a study on Niemann-Pick Disease, Type C
A prospective, randomised, double-blind, placebo controlled therapeutic study in patients with confirmed diagnosis of NiemannPick disease type C (NPC). The purpose of this study is to assess the efficacy and safety of arimoclomol (compared to placebo) when it is administered as an add-on therapy to the patient's current prescribed best standard of care; patient's standard of care may, or may not, include miglustat.
Arimoclomol Prospective Doubleblind, Randomised, Placebo-controlled Study in Patients Diagnosed With NiemannPick Disease Type C
A prospective, randomised, double-blind, placebo controlled therapeutic study in patients with confirmed diagnosis of NiemannPick disease type C (NPC).
Patients must either 1) have completed Visit 2 (end of study [EOS]) of the CTORZYNPC001 study or 2) meet the eligibility criteria of this study including a requirement of stable treatment with miglustat for 6 months (if on miglustat therapy) prior to enrolment into the study.
The purpose of this study is to assess the efficacy and safety of arimoclomol (compared to placebo) when it is administered as an add-on therapy to the patient's current prescribed best standard of care; patient's standard of care may, or may not, include miglustat.
Patients will be randomised to receive placebo or arimoclomol (with an allocation ratio of 1:2).
Pharmacokinetic evaluation(age below 12):
To confirm the selected dose, patients less than 12 years of age will undergo an arimoclomol single-dose pharmacokinetic (PK) evaluation before randomisation and the start of continuous (multiple dosing) treatment.
Early Escape Clause:
In patients whose disease progression is too severe and/or too fast, the "early escape clause" will allow the Investigator to apply the escape route which implies that the patient can be treated with arimoclomol (as per blinded phase study schedule) and be followed up on an annual basis until arimoclomol has received EU MA or until the analysis of data from the controlled, 12 month blinded phase study period does not support the efficacy and/or safety of arimoclomol.
The duration of the blinded phase study period will be 12 months.
Following this, all patients will be offered to continue into the extension phase of the study where every patient will receive arimoclomol and be followed up and attend site visits at 18 months and 24 months (after randomisation) and then on an annual basis thereafter.The extension phase runs until arimoclomol has received Regulatory Approval or until the analysis of data from the controlled, blinded phase 12 month study period does not support the efficacy and/or safety of arimoclomol.
Niemann-Pick Disease, Type C NPC1 Niemann-Pick Type C Niemann-Pick arimoclomol lysosomal storage disorder lysosomal storage disease NPC2 NP-C
Open to people ages 2–18
EITHER NP-C patients who have entered the CTORZYNPC001 study and who have completed Visit 2 (EOS) of the CTORZYNPC001 study.
NPC patients who did not enter or complete the CTORZYNPC001 study but are fulfilling all of criteria listed below:
◦Diagnosis of NPC1 or NPC2;
NPC diagnosis confirmed by:
If a patient is under prescribed treatment with miglustat, it has to be under stable dose of the medication for at least 6 continuous months prior to inclusion in the CTORZYNPC002 study;
o If a patient has been discontinued from prescribed treatment with miglustat,they must have been discontinued for at least 3 continuous months prior to inclusion in the CT-ORZY-NPC-002 study;
Highly effective birth control methods include: Combined (oestrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation (oral,intravaginal or transdermal); progestogen-only hormonal contraception associated with inhibition of ovulation (oral, injectable or implantable); intrauterine device (IUD);intrauterine hormone-releasing system (IUS); bilateral tubal occlusion; and vasectomised partner.
All sexually active male patients with female partners of child-bearing potential(post-menarchal) must use a condom with or without spermicide in addition to the birth control used by their partners during the study and until 3 months after the last dose of IMP.
Sexual abstinence is considered a highly effective birth control method only if it is defined as refraining from heterosexual intercourse during the study and for 1 week after the last dose of IMP (for female patients of child-bearing potential) and for 3 months after the last dose of IMP (for male patients with female partners of child-bearing potential). The reliability of sexual abstinence needs to be evaluated by the Investigator in relation to the duration of the clinical trial and the preferred and usual lifestyle of the patient.
•Ability to comply with the protocol-specified procedures/evaluations and scheduled visits.
Treatment with any investigational drug during the study or in the 4 weeks prior to entering the study.
This includes treatment with any investigational drug during the study in an attempt to treat NP-C;
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