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Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Male or female aged ≥6 years with a confirmed diagnosis of A-T at the time of signing informed consent
Patients must fall within specified SARA score and be able to perform specific tests
Timeline
Screening 3 weeks
Treatment Varies
Follow Up ci-cs comparing baseline (day 1) with ib1001 verses the end of 6-weeks treatment with ib1001 (approximately day 42) minus the ci-cs comparing the end of 6-weeks treatment with ib1001 (approximately day 42) verses the end of 6-weeks post-treatment washout
Awards & highlights
Study Summary
This trial will assess the safety and efficacy of N-Acetyl-L-Leucine (IB1001) for the treatment of Ataxia-Telangiectasia (A-T). There are two phases to the study: the Parent Study and the Extension Phase. The Parent Study will evaluate the short-term safety and efficacy of IB1001, while the Extension Phase will assess the long-term safety and efficacy of IB1001.
Who is the study for?
This trial is for males and females aged 6 or older diagnosed with Ataxia-Telangiectasia (A-T). Participants must have a specific level of disease severity, weigh at least 15 kg, and be willing to follow study procedures. Women who can bear children must use effective contraception or abstain from sex.Check my eligibility
What is being tested?
The trial is testing N-Acetyl-L-Leucine (IB1001) in two phases: the Parent Study for symptomatic treatment of A-T, and the Extension Phase for long-term safety and potential disease-modifying effects.See study design
What are the potential side effects?
Potential side effects are not detailed here but monitoring for safety is a key part of this study. Given it's an open-label trial, any observed side effects will be reported by participants throughout the study duration.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I am 6 years or older and have been diagnosed with A-T.
Select...
My SARA score is within the required range and I can perform certain physical tests.
Select...
I am a woman who cannot have children because I had surgery over 6 months ago or have not had a period for at least 1 year.
Select...
I weigh at least 15 kg.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ ci-cs comparing baseline (day 1) with ib1001 verses the end of 6-weeks treatment with ib1001 (approximately day 42) minus the ci-cs comparing the end of 6-weeks treatment with ib1001 (approximately day 42) verses the end of 6-weeks post-treatment washout
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~ci-cs comparing baseline (day 1) with ib1001 verses the end of 6-weeks treatment with ib1001 (approximately day 42) minus the ci-cs comparing the end of 6-weeks treatment with ib1001 (approximately day 42) verses the end of 6-weeks post-treatment washout
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Clinical Impression of Change in Severity (CI-CS)
Secondary outcome measures
Change in the Scale for Assessment and Rating of Ataxia (SARA) score
EuroQuol- 5 Dimension (EQ-5D) Quality of Life Scale
Investigator's Clinical Global Impressions (CGI)
+3 moreSide effects data
From 2016 Phase 2 & 3 trial • 77 Patients • NCT0076828717%
Headache
16%
Arthralgia
13%
Pyrexia
12%
Nasopharyngitis
10%
Limb injury
9%
Dairrhoea
8%
Vomiting
8%
Insomnia
8%
Nasal congestion
8%
Oropharyngeal pain
6%
Hypertension
6%
Upper respiratory tract infection
6%
Dizziness
6%
Cough
5%
Procedural pain
5%
Constipation
5%
Arthritis
5%
Back pain
5%
Nausea
5%
Contusion
5%
Pain in extremity
5%
Joint injury
1%
Abdominal pain
1%
Diverticulitis
1%
Wound infection
1%
Postoperative wound infection
1%
Skin laceration
1%
Lumbar vertebral fracture
1%
Periprosthetic fracture
1%
Mental status changes
1%
Femur fracture
1%
Haematoma
100%
80%
60%
40%
20%
0%
Study treatment Arm
IB1001 Safety Population
Trial Design
2Treatment groups
Experimental Treatment
Active Control
Group I: Treatment with IB1001Experimental Treatment1 Intervention
6-weeks treatment with IB1001 administered orally.
Patients ≥13 years old will receive a total daily dose of 4 g/day (administered as 3 doses per day).
Patients 6-12 years old will receive weight-tiered doses:
Patients aged 6-12 years weighing 15 to <25 kg will take 2 g per day: 1 g in the morning and 1 g in the evening.
Patients aged 6-12 years weighing 25 to <35 kg will take 3 g per day: 1 g in the morning, 1 g in the afternoon, and 1 g in the evening.
Patients aged 6-12 years weighing ≥35 kg will take 4 g per day: 2 g in the morning, 1g in the afternoon and 1 g in the evening (as per adults)
After the 6-week treatment period, patients will enter a 6-week post-treatment washout period.
Group II: Post-Treatment WashoutActive Control1 Intervention
After the 6-week treatment period, patients will enter a 6-week post-treatment washout period.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Trenonacog alfa
Not yet FDA approved
Find a Location
Who is running the clinical trial?
IntraBio IncLead Sponsor
3 Previous Clinical Trials
116 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I have not been sexually active or have used effective birth control for the required time.I am 6 years or older and have been diagnosed with A-T.You have a history of using drugs or alcohol in a way that may cause dependence or harm.I have chronic diarrhea, unexplained vision loss, cancer, or need insulin for diabetes.I do not have any symptoms.I agree to use contraception or abstain from sex during and 90 days after the study.I am willing to share my current medications for A-T and keep my treatment consistent during the study.You have important abnormalities in your blood, urine, or other lab tests.My SARA score is within the required range and I can perform certain physical tests.I show symptoms of A-T but my genetic test for A-T was negative.I have arthritis or a condition that affects my ability to move.I am allergic to N-Acetyl-Leucine or Ora-Blend ingredients.I am willing and able to stop taking certain medications for 6 weeks before my first visit and can stay off them until my sixth visit.I am a woman who cannot have children because I had surgery over 6 months ago or have not had a period for at least 1 year.I weigh at least 15 kg.I agree not to donate sperm for 90 days after my last dose.
Research Study Groups:
This trial has the following groups:- Group 1: Treatment with IB1001
- Group 2: Post-Treatment Washout
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
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.
Is IB1001 a danger to human health?
"IB1001 has undergone some safety testing and received a score of 2."
Answered by AI
Are there any unfilled positions in this trial for potential participants?
"Yes, as indicated on the website clinicaltrials.gov, this study is recruiting patients at this time. The trial was first posted on 1/8/2020 and updated most recently on 8/2/2022; currently, 39 participants are needed from a single location."
Answered by AI
Have there been similar medical studies before?
"IB1001 has been researched since 2019, when the first sponsored trial by IntraBio Inc. took place. The study involved 39 people and was completed in that same year. After the initial success of the Phase 1 IB1001 drug approval, there have been 4 active studies conducted in 7 cities across 9 countries."
Answered by AI
How many study participants are needed for this clinical trial?
"Indeed, the clinical trial is still recruiting patients as of 8/2/2022. The posting for this study went up on 1/8/2020. At present, they are looking for 39 more participants from a single site."
Answered by AI
What is the scientific community's consensus on IB1001?
"As of right now, there are 4 clinical trials that are still ongoing and investigating IB1001. One of these trials is in Phase 3. Most of the research being conducted for IB1001 is taking place in Manchester, Greater Manchester; however, 38 different locations worldwide are running studies concerning IB1001."
Answered by AI
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