A multicenter, randomized, double-blind, placebo-controlled study to investigate the efficacy and safety of FAB122 in patients with Amyotrophic Lateral Sclerosis.
- Conditions
- 10029317Disease of nerve cells that control musclesNeurodegenerative syndrome
- Registration Number
- NL-OMON54862
- Lead Sponsor
- Ferrer Internacional, S.A.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Withdrawn
- Sex
- Not specified
- Target Recruitment
- 10
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 *3 months,
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: hormonal contraception (oral, implanted, injected or other
hormonal (e.g., patch or contraceptive ring) contraception), intrauterine
device in place for *3 months, barrier method in conjunction with spermicide OR
use of appropriate
double barrier contraception as per local regulations or guidelines;
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. Depend on mechanical ventilation (invasive or non-invasive) or require
tracheostomy at Screening;
9. Renal impairment as indicated by a creatinine clearance of less than 50
mL/min as calculated by the Cockcroft Gault equation;
10. Subject has a history of clinically significant hepatic disease, hepatitis
or biliary tract disease, 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. Unstable
psychiatric illness defined as 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;
15. Current substance or alcohol dependence.
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 of treatment.</p><br>
- Secondary Outcome Measures
Name Time Method