Trial · NCT03847909
A Study to Evaluate DCR-PHXC in Children and Adults With Primary Hyperoxaluria Type 1 and Primary Hyperoxaluria Type 2
NCT03847909, Phase 2, Completed, Novo Nordisk A/S; results first posted 2024-05-22
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Study to Evaluate DCR-PHXC in Children and Adults With Primary Hyperoxaluria Type 1 and Primary Hyperoxaluria Type 2. Trials, record NCT03847909. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03847909
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study to Evaluate DCR-PHXC in Children and Adults With Primary Hyperoxaluria Type 1 and Primary Hyperoxaluria Type 2
- NCT number
- NCT03847909
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 35 participants, actual
- Lead sponsor
- Novo Nordisk A/S
- Conditions
- Primary Hyperoxaluria Type 1 (PH1), Primary Hyperoxaluria Type 2 (PH2), Kidney Diseases, Urologic Diseases, Genetic Disease
- Interventions
- DCR-PHXC, Sterile Normal Saline (0.9% NaCl)
- First posted
- 2019-02-20
- Last updated
- 2024-05-22
- Results first posted
- 2024-05-22
Source ClinicalTrials.gov
Registered outcomes37
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | AUC From Day 90 To Day 180, Based on Percent Change From Baseline in 24-Hour Uox | From Day 90 to 180 | The AUC of 24-hour urinary oxalate (Uox) from Day 90 to Day 180, based on percent change from baseline, was compared between the active treatment group and placebo group. A multiple imputation approach was used to handle missing Uox data and then calculate the AUC. |
| Secondary | Percentage of Participants Whose 24-hour Uox Values Normalized or Near-normalized on at Least 2 Consecutive Visits | From Day 90 to 180 | Percentage of participants whose 24-hour Uox values normalized or near-normalized on at least 2 consecutive visits are presented. Normalization of Uox was defined as less than (\<) 0.46 millimole per 24 hours (mmol/24 hours) and near normalization was defined as greater than or equal to (\>=) 0.46 to \< 0.60 mmol/24 hours (values adjusted per 1.73 square meter \[1.73 m\^2\] body surface area \[BSA\] in participants aged \<18 years). |
| Secondary | Percent Change From Baseline to Day 180 in the Summed Surface Area of Kidney Stones | Baseline, Day 180 | Percent change from baseline to Day 180 in the summed surface area measured in millimetre square (mm\^2) of kidney stones is presented. |
| Secondary | Percent Change From Baseline to Day 180 in the Number of Kidney Stones | Baseline, Day 180 | Percent change from baseline to Day 180 in the number of kidney stones is presented. |
| Secondary | Percent Change From Baseline to Day 180 in Plasma Oxalate (For Adults Only) | Baseline, Day 180 | Percent change from baseline to Day 180 in plasma oxalate (for adults only) is presented. |
| Secondary | Rate of Change in Estimated Glomerular Filtration Rate (eGFR) From Baseline to Day 180 | Baseline, Day 180 | Monthly rate of eGFR change is presented. eGFR was calculated using Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) and creatinine-based equation. |
| Secondary | Number of Treatment Emergent Adverse Events (TEAEs) And Serious Treatment Emergent Adverse Events (TEAEs) | From Baseline up to Day 180 | Number of TEAEs and TESAEs are presented. An adverse event (AE) is any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. An SAE was defined as any untoward medical occurrence that, at any dose: results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalisation, results in persistent disability/incapacity or is a congenital anomaly/birth defect. TEAE was defined as any AE with an onset date/time on or after administration (including any partial administration) of the first dose of study intervention and through the study completion date from the end of study case report form (CRF). |
| Secondary | Change From Baseline in Electrocardiogram (ECG): Heart Rate | Baseline, Day 180 | Change from baseline in heart rate is presented. |
| Secondary | Change From Baseline in ECG: PR Interval, QRS Duration, QT Interval, QTcB Interval, QTcF Interval and RR Interval | Baseline, Day 180 | Change from baseline in PR interval, QRS duration, QT interval, QTcB interval, QTcF interval and RR interval is presented. |
| Secondary | Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination | Baseline up to Day 180 | Number of participants who had most abnormal post-baseline shift in physical examination are presented. Physical examination shifts were categories into 4 categories: 1) missing; 2) normal; 3) abnormal-not clinically significant (NCS) and 4) abnormal-clinically significant (CS). Each category was presented according body systems including: 1) eyes, ears, nose and throat; 2) chest/respiratory; 3) heart/cardiovascular; 4) gastrointestinal/liver; 5) musculoskeletal/extremities; 6) dermatological/skin; 7) thyroid/neck; 8) lymph nodes; 9) neurological. |
1 to 10 of 37Source ClinicalTrials.gov
See all 37 registered outcomesOutcomes37
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | AUC From Day 90 To Day 180, Based on Percent Change From Baseline in 24-Hour Uox | From Day 90 to 180 | MITT population included all participants in the ITT population who had at least one efficacy assessment after the Day 90 dosing visit, where the ITT population included all participants who were randomized and had at least one post-baseline efficacy assessment. | Percent change in 24-hour Uox AUC | LEAST_SQUARES_MEAN |
| SECONDARY | Percentage of Participants Whose 24-hour Uox Values Normalized or Near-normalized on at Least 2 Consecutive Visits | From Day 90 to 180 | Modified Intent-to-treat population (MITT) included all participants in the intent-to-treat (ITT) population who had at least one efficacy assessment after the Day 90 dosing visit where ITT population included all participants who were randomized and had at least one post-baseline efficacy assessment. | Percentage of participants | NUMBER |
| SECONDARY | Percent Change From Baseline to Day 180 in the Summed Surface Area of Kidney Stones | Baseline, Day 180 | The ITT population included all participants who were randomised and had at least one post-baseline efficacy assessment. Number of participants analysed signifies participants with available data. | Percent change in summed surface area | MEDIAN |
| SECONDARY | Percent Change From Baseline to Day 180 in the Number of Kidney Stones | Baseline, Day 180 | The ITT population included all participants who were randomised and had at least one post-baseline efficacy assessment. Number of participants analysed signifies participants with available data. | Percent change in kidney stone numbers | MEDIAN |
| SECONDARY | Percent Change From Baseline to Day 180 in Plasma Oxalate (For Adults Only) | Baseline, Day 180 | Analysis population included all adult participants from ITT population who were randomised and had at least one post-baseline efficacy assessment. Number of participants analysed signifies participants with available data. | Percent change in plasma oxalate | MEDIAN |
| SECONDARY | Rate of Change in Estimated Glomerular Filtration Rate (eGFR) From Baseline to Day 180 | Baseline, Day 180 | The ITT population included all participants who were randomised and had at least one post-baseline efficacy assessment. | mL/minute/1.73 m^2 | MEAN |
| SECONDARY | Number of Treatment Emergent Adverse Events (TEAEs) And Serious Treatment Emergent Adverse Events (TEAEs) | From Baseline up to Day 180 | The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. | Events | NUMBER |
| SECONDARY | Change From Baseline in Electrocardiogram (ECG): Heart Rate | Baseline, Day 180 | The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. Number of participants analysed signifies participants with available data. | beats per minute (beats/min) | MEAN |
| SECONDARY | Change From Baseline in ECG: PR Interval, QRS Duration, QT Interval, QTcB Interval, QTcF Interval and RR Interval | Baseline, Day 180 | The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. Number of participants analysed signifies participants with available data. | milliseconds (msec) | MEAN |
| SECONDARY | Number of Participants With Most Abnormal Post-Baseline Shift in Physical Examination | Baseline up to Day 180 | The safety population included all participants randomly assigned to study intervention and who took at least 1 partial or full dose of study intervention. | Participants | COUNT_OF_PARTICIPANTS |
1 to 10 of 37Source ClinicalTrials.gov
See all 37 outcomesResults by group198
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| AUC From Day 90 To Day 180, Based on Percent Change From Baseline in 24-Hour Uox | DCR-PHXC | 3507.4 | Standard Error 788.49 | Percent change in 24-hour Uox AUC | |
| AUC From Day 90 To Day 180, Based on Percent Change From Baseline in 24-Hour Uox | Placebo | -1664.4 | Standard Error 1189.96 | Percent change in 24-hour Uox AUC | |
| Percentage of Participants Whose 24-hour Uox Values Normalized or Near-normalized on at Least 2 Consecutive Visits | DCR-PHXC | 50 | Percentage of participants | ||
| Percentage of Participants Whose 24-hour Uox Values Normalized or Near-normalized on at Least 2 Consecutive Visits | Placebo | 0 | Percentage of participants | ||
| Percent Change From Baseline to Day 180 in the Summed Surface Area of Kidney Stones | DCR-PHXC | -2.13 | Full Range -100.0 to 228.6 | Percent change in summed surface area | |
| Percent Change From Baseline to Day 180 in the Summed Surface Area of Kidney Stones | Placebo | 21.77 | Full Range -24.3 to 1500.0 | Percent change in summed surface area | |
| Percent Change From Baseline to Day 180 in the Number of Kidney Stones | DCR-PHXC | 0.00 | Full Range -100.0 to 200.0 | Percent change in kidney stone numbers | |
| Percent Change From Baseline to Day 180 in the Number of Kidney Stones | Placebo | 0.00 | Full Range -94.1 to 100.0 | Percent change in kidney stone numbers | |
| Percent Change From Baseline to Day 180 in Plasma Oxalate (For Adults Only) | DCR-PHXC | -25.00 | Full Range -61.9 to 22.2 | Percent change in plasma oxalate | |
| Percent Change From Baseline to Day 180 in Plasma Oxalate (For Adults Only) | Placebo | -0.00 | Full Range -25.0 to 350.0 | Percent change in plasma oxalate |
1 to 10 of 198Source ClinicalTrials.gov
See all 198 results by groupParticipant flow12
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | DCR-PHXC | 23 | |
| Overall Study | STARTED | Placebo | 12 | |
| Overall Study | Safety Population | DCR-PHXC | 23 | |
| Overall Study | Safety Population | Placebo | 12 | |
| Overall Study | Intent to Treat (ITT) Population | DCR-PHXC | 23 | |
| Overall Study | Intent to Treat (ITT) Population | Placebo | 12 | |
| Overall Study | Modified ITT (MITT) Population | DCR-PHXC | 22 | |
| Overall Study | Modified ITT (MITT) Population | Placebo | 12 | |
| Overall Study | COMPLETED | DCR-PHXC | 22 | |
| Overall Study | COMPLETED | Placebo | 11 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 participant flowAdverse events92
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Injection site erythema | General disorders | DCR-PHXC | 5 | 23 | |
| other | Injection site erythema | General disorders | Placebo | 0 | 12 | |
| other | Injection site pain | General disorders | DCR-PHXC | 2 | 23 | |
| other | Injection site pain | General disorders | Placebo | 0 | 12 | |
| other | Fatigue | General disorders | DCR-PHXC | 1 | 23 | |
| other | Fatigue | General disorders | Placebo | 2 | 12 | |
| other | Dysmenorrhoea | Reproductive system and breast disorders | DCR-PHXC | 2 | 23 | |
| other | Dysmenorrhoea | Reproductive system and breast disorders | Placebo | 0 | 12 | |
| other | Menstrual disorder | Reproductive system and breast disorders | DCR-PHXC | 0 | 23 | |
| other | Menstrual disorder | Reproductive system and breast disorders | Placebo | 1 | 12 |
1 to 10 of 92Source ClinicalTrials.gov
See all 92 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | DCR-PHXC | 0 | 23 |
| Total, all-cause mortality | Placebo | 0 | 12 |
| Total, other adverse events | DCR-PHXC | 16 | 23 |
| Total, other adverse events | Placebo | 10 | 12 |
| Total, serious adverse events | DCR-PHXC | 1 | 23 |
| Total, serious adverse events | Placebo | 2 | 12 |
Source ClinicalTrials.gov
Papers3
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 comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| DCR-PHXC vs Placebo: deaths from any cause | Trial comparisons |
| DCR-PHXC vs Placebo: serious adverse events | Trial comparisons |
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/NCT03847909
GET https://conductscience.org/api/trials/v1/NCT03847909/registered?page=2