A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C
ConditionNiemann-Pick Disease, Type C
Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age4+
SponsorIntraBio Inc
About this trial
A pivotal, randomized, double-blind, placebo controlled, multi-center therapeutic study for patients age 4 and older with a confirmed diagnosis of Niemann Pick disease type C (NPC). The objective of this study is to evaluate the safety, tolerability and efficacy of N-acetyl-L-leucine (IB1001) compared to standard of care.
Following this randomized, double-blind, placebo-controlled "Parent Study", an extension phase is conducted for (1) patients who completed the "Parent Study" and (2) patients who are enrolled directly into the Extension Phase. Currently, the Extension Phase provides patients with 3 years of open-label treatment.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Written informed consent signed by the patient and/or their legal representative/ parent/ impartial witness
Male or female aged ≥4 years with a confirmed genetic diagnosis of NPC at the time of signing informed consent.
intrauterine device (IUD);
surgical sterilization of the partner (vasectomy for 6 months minimum);
Disqualifiers
Acetyl-Leucine (DL-, L-, D-) or derivatives.
Excipients the IB1001 sachet (namely isomalt, hypromellose, and strawberry flavour).
Excipients the placebo sachet (namely isomalt, hypromellose, strawberry flavour, citric acid, microcrystalline cellulose, lactose, denatonium benzoate).
Simultaneous participation in another clinical study or participation in any clinical study involving administration of an investigational medicinal product (IMP; 'study drug') for at least 42 days prior to Visit 1. At the discretion of the investigator, Medical Monitor, and Sponsor, the washout period for specific IMPs may be longer based on the pharmacological activity and pharmacokinetics of the drug.
Trial design
Design model
Crossover
Treatments tested in this trial
N-Acetyl-L-Leucine
DrugN-Acetyl-L-Leucine is a modified amino-acid ester that is orally administered (granules for suspension in a sachet)
Placebo
Other interventionMatching Placebo Sachet
Treatment groups
53 Participants
are divided into 2 treatment groupsGroup A: N-acetyl-L-leucine (IB1001)Experimental treatment 1 intervention
Group B: Placebo comparatorPlacebo comparator 1 intervention
Trial outcomes
Primary outcomes
1
Scale for the Assessment and Rating of Ataxia (all jurisdictions except US)
Time frame
End of Period I (week 12) vs. End of Period 2 (week 24)2
Modified Scale for the Assessment and Rating of Ataxia (US only)
Time frame
End of Period I (week 12) vs. End of Period 2 (week 24)Secondary outcomes
1
Spinocerebellar Ataxia Functional Index (SCAFI)
Time frame
End of Period I (week 12) vs. End of Period 2 (week 24)2
Modified Disability Rating Scale
Time frame
End of Period I (week 12) vs. End of Period 2 (week 24)3
Physician's / Caregiver's / Patient's Clinical Global Impressions (CGI)
Time frame
End of Period I (week 12) vs. End of Period 2 (week 24)4
EuroQuol- 5 Dimension (EQ-5D) Quality of Life Scale
Time frame
End of Period I (week 12) vs. End of Period 2 (week 24)Other outcomes
1
Niemann-Pick disease type C Clinical Severity Scale (NPC-CSS)
Time frame
End of Period I (week 12) vs. End of Period 2 (week 24)2
5-Domain Niemann-Pick disease type C Clinical Severity Scale (NPC-CSS)
Time frame
End of Period I (week 12) vs. End of Period 2 (week 24)3
Investigator's / Caregiver's / Patient's Clinical Global Impressions (CGI)
Time frame
End of Period I (week 12) vs. End of Period 2 (week 24)4
Modified Scale for the Assessment and Rating of Ataxia (all jurisdictions except US)
Time frame
End of Period I (week 12) vs. End of Period 2 (week 24)Sponsors and contacts
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Source ClinicalTrials.gov