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New treatment option for Niemann-Pick Disease, Type C
Official title A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C
ClinicalTrials.gov ID: NCT05163288
What this study is testing
What is N-Acetyl-L-Leucine?
N-Acetyl-L-Leucine is an investigational medicine, given as a pill taken by mouth, being studied as a potential treatment for niemann-pick disease, type c.
Also referred to as IB1001.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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 3: a large, late-stage study
- You might receive a placebo (an inactive treatment) instead of the study drug, decided by chance. You may not know which one you got.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 4 and older
You may be able to join if
- 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.
- Females of childbearing potential, defined as a premenopausal female capable of becoming pregnant, will be included if they are either sexually...
- intrauterine device (IUD);
- surgical sterilization of the partner (vasectomy for 6 months minimum);
You likely can't join if
- Patients who are unable to consistently Patients who have any known hypersensitivity or history of hypersensitivity to:
- 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...
- Simultaneous participation in another clinical study or participation in any clinical study involving administration of an investigational medicinal...
- Patients with a physical, cognitive, or psychiatric condition which, at the investigator's discretion and in consultation with the Medical Monitor...
See the full eligibility criteria
Who can join
- 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.
- 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...
- intrauterine device (IUD);
- surgical sterilization of the partner (vasectomy for 6 months minimum);
- combined (estrogen or progestogen containing) hormonal contraception associated with the inhibition of ovulation (either oral, intravaginal, or transdermal);
- progestogen only hormonal contraception associated with the inhibition of ovulation (either oral, injectable, or implantable);
- intrauterine hormone releasing system (IUS);
- bilateral tubal occlusion.
- Females of non-childbearing potential who have undergone one of the following sterilization procedures at least 6 months prior to the first dose:
- hysteroscopic sterilization;
- bilateral tubal ligation or bilateral salpingectomy;
- hysterectomy;
- 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...
- 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...
- If male, patient agrees not to donate sperm from the first dose until 90 days after their last dose.
- 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...
- Weight ≥15 kg at screening.
- 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...
- The Investigator does not believe the medication/therapy will interfere with the study protocol/results
- Patients have been on a stable dose/duration and type of therapy for at least 42 days before Visit 1 (Baseline 1)
- Patients are willing to maintain a stable dose/do not change their therapy throughout the duration of the study.
- 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...
What rules you out
- Patients who are unable to consistently Patients who have any known hypersensitivity or history of hypersensitivity to:
- 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...
- 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...
- Known or persistent use, misuse, or dependency of medication, drugs, or alcohol.
- Current or planned pregnancy or women who are breastfeeding.
- 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.
- 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...
- 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.
- N-Acetyl-DL-Leucine (e.g. Tanganil®);
- N-Acetyl-L-Leucine (prohibited if not provided as IMP in the IB1001-301 trial);
- Sulfasalazine;
- Rosuvastatin. \-
The study team makes the final eligibility decision.
Where it's taking place
- Rochester, Minnesota, United States
- Parkville, Victoria, Australia
- Prague, Czechia
- Giessen, Germany
- Hamburg, Germany
- Höchheim, Germany
- München, Germany
- Münster, Germany
- Amsterdam, Netherlands
- Bratislava, Slovakia
- Bern, Switzerland
- London, United Kingdom
- Manchester, United Kingdom
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 4 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Rochester, Minnesota, United States; Parkville, Victoria, Australia; Prague, Czechia; Giessen, Germany; Hamburg, Germany; Höchheim, Germany and 7 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.