nedosiran for Primary Hyperoxaluria

Phase-Based Progress Estimates
1
Effectiveness
2
Safety
Clinical Research Site, Hamilton, CanadaPrimary Hyperoxaluria+3 Morenedosiran - Drug
Eligibility
< 18
All Sexes
What conditions do you have?
Select

Study Summary

This trial will study the effects of nedosiran in children with Primary Hyperoxaluria, a kidney disease.

Eligible Conditions
  • Primary Hyperoxaluria
  • Primary Hyperoxaluria Type 3
  • Primary Hyperoxaluria Type 1
  • Primary Hyperoxaluria Type 2

Treatment Effectiveness

Effectiveness Progress

1 of 3

Similar Trials

1
NOV-001
1
Moderately High Oxalate (MOx) Diet
1
FB-001 Healthy Volunteer

Study Objectives

2 Primary · 24 Secondary · Reporting Duration: 180 days

180 days
Change from Baseline in Plasma Oxalate Concentration
Change from Baseline number of kidney stones
Change from baseline PedsQL™
Change from baseline WPAI
Efficacy: Absolute change in urinary oxalate to creatinine ratio
Efficacy: Percent change in urinary oxalate to creatinine ratio
Efficacy: eGFR changes
Safety: Changes from baseline in ECG: PR interval
Safety: Changes from baseline in ECG: QRS duration
Safety: Changes from baseline in ECG: QT interval
Safety: Changes from baseline in ECG: Rhythm
Safety: Changes from baseline in ECG: Ventricular Rate
Safety: Changes from baseline in Vitals: blood pressure
Safety: Changes from baseline in Vitals: pulse rate
Safety: Changes from baseline in Vitals: respiratory rate
Safety: Changes from baseline in Vitals: temperature
Safety: Changes from baseline: Labs - Antibody
Safety: Changes from baseline: Labs - Clinical Chemistry
Blood coagulation tests
Safety: Changes from baseline: Labs - Hematology
Safety: Changes from baseline: Labs - Plasma Oxalate
Safety: Changes from baseline: Labs - Urinalysis
Safety: Changes from baseline: Labs - Urine Oxalate
Safety: Changes from baseline: Physical Exam
Safety: Incidence of Events
To characterize the PK of DCR PHXC in patients with PH by observing maximum concentration (Tmax).
Dacryocystorhinostomy
To characterize the PK of DCR PHXC in patients with PH by observing minimum concentration (Cmin).
Dacryocystorhinostomy
To characterize the PK of DCR PHXC in patients with PH by observing terminal elimination half-life (t1/2).

Trial Safety

Safety Progress

2 of 3
This is further along than 68% of similar trials

Similar Trials

1
NOV-001
1
Moderately High Oxalate (MOx) Diet
1
FB-001 Healthy Volunteer

Trial Design

1 Treatment Group

Open-Label DCR-PHXC
1 of 1

Experimental Treatment

20 Total Participants · 1 Treatment Group

Primary Treatment: nedosiran · No Placebo Group · Phase 2

Open-Label DCR-PHXC
Drug
Experimental Group · 1 Intervention: nedosiran · Intervention Types: Drug

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: 180 days

Who is running the clinical trial?

Dicerna Pharmaceuticals, Inc.Lead Sponsor
14 Previous Clinical Trials
512 Total Patients Enrolled
2 Trials studying Primary Hyperoxaluria
60 Patients Enrolled for Primary Hyperoxaluria
Alexandra Haagensen, MD, MBAStudy ChairDicerna Pharmaceuticals, Inc.
3 Previous Clinical Trials
53 Total Patients Enrolled
1 Trials studying Primary Hyperoxaluria
17 Patients Enrolled for Primary Hyperoxaluria
Sarb Shergill, PhDStudy DirectorDicerna Pharmaceuticals, Inc.
2 Previous Clinical Trials
669 Total Patients Enrolled
1 Trials studying Primary Hyperoxaluria
17 Patients Enrolled for Primary Hyperoxaluria

Eligibility Criteria

Age < 18 · All Participants · 15 Total Inclusion Criteria

Mark “Yes” if the following statements are true for you:
Both males and females can participate.
References

Frequently Asked Questions

How many participants can potentially join this trial?

"Affirmative, the information available on clinicaltrials.gov indicates that this study is still in progress. Initially posted on February 22nd 2022 and recently modified on November 30th 2022, it aims to recruit 20 people from 3 different medical centres." - Anonymous Online Contributor

Unverified Answer

To what extent is nedosiran hazardous to human health?

"Nedosiran has been assigned a score of 2, as there is evidence to suggest it is safe but no clinical trials have yet tested its efficacy." - Anonymous Online Contributor

Unverified Answer

What are the primary aims of this clinical research project?

"This clinical trial is scheduled to last for 180 days and will measure the efficacy of a particular medical intervention via % change in urinary oxalate-creatinine ratio. Secondary goals include characterizing safety using changes from baseline in laboratory tests, blood pressure readings, and individual pharmacokinetic parameters (AUC)." - Anonymous Online Contributor

Unverified Answer

Are there still opportunities for applicants to join this medical experiment?

"Affirmative. The information on clinicaltrials.gov reveals that the trial is actively seeking participants; this study, which was initially posted on February 22nd 2022, has been recently modified as of November 30th 2022." - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

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