Primary:The primary objective of the study is to evaluate the long-term safety profile of dexpramipexole in subjects with ALS.Secondary:The secondary objective of this study is to evaluate the longterm efficacy of dexpramipexole in this study…
ID
Source
Brief title
Condition
- Neuromuscular disorders
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
Primary:
The following primary endpoints will be evaluated:
• incidence of adverse events (AEs) and serious adverse events (SAEs)
• discontinuation of study treatment due to an AE
• changes in vital signs, clinical laboratory assessments (hematology, blood
chemistry, and urinalysis), 12-lead electrocardiograms (ECGs) and body weight
• incidence of laboratory abnormalities
Secondary outcome
Secondary:
The following secondary endpoints will be evaluated:
• change in ALS Functional Rating Scale-Revised (ALSFRS-R) score
• decline in sniff nasal inspiratory pressure (SNIP)
• time to death
• time to death or death equivalent (tracheostomy or permanent assisted
ventilation [PAV], defined as use of noninvasive ventilation [NIV] for >= 22
hours per day for >= 10 days
Background summary
Currently, only 1 medicine, Rilutek® (riluzole, Sanofi Aventis, approved by the
United States [US] Food and Drug Administration in 1995 and the European
Medicines Agency in 1996), is available for the treatment of ALS. Considering
the seriousness of the disease, the lack of robust efficacy of riluzole, and
limited options for further treatment, there remains a pressing unmet medical
need for effective and safe treatments for ALS. In a 2-part Phase 2 study
conducted by Knopp Neurosciences Inc. (KNS 760704-CL201 [CL201]),
dexpramipexole appeared to be well tolerated at doses ranging from 25 mg to 150
mg twice daily. Study results also showed a dose-dependent trend in slowing the
rate of functional decline, as measured by change in the ALS Functional Rating
Scale-Revised (ALSFRS-R), and a trend toward reduction in mortality of the 150
mg twice daily group compared to the 25 mg twice daily group.
This extension study will further evaluate the long-term safety and efficacy
profile of dexpramipexole.
Study objective
Primary:
The primary objective of the study is to evaluate the long-term safety profile
of dexpramipexole in subjects with ALS.
Secondary:
The secondary objective of this study is to evaluate the longterm efficacy of
dexpramipexole in this study population using clinical endpoints measuring
function and survival.
Study design
An Open-Label, Multicenter, Extension Study to Evaluate the Long-Term Safety
and Efficacy of Dexpramipexole (BIIB050) in Subjects With Amyotrophic Lateral
Sclerosis
Intervention
All subjects will receive 300 mg dexpramipexole per day (150 mg given twice
daily).
Study burden and risks
After taking the study medication the patient will be asked to return to the
clinic on the following times:
• at Month 1, Month 2, Month 4, and Month 6
• once every 4 months from Month 10 through Month 34 (at Months 10, 14, 18, 22,
26, 30, and 34)
• at Month 36 (or within 7 days after your last dose of study medicine if you
stop study medicine before the end of the study)
• 1 month after your last dose of study medicine.
Every other month, the research personnel will conduct evaluations by calling
you and/or visiting the patients at home. The patients will be contacted by
the study staff at Months 3 and 5, and every 4 months from Month 8 through
Month 32 (at Months 8, 12, 16, 20, 24, 28, and 32).
• Medical history update and physical examination
• Blood pressure, heart rate, respiration rate, temperature, weight, and height
• Blood tests
• Urine tests
• Pregnancy tests (if applicable)
• Blood tests to check the level of study medicine in your blood (only if you
have a serious side effect)
• Electrocardiogram (ECG)
• Lung function tests
• Questions about difficulties you may have had, how well you feel, and how
well you are able to do your usual activities
• Questions about if you are taking the study medicine as instructed
• Questions about any other treatments you are taking
• Questions about if you have any new side effects
Innovation House, 70 Norden Road 70
Maidenhead SL6 4AY
GB
Innovation House, 70 Norden Road 70
Maidenhead SL6 4AY
GB
Listed location countries
Age
Inclusion criteria
1. Subject has the ability to understand the purpose and risks of the study and provide signed and dated informed consent (or have the consent confirmed by a witness if unable to write) and authorization to use protected health information (PHI) in accordance with national and local subject privacy regulations.;2. Subject was enrolled in either Study CL211 or Study 223AS302.;3. Subject has completed their last visit in Study CL211 or Study 223AS302.;4. Subjects of childbearing potential must practice effective contraception during the study and be willing and able to continue contraception for 1 month (females) or 3 months (males) after their last dose of study treatment.
Exclusion criteria
1. Subject withdrew prematurely from Study CL211 or Study 223AS302.;2. Subject permanently discontinued study treatment in Study CL211 or Study 223AS302 for any reason other than enrollment into this study.;3. Subject from Study CL211 or Study 223AS302 has a significant change in medical history (including laboratory tests or a clinically significant condition) that in the opinion of the Investigator would impair the subject*s medical fitness for participation and preclude treatment.;4. Female subject who is pregnant or breastfeeding.;5. Subject is currently enrolled in any investigational drug study other than Study CL211 or Study 223AS302.;6. Subject is taking pramipexole, other dopamine agonists, any other agent with dopaminergic activity, or any other disallowed concomitant medication;7. Subject is unwilling or unable to comply with the requirements of the protocol including the presence of any condition (physical, mental, or social) that is likely to affect the subject*s ability to comply with the protocol. At a minimum, subjects who are not able to travel to the study site must be willing to agree to remote blood draws for clinical laboratory evaluations and telephone visits to report AEs, concomitant medications, and ALSFRS-R scores.
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 | EUCTR2011-006119-70-NL |
| CCMO | NL40561.041.12 |