There are three parts to the AIDNPC program:

1.     The first is an ‘observational’ study (NPC-001) to establish each patient’s baseline disease progression. This study has now completed enrolment.

2.     The second is the ‘interventional’ study (NPC-002), in which patients are treated with either arimoclomol or placebo. This has been designed to randomise patients 2:1, meaning that two patients will receive arimoclomol for every one patient receiving placebo. The trial is double-blind, meaning that neither doctors not patients will know whether they are on the treatment or placebo arm.

3.     The final part is an open-label extension follow up.

Purpose of the interventional trial

The purpose of this trial is to assess the efficacy and safety of arimoclomol, compared to placebo, in the treatment of Niemann-Pick type C, when it is administered in addition to the patient's current prescribed best standard of care. The standard of care may, or may not, include miglustat. This is a prospective, randomised, double-blind, placebo controlled therapeutic trial in patients with a confirmed diagnosis of Niemann-Pick disease type C (NPC).

Design & end points

Primary outcome measure

The primary outcome is a change in the severity of disease. The efficacy of arimoclomol will be evaluated based on the collective set of results including clinical scores and biomarkers.

Secondary outcome measures

Key secondary outcome measures are:

  • Changes in the Niemann Pick type C Clinical Database score

  • Changes in Quality of Life

  • Changes in the 9HPT (9-Hole Peg Test)

Trial size

50 patients was enrolled in the interventional trial.

Start and finish dates

The interventional trial began in June 2016 and the Phase II/III full data set was reported in Q1 2019.

Administration of study medication

The study medication, both arimoclomol and placebo, is administered as capsules for oral administration, three times daily.


Pharmacokinetic evaluation

To ensure correct dosing, patients younger than 12 years of age will undergo an arimoclomol single-dose pharmacokinetic (PK) evaluation before randomisation and the start of study treatment.


Trial duration

The duration of the blinded phase interventional 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. The follow up will involve attendance at site visits at 18 months and 24 months after randomisation, and then on an annual basis thereafter. The extension phase will continue 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.



Participating sites

United States

1  UCSF Benioff Children's Hospital Oakland

2  Mayo Clinic Children's Center


3  University Hospital Copenhagen (Rigshospitalet)


4  CHU de Montpellier

5  Hôpital Trousseau Germany


6  Villa Metabolica Mainz

7  Dr. von Haunersches Kinderspital der Universität München


8  Ospedale Pediatrico Bambino Gesù

9  Azienda Ospedaliero-Universitaria "Santa Maria della Misericordia" di Udin


10  The Children´s Memorial Istitute Warsaw


11  Hospital Vall D'Hebron


12  INSELSPITAL University Hospital Bern

United Kingdom

13  Birmingham Children's Hospital

14  Great Ormond Street Hospital

About NPC

Niemann-Pick type C (NPC) is a lysosomal storage disease affecting around 1 in 150,000 newborns and is caused by mutations in the NPC1 or NPC2 genes. It is marked by an accumulation of lipid molecules in structures called lysosomes, inside cells of internal organs and the central nervous system.

NPC results in a range of motor and cerebral impairments including progressive loss of muscle control and intellectual capacity. Although symptom onset and progression is highly variable, NPC diagnosis is usually in childhood, and the prognosis is poor: the majority of NPC patients die before the age of 20 and very few live into middle age.

For support relating to Niemann-Pick type C, please contact the INPDA