A Phase 3 placebo controlled study to evaluate safety and efficacy of Amifampridine phosphate.
- Conditions
- MedDRA version: 14.1Level: SOCClassification code 10029205Term: Nervous system disordersSystem Organ Class: 10029205 - Nervous system disordersambert-Eaton Myasthenic Syndrome (LEMS)Therapeutic area: Body processes [G] - Digestive System and Oral Physiological Phenomena [G10]MedDRA version: 14.1Level: LLTClassification code 10028415Term: MyastheniaSystem Organ Class: 10028395 - Musculoskeletal and connective tissue disorders
- Registration Number
- EUCTR2010-021850-20-IT
- Lead Sponsor
- BIOMARIN PHARMACEUTICAL INC.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 60
=18 years of age. - Confirmed diagnosis of LEMS as documented by acquired (typical) proximal muscle weakness and at least 1 of the following: Nerve conduction findings (CMAP that increases at least 2-fold after maximum voluntary contraction of the tested muscle) Positive anti-P/Q type voltage-gated calcium-channel antibody test - If currently receiving treatment with amifampridine for LEMS, a normal resp function as deined by an FVC >= 80% of predicted - If not currently receving amifampridine and pt has no history of othr current respiratory disease a FVC >= 60& i predicted- Completion of anti-cancer treatment at least 3 months (90 days) prior to Screening - A QMG score of = 5 is required for patients without any prior symptomatic treatment for LEMS - If currently receiving treatment for LEMS, patients must present with some signs and/or symptoms consistent with LEMS - Normal respiratory function as defined by a forced vital capacity > 80% predicted (score of 0 on this dimension of QMG) - Normal swallowing function as defined by the ability to swallow 4 ounces of water without coughing or throat clearing (score of 0 on this dimension of QMG) - If receiving peripherally acting cholinesterase inhibitors (eg, pyridostigmine), a stable dose of cholinesterase inhibitors is required for at least 7 days prior to Screening - If receiving permitted oral immunosuppressants (eg, prednisone or other corticosteroid, azathioprine, mycophenolate), a stable dose is required for at least 90 days prior to Screening - Negative pregnancy test for females of childbearing potential at Screening - If sexually active, willing to use 2 acceptable methods of contraception from Screening until 3 months after the last dose - Willing to perform all study procedures as physically possible - Willing and able to provide written informed consent after the nature of the study has been explained and prior to the start of any research-related procedures.
Are the trial subjects under 18? no
Number of subjects for this age range: 0
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 15
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 15
History of epilepsy or seizure - Known active brain metastasis - cuncurrent use of fampridine and ay other 3,4 diaminopridine other than IP provided - - Use of medications known to lower the epileptic threshold within 7 days or 5 half-lives, whichever is longer, prior to Screening - Use of medications which inhibit neuromuscular junction function within 7 days or 5 half-lives, whichever is longer, prior to Screening - Use of IVIG, plasmapheresis (plasma exchange), or immunoadsorption within 90 days prior to Screening - Use of guanidine hydrochloride within 7 days of Screening - Use of rituximab within 12 months prior to Screening - History of drug allergy to any pyridine-containing substances or any amifampridine phosphate excipient(s) - Use of any other investigational product other than amifampridine phosphate or investigational medical device within 30 days prior to Screening or requirement for any investigational agent prior to completion of all scheduled study assessments - Treatment with a concomitant medication that prolongs the QT wave QT/QTc wave corrected for heart rate (QTc) interval within 7 days or 5 half-lives, whichever is longer, prior to Screening - Treatment with sultopride within 7 days prior to Screening - sinus arrhythmias - excessive heart rate variation at rest -QT wave corrected for heart rate using Bazzzatt`s formula (QTBC) interval > 450 msec confirmed by a rpeat ECG - PR inteval > 210 msec - QRS interval > 120 msec - Early polarisation pattern that increase the risk of partecipating in the study - Documented history of arrhythmias - Breastfeeding or pregnant at Screening or planning to become pregnant (self or partner) at any time during the study. Male patients with breastfeeding partners are not excluded from the study - Likely or expected to require treatment for cancer within 3 months (90 days) after entering Screening - History of severe renal impairment or evidence of severe renal impairment on Screening laboratory tests- Screening laboratory tests for hepatic impairment; In pt with without cancer , ALT, AST and or total bilirubin >ULN - in pt with cancer ALT, AST > 5xupper limit of normal (ULN) and or total bilirubin > 3 x ULN History of uncontrolled asmtma - Basline vital capacity < 1500 ml
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method