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Amino Acid Derivative

IB1001 for Ataxia

Phase 2

Recruiting

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 ≥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

Must not have

Patients with chronic diarrhea, unexplained visual loss, malignancies, or insulin-dependent diabetes mellitus

Patient has clinical features of A-T, but a completely negative result on a previous genetic test for A-T

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

No Placebo-Only Group

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.

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.

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 below

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I am 6 years or older and have been diagnosed with A-T.

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My SARA score is within the required range and I can perform certain physical tests.

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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.

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I weigh at least 15 kg.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have chronic diarrhea, unexplained vision loss, cancer, or need insulin for diabetes.

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I show symptoms of A-T but my genetic test for A-T was negative.

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I have arthritis or a condition that affects my ability to move.

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I am allergic to N-Acetyl-Leucine or Ora-Blend ingredients.

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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.

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

This trial's timeline: 3 weeks for screening, Varies for treatment, and 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 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Clinical Impression of Change in Severity (CI-CS)

Secondary study objectives

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 more

Side effects data

From 2016 Phase 2 & 3 trial • 77 Patients • NCT00768287

17%

Headache

16%

Arthralgia

13%

Pyrexia

12%

Nasopharyngitis

10%

Limb injury

Dairrhoea

Vomiting

Insomnia

Nasal congestion

Oropharyngeal pain

Dizziness

Hypertension

Upper respiratory tract infection

Cough

Constipation

Nausea

Procedural pain

Back pain

Arthritis

Contusion

Pain in extremity

Joint injury

Diverticulitis

Skin laceration

Periprosthetic fracture

Abdominal pain

Mental status changes

Lumbar vertebral fracture

Wound infection

Femur fracture

Postoperative wound infection

Haematoma

100%

80%

60%

40%

20%

Study treatment Arm

IB1001 Safety Population

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

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

0 / 9Eligibility criteria met