Open-label, Non-randomised Extension Trial to Assess the Long-Term Safety and Efficacy of 1200 mg/day Arimoclomol 400 mg Three Times a Day (t.i.d.) in Subjects with Amyotrophic Lateral Sclerosis (ALS) who have Completed the ORARIALS-01 Trial
- Conditions
- neurodegenerative disorder and Lou Gehrig's disease10029305
- Registration Number
- NL-OMON55065
- Lead Sponsor
- Orphazyme A/S
- Brief Summary
Trial is onging in other countries
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 10
1. Subject is able to comprehend and is willing to provide written informed
consent and is capable and willing to comply with trial procedures or in the
circumstance that the subject is incompetent, informed consent/assent is
provided in accordance with local regulation and/or procedures.
2. Subject has completed the ORARIALS 01 trial (i.e., met one of the surrogate
survival endpoints of tracheostomy or PAV or has completed the 76 weeks
randomised treatment period).
3. Subject completed ORARIALS-01 while on treatment, where on treatment is
defined as having taken the last dose of IMP within 2 weeks of the End of Trial
visit. (whether at week 76 or prior).
1. Known or suspected allergy or intolerance to the IMP (arimoclomol or
constituents).
2. Exposure to any other investigational treatment, advanced therapy medicinal
product (ATMP) or use of any other prohibited concomitant medications (see
section 6.8)
3. Women who are lactating or pregnant, or men or women unwilling to use a
highly effective method of birth control if not surgically sterile (defined as
bilateral tubal ligation, bilateral oophorectomy, or hysterectomy for women;
vasectomy for men) for female participants until 4 weeks after last dose and
for male participants until 3 months after last dose. Pre-menopausal women must
have a negative pregnancy test prior to dosing with trial medication.
Acceptable methods of birth control are:
a. Hormonal methods associated with inhibition of ovulation such as oral,
implantable, injectable, or transdermal contraceptives for a minimum of 1 full
cycle (based on the subject*s usual menstrual cycle period) before IMP
administration.
b. Total abstinence from sexual intercourse since the last menses before IMP
administration. (The reliability of sexual abstinence needs to be evaluated in
relation to the duration of the clinical trial and the preferred and usual
lifestyle of the subject. Periodic abstinence methods [calendar, symptothermal,
post ovulation methods] are not acceptable methods of contraception).
c. IUD or IUS.
4. Any of the following medically significant conditions:
a. Clinically significant renal or hepatic disease OR clinical laboratory
assessment (results * 3 times the upper limit of normal [ULN] for aspartate
aminotransferase and/or alanine aminotransferase, bilirubin * 2 times the ULN,
or creatinine * 1.5 times the ULN).
b. Any new condition or worsening of existing condition which, in the
opinion of the investigator
would put the subject at undue risk.
5. Any serious adverse event or moderate/severe adverse event from the
ORARIALS-01 trial which is ongoing at the time of transitioning to ORARIALS-02
and assessed as probably related to IMP.
6. Subjects who exceed 60 days from the End of Trial Visit date of the
ORARIALS-01 trial at the time of enrolment.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>Primary endpoints:<br /><br><br /><br>* Incidence and severity of TEAEs over a treatment period of 76 weeks<br /><br>* Mean and change from Baseline (of the present trial) to Week 76 (or End of<br /><br>Trial - Treatment Period 1) in clinical safety laboratory tests and vital signs<br /><br>* Incidence of potentially clinically significant abnormalities in clinical<br /><br>safety laboratory tests and vital signs over a treatment period of 76 weeks<br /><br>* C-SSRS over a treatment period of 76 weeks</p><br>
- Secondary Outcome Measures
Name Time Method <p>Secondary endpoints:<br /><br><br /><br>* Time to PAV/tracheostomy/death (for subjects entering this trial having<br /><br>completed 76 weeks of randomised treatment in ORARIALS-01)<br /><br>* Change in ALSFRS R from Baseline (of the present trial) to week 76 (End of<br /><br>Trial - Treatment Period 1)<br /><br>* Change in SVC from Baseline (of the present trial) to the week 76 (End of<br /><br>Trial - Treatment Period 1) (for subjects who did not meet the survival<br /><br>endpoint in the ORARIALS-01 trial)</p><br>