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.~Aim:~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.~Randomisation:~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.~Study duration:~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.~The CT-ORZY-NPC-002 protocol has been updated to include a paediatric sub-study including new and naïve patients aged 6 to <24 months at study enrolment.~Aim:~The purpose of the paediatric sub-study, is to assess the safety and tolerability of 36 months of open-label arimoclomol when 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.~The Paediatric sub-study will run at the open sites participating in the main study. A total of 3-5 patients are planned to be enrolled. All patients will be treated with arimoclomol.~Inclusion criteria:~Diagnosis of NPC1 or NPC2;~NPC diagnosis confirmed by:~Genetically confirmed (deoxyribonucleic acid [DNA] sequence analysis) by mutations in both alleles of NPC1 or NPC2, OR~Mutation in only one allele of NPC1 or NPC2 plus either positive filipin staining or elevated cholestane triol/oxysterols (>2 x upper limit of normal).~Males and females aged 6 to <24 months, with a cap of maximum 3 patients above 18 months~Treated or not treated with miglustat;~If a patient is on prescribed treatment with miglustat, the dose must have been stable for at least 1 month prior to inclusion in the paediatric sub-study~If a patient has been discontinued from prescribed treatment with miglustat, they must have been discontinued for at least 1 month prior to inclusion in the paediatric sub-study~The Legal Authorised Representative (LAR) has read and signed the Informed Consent Form (ICF) prior to any study-related procedures~The LAR agrees for the patient to participate in all aspects of the trial design~Exclusion Criteria:~◦ Recipient of a liver transplant or a planned liver transplant~The Aspartate Transaminase (AST) and/or Alanine Transaminase (ALT) >3 x Upper Limit of Normal (ULN) for age and gender~Renal insufficiency with serum creatinine level >1.5 x ULN~Patients with known causes of active liver disease or prolonged icterus or malformation of organs other than NPC~Patient was born before 37 weeks gestation~Patient weight <5 kg at study enrollment~Patient is diagnosed with severe intra-uterine growth restriction~Patient has severe neurological symptoms~Patient has received or plans to receive a bone marrow transplant~Arms and Intervention:~arm open label treatment with arimoclomol capsules 100 mg (dispersed in water) for oral administration (3 times daily). Doses: The dose in mL is based on the patient's weight in kg.~Randomization: Open Label~Pharmacokinetic: To confirm the selected dose, patients will undergo an arimoclomol single-dose pharmacokinetic (PK) evaluation before the start of continuous treatment.~Outcome measures: Primary/Safety Outcome Measures~Collection of safety data:~Adverse events (AEs)~Vital signs~[Time Frame: Screening (V1), to week 1(V2 baseline), weeks 2, 4, 12, 24, 36, 48, 72, 96, 120 and 144 after baseline]~o Haematology~o Clinical chemistry~[Time Frame: Screening (V1), to weeks 2, 4, 12, 24, 36, 48, 72, 96, 120 and 144 after baseline - V2]~Secondary Outcome Measures • Clinical signs and symptoms captured through physical examination,~Change from baseline in patient weight measured in kg~[Time Frame: Screening (Visit 1) to Baseline V2- 1 week after V1, 1, 3, 6, 9, 12, 15, 18, 24, 30 and 36 months after baseline]~• Change from baseline in patient height measured in meter~[Time Frame: Screening (Visit 1) to Baseline V2- 1 week after V1, 3, 6, 12 , 18, 24, 30 and 36 months after baseline]~Change in Developmental delay scoring, using the Bayley III score~Changes from baseline in the size of the liver and spleen assed by ultrasound~[Time Frame: Baseline, months 6,12, and 18 (Visit 2, 6, 8, 9)]
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.~The CT-ORZY-NPC-002 study has been expanded to include an open label paediatric sub-study including patients aged 6 to <24 months at study enrolment.