To provide continuing open-label treatment with bardoxolone methyl as part of this extended access program while collecting ongoing safety and tolerability data of bardoxolone methyl.
ID
Source
Brief title
Condition
- Vascular hypertensive disorders
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
Primary safety endpoint:
Frequency, intensity, and relationship to study drug of adverse events (AEs)
and serious adverseevents (SAEs), and change from baseline in the following
assessments: physical examinations, vital sign measurements, BNP, N-terminal
pro-brain natriuretic peptide (NT-proBNP), and
weight.
Secondary outcome
not applicable
Background summary
The established pharmacologic effects of bardoxolone methyl, including the
suppression of pathologic NF-*B signaling and inflammation and mitochondrial
dysfunction, are directly applicable to the treatment of PH. Despite available
therapies, the prognosis for PH remains poor, especially for patients with CTD
and ILD. Given the severity of the underlying disease, this study seeks to
offer patients who previously participated in clinical studies extended access
to bardoxolone methyl until it is available through commercial channels
(including reimbursement).
Study objective
To provide continuing open-label treatment with bardoxolone methyl as part of
this extended access
program while collecting ongoing safety and tolerability data of bardoxolone
methyl.
Study design
extended access program - open label study
Intervention
Patients will start dosing at 10 mg of bardoxolone methyl every other day, then
begin once dailydosing at Week 4. Dose de-escalation (down to 5 mg) is
permitted during the study, if indicated clinically.
Study burden and risks
The most common adverse events of bardoxolone methyl are:
* Muscle spasm
* Hypomagnesemia (low level of magnesium in the blood), which primarily
occurred in diabetic chronic kidney disease patients and was not associated
with loss of magnesium into the urine or loss of magnesium inside cells
* Nausea
* Decreased appetite
* Weight decreased associated with a reduction in waist circumference, which
primarily occurred in obese patients
* Fatigue
Less common adverse events of bardoxolone methyl are:
* Cough
* Dysgeusia (persistent metaalic taste in the mouth)
* Transient increased levels of liver enzymes, which have not been associated
with liver injury in any patients
* Headache
Some participants with Stage 4 chronic kidney disease and a medical history of
heart failure in previous studies experienced fluid overload. The current trial
will employ risk mitigation procedures to reduce the potential for bardoxolone
methyl induces fluid overload.
Th prognosis for patients with PAH is poor. The treatments that are available
at the moment, do mostly not lead to functional improvements. Previous studies
suggest that treatment with bardoxolone methyl may be beneficial to patients in
combination with the optimal treatment with vasodilators. Patients
participating in the study will be closely monitored en will continue to
receive their standard of care treatment.
2801 Gateway Drive Suite 150
Irving TX 75063
US
2801 Gateway Drive Suite 150
Irving TX 75063
US
Listed location countries
Age
Inclusion criteria
Treatment-compliant patients who are participating in qualifying ongoing
studies and have completed required End-of-Treatment and/or Follow-up visits in
a prior clinical study with bardoxolone methyl
Exclusion criteria
1. Participation in other investigational clinical studies involving
interventional products being tested or used in a way different from the
approved form or when used for an unapproved indication, 2. Patients who have
an ongoing SAE from a clinical study that is assessed by the investigator as
related to bardoxolone methyl, 3. Unwilling to practice acceptable methods of
birth control (both males who have partners of childbearing potential and
females of childbearing potential) while taking study drug, 4. Women who are
pregnant or breastfeeding, 5. Patient is, in the opinion of the investigator,
unable to comply with the requirements of the study protocol or is unsuitable
for the study for any reason, 6. Known hypersensitivity to any component of the
study drug
Design
Recruitment
Medical products/devices used
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 |
|---|---|
| EudraCT | EUCTR2016-004365-16-NL |
| CCMO | NL60468.056.17 |