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Amino Acid Derivative

IB1001 for Tay-Sachs Disease

Q: How extensive is the recruitment process for this research endeavor?

This clinical trial is presently not accepting participants. Initially posted on June 7th 2019, this study was last updated in August 2nd 2022. If you're searching for alternative trials, there are 11 studies recruiting patients with Tay-Sachs disease and 3 trials involving IB1001 looking for volunteers.

Q: Does this trial represent a pioneering endeavor?

Since 2019, IntraBio Inc. has been researching IB1001 and was the initial sponsor of its first trial in that same year with a participant pool of 39 individuals. This preliminary study led to Phase 2 approval for this drug candidate and now there are three trials running simultaneously across seven cities and nine nations.

Q: What prior investigations have utilized IB1001 as a research tool?

At present, there are 3 medical studies being conducted to assess the efficacy of IB1001. Out of those trials, one is in Phase 3 clinicals. Most research sites for this drug are located in Rochester <a href="https://www.withpower.com/clinical-trials/minnesota">Minnesota</a>; however, the total number of trial locations extends to 29.

Q: Has IB1001 received the necessary governmental authorization to be available on the market?

As this is a Phase 2 medical trial, there exists some evidence that IB1001 is safe for use. Our team at Power gave it an overall safety score of 2 out of 3.

Q: Are there presently any open spots for participants in this experiment?

This research is no longer actively seeking participants. The post was initially made on June 7th 2019 and the last update occurred on August 2nd 2022. Currently, there are 11 studies accepting patients with Tay-Sachs disease and 3 trials recruiting for IB1001.

Phase 2

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

Timeline

Screening 3 weeks

Treatment Varies

Follow Up (ci-cs comparing baseline [day 1] with ib1001 versus the end of 6-weeks treatment with ib1001 [approximately day 42]) minus (ci-cs comparing the end of 6-weeks treatment with ib1001 [approximately day 42] versus end of 6-weeks post-treatment washout);

Awards & highlights

Study Summary

This trial is assessing the safety and efficacy of N-Acetyl-L-Leucine (IB1001) for the treatment of GM2 Gangliosidosis.

Eligible Conditions

Tay-Sachs Disease
Sandhoff Disease

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ (ci-cs comparing baseline [day 1] with ib1001 versus the end of 6-weeks treatment with ib1001 [approximately day 42]) minus (ci-cs comparing the end of 6-weeks treatment with ib1001 [approximately day 42] versus end of 6-weeks post-treatment washout);

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~(ci-cs comparing baseline [day 1] with ib1001 versus the end of 6-weeks treatment with ib1001 [approximately day 42]) minus (ci-cs comparing the end of 6-weeks treatment with ib1001 [approximately day 42] versus 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 versus the end of 6-weeks treatment with ib1001 [approximately day 42]) minus (ci-cs comparing the end of 6-weeks treatment with ib1001 [approximately day 42] versus end of 6-weeks post-treatment washout); for reporting.

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) [Fields et al 2021]

Secondary outcome measures

EuroQuol- 5 Dimension (EQ-5D) Quality of Life Scale: Visual Analogue Scale (VAS)

Investigator's Clinical Global Impressions of Change (CGI-c)

Key Secondary Endpoint: CI-CS Score Reclassified on a 3-Point Scale

+8 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

Hypertension

Upper respiratory tract infection

Dizziness

Cough

Procedural pain

Constipation

Arthritis

Back pain

Nausea

Contusion

Pain in extremity

Joint injury

Abdominal pain

Diverticulitis

Wound infection

Postoperative wound infection

Skin laceration

Lumbar vertebral fracture

Periprosthetic fracture

Mental status changes

Femur fracture

Haematoma

100%

80%

60%

40%

20%

Study treatment Arm

IB1001 Safety Population

Trial Design

2Treatment groups

Experimental Treatment

Active Control

Group I: Treatment with IB1001Experimental Treatment1 Intervention

Parent Study: 6-weeks treatment with IB1001 administered orally. Extension Phase: 1-year 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)

Group II: Post-Treatment WashoutActive Control1 Intervention

After the Parent Study 6-week treatment period, and Extension Phase one-year 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

Do I qualify?Apply below to find out

Find a Location

Who is running the clinical trial?

IntraBio IncLead Sponsor

3 Previous Clinical Trials

125 Total Patients Enrolled

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

How extensive is the recruitment process for this research endeavor?

"This clinical trial is presently not accepting participants. Initially posted on June 7th 2019, this study was last updated in August 2nd 2022. If you're searching for alternative trials, there are 11 studies recruiting patients with Tay-Sachs disease and 3 trials involving IB1001 looking for volunteers."

Answered by AI

Does this trial represent a pioneering endeavor?

"Since 2019, IntraBio Inc. has been researching IB1001 and was the initial sponsor of its first trial in that same year with a participant pool of 39 individuals. This preliminary study led to Phase 2 approval for this drug candidate and now there are three trials running simultaneously across seven cities and nine nations."

Answered by AI

What prior investigations have utilized IB1001 as a research tool?

"At present, there are 3 medical studies being conducted to assess the efficacy of IB1001. Out of those trials, one is in Phase 3 clinicals. Most research sites for this drug are located in Rochester Minnesota; however, the total number of trial locations extends to 29."

Answered by AI

Has IB1001 received the necessary governmental authorization to be available on the market?

"As this is a Phase 2 medical trial, there exists some evidence that IB1001 is safe for use. Our team at Power gave it an overall safety score of 2 out of 3."

Answered by AI

Are there presently any open spots for participants in this experiment?

"This research is no longer actively seeking participants. The post was initially made on June 7th 2019 and the last update occurred on August 2nd 2022. Currently, there are 11 studies accepting patients with Tay-Sachs disease and 3 trials recruiting for IB1001."

Answered by AI

Recent research and studies

TrialsJournal

A Master Protocol to Investigate a Novel Therapy Acetyl-l-leucine for Three Ultra-rare Neurodegenerative Diseases: Niemann-pick Type C, the GM2 Gangliosidoses, and Ataxia Telangiectasia

Fields, T., M. Patterson, T. Bremova-Ertl, G. Belcher, I. Billington, G. C. Churchill, W. Davis, et al.. 2021. “A Master Protocol to Investigate a Novel Therapy Acetyl-l-leucine for Three Ultra-rare Neurodegenerative Diseases: Niemann-pick Type C, the GM2 Gangliosidoses, and Ataxia Telangiectasia”. Trials. Springer Science and Business Media LLC. doi:10.1186/s13063-020-05009-3.

Scientific ReportsJournal

Acetylation Turns Leucine into a Drug by Membrane Transporter Switching

Churchill, Grant C., Michael Strupp, Cailley Factor, Tatiana Bremova-Ertl, Mallory Factor, Marc C. Patterson, Frances M. Platt, and Antony Galione. 2021. “Acetylation Turns Leucine into a Drug by Membrane Transporter Switching”. Scientific Reports. Springer Science and Business Media LLC. doi:10.1038/s41598-021-95255-5.

clinicaltrials.govStudy

N-Acetyl-L-Leucine for GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease)

2019. "N-Acetyl-L-Leucine for GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03759665.