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Amino Acid Derivative

IB1001 for Niemann-Pick Disease

Q: To what extent does IB1001 pose a threat to the health of those who take it?

Our research team has attributed a rating of 2 to the safety profile of IB1001, as it is currently in Phase 2 testing and there are limited trial results that demonstrate efficacy.

Q: Is this the pioneering instance of its kind in terms of clinical trials?

Currently, four separate trials of IntraBio Inc's IB1001 medication are running in seven cities across nine distinct nations. The first trial of this drug began in 2019 and was sponsored by the aforementioned company. It included 39 participants who ultimately reached Phase 2 approval stage before conclusion. Since then, two more research projects have been completed.

Q: Could you provide a summary of research conducted on IB1001?

Currently, 4 trials are running with IB1001 as the focus. Out of these experiments, 1 is in its final phase. Although Barcelona and <a href="https://www.withpower.com/clinical-trials/minnesota">Minnesota</a> play a major role in this research effort, there are 38 other sites contributing to the clinical trial process.

Q: Are there still opportunities to join this research effort?

At this time, we are not accepting applications for the trial posted on September 4th 2019 and last updated December 5th 2022. For individuals seeking other trials related to Niemann-Pick Disease Type C or IB1001 there are currently 63 and 4 studies respectively that require participants.

Q: What is the participant capacity for this medical research?

Unfortunately, this clinical trial is no longer accepting participants. Initially posted on September 4th 2019 and last updated December 5th 2022, it has since been closed to recruitment. If you are looking for additional studies related to Niemann-Pick Disease Type C or IB1001 there are currently 63 and 4 trials actively enrolling patients respectively.

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 the ci-cs comparing the end of 6-weeks treatment with ib1001 (approximately day 42) versus the end of 6-weeks post-treatment washout

Awards & highlights

Study Summary

This trial is designed to study the safety and effectiveness of a new drug called N-Acetyl-L-Leucine (IB1001) for treating Niemann-Pick type C disease (NPC). The trial has two phases: the Parent Study and the Extension Phase. The Parent Study will evaluate the safety and efficacy of IB1001 for the symptomatic treatment of NPC. The Extension Phase will evaluate the long-term safety and efficacy of IB1001 for the neuroprotective, disease-modifying treatment of NPC.

Eligible Conditions

Niemann-Pick Disease

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline to end of treatment with ib1001 (parent study 6-weeks treatment);end of treatment with ib1001 to the end of post 6-week treatment washout

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline to end of treatment with ib1001 (parent study 6-weeks treatment);end of treatment with ib1001 to the end of post 6-week treatment washout

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline to end of treatment with ib1001 (parent study 6-weeks treatment);end of treatment with ib1001 to the end of post 6-week 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 both 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

122 Total Patients Enrolled

Frequently Asked Questions

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

To what extent does IB1001 pose a threat to the health of those who take it?

"Our research team has attributed a rating of 2 to the safety profile of IB1001, as it is currently in Phase 2 testing and there are limited trial results that demonstrate efficacy."

Answered by AI

Is this the pioneering instance of its kind in terms of clinical trials?

"Currently, four separate trials of IntraBio Inc's IB1001 medication are running in seven cities across nine distinct nations. The first trial of this drug began in 2019 and was sponsored by the aforementioned company. It included 39 participants who ultimately reached Phase 2 approval stage before conclusion. Since then, two more research projects have been completed."

Answered by AI

Could you provide a summary of research conducted on IB1001?

"Currently, 4 trials are running with IB1001 as the focus. Out of these experiments, 1 is in its final phase. Although Barcelona and Minnesota play a major role in this research effort, there are 38 other sites contributing to the clinical trial process."

Answered by AI

Are there still opportunities to join this research effort?

"At this time, we are not accepting applications for the trial posted on September 4th 2019 and last updated December 5th 2022. For individuals seeking other trials related to Niemann-Pick Disease Type C or IB1001 there are currently 63 and 4 studies respectively that require participants."

Answered by AI

What is the participant capacity for this medical research?

"Unfortunately, this clinical trial is no longer accepting participants. Initially posted on September 4th 2019 and last updated December 5th 2022, it has since been closed to recruitment. If you are looking for additional studies related to Niemann-Pick Disease Type C or IB1001 there are currently 63 and 4 trials actively enrolling patients respectively."

Answered by AI