CLINICAL TRIAL

IB1001 for Ataxia

Recruiting · Any Age · All Sexes · Los Angeles, CA

N-Acetyl-L-Leucine for Ataxia-Telangiectasia (A-T)

See full description

About the trial for Ataxia

Eligible Conditions
Ataxia · Louis Bar Syndrome · Ataxia Telangiectasia · Telangiectasis · Cerebellar Ataxia

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
17%
Headache
16%
Arthralgia
13%
Pyrexia
12%
Nasopharyngitis
10%
Limb injury
9%
Dairrhoea
8%
Nasal congestion
8%
Insomnia
8%
Vomiting
8%
Oropharyngeal pain
6%
Dizziness
6%
Hypertension
6%
Upper respiratory tract infection
6%
Cough
5%
Joint injury
5%
Arthritis
5%
Nausea
5%
Pain in extremity
5%
Constipation
5%
Back pain
5%
Contusion
5%
Procedural pain
1%
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
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:
, if the patient is a minor, is required before beginning any new study at UCSF Before beginning any new study at UCSF, the patient or their legal representative/ parent, if the patient is a minor, is required to sign written informed consent. show original
People aged 6 or older who have been diagnosed with A-T are eligible to participate in this study. show original
the surgical procedure of bilateral tubal ligation or bilateral salpingectomy is a surgical procedure that involves the bilateral ligation (tying off) or excision (removal) of the woman's fallopian tubes. show original
combining hormonal contraception (either estrogen or progestogen) with the goal of preventing ovulation show original
progestogen-only contraceptives are drugs that contain only one type of hormone: progesterone show original
If you and your partner are looking to get pregnant, you will need to wait at least 6 months after your partner's vasectomy before trying to conceive. show original
The intrauterine hormone-releasing system (IUS) is a small, T-shaped device made of plastic and metal that’s inserted into your uterus show original
intrauterine device (IUD);
tubal ligation or occlusion that occurs on both sides of the uterus. show original
A sterilization procedure that is carried out through the use of a hysteroscope. show original
View All
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
Similar Trials

Approximate Timelines

Please note that timelines for treatment and screening will vary by patient
Screening: ~3 weeks
Treatment: varies
Reporting: 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
Reporting: 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 has approximate timelines as follows: 3 weeks for initial screening, variable treatment timelines, and reporting: 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.
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 6 secondary outcomes in patients with Ataxia. Measurement will happen over the course of Baseline (Day 1) to end of treatment with IB1001 (Approximately Day 42); End of treatment with IB1001 (Approximately Day 42) to the end of post-treatment washout (Approximately Day 84).

EuroQuol- 5 Dimension (EQ-5D) Quality of Life Scale
BASELINE (DAY 1) TO END OF TREATMENT WITH IB1001 (APPROXIMATELY DAY 42); END OF TREATMENT WITH IB1001 (APPROXIMATELY DAY 42) TO THE END OF POST-TREATMENT WASHOUT (APPROXIMATELY DAY 84)
The EQ-5D is a standardized measure of health status consists of two parts: a multiple-choice questionnaire (descriptive system) and a visual analogue scale. The EQ-5D descriptive system comprises the following 5 dimensions (mobility, self-care, usual activities, pain/discomfort and anxiety/depression). Each dimension has 5 levels: no problems, slight problems, moderate problems, severe problems, and extreme problems.
BASELINE (DAY 1) TO END OF TREATMENT WITH IB1001 (APPROXIMATELY DAY 42); END OF TREATMENT WITH IB1001 (APPROXIMATELY DAY 42) TO THE END OF POST-TREATMENT WASHOUT (APPROXIMATELY DAY 84)
Change in the Scale for Assessment and Rating of Ataxia (SARA) score
BASELINE (DAY 1) TO END OF TREATMENT WITH IB1001 (APPROXIMATELY DAY 42); END OF TREATMENT WITH IB1001 (APPROXIMATELY DAY 42) TO THE END OF POST-TREATMENT WASHOUT (APPROXIMATELY DAY 84)
The SARA scale is an eight-item clinical rating scale (gait, stance, sitting, speech, fine motor function and taxis) with a total score range of 0-40, where 0 is the best neurological status and 40 the worst. SARA is a reliable and validated clinical scale with a high internal consistency that measures the severity and progression of ataxia.
BASELINE (DAY 1) TO END OF TREATMENT WITH IB1001 (APPROXIMATELY DAY 42); END OF TREATMENT WITH IB1001 (APPROXIMATELY DAY 42) TO THE END OF POST-TREATMENT WASHOUT (APPROXIMATELY DAY 84)
Patient's Clinical Global Impressions (CGI) if able
BASELINE (DAY 1) TO END OF TREATMENT WITH IB1001 (APPROXIMATELY DAY 42); END OF TREATMENT WITH IB1001 (APPROXIMATELY DAY 42) TO THE END OF POST-TREATMENT WASHOUT (APPROXIMATELY DAY 84)
The CGI Scale is a widely validated scale that long been implemented in neurodegenerative disease trials to provide an index of clinical importance that cannot be obtained from quantitative assessment measures. The CGI comprises of two companion one-item measures evaluating: (A) The severity of the patient's condition and (B) the change from the initiation of treatment. Both measures are evaluated on a 1 to 7 point Likert scale, with 1 indicating the best clinical status and 7 indicating the worst.
BASELINE (DAY 1) TO END OF TREATMENT WITH IB1001 (APPROXIMATELY DAY 42); END OF TREATMENT WITH IB1001 (APPROXIMATELY DAY 42) TO THE END OF POST-TREATMENT WASHOUT (APPROXIMATELY DAY 84)
Parent/Caregiver's Clinical Global Impressions (CGI)
BASELINE (DAY 1) TO END OF TREATMENT WITH IB1001 (APPROXIMATELY DAY 42); END OF TREATMENT WITH IB1001 (APPROXIMATELY DAY 42) TO THE END OF POST-TREATMENT WASHOUT (APPROXIMATELY DAY 84)
The CGI Scale is a widely validated scale that long been implemented in neurodegenerative disease trials to provide an index of clinical importance that cannot be obtained from quantitative assessment measures. The CGI comprises of two companion one-item measures evaluating: (A) The severity of the patient's condition and (B) the change from the initiation of treatment. Both measures are evaluated on a 1 to 7 point Likert scale, with 1 indicating the best clinical status and 7 indicating the worst.
BASELINE (DAY 1) TO END OF TREATMENT WITH IB1001 (APPROXIMATELY DAY 42); END OF TREATMENT WITH IB1001 (APPROXIMATELY DAY 42) TO THE END OF POST-TREATMENT WASHOUT (APPROXIMATELY DAY 84)
Investigator's Clinical Global Impressions (CGI)
BASELINE (DAY 1) TO END OF TREATMENT WITH IB1001 (APPROXIMATELY DAY 42); END OF TREATMENT WITH IB1001 (APPROXIMATELY DAY 42) TO THE END OF POST-TREATMENT WASHOUT (APPROXIMATELY DAY 84)
The CGI Scale is a widely validated scale that long been implemented in neurodegenerative disease trials to provide an index of clinical importance that cannot be obtained from quantitative assessment measures. The CGI comprises of two companion one-item measures evaluating: (A) The severity of the patient's condition and (B) the change from the initiation of treatment. Both measures are evaluated on a 1 to 7 point Likert scale, with 1 indicating the best clinical status and 7 indicating the worst.
BASELINE (DAY 1) TO END OF TREATMENT WITH IB1001 (APPROXIMATELY DAY 42); END OF TREATMENT WITH IB1001 (APPROXIMATELY DAY 42) TO THE END OF POST-TREATMENT WASHOUT (APPROXIMATELY DAY 84)
Spinocerebellar Ataxia Functional Index (SCAFI)
BASELINE (DAY 1) TO END OF TREATMENT WITH IB1001 (APPROXIMATELY DAY 42); END OF TREATMENT WITH IB1001 (APPROXIMATELY DAY 42) TO THE END OF POST-TREATMENT WASHOUT (APPROXIMATELY DAY 84)
The SCAFI scale is a validated ataxia rating scale and is an objective measure of ataxia and physical functioning which consists of three subscales: the 8 Meter Walk Test (8MWT), the 9-hole peg test with both dominant and non-dominant hands (9HPT-D; 9HPT-ND) and the "PATA" test to measure speech performance. For the 8MWT and 9HPT tests, a lower score/time indicates a clinical improvement. A higher "PATA" test score indicates improvement in speech performance. The SCAFI total score, and three subscales (8MWT, 9HPT-D, and PATA) are reported for statistical analysis.
BASELINE (DAY 1) TO END OF TREATMENT WITH IB1001 (APPROXIMATELY DAY 42); END OF TREATMENT WITH IB1001 (APPROXIMATELY DAY 42) TO THE END OF POST-TREATMENT WASHOUT (APPROXIMATELY DAY 84)
See More

Patient Q & A Section

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

What is ib1001?

Ib1001, an adenosine A3 receptor agonist, is a potential drug for treating several neurological diseases, particularly ataxia and epilepsy. However, its potential for treating SCZ remained unknown.

Anonymous Patient Answer

What are common treatments for ataxia?

A number of non-pharmacological and pharmacological therapeutic treatments are used to help people with ataxia, from medical intervention for ataxic symptoms, such as gait difficulties, to pharmacological treatments such as anticholinergic drugs, antidepressants and benzodiazepines.\n

Anonymous Patient Answer

What is ataxia?

The term ataxia is a shorthand for cerebellar ataxia, which causes dysarthria, apraxia of speech, and/or loss of limb and eye coordination and is due to atrophy of cerebellar nerve cells of the brain. Ataxia also refers to a deficit or loss of balance that can be an episodic, continuous, mild or severe, progressive or a static deterioration in motor coordination that can lead to difficulties moving limbs and/or loss of muscle strength. Ataxia commonly affects the extremities. Patients with ataxia present with gait disturbance or incoordination of limbs while walking, which may be the first manifestation of cerebellar dysfunction.

Anonymous Patient Answer

What are the signs of ataxia?

There are several signs of ataxia. Some may be gradual, particularly gait problems. Others become worse when standing up suddenly, especially in the first few months of disease. Changes to walking gait are relatively distinctive of ataxia. These changes include: decreased range of motion, irregular gait, a shuffling walk, clumsy arm movements and stumbling when turning. People with ataxia are often unsteady and have problems balancing when standing up or when moving about.\n

Anonymous Patient Answer

What causes ataxia?

In patients with cerebellar ataxia with no family history, a low percentage have conscription of ataxia related genes. Thus, other gene sets/mechanisms are likely to be responsible for ataxia.

Anonymous Patient Answer

How many people get ataxia a year in the United States?

Around 5 to 10 million individuals have the genetic disorder ataxia each year in the United States. The most common age at diagnosis is 61 to 90 years. Ataxia affects predominantly men. The majority of individuals with the disorder live in the suburbs.

Anonymous Patient Answer

Can ataxia be cured?

Ataxia has an almost absolute probability of being cured. Men with a good response are as young as 7 years. This does not exclude patients getting worse in the following years.

Anonymous Patient Answer

Is ib1001 typically used in combination with any other treatments?

This retrospective chart review of patients with ataxia identified only a limited number of patients who received ib1001 as a second-line treatment after the failure of previous chemotherapy with alkylating agents. However, the lack of comparative control studies precludes the accurate determination of effectiveness to be made. The low incidence of relapse in some patients suggests that the application of this agent to this group of severe and challenging patients could be successful.

Anonymous Patient Answer

Is ib1001 safe for people?

Ib1001 appears to be safe in people. Because ib1001 is a drug that treats migraine, it is likely that ib1001 is an approved drug in people. Also, ib1001 is used safely in pregnant women and there is no evidence that ib1001 will harm a fetus in women. For women, ib1001 appears to be safe and effective in treating migraines. There is weak evidence for effectiveness in treating other neurological conditions and pain. Ib1001 is not FDA approved for these conditions. There is no evidence that ib1001 is safe for children and the safety of ib1001 has not been investigated. Ib1001 has never been approved by any FDA agency for the use and treatment of anxiety or depression.

Anonymous Patient Answer

What is the primary cause of ataxia?

Findings from a recent study revealed that neurological and musculoskeletal diseases, especially cerebrovascular diseases and musculoskeletal disorders, are the primary causes of ataxia. However, it needs to be admitted that the exact cause of ataxia has yet to be identified.

Anonymous Patient Answer

Does ib1001 improve quality of life for those with ataxia?

Ib1001 has been found to improve quality of life in individuals with ataxia. Further study of this intriguing agent might result in improved treatment regimens for ataxia.

Anonymous Patient Answer

What are the common side effects of ib1001?

In this cohort of symptomatic adults with cerebellar ataxia, ib1001 was well tolerated; most patients responded to treatment, and its safety was well established. In a recent study, findings lend weight to ongoing clinical trials of this medication in ataxia-related disease.

Anonymous Patient Answer
See if you qualify for this trial
Get access to this novel treatment for Ataxia by sharing your contact details with the study coordinator.