Primary Objective• To collect data regarding changes in oxalate, glycolate, and other metabolites over time in subjects with primary hyperoxaluria type 1 (PH1) who may be considered for clinical trials of DCR PH1Secondary Objectives• To collect data…
ID
Source
Brief title
Condition
- Other condition
Synonym
Health condition
autosomal recessive disorders
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
N.A.
Secondary outcome
N.A.
Background summary
PHYOS is an observational study to collect data regarding changes in oxalate,
glycolate, and other biochemical parameters in patients with PH1 who may be
considered for upcoming clinical trials of DCR-PH1, a novel experimental agent
being developed for the treatment of PH1.
Study objective
Primary Objective
• To collect data regarding changes in oxalate, glycolate, and other
metabolites over time in subjects with primary hyperoxaluria type 1 (PH1) who
may be considered for clinical trials of DCR PH1
Secondary Objectives
• To collect data regarding clinical manifestations associated with PH1
• To collect data regarding fluid intake using a diary for self-reporting by
subjects
• To collect data regarding quality of life (QOL) in patients with PH1
Study design
This is an observational study: prospective data collection will continue up to
6 months per subject, during which subjects will be seen in clinic at
Screening, Day 1, Month 4 and Month 6 (± 15 days).
Study burden and risks
No investigational drugs will be administered.
Collection of blood samples may cause mild pain, redness, bruising and or
irritation at the injection site.
Cambridgepark Drive 87
Cambridge MA 02140
US
Cambridgepark Drive 87
Cambridge MA 02140
US
Listed location countries
Age
Inclusion criteria
- Diagnosis of PH1, confirmed by genotyping for mutations in the AGXT gene.
- Urine oxalate excretion >=0.7 mmol per 1.73 m2 body surface area (BSA) in 24 hours.
- Estimated glomerular filtration rate (eGFR) >=40 mL/min per 1.73 m2 BSA.
Exclusion criteria
- Prior renal and/or hepatic transplantation, or patients undergoing dialysis.
- Pregnancy or lactation at the time of screening or enrollment.
- Any significant illness, organ system dysfunction, or other condition that, in the opinion of the Investigator, would interfere with the subject*s ability to comply with the protocol requirements, including the ability to attend all visits and undergo all assessments.
Design
Recruitment
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| CCMO | NL54393.018.15 |