IB1001 Clinical Trials

IB1001 Clinical Trials

The IB1001-201 (NPC) and IB1001-202 (GM2) clinical trials are currently active and enrolling patients.

Information about each active clinical center, including contact details, can be found on ClinicalTrials.Gov: 

IB1001-201 – Effects of N-Acetyl-L-Leucine on Niemann Pick Type C Disease: A Multinational, Multicenter, Open-label, Rater-blinded Phase II Study

 IB1001-201 ClinicalTrials.gov (NCT03759639)

IB1001-202 – Effects of N-Acetyl-L-Leucine on GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease): A Multinational, Multicenter, Open-label, Rater-blinded Phase II Study

IB1001-202 ClinicalTrials.gov (NCT03759665)

IB1001-203 Effects of N-Acetyl-L-Leucine on Ataxia-Telangiectasia (A-T): A Multinational, Multicenter, Open-label, Rater-blinded Phase II Study

IB1001-203 ClinicalTrials.gov (NCT03759678)

FREQUENTLY ASKED QUESTIONS

What are the IB1001 Clinical Studies?

IB1001-201 (NPC), IB1001-202 (GM2), and IB1001-203 (A-T) are three parallel studies which utilize a master protocol to investigate IB1001 for the treatment of three rare, serious, diseases: NPC, GM2, and A-T.

What is the goal of the IB1001 studies?

The goal of the studies is to gather scientific evidence to demonstrate that IB1001 is safe and effective at treating symptoms and improving functioning and quality of life in patients with NPC, GM2, and A-T.

These assessments will be made based on the clinical outcomes measurements such as the 8 Meter Walk Test (8MWT) and 9 Hole Peg Test (9HPT), the Scale for Assessment and Rating of Ataxia (SARA), Clinical Global Impression (CGI) Scales (by Physicians, Caregivers, and Patients), and Quality of Life Questionnaires.

In addition, the study aims to further characterize the safety and tolerability profile of IB1001 for the treatment of NPC, GM2, and A-T.

How long are the IB1001 studies?

Following a screening period which will last approximately 2 – 8 weeks, patients will take part in the studies for approximately 12 weeks.

During the study period, there will be 6 -7 visits to the study site, depending on the duration of the screening period.

At trial sites that are able, 1-year rollover studies are planned to be conducted.

How many patients will be enrolled?

Each study will recruit approximately 30 patients, for a total of approximately 90 patients:

IB1001-201 (NPC): 30 Patients Approximately

IB1001-202 (GM2): 30 Patients Approximately

IB1001-203 (A-T): 30 Patients Approximately

Will all patients receive treatment?

All patients enrolled in the trials will receive treatment with IB1001. There is no placebo arm.

 Where are the trial sites?

The trials will be conducted at key specialist centers to clearly establish the safety and efficacy of IB1001 for the treatment of these rare, life-threatening conditions.

Clinical trial sites are currently planned to be established in the United States, United Kingdom, Spain, Slovakia, and Germany. Clinical trial sites may vary for each specific study by country/location.

ClinicalTrials.Gov will be updated regularly with details on clinical trial centers for each study in each country.

Who is eligible to participate in the trials?

In Europe, patients ages 6 and older with a confirmed genetic diagnosis of Niemann-Pick disease Type C (NPC), GM2 Gangliosidosis (Tay-Sachs or Sandhoff Disease), or Ataxia-Telangiectasia (A-T) may be accepted to participate in the trials provided they meet the eligibility criteria as determined by the doctors involved in the trials.

In the United States, patients ages 18 and older with a confirmed genetic diagnosis of Niemann-Pick disease Type C (NPC), GM2 Gangliosidosis (Tay-Sachs or Sandhoff Disease), or Ataxia-Telangiectasia (A-T) may be accepted to participate in the trials provided they meet the eligibility criteria as determined by the doctors involved in the trials.

The full eligibility criteria for the studies can be found at ClinicalTrials.Gov

Why are the eligibility requirements different in the US and Europe?

The eligibility requirements vary according to each national regulatory requirements, and the informed assessment of each national agency responsible for approving the clinical trials.

Who will be selecting the participants?

The Principal Investigator at each clinical site is responsible for determining if a patient is eligible to participate in the clinical trial.

Will patients have to cover the costs to travel to participate in the trial?

Patients will be reimbursed for reasonable out-of-pocket expenses, such as travel expenses that will be incurred for participating in the trial.

When will the trials start?

Enrollment in the IB1001-201 and IB1001-202 studies is ongoing. Enrollment in the IB1001-203 study is expected to begin in Q3 2019.

All announcements or statements regarding the status of Approvals will be highlighted in the News section of our website. 

IB1000s Series