| 1 |
PHYOX1, NCT03392896
|
completed 11/19/2019 |
25 normal healthy volunteers;
18 PH1 or PH2 patients |
placebo-controlled, single-blind,
single-center study to evaluate
the safety, pharmacokinetics, and tolerability; open-label multicenter
study to evaluate the safety, tolerability, pharmacokinetics, and
pharmacodynamics of single ascending doses |
no significant
drug related ADRs were observed; the most common
ADRs were mild to moderate injection site reactions that were resolved
spontaneously; four patients experienced severe ADRs that were not
considered treatment-related and have since resolved |
| 1 |
PHYOX4, NCT04555486
|
completed 09/07/2021 |
6 PH3 patients |
placebo-controlled, double-blind, multicenter study to evaluate
the safety, tolerability, pharmacokinetics, and pharmacodynamics of
a single dose |
primary end point was met with nedosiran,
demonstrating a good
safety profile in patients with PH3; all ADRs reported were mild,
with the most common being back pain; the secondary end point was
not met; however, all patients showed decreased levels of UOx compared
to baseline levels, demonstrating the efficacy of nedosiran |
| 2 |
PHYOX2, NCT03847909
|
completed 06/29/2021 |
35 PH1 or PH2 patients |
placebo-controlled, double-blind,
multicenter study to evaluate
the efficacy, safety, and tolerability |
the primary end
point was met with patients who received nedosiran
having a 57.5% greater daily reduction in UOx compared to placebo;
the secondary end point was also met with 50% of the patients given
nedosiran achieving and maintaining a normal or near-normal UOx level
compared to placebo; nedosiran was generally well tolerated, with
the most common ADRs being injection site reactions that resolved
spontaneously; three patients who received nedosiran reported kidney
stones |
| 2 |
PHYOX7, NCT04580420
|
estimated completion 05/2025 |
12 PH1 or PH2 patients with severe renal impairment
with or
without dialysis |
open-label study to evaluate the safety
and efficacy |
not reported yet |
| 2 |
PHYOX8, NCT05001269
|
estimated completion 12/2022 |
10 HP1 or HP2 pediatric patients
from birth to 5 years and
relatively intact renal function |
open-label multicenter
study to evaluate the safety, pharmacokinetics,
and efficacy |
not reported yet |
| 3 |
PHYOX3, NCT04042402
|
estimated completion 12/2023 |
50 PH1 or PH2 patients from
the previous phase I and II studies |
open-label roll-over
study to evaluate the long-term safety
and efficacy |
an interim data report shows that nedosiran
is generally well
tolerated, and there have been no serious adverse events thus far;
all participants who have received nedosiran achieved normal or near-normal
UOx by day 180 |
