A multicenter, randomized, double-blind, placebo-controlled study to investigate the efficacy and safety of FAB122 in patients with Amyotrophic Lateral Sclerosis
- Conditions
- Disease of nerve cells that control musclesNeurodegenerative syndrome10029317
- Registration Number
- NL-OMON54334
- Lead Sponsor
- Ferrer Internacional, S.A.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 25
1. Age 18 - 80 years (both inclusive), male or female;
2. Diagnosis of definite, probable, probable laboratory supported or possible
ALS as based on the El Escorial and the revised Airlie House diagnostic
criteria for ALS;
3. Onset of first symptoms* no longer than 24 months prior to randomization;
*Date of onset is the date the patient reported one or more of the following
symptoms: Muscle weakness in limbs; speech/swallowing difficulties; respiratory
symptoms: dyspnea was noticed
4. Slow Vital Capacity (SVC) equal to or more than 70% of the predicted normal
value for gender, height and age at screening visit;
5. Change in ALSFRS-R score between 0.35 points and 1.5 points per month (both
inclusive) in the period from onset of first symptoms to the Screening visit;
6. Patients on riluzole should be on stable doses >=30 days prior to the
baseline visit and this dose should be maintained during the entire trial.
7. 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) or practicing 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 >=3months,
intrauterine hormone-releasing system, bilateral tubal occlusion or
vasectomised partner.;
8. 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;
9. Capable of providing informed consent and complying with trial procedures.
1. Diagnosis of Primary Lateral Sclerosis;
2. Diagnosis of Frontotemporal Dementia;
3. Diagnosis of other neurodegenerative diseases (e.g. Parkinson disease,
Alzheimer disease);
4. Diagnosis of polyneuropathy;
5. Other causes of neuromuscular weakness;
6. Have a significant pulmonary disorder not attributed to ALS and/or require
treatment interfering with the evaluation of ALS on respiratory function;
7. Use of intravenous (IV) edaravone within 6 months of the screening visit;
8. Use of mechanical ventilation (invasive or non-invasive) at Screening;
9. Renal impairment as indicated by a creatinine clearance of less than 50
mL/min;
10. Subject has a history of clinically significant hepatic disease, hepatitis
or biliary tract disease, ALT/AST levels >= 3xULN, bilirubin levels >=2xULN or
subject has a positive screening test for HIV, hepatitis B or C;
11. Presence of any of the following clinical conditions:
a. Unstable cardiac, pulmonary, endocrine, hematologic or active infectious
disease
b. Severe active psychiatric illness e.g psychosis, untreated major depression
within 90 days of the screening visit
c. Significant cognitive impairment, clinical dementia or psychiatric illness
d. Cancer that is currently under active treatment or is likely to require
treatment during the trial that may alter the subject*s function and interfere
with assessment of ALS disease progression.
12. Any comorbidity that may interfere with the functions as scored with the
ALSFRS-R;
13. History of known sensitivity or intolerability to edaravone, to any related
compound, or to any of the excipients;
14. Exposure to any investigational drug within 30 days of the screening visit
or 5 half-lives, whichever is longer;
15. Current substance or alcohol dependence;
16. For patients undergoing optional CSF sampling: any condition that according
to the investigator criteria is contraindicated for the procedure (e.g.
space-occupying lesion with mass effect, increase of intracranial pressure due
to increased CSF pressure; posterior fossa mass; Arnold-Chiari malformation;
anticoagulant medication; coagulopathy; uncorrected bleeding diathesis;
congenital spine abnormality; and skin infection at puncture site.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>Change from baseline in Amyotrophic Lateral Sclerosis Functional Rating Scale -<br /><br>Revised (ALSFRS-R) score after 48 weeks.</p><br>
- Secondary Outcome Measures
Name Time Method