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Amino Acid
N-Acetyl-L-Leucine for Niemann-Pick Disease Type C
Phase 3
Waitlist Available
Research Sponsored by IntraBio Inc
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Male or female aged ≥4 years with a confirmed genetic diagnosis of NPC at the time of signing informed consent
Patients must fall within specified SARA score and Gait subtest or 9-Hole Peg Test with Dominant Hand (9HPT-D) ranges
Timeline
Screening 3 weeks
Treatment Varies
Follow Up end of period i (week 12) vs. end of period 2 (week 24)
Awards & highlights
Study Summary
This trial is testing a new drug for Niemann Pick disease type C, to see if it is safe, effective, and better than the current standard of care.
Who is the study for?
This trial is for individuals aged 4 and older with a confirmed genetic diagnosis of Niemann-Pick disease type C. Participants must weigh at least 15 kg, have stable medication doses, and not be pregnant or breastfeeding. Men must agree to use contraception, and women who can bear children must either abstain from sex or use effective birth control.Check my eligibility
What is being tested?
The study tests the safety and effectiveness of N-Acetyl-L-Leucine (IB1001) compared to a placebo in treating Niemann-Pick disease type C. It's randomized, meaning participants are randomly assigned to receive either the drug or placebo without choosing which one.See study design
What are the potential side effects?
While specific side effects aren't listed here, common ones may include digestive issues like nausea or diarrhea, headaches, dizziness, potential allergic reactions if sensitive to ingredients in IB1001 or similar substances.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I am 4 years or older and have a confirmed genetic diagnosis of NPC.
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My balance and walking or hand function scores are within a specific range.
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I weigh at least 15 kg.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ end of period i (week 12) vs. end of period 2 (week 24)
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~end of period i (week 12) vs. end of period 2 (week 24)
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Modified Scale for the Assessment and Rating of Ataxia (US only)
Scale for the Assessment and Rating of Ataxia (all jurisdictions except US)
Secondary outcome measures
EuroQuol- 5 Dimension (EQ-5D) Quality of Life Scale
Modified Disability Rating Scale
Physician's / Caregiver's / Patient's Clinical Global Impressions (CGI)
+2 moreOther outcome measures
5-Domain Niemann-Pick disease type C Clinical Severity Scale (NPC-CSS)
Investigator's / Caregiver's / Patient's Clinical Global Impressions (CGI)
Modified Scale for the Assessment and Rating of Ataxia (all jurisdictions except US)
+1 moreTrial Design
2Treatment groups
Experimental Treatment
Placebo Group
Group I: N-acetyl-L-leucine (IB1001)Experimental Treatment1 Intervention
Oral administration (granule in a sachet for suspension in water, orange juice, or almond milk). Patients ≥13 years old will receive a total daily dose of 4 g/day (administered as 3 doses per day); patients <13 will receive weight-tiered doses.
Group II: Placebo comparatorPlacebo Group1 Intervention
Oral administration (granule in a sachet for suspension in water, orange juice, or almond milk). Patients ≥13 years old will receive a total daily dose of 4 g/day (administered as 3 doses per day); patients <13 will receive weight-tiered doses.
Find a Location
Who is running the clinical trial?
IntraBio IncLead Sponsor
3 Previous Clinical Trials
102 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I have not been in a drug study within the last 42 days.I do not have any health conditions that could risk my safety or affect the study's results.I am 4 years or older and have a confirmed genetic diagnosis of NPC.I can avoid certain medications for 42 days before my first visit and until my sixth visit.I agree to share my current NPC symptom treatments and keep doses consistent.I had a vasectomy over 6 months ago and will use a condom during sex.My balance and walking or hand function scores are within a specific range.I weigh at least 15 kg.I agree to use contraception or abstain from sex during the study and for 90 days after.I am a woman who cannot have children because I had a sterilization procedure over 6 months ago.I am not sexually active or I use effective birth control.You regularly use, misuse, or depend on medication, drugs, or alcohol.I have arthritis or a condition that affects my ability to move.
Research Study Groups:
This trial has the following groups:- Group 1: N-acetyl-L-leucine (IB1001)
- Group 2: Placebo comparator
Awards:
This trial has 1 awards, including:- Pivotal Trial - The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Are new participants being recruited for this experiment?
"This clinical trial, which can be found on clinicaltrials.gov, is actively searching for candidates. The study was initially posted on 6/30/2022 and was most recently updated on 10/20/2022."
Answered by AI
Has N-Acetyl-L-Leucine been cleared for use by the FDA?
"N-Acetyl-L-Leucine is in Phase 3 clinical trials, so there is some efficacy data and multiple rounds of safety data. We feel confident giving it a score of 3."
Answered by AI
Has this type of research been conducted before?
"Since 2019, N-Acetyl-L-Leucine has been the focus of 4 different live clinical trials spanning 7 cities and 9 countries. This interest in the medication was sparked by a Phase 1 study conducted in 2019 that was sponsored by IntraBio Inc."
Answered by AI
What has been the outcome of research on N-Acetyl-L-Leucine in the past?
"N-Acetyl-L-Leucine was first observed in a clinical setting at Mayo Clinic in 2019. As of right now, there have been 18,291 completed trials involving this medication. Additionally, there are 4 on-going studies that are actively recruiting participants; many of these trials are based out of Rochester, Minnesota."
Answered by AI
How many patients will be participating in this clinical trial?
"That is correct, the most recent information on clinicaltrials.gov states that this trial is looking for 53 individuals to participate. The study was first posted on 6/30/2022 and updated last on 10/20/2020. There are currently 2 locations where recruitment is taking place."
Answered by AI
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