Overview

A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C

Status:
Not yet recruiting
Trial end date:
2024-04-01
Target enrollment:
0
Participant gender:
All
Summary
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.
Phase:
Phase 3
Accepts Healthy Volunteers?
No
Details
Lead Sponsor:
IntraBio Inc
Criteria
Inclusion Criteria:

1. Written informed consent signed by the patient and/or their legal representative/
parent/ impartial witness

2. Male or female aged ≥4 years with a confirmed genetic diagnosis of NPC at the time of
signing informed consent.

3. Females of childbearing potential, defined as a premenopausal female capable of
becoming pregnant, will be included if they are either sexually inactive (sexually
abstinent for 14 days prior to the first dose and confirm to continue through 28 days
after the last dose) or using one of the following highly effective contraceptives
(i.e. results in <1% failure rate when used consistently and correctly) 14 days prior
to the first dose continuing through 28 days after the last dose:

1. intrauterine device (IUD);

2. surgical sterilization of the partner (vasectomy for 6 months minimum);

3. combined (estrogen or progestogen containing) hormonal contraception associated
with the inhibition of ovulation (either oral, intravaginal, or transdermal);

4. progestogen only hormonal contraception associated with the inhibition of
ovulation (either oral, injectable, or implantable);

5. intrauterine hormone releasing system (IUS);

6. bilateral tubal occlusion.

4. Females of non-childbearing potential who have undergone one of the following
sterilization procedures at least 6 months prior to the first dose:

1. hysteroscopic sterilization;

2. bilateral tubal ligation or bilateral salpingectomy;

3. hysterectomy;

4. bilateral oophorectomy; OR be postmenopausal with amenorrhea for at least 1 year
prior to the first dose and follicle stimulating hormone (FSH) serum levels
consistent with postmenopausal status. FSH analysis for postmenopausal women will
be done at screening. FSH levels should be in the postmenopausal range as
determined by the central laboratory.

5. Non-vasectomized male patient agrees to use a condom with spermicide or abstain from
sexual intercourse during the study until 90 days beyond the last dose of study
medication and the female partner agrees to comply with inclusion criteria 3 or 4.

For a vasectomized male who has had his vasectomy 6 months or more prior to study
start, it is required that they use a condom during sexual intercourse. A male who has
been vasectomized less than 6 months prior to study start must follow the same
restrictions as a non-vasectomized male.

6. If male, patient agrees not to donate sperm from the first dose until 90 days after
their last dose.

7. Patients must fall within:

a) A SARA score of 7 ≤ X ≤ 34 points (out of 40) AND b) Either: i. Within the 2-7
range (0-8 range) of the Gait subtest of the SARA scale OR ii. Be able to perform the
9-Hole Peg Test with Dominant Hand (9HPT-D) (SCAFI subtest) in 20 ≤ X ≤150 seconds.

8. Weight ≥15 kg at screening.

9. Patients are willing to disclose their existing medications/therapies for (the
symptoms) of NPC including those on the prohibited medication list. Non-prohibited
medications/therapies (authorized medicines for NPC [e.g. miglustat], speech therapy,
and physiotherapy) are permitted provided:

1. The Investigator does not believe the medication/therapy will interfere with the
study protocol/results

2. Patients have been on a stable dose/duration and type of therapy for at least 42
days before Visit 1 (Baseline 1)

3. Patients are willing to maintain a stable dose/do not change their therapy
throughout the duration of the study.

10. An understanding of the implications of study participation, provided in the written
patient information and informed consent by patients or their legal
representative/parent, and demonstrates a willingness to comply with instructions and
attend required study visits (for children this criterion will also be assessed in
parents or appointed guardians).

Exclusion Criteria:

1. Patients who are unable to consistently Patients who have any known hypersensitivity
or history of hypersensitivity to:

1. Acetyl-Leucine (DL-, L-, D-) or derivatives.

2. Excipients the IB1001 sachet (namely isomalt, hypromellose, and strawberry
flavour).

3. Excipients the placebo sachet (namely isomalt, hypromellose, strawberry flavour,
citric acid, microcrystalline cellulose, lactose, denatonium benzoate).

2. 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.

3. Patients with a physical, cognitive, or psychiatric condition which, at the
investigator's discretion and in consultation with the Medical Monitor and Sponsor (as
applicable), may put the patient at risk, may confound the study results, or may
interfere with the patient's participation in the clinical study, i.e. reliably
perform study assessments.

4. Known or persistent use, misuse, or dependency of medication, drugs, or alcohol.

5. Current or planned pregnancy or women who are breastfeeding.

6. Patients with severe vision or hearing impairment (that is not corrected by glasses or
hearing aids) that, at the investigator's discretion, interferes with their ability to
perform study assessments.

7. Patients who have been diagnosed with arthritis or other musculoskeletal disorders
affecting joints, muscles, ligaments, and/or nerves that by themselves affects
patient's mobility and, at the investigator's discretion, interferes with their
ability to perform study assessments.

8. Patients unwilling and/or not able to undergo a 42 day period from any of the
following prohibited medication prior to Visit 1 (Baseline 1) and remain without
prohibited medication through Visit 6.

1. N-Acetyl-DL-Leucine (e.g. Tanganil®);

2. N-Acetyl-L-Leucine (prohibited if not provided as IMP in the IB1001-301 trial);

3. Sulfasalazine;

4. Rosuvastatin.

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