Primary Hyperoxaluria Type 1Phase 1NCT02795325
A Study of DCR-PH1 in Patients With Primary Hyperoxaluria Type 1 (PH1)
A Phase 1 study testing DCR-PH1 in people with Primary Hyperoxaluria Type 1, run by Dicerna Pharmaceuticals, Inc., a Novo Nordisk company at 2 sites in 2 countries.
Trial summary
A phase 1 study of DCR-PH1 in patients with primary hyperoxaluria type 1 (PH1) to determine the safety, tolerability, pharmacokinetic (PK) and pharmacodynamics (PD) effects of DCR-PH1 administered via-intravenous infusion (IV) Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Primary Hyperoxaluria Type 1
- What is being tested?
- DCR-PH1
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 41 participants
- Where
- 2 sitesin 2 countries
- Study dates
- 13 May 2016to 14 Oct 2016
- 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 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Male or female, at least 12 years of age
- Diagnosis of PH1, confirmed by genotyping
- 24-hour urine oxalate excretion as defined in the protocol
- eGFR ≥ 40 mL/min normalized to 1.73 m2 BSA
- Written informed consent for adults (≥18 years old, or per local regulatory requirement); written informed assent for adolescents (12 to <18 years old, or per local regulatory requirement)
Who cannot join
- Prior renal and/or hepatic transplantation
- Participation in any clinical study involving administration of any investigational drug within the 30 days before enrollment
- Pregnancy or lactation at the time of screening or enrollment
- Women of child-bearing potential must have a negative pregnancy test, cannot be breastfeeding and must be willing to use contraception
- Patients with a known history of human immunodeficiency virus (HIV) or active infection with hepatitis B virus or hepatitis C virus
- Moderate to severe liver impairment
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- Liver function test abnormalities: alanine transaminases (ALT) and/or aspartate transaminases (AST) > 2 times upper limit of normal (ULN)
- History of severe reaction to a liposomal product or a known hypersensitivity to lipid products.
- Unable to collect required study samples or follow study procedures
- No clinically significant health concerns
Trial locations and contacts
2 sites in 2 countries.
2 sites in 2 countries
About this study
Official title: A Phase 1 Study of DCR-PH1 in Patients With Primary Hyperoxaluria Type 1 (PH1)
A phase 1 study of DCR-PH1 in patients with primary hyperoxaluria type 1 (PH1) to determine the safety, tolerability, pharmacokinetic (PK) and pharmacodynamics (PD) effects of DCR-PH1 administered via-intravenous infusion (IV)
- Conditions
- Primary Hyperoxaluria Type 1
- Being tested
- DCR-PH1, Placebo
- Keywords
- Primary Hyperoxaluria Type 1, PH1, Calcium Oxalate Stones, Kidney Stones, Liver Enzyme Deficiency, Genetic Diseases, Inborn Kidney Diseases, Metabolic Diseases, Inborn Errors, Urological Diseases, Carbohydrate Metabolism, Hyperoxaluria, AGT, RNAi, siRNA, DCR-PH1
- Medical subject headings
- Primary hyperoxaluria type 1, Kidney Calculi, Genetic Diseases, Inborn, Metabolic Diseases, Urologic Diseases, Hyperoxaluria
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DCR-PH1Drug
IV infusion of DCR-PH1
PlaceboOther
| Study group | Role | What they receive |
|---|---|---|
| PH Patients | Experimental | Not described Receives: Drug: DCR-PH1 |
| Healthy Volunteers | Experimental | Not described Receives: Drug: DCR-PH1; Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
The safety of DCR-PH1 evaluated by the proportion of subjects that experience adverse events (AEs)
Measured: Through Day 29
Secondary outcomes (8)
Profile of pharmacokinetics (PK) of DCR-PH1 - Cmax
Measured: Through Day 29
Profile of pharmacokinetics (PK) of DCR-PH1 - tmax
Measured: Through Day 29
Profile of pharmacokinetics (PK) of DCR-PH1 - AUC
Measured: Through Day 29
Profile of pharmacokinetics (PK) of DCR-PH1 - t½
Measured: Through Day 29
The effect of DCR-PH1 on plasma glycolate levels
Measured: Through Day 29
The effect of DCR-PH1 on urine glycolate levels
Measured: Through Day 29
The effect of DCR-PH1 on plasma oxalate levels
Measured: Through Day 29
The effect of DCR-PH1 on urine oxalate levels
Measured: Through Day 29
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Jun 2016 (estimated)
- Study start13 May 2016
- Primary completion14 Oct 2016
- Study completion14 Oct 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Primary Hyperoxaluria Type 1Studies 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
- Masking
- None (open label)
- Enrolment
- 41 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
- NCT02795325
- Sponsor's protocol ID
- DCR-PH1-101
- Status last verified
- Jul 2024
- First submitted
- 18 May 2016
- Last updated
- 11 Jul 2024
- Results posted
- No
Publications
Martin-Higueras C, Luis-Lima S, Salido E. Glycolate Oxidase Is a Safe and Efficient Target for Substrate Reduction Therapy in a Mouse Model of Primary Hyperoxaluria Type I. Mol Ther. 2016 Apr;24(4):719-25. doi: 10.1038/mt.2015.224. Epub 2015 Dec 22.
PubMed 26689264Milliner DS. siRNA Therapeutics for Primary Hyperoxaluria: A Beginning. Mol Ther. 2016 Apr;24(4):666-7. doi: 10.1038/mt.2016.50. No abstract available.
PubMed 27081720Dutta C, Avitahl-Curtis N, Pursell N, Larsson Cohen M, Holmes B, Diwanji R, Zhou W, Apponi L, Koser M, Ying B, Chen D, Shui X, Saxena U, Cyr WA, Shah A, Nazef N, Wang W, Abrams M, Dudek H, Salido E, Brown BD, Lai C. Inhibition of Glycolate Oxidase With Dicer-substrate siRNA Reduces Calcium Oxalate Deposition in a Mouse Model of Primary Hyperoxaluria Type 1. Mol Ther. 2016 Apr;24(4):770-8. doi: 10.1038/mt.2016.4. Epub 2016 Jan 13.
PubMed 26758691
Study at a glance
From the registry record, last updated by the sponsor on 11 Jul 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1 Study of DCR-PH1 in Patients With Primary Hyperoxaluria Type 1 (PH1)
- ClinicalTrials.gov ID
- NCT02795325
- Acronym / sponsor ID
- DCR-PH1-101
- Conditions
- Primary Hyperoxaluria Type 1
- Keywords
- Primary Hyperoxaluria Type 1, PH1, Calcium Oxalate Stones, Kidney Stones, Liver Enzyme Deficiency, Genetic Diseases, Inborn Kidney Diseases, Metabolic Diseases
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Terminated
- Enrolment
- 41 participants (actual)
- Lead sponsor
- Dicerna Pharmaceuticals, Inc., a Novo Nordisk companyIndustry (pharma, biotech, devices)
- Study start
- 13 May 2016 (actual)
- Primary completion
- 14 Oct 2016 (actual)
- Study completion
- 14 Oct 2016 (actual)
- First posted
- 10 Jun 2016 (estimated)
- 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.
A phase 1 study of DCR-PH1 in patients with primary hyperoxaluria type 1 (PH1) to determine the safety, tolerability, pharmacokinetic (PK) and pharmacodynamics (PD) effects of DCR-PH1 administered via-intravenous infusion (IV)
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| PH Patients | Experimental | Drug: DCR-PH1 |
| Healthy Volunteers | Experimental | Drug: DCR-PH1, Other: Placebo |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Male or female, at least 12 years of age
- Diagnosis of PH1, confirmed by genotyping
- 24-hour urine oxalate excretion as defined in the protocol
- eGFR ≥ 40 mL/min normalized to 1.73 m2 BSA
- Written informed consent for adults (≥18 years old, or per local regulatory requirement); written informed assent for adolescents (12 to <18 years old, or per local regulatory requirement)
Key exclusion criteria (10 listed)
- Prior renal and/or hepatic transplantation
- Participation in any clinical study involving administration of any investigational drug within the 30 days before enrollment
- Pregnancy or lactation at the time of screening or enrollment
- Women of child-bearing potential must have a negative pregnancy test, cannot be breastfeeding and must be willing to use contraception
- Patients with a known history of human immunodeficiency virus (HIV) or active infection with hepatitis B virus or hepatitis C virus
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-PH1IV infusion of DCR-PH1 | Drug | Healthy Volunteers, PH Patients |
| Placebo | Other | Healthy Volunteers |
Primary outcome
The safety of DCR-PH1 evaluated by the proportion of subjects that experience adverse events (AEs)
Time frame: Through Day 29
8 secondary outcomes
Profile of pharmacokinetics (PK) of DCR-PH1 - Cmax
Time frame: Through Day 29
Profile of pharmacokinetics (PK) of DCR-PH1 - tmax
Time frame: Through Day 29
Profile of pharmacokinetics (PK) of DCR-PH1 - AUC
Time frame: Through Day 29
Profile of pharmacokinetics (PK) of DCR-PH1 - t½
Time frame: Through Day 29
The effect of DCR-PH1 on plasma glycolate levels
Time frame: Through Day 29
The effect of DCR-PH1 on urine glycolate levels
Time frame: Through Day 29
The effect of DCR-PH1 on plasma oxalate levels
Time frame: Through Day 29
The effect of DCR-PH1 on urine oxalate levels
Time frame: Through Day 29
Results and safety
The study was stopped early. Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Jul 2024.
- Background: PubMed 26689264Martin-Higueras C, Luis-Lima S, Salido E. Glycolate Oxidase Is a Safe and Efficient Target for Substrate Reduction Therapy in a Mouse Model of Primary Hyperoxaluria Type I. Mol Ther. 2016 Apr;24(4):719-25. doi: 10.1038/mt.2015.224. Epub 2015 Dec 22.
- Background: PubMed 27081720Milliner DS. siRNA Therapeutics for Primary Hyperoxaluria: A Beginning. Mol Ther. 2016 Apr;24(4):666-7. doi: 10.1038/mt.2016.50. No abstract available.
- Background: PubMed 26758691Dutta C, Avitahl-Curtis N, Pursell N, Larsson Cohen M, Holmes B, Diwanji R, Zhou W, Apponi L, Koser M, Ying B, Chen D, Shui X, Saxena U, Cyr WA, Shah A, Nazef N, Wang W, Abrams M, Dudek H, Salido E, Brown BD, Lai C. Inhibition of Glycolate Oxidase With Dicer-substrate siRNA Reduces Calcium Oxalate Deposition in a Mouse Model of Primary Hyperoxaluria Type 1. Mol Ther. 2016 Apr;24(4):770-8. doi: 10.1038/mt.2016.4. Epub 2016 Jan 13.
- Related InfoLink provided by the sponsor.
- Related InfoLink provided by the sponsor.
- Related InfoLink provided by the sponsor.
- Related InfoLink provided by the sponsor.
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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.
2 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing DCR-PH1 in people with Primary Hyperoxaluria Type 1, run by Dicerna Pharmaceuticals, Inc., a Novo Nordisk company at 2 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 10 exclusion criteria. 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: stopped early, as of the sponsor's last update on 11 Jul 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in 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. 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 was stopped early. Results are not available in the source record.
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?
The safety of DCR-PH1 evaluated by the proportion of subjects that experience adverse events (AEs) (time frame: Through Day 29). 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 NCT02795325, which also lists 3 linked publications.
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 11 Jul 2024.