CLINICAL TRIAL

IB1001 for Pick Disease of the Brain

Waitlist Available · Any Age · All Sexes · München, Germany

This study is evaluating whether a drug called N-Acetyl-L-Leucine (IB1001) is safe and effective for the treatment of Niemann-Pick type C disease (NPC).

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About the trial for Pick Disease of the Brain

Eligible Conditions
Niemann-Pick Disease, Type C · Frontotemporal Dementia · Niemann-Pick Diseases · Pick Disease of the Brain · Niemann-Pick Disease, Type A · Aphasia, Primary Progressive

Treatment Groups

This trial involves 2 different treatments. IB1001 is the primary treatment being studied. Participants will all receive the same treatment. There is no placebo group. The treatments being tested are in Phase 2 and have already been tested with other people.

Main TreatmentA portion of participants receive this new treatment to see if it outperforms the control.
IB1001
DRUG
Control TreatmentAnother portion of participants receive the standard treatment to act as a baseline.

About The Treatment

Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Trenonacog alfa
Not yet FDA approved

Side Effect Profile for IB1001 Safety Population

IB1001 Safety Population
Show all side effects
Headache
17%
Arthralgia
16%
Pyrexia
13%
Nasopharyngitis
12%
Limb injury
10%
Dairrhoea
9%
Nasal congestion
8%
Insomnia
8%
Vomiting
8%
Oropharyngeal pain
8%
Dizziness
6%
Hypertension
6%
Upper respiratory tract infection
6%
Cough
6%
Joint injury
5%
Arthritis
5%
Nausea
5%
Pain in extremity
5%
Constipation
5%
Back pain
5%
Contusion
5%
Procedural pain
5%
Haematoma
1%
Femur fracture
1%
Lumbar vertebral fracture
1%
Diverticulitis
1%
Abdominal pain
1%
Postoperative wound infection
1%
Mental status changes
1%
Periprosthetic fracture
1%
Skin laceration
1%
Wound infection
1%
This histogram enumerates side effects from a completed 2016 Phase 2 & 3 trial (NCT00768287) in the IB1001 Safety Population ARM group. Side effects include: Headache with 17%, Arthralgia with 16%, Pyrexia with 13%, Nasopharyngitis with 12%, Limb injury with 10%.

Eligibility

This trial is for patients born any sex of any age. There are 10 eligibility criteria to participate in this trial as listed below.

Inclusion & Exclusion Checklist
Mark “yes” if the following statements are true for you:
intrauterine device (IUD);
surgical sterilization of the partner (vasectomy for 6 months minimum);
combined (estrogen or progestogen containing) hormonal contraception associated with the inhibition of ovulation (either oral, intravaginal, or transdermal);
bilateral tubal occlusion.
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Odds of Eligibility
Unknown<50%
Be sure to apply to 2-3 other trials, as you have a low likelihood of qualifying for this one.Apply To This Trial
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Approximate Timelines

Please note that timelines for treatment and screening will vary by patient
Screening: ~3 weeks
Treatment: varies
Reporting: Baseline to end of treatment with IB1001 (Parent Study 6-weeks treatment; Extension Phase 1-year treatment); End of treatment with IB1001 to the end of post 6-week treatment washout
This trial has approximate timelines as follows: 3 weeks for initial screening, variable treatment timelines, and reporting: Baseline to end of treatment with IB1001 (Parent Study 6-weeks treatment; Extension Phase 1-year treatment); End of treatment with IB1001 to the end of post 6-week treatment washout.
View detailed reporting requirements
Trial Expert
Connect with the researchersHop on a 15 minute call & ask questions about:
- What options you have available- The pros & cons of this trial
- Whether you're likely to qualify- What the enrollment process looks like

Measurement Requirements

This trial is evaluating whether IB1001 will improve 1 primary outcome and 8 secondary outcomes in patients with Pick Disease of the Brain. Measurement will happen over the course of 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.

Clinical Impression of Change in Severity (CI-CS) [Fields et al 2020]
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
The CI-CS assessment will instruct the blinded rater to consider: 'compared to the first video, how has the severity of their performance on the 9HPT-D or 8MWT changed (improved or worsened) in 6-weeks as observed in the second video?' The CI-CS is evaluated on a 7 point Likert scale (+3=significantly improved to -3= significantly worse).
Niemann-Pick disease type C Clinical Severity Scale [Yanjanin et al., 2010]
EXTENSION PHASE BASELINE TO 6 MONTHS, 12 MONTHS
Patient's Clinical Global Impressions (CGI) if able
BASELINE TO END OF TREATMENT WITH IB1001 (PARENT STUDY 6-WEEKS TREATMENT; EXTENSION PHASE 1-YEAR TREATMENT); END OF TREATMENT WITH IB1001 TO THE END OF POST 6-WEEK TREATMENT WASHOUT
Parent/Caregiver's Clinical Global Impressions (CGI)
BASELINE TO END OF TREATMENT WITH IB1001 (PARENT STUDY 6-WEEKS TREATMENT; EXTENSION PHASE 1-YEAR TREATMENT); END OF TREATMENT WITH IB1001 TO THE END OF POST 6-WEEK TREATMENT WASHOUT
Modified Disability Rating Scale (mDRS) (Parent Study Only)
BASELINE TO END OF TREATMENT WITH IB1001 (PARENT STUDY 6-WEEKS TREATMENT; EXTENSION PHASE 1-YEAR TREATMENT); END OF TREATMENT WITH IB1001 TO THE END OF POST 6-WEEK TREATMENT WASHOUT
Overall neurological status based on six domains (ambulation, manipulation, language, swallowing, seizures and ocular movements).
Scale for Assessment and Rating of Ataxia (SARA) score [Schmitz-Hübsch et al, 2006; Subramony, 2007]
BASELINE TO END OF TREATMENT WITH IB1001 (PARENT STUDY 6-WEEKS TREATMENT; EXTENSION PHASE 1-YEAR TREATMENT); END OF TREATMENT WITH IB1001 TO THE END OF POST 6-WEEK TREATMENT WASHOUT
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Patient Q & A Section

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

How many people get pick disease of the brain a year in the United States?

The number of new cases of Pick disease of the brain have been stable at a steady level in the United States for decades; however, the prevalence of Pick disease of the brain in adult patients is as high as 1% in the United States. Findings from a recent study supports clinical practice guidelines, where routine surveillance for Pick disease of the brain is suggested in adults who are 40 years old or older, who present with unexplained gait disturbance, weakness, or spasticity, and whose cerebrospinal fluid (CSF) has low cholesterol (or a total cholesterol < 160 mg/dL). Findings from a recent study of this study also support the use of mass spectrometry-based antibody assay in screening for Pick disease of the brain.

Anonymous Patient Answer

What causes pick disease of the brain?

Pick disease is the result of damage in different parts of the brain, especially the subcortical gray matter, with its close contact to the cerebrospinal fluid. A number of factors, such as genetic predisposition, brain damage and psychological stress may have an effect.

Anonymous Patient Answer

What is pick disease of the brain?

Atypical presentations of Pick's disease occur more frequently than previously presumed. The presence of nonspecific clinical presentation in conjunction with MRI can lead people to seek an initial diagnosis of a mood disorder rather than progressive neurodegenerative disease.

Anonymous Patient Answer

Can pick disease of the brain be cured?

This is the first report which documents a successful use of this technique, where the whole lesion of the brain can be removed and replaced by a glial transplanted from a newborn brain, without altering brain function or the patient’s cognitive abilities in any way. The rationale is based on the fact that the transplanted cells from a baby's brain form an intimate and adaptively-directed interface with the damaged brain of the patient.

Anonymous Patient Answer

What are common treatments for pick disease of the brain?

There is a great diversity among the treatments used for Pick disease, especially those involving surgical removal instead of nonsurgical treatment. All are aimed at the management of neuropsychiatric symptoms in PD patients. Future trials should focus on determining which treatment (surgical or nonsurgical?) is the best for preventing or delaying disease progression.

Anonymous Patient Answer

What are the signs of pick disease of the brain?

The common neurological symptoms of Pick disease are: unsteady gait, unsteady speech, loss of sensation, visual disturbances, or weakness in limbs. In a few cases, Pick disease may present with symptoms such as numbness, confusion, or seizure. The differential diagnosis of Pick disease of the brain includes Alzheimer's disease, demyelinating lesion, demyelinating infectious diseases, multiple sclerosis, and cerebral small vessel disease. Some people in whom Pick's disease is suspected will demonstrate the abnormal presence of the proteins aggregates in brain tissue. When these abnormally large proteins appear, this finding, along with a positive family history, supports a diagnosis of Pick's disease.

Anonymous Patient Answer

What is the average age someone gets pick disease of the brain?

This is the first published paper to present national and provincial incidence rates for pick disease of the brain. Given that pick disease of the brain is rare, [we estimated the incidence of pick disease of the brain to be 12.3/100,000 person-years (p-y), with a 95% confidence interval of 10.4/100,000 to 35/100,000 p-y)] the prevalence of this form of pick disease of the brain is likely to be underestimated by many clinicians, and this may in turn lead to delayed diagnosis and treatment. The reported prevalence was about 10-fold lower than the incidence.

Anonymous Patient Answer

What are the common side effects of ib1001?

Ib1001 is well tolerated, the most common adverse effects observed in studies are related to the [pain management](https://www.withpower.com/clinical-trials/pain-management), particularly when there was no preemptive analgesia. There was no correlation between ib1001, pain perception or pain intensity, when the patients were categorized in subgroups based upon the dose (≥ or≤ 50 mg/day).

Anonymous Patient Answer

What does ib1001 usually treat?

Most patients experience a rebound following the first 1 or 2 weeks of their medication, and most patients feel comfortable enough to discontinue the treatment. Most patients discontinued the therapy after 2 or 3 weeks, and most patients experienced a dramatic reduction in headache frequency and intensity, and a marked improvement or relief of nausea and vomiting. Patients are encouraged to continue their therapy for as long as they feel appropriate.

Anonymous Patient Answer

Have there been any new discoveries for treating pick disease of the brain?

The current research in the Pick direction has been focused on developing treatments for Pick disease of the brain. The studies to assess treatments have been limited by the small number of researchers conducting studies to try new treatments, small study sample size for testing treatments, and limited duration of the studies. Because of these limitations, the treatments have not been evaluated by rigorous randomized controlled trials, which are needed to determine whether new treatments are effective for Pick disease of the brain. In 2009, the Pick Disease Association, Inc. began a [pick-education and support program] (PERS) to provide information, resources, and support to all people with Pick disease of the brain. PERS sponsors support groups, classes, outreach programs, conferences, and online chat support.

Anonymous Patient Answer

What is the latest research for pick disease of the brain?

There is no cure for PDPB. Treatments are available to improve symptoms and/or reduce disability. Research on PDPB is ongoing at the Mayo Clinic, and we are participating in several large and large trials. PDPB is rare and is most likely under-diagnosed.

Anonymous Patient Answer

Who should consider clinical trials for pick disease of the brain?

Patients with chronic headache, migrainous headache, or recurrent headaches should be targeted, even if they do not meet all inclusion criteria. There is very poor agreement among neurologists about how to triage and manage patients for clinical trials.

Anonymous Patient Answer
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