To evaluate the long-term safety of FAB122 in patients with ALS.
ID
Source
Brief title
Condition
- Neuromuscular disorders
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
Nature, frequency and severity of Treatment Emergent Adverse Events
Secondary outcome
1. Mortality-adjusted change from baseline in ALSFRS-R total score until the
end of the study;
2. Survival time, i.e. time to death from any cause, tracheostomy or initiation
of non-invasive ventilation for more than 20 hours a day for more than 10
consecutive days until the end of the study;
3. Mean change in norm-standardized ECAS total score;
4. Change from baseline in the total score on the ALS Assessment
Questionnaire-40-Item (ALSAQ-40) until the end of the study;
5. Change from baseline in EuroQoL - 5 Dimensions - 5 Levels (EQ-5D-5L) until
the end of the study;
6. Change from baseline in Health related QoL Visual Analogue Scale (VAS) score
until the end of the study;
7. Change from baseline in the prognostic ALS biomarker neurofilament light
(NFL);
8. Change from baseline in the ALS biomarkers creatinine and creatinine kinase;
9. Change from baseline in the ALS biomarker Urinary extracellular domain of
neurotrophin receptor p75 (Urinary P75ECD);
10. Change from baseline of oxidative stress biomarker 8-hydroxyguanosine
(8-OHdG);
11. Cost-Utility analysis of treatment with FAB122.
Safety Endpoints:
1. Parameters derived from in vital signs and 12-lead electrocardiogram (ECG);
2. Parameters derived from laboratory tests (hematology, biochemistry,
urinalysis);
3. Proportion of patients that drop out due to adverse events.
Background summary
As ALS is a rapidly progressive and fatal disease, this study is designed as an
open-label extension (OLE) study to provide longer term access to daily oral
edaravone to patients who have demonstrated a good tolerance in the ADORE trial
(either FAB122 or placebo arm) during 48 weeks and up to 72 weeks.
The good tolerance for each patient who could be included in the current OLE
study will be evaluated by the investigator, according to the individual
favourable benefit/risk ratio which will be based on the available data.
This OLE study will also assess longer term safety, survival and therapeutic
potential of daily oral edaravone.
Study objective
To evaluate the long-term safety of FAB122 in patients with ALS.
Study design
Multicenter, multinational, open-label Phase III extension study to investigate
the long-term safety of 100 mg FAB122 once daily as oral formulation in ALS
patients.
All patients participating in the ADORE study will be invited to roll over to
FAB122 until market authorization is obtained or in case the objectives of the
main study are not met (end of OLE study), provided good tolerance and safety
is proven. Patients that discontinued treatment in the main ADORE study for
other than safety reasons, will be also invited to re-start treatment with
FAB122 in the OLE study.
Patients not willing to continue receiving active treatment in the extension
study or that had already discontinued study treatment during the course of the
main ADORE study for safety reasons, will be asked to be contacted by phone and
followed up for vital status.
Subjects rolling over active treatment will visit the clinic at Baseline
(whenever possible, Visit 6 or 8 of the main study) and every 3 months
thereafter.
Intervention
Fasted daily dose of 100 mg FAB122 granules for oral solution in single
sachets, which has to be dissolved in 100 mL water prior to administration
Study burden and risks
Benefits
Your current condition of ALS will be assessed carefully. FAB122 may slow down
ALS disease progression, but this is not certain. It may be that participation
in this study does not provide you any benefit for your health. However, you
will contribute to increase the knowledge about the treatment of ALS.
Drawbacks
Drawbacks of participation in the study may be:
- Possible side effects of FAB122
- Possible adverse effects/discomforts of the tests and procedures applied in
the study
- Taking medication according to study procedures.
Participation in the study also means:
- That you have to invest time in participation in the study
- That you have to attend (additional) clinic visits and undergo testing.
Diagonal 549
Barcelona 08029
ES
Diagonal 549
Barcelona 08029
ES
Listed location countries
Age
Inclusion criteria
All patients:
1. who completed the full study period in the main ADORE study
(FAB122-CT-2001);
2. whom the investigator has no concern and judges tolerable for the continued
treatment with FAB122 from a risk and benefit point of view;
3. a female subject should not be able to become pregnant and needs to meet at
least one of the following criteria:
- female subject who is not of reproductive potential is eligible without
requiring the use of contraception. A woman is considered not having
childbearing potential when becoming post-menopausal unless permanently
sterile. Permanent sterilisation methods include hysterectomy, bilateral
salpingectomy and bilateral oophorectomy. A postmenopausal state is defined as
no menses for 12 months without an alternative medical cause.
- female who is of reproductive potential and has a negative pregnancy test at
screening and at baseline and is non-lactating. A female subject who is of
reproductive potential agrees to use (or have their partner use) adequate birth
control methods starting from the time of consent through 30 days after the
last dose of study therapy. Longer periods of birth control may be required
per local requirements. Acceptable methods of birth control include combined
(estrogen and progestogen containing) hormonal contraception associated with
inhibition of ovulation (oral, intravaginal, transdermal), progestogen-only
hormonal contraception associated with inhibition of ovulation (oral,
injectable, implantable), intrauterine device in place for >=3 months,
intrauterine hormone-releasing system, bilateral tubal occlusion or
vasectomised partner.
4. a male patient must:
- agree he will not donate sperm during the study and until 104 days after the
last dose, AND use a condom during sexual intercourse with pregnant or
non-pregnant women of childbearing potential (WOCBP) partner even if he is
vasectomized.
- in addition WOCBP partner of the male patient must use the following
acceptable methods of birth control during the study and until 104 days after
the last dose: combined (estrogen and progestogen containing) hormonal
contraception associated with inhibition of ovulation (oral, intravaginal,
transdermal), progestogen-only hormonal contraception associated with
inhibition of ovulation (oral, injectable, implantable), intrauterine device in
place for >=3 months, intrauterine hormone-releasing system, bilateral tubal
occlusion or vasectomised partner;
5. providing informed consent.
Exclusion criteria
This study has no exclusion criteria. The study is an open-label extension of
an already existing study.
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 | EUCTR2022-003050-32-NL |
| ClinicalTrials.gov | NCT05178810 |
| CCMO | NL83050.100.22 |