are performed monthly for 6 months (or quarterly for PH1 multidose rollovers), quarterly for 2 1/2 years (or monthly for PH2/PH3 multidose rollovers until Month 12), and every 6 months for 3 years after that. | The proportion of participants with a 24 hour Uox level (\< 0.46 mmol/24 hours or ≥ 0.46 - \< 0.60 mmol/24 hours \[adjusted per 1.73 m2 body surface area (BSA) in participants aged \< 18 years\]) at each assessment time point throughout the study in PH1, PH2, and PH3 participant subgroups |
| Secondary | To identify the percentage of participants with spot urinary oxalate-to-creatinine ratio ≤ the ULN or ≤ 1.5 x ULN | Spot urine collections are performed monthly for 6 months (or quarterly for PH1 multidose rollovers), quarterly for 2 1/2 years (or monthly for PH2/PH3 multidose rollovers until Month 12), and every 6 months for 3 years after that. | The percentage of participants with spot urinary oxalate-to-creatinine ratio ≤ the ULN or ≤ 1.5 x ULN at each assessment time point throughout the study in PH1, PH2, and PH3 participant subgroups |
| Secondary | To assess the effect of DCR-PHXC on stone events in patients with PH | Evaluated yearly for 6 years | Change from Baseline in the number of stone events over a 12-month period, annually in Year 1, Year 2, etc. in PH1, PH2, and PH3 participant subgroups |
| Secondary | To assess the effect of DCR-PHXC on stone burden grade in patients with PH | Evaluated yearly for 6 years | Change from Baseline in the stone burden grade at Year 1, Year 2, etc. in PH1, PH2, and PH3 participant subgroups |
| Secondary | To assess the effect of DCR-PHXC in nephrocalcinosis grade in patients with PH | Evaluated yearly for 6 years | Change from Baseline in nephrocalcinosis grade at Year 1, Year 2, etc. in PH1, PH2, and PH3 participant subgroups |
1 to 10 of 25Source ClinicalTrials.gov
See all 25 registered outcomesOutcomes0
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
None in this release.
Results by group0
Each measurement the trial posted, group by group, in the words and numbers it posted.
None in this release.
Participant flow0
How many started, completed or left each group, period by period.
None in this release.
Adverse events0
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
None in this release.
Adverse event totals0
The totals the trial posted for each group.
None in this release.
Papers2
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT04042402
GET https://conductscience.org/api/trials/v1/NCT04042402/registered?page=2