Episodic Ataxia Type 2Phase 24AP in EA2NCT01543750
4-Aminopyridine in Episodic Ataxia Type 2
A Phase 2 study testing 4-Aminopyridine in people with Episodic Ataxia Type 2, run by University of California, Los Angeles at 3 sites in United States.
Trial summary
Episodic ataxia type 2 (EA2) is a rare familial neurological condition characterized by debilitating episodes of vertigo and imbalance. Since the serendipitous discovery of dramatic response of EA2 to acetazolamide, acetazolamide has been the first-line treatment for EA2. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Episodic Ataxia Type 2
- What is being tested?
- 4-Aminopyridine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 0 participants
- Where
- 3 sitesUnited States
- Study dates
- Start not givento not given
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients will be included if they:
- Have EA2 genetically confirmed to harbor mutations in CACNA1A
- Are ≥ 18 years of age
- Are not taking acetazolamide (because of intolerance, poor response, or allergy)
- Are able to maintain a daily log of ataxia episode(s) and report daily by using an Interactive Voice Recording System (IVR) throughout the study
- Experience ≥ 3 ataxia episodes per month during the two-month screening period to qualify for randomization
Who cannot join
- Patients will be excluded if they:
- Have seizures or a history of seizures
- Have first-degree relatives with EA2 and seizures
- Have renal disease with impaired function (Creatinine clearance CrCl≤50ml/min)
- Are pregnant or breast feeding (women of childbearing age will be tested for pregnancy and must be using birth control)
- Are unable to comply with the study requirement
Trial locations and contacts
3 sites in 1 country.
United States3 sites
University of California, Los Angeles (UCLA)
Los Angeles, California, 90095
University of Rochester School of Medicine
Rochester, New York, 14642
University of South Florida
Tampa, Florida, 33612
About this study
Official title: Phase 2 Study of 4-Aminopyridine for the Treatment of Episodic Ataxia Type 2
Episodic ataxia type 2 (EA2) is a rare familial neurological condition characterized by debilitating episodes of vertigo and imbalance. Since the serendipitous discovery of dramatic response of EA2 to acetazolamide, acetazolamide has been the first-line treatment for EA2. Yet, for those patients who do not respond to or cannot tolerate acetazolamide, there is no alternative treatment. The purpose of this randomized trial is to test whether 4-aminopyridine may reduce the ataxia episodes in EA2 as an alternative to acetazolamide. Funding Source - FDA OOPD
Read the detailed description
This study aims to determine whether 4-aminopyridine (4AP) can reduce attacks of ataxia in patients with episodic ataxia type 2 (EA2), a rare but often debilitating condition. Episodic ataxia (EA) is a group of inherited disorders characterized by recurrent, discrete episodes of vertigo and ataxia variably associated with progressive ataxia. EA2, the most common and the best characterized of all the EA syndromes, is caused by heterozygous mutations in CACNA1A, which encodes the main subunit of a neuronal voltage-gated calcium channel, Cav2.1.
Although observational data suggest symptomatic resolution with acetazolamide in many EA2 patients, the investigators found in our patient databases that at least a third of the EA2 patients continue to suffer debilitating ataxia attacks, either because of incomplete control while on acetazolamide or because of intolerability or hypersensitivity to acetazolamide. For these patients there is no alternative intervention. 4-Aminopyridine (4AP) has been found to be helpful in a handful of patients with EA2. Recently, dalfampridine, an extended release formulation of 4AP (AMPYRA) by Acorda Therapeutics, received FDA approval to improve gait in multiple sclerosis.
The investigators plan to recruit 20 subjects with genetically defined EA2 who suffer frequent ataxia episodes (at least 3 episodes a month) to conduct a randomized trial of 4AP to examine its efficacy and tolerability in EA2. Study subjects will be recruited at UCLA and the University of Rochester to participate in a randomized, double-blind, double-crossover trial of 4AP.Each treatment period is 2-months with a 1-week wash-out period in between each treatment period. Participating subjects will undergo standardized history and physical examination at the time of enrollment. Participants will log their ataxia attacks daily by interactive voice response (IVR) system and will be interviewed monthly for events and side effects/toxicity. Study visits will occur at the beginning and the end of the study.
- Conditions
- Episodic Ataxia Type 2
- Being tested
- 4-Aminopyridine, Placebo
- Keywords
- episodic ataxia, CACNA1A mutations
- Medical subject headings
- Episodic Ataxia, Type 2, Episodic Ataxia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
4-AminopyridineDrug
PlaceboDrug
Placebo
| Study group | Role | What they receive |
|---|---|---|
| Study Medication | Experimental | 4-aminopyridine 10mg twice daily for 8 weeks Receives: Drug: 4-Aminopyridine |
| Placebo | Experimental | placebo twice daily for 8 weeks Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
the frequency of ataxia episodes
Trial participants have frequent episodes of ataxia at baseline. The participants will document daily whether ataxia events occurred during the 2-month screening period and the 9-month study period by calling a toll-free number and participating in an Interactive Voice Response (IVR) system.
Measured: 11 months
Secondary outcomes (6)
impact on daily activities
Participants will use IVR to log the impact (on a scale of 0-3) of ataxia events, if any, on their daily activities:
- (0) No impact
- (1) Mild
- (2) Moderate
- (3) Severe
Measured: 11 months
duration of ataxia episodes
Study Participants will use IVR daily to log the duration of ataxia events, if any, in hours.
Measured: 11 months
severity of ataxia episodes
Study Participants will use IVR daily to log the severity of ataxia events, if any, on a scale of 1-9:
(1) mild (9) very severe
Measured: 11 months
treatment satisfaction
The study participant will respond by phone interview to the 11-item Treatment Satisfaction Questionnaire for Medication (TSQM Version 2) at the end of each of the four treatment periods.
Measured: 9 months
Toxicity
The study participant will be interviewed by phone regarding toxicity using the [Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0] at two different time points (4 weeks, 8 weeks) of each 8-week Treatment Period. Spectrum and severity of toxicity and the prevalence among study participants will be documented.
Measured: 9 months
Side Effects
The study participant will log side effects as they occur (reporting the seizures or other severe side effects immediately to Investigators) and will be interviewed by phone regarding side effects at two different time points (4 weeks, 8 weeks) of each 8-week Treatment Period. Spectrum of side effects and the prevalence among those treated will be documented.
Measured: 9 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Mar 2012 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (31)Papers the sponsor has linked to this study.
- Other trials for Episodic Ataxia Type 2Studies of the same condition, open and under way.
- More trials run by University of California, Los AngelesThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of California, Los Angeles
- Sponsor type
- University, hospital or other
- Collaborators
- University of Rochester, University of South Florida
- Responsible party
- Sponsor
- Investigators
- Joanna C Jen, MD PhD, Principal investigator, University of California, Los Angeles
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01543750
- Sponsor's protocol ID
- CINCH-EA2
- Other IDs
- R01FD003923
- Acronym
- 4AP in EA2
- Status last verified
- Nov 2020
- First submitted
- 27 May 2011
- Last updated
- 16 Nov 2020
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Atkinson MJ, Sinha A, Hass SL, Colman SS, Kumar RN, Brod M, Rowland CR. Validation of a general measure of treatment satisfaction, the Treatment Satisfaction Questionnaire for Medication (TSQM), using a national panel study of chronic disease. Health Qual Life Outcomes. 2004 Feb 26;2:12. doi: 10.1186/1477-7525-2-12.
PubMed 14987333Bever CT Jr, Young D, Anderson PA, Krumholz A, Conway K, Leslie J, Eddington N, Plaisance KI, Panitch HS, Dhib-Jalbut S, et al. The effects of 4-aminopyridine in multiple sclerosis patients: results of a randomized, placebo-controlled, double-blind, concentration-controlled, crossover trial. Neurology. 1994 Jun;44(6):1054-9. doi: 10.1212/wnl.44.6.1054.
PubMed 8208399DuMouchel W, Groer PG. A Bayesian methodology for scaling radiation studies from animals to man. Health Phys. 1989;57 Suppl 1:411-8. doi: 10.1097/00004032-198907001-00058.
PubMed 2606701Glasauer S, Strupp M, Kalla R, Buttner U, Brandt T. Effect of 4-aminopyridine on upbeat and downbeat nystagmus elucidates the mechanism of downbeat nystagmus. Ann N Y Acad Sci. 2005 Apr;1039:528-31. doi: 10.1196/annals.1325.060.
PubMed 15827015Griggs RC, Moxley RT 3rd, Lafrance RA, McQuillen J. Hereditary paroxysmal ataxia: response to acetazolamide. Neurology. 1978 Dec;28(12):1259-64. doi: 10.1212/wnl.28.12.1259.
PubMed 366453Imbrici P, Jaffe SL, Eunson LH, Davies NP, Herd C, Robertson R, Kullmann DM, Hanna MG. Dysfunction of the brain calcium channel CaV2.1 in absence epilepsy and episodic ataxia. Brain. 2004 Dec;127(Pt 12):2682-92. doi: 10.1093/brain/awh301. Epub 2004 Oct 13.
PubMed 15483044Harno H, Heikkinen S, Kaunisto MA, Kallela M, Hakkinen AM, Wessman M, Farkkila M, Lundbom N. Decreased cerebellar total creatine in episodic ataxia type 2: a 1H MRS study. Neurology. 2005 Feb 8;64(3):542-4. doi: 10.1212/01.WNL.0000150589.26350.3D.
PubMed 15699392Hoebeek FE, Stahl JS, van Alphen AM, Schonewille M, Luo C, Rutteman M, van den Maagdenberg AM, Molenaar PC, Goossens HH, Frens MA, De Zeeuw CI. Increased noise level of purkinje cell activities minimizes impact of their modulation during sensorimotor control. Neuron. 2005 Mar 24;45(6):953-65. doi: 10.1016/j.neuron.2005.02.012.
PubMed 15797555Jen J, Kim GW, Baloh RW. Clinical spectrum of episodic ataxia type 2. Neurology. 2004 Jan 13;62(1):17-22. doi: 10.1212/01.wnl.0000101675.61074.50.
PubMed 14718690Jen JC, Graves TD, Hess EJ, Hanna MG, Griggs RC, Baloh RW; CINCH investigators. Primary episodic ataxias: diagnosis, pathogenesis and treatment. Brain. 2007 Oct;130(Pt 10):2484-93. doi: 10.1093/brain/awm126. Epub 2007 Jun 15.
PubMed 17575281Jouvenceau A, Eunson LH, Spauschus A, Ramesh V, Zuberi SM, Kullmann DM, Hanna MG. Human epilepsy associated with dysfunction of the brain P/Q-type calcium channel. Lancet. 2001 Sep 8;358(9284):801-7. doi: 10.1016/S0140-6736(01)05971-2.
PubMed 11564488Lohle M, Schrempf W, Wolz M, Reichmann H, Storch A. Potassium channel blocker 4-aminopyridine is effective in interictal cerebellar symptoms in episodic ataxia type 2--a video case report. Mov Disord. 2008 Jul 15;23(9):1314-6. doi: 10.1002/mds.22071.
PubMed 18442126March B, Cardi T. Assessment of the cardiac safety of fampridine-SR sustained-release tablets in a thorough QT/QTc evaluation at therapeutic and supratherapeutic doses in healthy individuals. Expert Opin Investig Drugs. 2009 Dec;18(12):1807-15. doi: 10.1517/13543780903443096.
PubMed 19938897McEvoy KM, Windebank AJ, Daube JR, Low PA. 3,4-Diaminopyridine in the treatment of Lambert-Eaton myasthenic syndrome. N Engl J Med. 1989 Dec 7;321(23):1567-71. doi: 10.1056/NEJM198912073212303.
PubMed 2555713Ophoff RA, Terwindt GM, Vergouwe MN, van Eijk R, Oefner PJ, Hoffman SM, Lamerdin JE, Mohrenweiser HW, Bulman DE, Ferrari M, Haan J, Lindhout D, van Ommen GJ, Hofker MH, Ferrari MD, Frants RR. Familial hemiplegic migraine and episodic ataxia type-2 are caused by mutations in the Ca2+ channel gene CACNL1A4. Cell. 1996 Nov 1;87(3):543-52. doi: 10.1016/s0092-8674(00)81373-2.
PubMed 8898206
Study at a glance
From the registry record, last updated by the sponsor on 16 Nov 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 2 Study of 4-Aminopyridine for the Treatment of Episodic Ataxia Type 2
- ClinicalTrials.gov ID
- NCT01543750
- Acronym / sponsor ID
- 4AP in EA2 / CINCH-EA2
- Conditions
- Episodic Ataxia Type 2
- Keywords
- episodic ataxia, CACNA1A mutations
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Withdrawn
- Enrolment
- 0 participants (actual)
- Lead sponsor
- University of California, Los AngelesUniversity, hospital or other
- Collaborators
- University of Rochester, University of South Florida
- First posted
- 5 Mar 2012 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Episodic ataxia type 2 (EA2) is a rare familial neurological condition characterized by debilitating episodes of vertigo and imbalance. Since the serendipitous discovery of dramatic response of EA2 to acetazolamide, acetazolamide has been the first-line treatment for EA2. Yet, for those patients who do not respond to or cannot tolerate acetazolamide, there is no alternative treatment. The purpose of this randomized trial is to test whether 4-aminopyridine may reduce the ataxia episodes in EA2 as an alternative to acetazolamide. Funding Source - FDA OOPD
Detailed description
This study aims to determine whether 4-aminopyridine (4AP) can reduce attacks of ataxia in patients with episodic ataxia type 2 (EA2), a rare but often debilitating condition. Episodic ataxia (EA) is a group of inherited disorders characterized by recurrent, discrete episodes of vertigo and ataxia variably associated with progressive ataxia. EA2, the most common and the best characterized of all the EA syndromes, is caused by heterozygous mutations in CACNA1A, which encodes the main subunit of a neuronal voltage-gated calcium channel, Cav2.1.
Although observational data suggest symptomatic resolution with acetazolamide in many EA2 patients, the investigators found in our patient databases that at least a third of the EA2 patients continue to suffer debilitating ataxia attacks, either because of incomplete control while on acetazolamide or because of intolerability or hypersensitivity to acetazolamide. For these patients there is no alternative intervention. 4-Aminopyridine (4AP) has been found to be helpful in a handful of patients with EA2. Recently, dalfampridine, an extended release formulation of 4AP (AMPYRA) by Acorda Therapeutics, received FDA approval to improve gait in multiple sclerosis.
The investigators plan to recruit 20 subjects with genetically defined EA2 who suffer frequent ataxia episodes (at least 3 episodes a month) to conduct a randomized trial of 4AP to examine its efficacy and tolerability in EA2. Study subjects will be recruited at UCLA and the University of Rochester to participate in a randomized, double-blind, double-crossover trial of 4AP.Each treatment period is 2-months with a 1-week wash-out period in between each treatment period. Participating subjects will undergo standardized history and physical examination at the time of enrollment. Participants will log their ataxia attacks daily by interactive voice response (IVR) system and will be interviewed monthly for events and side effects/toxicity. Study visits will occur at the beginning and the end of the study.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Study Medication | Experimental | Drug: 4-Aminopyridine4-aminopyridine 10mg twice daily for 8 weeks |
| Placebo | Experimental | Drug: Placeboplacebo twice daily for 8 weeks |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Have EA2 genetically confirmed to harbor mutations in CACNA1A
- Are ≥ 18 years of age
- Are not taking acetazolamide (because of intolerance, poor response, or allergy)
- Are able to maintain a daily log of ataxia episode(s) and report daily by using an Interactive Voice Recording System (IVR) throughout the study
- Experience ≥ 3 ataxia episodes per month during the two-month screening period to qualify for randomization
Key exclusion criteria (6 listed)
- Have seizures or a history of seizures
- Have first-degree relatives with EA2 and seizures
- Have renal disease with impaired function (Creatinine clearance CrCl≤50ml/min)
- Are pregnant or breast feeding (women of childbearing age will be tested for pregnancy and must be using birth control)
- Are unable to comply with the study requirement
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| 4-Aminopyridine | Drug | Study Medication |
| PlaceboPlacebo | Drug | Placebo |
Primary outcome
the frequency of ataxia episodes
Time frame: 11 months
Trial participants have frequent episodes of ataxia at baseline. The participants will document daily whether ataxia events occurred during the 2-month screening period and the 9-month study period by calling a toll-free number and participating in an Interactive Voice Response (IVR) system.
6 secondary outcomes
impact on daily activities
Time frame: 11 months
duration of ataxia episodes
Time frame: 11 months
severity of ataxia episodes
Time frame: 11 months
treatment satisfaction
Time frame: 9 months
Toxicity
Time frame: 9 months
Side Effects
Time frame: 9 months
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Nov 2020.
- Background: PubMed 14987333Atkinson MJ, Sinha A, Hass SL, Colman SS, Kumar RN, Brod M, Rowland CR. Validation of a general measure of treatment satisfaction, the Treatment Satisfaction Questionnaire for Medication (TSQM), using a national panel study of chronic disease. Health Qual Life Outcomes. 2004 Feb 26;2:12. doi: 10.1186/1477-7525-2-12.
- Background: PubMed 8208399Bever CT Jr, Young D, Anderson PA, Krumholz A, Conway K, Leslie J, Eddington N, Plaisance KI, Panitch HS, Dhib-Jalbut S, et al. The effects of 4-aminopyridine in multiple sclerosis patients: results of a randomized, placebo-controlled, double-blind, concentration-controlled, crossover trial. Neurology. 1994 Jun;44(6):1054-9. doi: 10.1212/wnl.44.6.1054.
- Background: PubMed 2606701DuMouchel W, Groer PG. A Bayesian methodology for scaling radiation studies from animals to man. Health Phys. 1989;57 Suppl 1:411-8. doi: 10.1097/00004032-198907001-00058.
- Background: PubMed 15827015Glasauer S, Strupp M, Kalla R, Buttner U, Brandt T. Effect of 4-aminopyridine on upbeat and downbeat nystagmus elucidates the mechanism of downbeat nystagmus. Ann N Y Acad Sci. 2005 Apr;1039:528-31. doi: 10.1196/annals.1325.060.
- Background: PubMed 366453Griggs RC, Moxley RT 3rd, Lafrance RA, McQuillen J. Hereditary paroxysmal ataxia: response to acetazolamide. Neurology. 1978 Dec;28(12):1259-64. doi: 10.1212/wnl.28.12.1259.
- Background: PubMed 15483044Imbrici P, Jaffe SL, Eunson LH, Davies NP, Herd C, Robertson R, Kullmann DM, Hanna MG. Dysfunction of the brain calcium channel CaV2.1 in absence epilepsy and episodic ataxia. Brain. 2004 Dec;127(Pt 12):2682-92. doi: 10.1093/brain/awh301. Epub 2004 Oct 13.
- Background: PubMed 15699392Harno H, Heikkinen S, Kaunisto MA, Kallela M, Hakkinen AM, Wessman M, Farkkila M, Lundbom N. Decreased cerebellar total creatine in episodic ataxia type 2: a 1H MRS study. Neurology. 2005 Feb 8;64(3):542-4. doi: 10.1212/01.WNL.0000150589.26350.3D.
- Background: PubMed 15797555Hoebeek FE, Stahl JS, van Alphen AM, Schonewille M, Luo C, Rutteman M, van den Maagdenberg AM, Molenaar PC, Goossens HH, Frens MA, De Zeeuw CI. Increased noise level of purkinje cell activities minimizes impact of their modulation during sensorimotor control. Neuron. 2005 Mar 24;45(6):953-65. doi: 10.1016/j.neuron.2005.02.012.
- Background: PubMed 14718690Jen J, Kim GW, Baloh RW. Clinical spectrum of episodic ataxia type 2. Neurology. 2004 Jan 13;62(1):17-22. doi: 10.1212/01.wnl.0000101675.61074.50.
- Background: PubMed 17575281Jen JC, Graves TD, Hess EJ, Hanna MG, Griggs RC, Baloh RW; CINCH investigators. Primary episodic ataxias: diagnosis, pathogenesis and treatment. Brain. 2007 Oct;130(Pt 10):2484-93. doi: 10.1093/brain/awm126. Epub 2007 Jun 15.
- Background: PubMed 11564488Jouvenceau A, Eunson LH, Spauschus A, Ramesh V, Zuberi SM, Kullmann DM, Hanna MG. Human epilepsy associated with dysfunction of the brain P/Q-type calcium channel. Lancet. 2001 Sep 8;358(9284):801-7. doi: 10.1016/S0140-6736(01)05971-2.
- Background: PubMed 18442126Lohle M, Schrempf W, Wolz M, Reichmann H, Storch A. Potassium channel blocker 4-aminopyridine is effective in interictal cerebellar symptoms in episodic ataxia type 2--a video case report. Mov Disord. 2008 Jul 15;23(9):1314-6. doi: 10.1002/mds.22071.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Joanna C Jen, MD PhD principal investigator
University of California, Los Angeles
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing 4-Aminopyridine in people with Episodic Ataxia Type 2, run by University of California, Los Angeles at 3 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 16 Nov 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
the frequency of ataxia episodes (time frame: 11 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01543750, which also lists 31 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Nov 2020.