| Trial ID: | L1307 |
| Source ID: | NCT04580420
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| Associated Drug: |
Dcr-Phxc
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| Title: |
Safety & Efficacy of DCR-PHXC in Patients With PH1 and ESRD
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| Acronym: |
PHYOX7
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| Status: |
RECRUITING
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| Study Results: |
NO
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| Results: |
|
| Conditions: |
Primary Hyperoxaluria Type 1|End Stage Renal Disease
|
| Interventions: |
DRUG: DCR-PHXC
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| Outcome Measures: |
Primary: Safety: Incidence of Events, To assess the efficacy of DCR-PHXC in lowering Pox in participants with PH1 and severe renal impairment, with or without hemodialysis or peritoneal dialysis., 180 days|Safety: Incidence of Events, Characterize the safety of DCR-PHXC in participants with PH1 or PH2 and severe renal impairment, with or without dialysis., 180 days | Secondary: Change from Baseline in Plasma Oxalate Concentration, To evaluate the effect of DCR-PHXC on Plasma Oxalate concentration from Baseline to day 180, 180 Days|To characterize the PK of DCR PHXC in patients with PH by observing secondary parameters of the area under the curve., Population and individual pharmacokinetic (PK) parameters for DCR PHXC, including secondary parameters of area under the curve (AUC), 180 days|To characterize the PK of DCR PHXC in patients with PH by observing maximum observed concentration (Cmax)., Population and individual pharmacokinetic (PK) parameters for DCR PHXC, including maximum observed concentration (Cmax), 180 days|To characterize the PK of DCR PHXC in patients with PH by observing minimum concentration (Cmin)., Population and individual pharmacokinetic (PK) parameters for DCR PHXC, including minimum observed concentration (Cmin), 180 days|To characterize the PK of DCR PHXC in patients with PH by observing maximum concentration (Tmax)., Population and individual pharmacokinetic (PK) parameters for DCR PHXC, including time to maximum concentration (Tmax), 180 days|To characterize the PK of DCR PHXC in patients with PH by observing terminal elimination half-life (t1/2)., Population and individual pharmacokinetic (PK) parameters for DCR PHXC, including terminal elimination half-life (t1/2), 180 days|Change in Frequency of Dialysis, Change from Baseline in the frequency of dialysis over 180 days, 180 days|Change in Duration of Dialysis, Change from Baseline in the duration of dialysis over 180 days, 180 days | Other: Change from Baseline in the Short Form (36) Health Survey (SF-36®) in adults, To evaluate the effect of DCR-PHXC on Quality of Life (QoL) assessments in patients with PH. The SF 36 is a set of generic, coherent, and easily administered quality-of-life measures that taps 8 health concepts: physical functioning, bodily pain, role limitations due to physical health problems, role limitations due to personal or emotional problems, emotional well-being, social functioning, energy/fatigue, and general health perceptions. It also includes a single item that provides an indication of perceived change in health. The 36 items are identical to the MOS SF 36 described in Ware and Sherbourne (1992). Participants respond to each item on a categorical scale. Categorical answers are transformed to a 0 to 100 range so that the lowest and highest possible scores are 0 and 100, respectively. All items are scored so that a high score defines a more favorable health state, 180 days|Change from Baseline in the EQ-5D-5L™ in adults, To evaluate the effect of DCR-PHXC on Quality of Life (QoL) assessments in patients with PH. The EQ-5D-5L consists of the EQ 5D descriptive system and the EQ visual analogue scale (EQ VAS). The descriptive system has 5 dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each dimension has 5 levels: no problems, slight problems, moderate problems, severe problems and extreme problems. The digits for the 5 dimensions can be combined into a 5-digit number that describes the participant's health state. The EQ VAS records the participant's self-rated health on a 20-cm vertical VAS, where the endpoints are labelled 'The best health you can imagine' and 'The worst health you can imagine.' Participants are asked to place an "X" on the line that represents their health on that day., 180 days|Change from Baseline in the Pediatric Quality of Life Inventory (PedsQL™) in children, To evaluate the effect of DCR-PHXC on Quality of Life (QoL) assessments in patients with PH. The 23-item PedsQL is comprised of 5 items in the Emotional, Social, and School Functioning dimensions (Psychosocial Health) and 8 items in the Physical Functioning (Physical Health) dimension. Items are reverse-scored on a 0 to 4 Likert scale and linearly transformed to a 0 to 100 scale, so that higher scores indicate better functioning and HRQOL. Scale Scores are the sum of the items in each dimension, divided by the number of items answered., 180 days|Changes from Baseline number of kidney stones, Evaluate the effect of DCR-PHXC on stone burden in participants with PH1 or PH2 and severe renal impairment through Kidney Ultrasound, 180 days|Changes from Baseline size of kidney stones, Evaluate the effect of DCR-PHXC on stone burden in participants with PH1 or PH2 and severe renal impairment through Kidney Ultrasound, 180 days
|
| Sponsor/Collaborators: |
Sponsor: Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
|
| Gender: |
ALL
|
| Age: |
CHILD, ADULT, OLDER_ADULT
|
| Phases: |
PHASE2
|
| Enrollment: |
28
|
| Study Type: |
INTERVENTIONAL
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| Study Designs: |
Allocation: NA|Intervention Model: SEQUENTIAL|Masking: NONE|Primary Purpose: TREATMENT
|
| Start Date: |
2021-04-15
|
| Completion Date: |
2032-01-30
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| Results First Posted: |
|
| Last Update Posted: |
2024-11-07
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| Locations: |
Clinical Trial Site, San Francisco, California, 94143, United States|Clinical Trial Site, Boston, Massachusetts, 02115, United States|Clinical Trial Site, Rochester, Minnesota, 55905, United States|Clinical Trial Site, New York, New York, 10016, United States|Clinical Trial Site, Bron, 69677, France|Clinical Trial Site, Paris, 75019, France|Clinical Trial Site, Bonn, 53127, Germany|Clinical Trial Site, Heidelberg, 69120, Germany|Clinical Trial Site, Roma, 00165, Italy|Clinical Trial Site, Beirut, 00001, Lebanon|Clinical Trial Site, Casablanca, 2025, Morocco|Clinical Trial Site, Oradea, 410469, Romania|Clinical Trial Site, Oradea, 410562, Romania|Clinical Trial Site, Barcelona, 08035, Spain|Clinical Trial Site, Santa Cruz De Tenerife, 38320, Spain|Clinical Trial Site, Dubai, +971, United Arab Emirates|Clinical Trial Site, London, NWG 2Q3, United Kingdom|Clinical Trial Site, London, WC1N3JH, United Kingdom
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| URL: |
https://clinicaltrials.gov/show/NCT04580420
|
