Ornithine Transcarbamylase DeficiencyNCT01569568

Investigation of Brain Nitrogen in Partial Ornithine Transcarbamylase Deficiency (OTCD) Using 1 H MRS, DTI, and fMRI

An observational study of Ornithine Transcarbamylase Deficiency, run by Andrea Gropman.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Trial summary

The purpose of this study is to use various types of MRI and cognitive testing to evaluate changes in the brain and cognitive function that occur in subjects with ornithine transcarbamylase deficiency (OTCD) relative to healthy individuals Read more about this study

Who is it for?: Ages 7 to 60 yearsHealthy volunteers can take part
Conditions the study is for: Ornithine Transcarbamylase Deficiency
What is being tested?: MRI scanning and Cognitive testing
Type of treatment: Lifestyle or therapy programme
How many people: 49 participants
Where: No sites listed yet
Study dates: Sep 2010to Aug 2014
Phase: Observational
Are there results?: Yes, posted in the registrySummary in the doctors' view

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 7 to 60 years
All sexes
Healthy volunteers accepted
Child, Adult

Study population: Males and females, ages 7-60 years with ornithine transcarbamylase deficiency Males and females, ages 7-60 years who are healthy controls without ornithine transcarbamylase deficiency

Who may be able to join

Subject inclusion criteria:
Patients with OTCD;
Age range: 7-60 years
Able to undergo neuroimaging safely (i.e. without presence of ferromagnetic devices)
Subject has a documented full scale IQ > 70
Control participant inclusion criteria:
Healthy males and females without metabolic disease aged 7-60 years
Subject has a documented full scale IQ > 70

Who cannot join

Subject exclusion criteria:
Mental retardation (i.e., Full Scale IQ< 70)
Age range <7 or >60 years
Presence of ferromagnetic device(s) that preclude safe imaging
Pregnant female
Control exclusion criteria:

Show all 10Show fewer

Subjects with a documented history of an intellectual deficit (i.e., Full Scale IQ< 70)
Age range <7 or >60 years
Presence of ferromagnetic device(s) that preclude safe imaging
Pregnant female

About this study

Read the detailed description

The overall goal of this project is to characterize metabolic, structural and cognitive changes in OTCD using 1H MRS, DTI, volumetric averaging and fMRI with cognitive testing of executive function measures to validate biomarkers for the effect of HA and its treatment on the brain.

The investigators will measure gln and mI in blood and brain (using 1H MRS) in affected participants, and mI in brain in controls, fractional anisotropy as a measure of white matter microstructural damage (by DTI) and brain activation pathways alterations with tasks probing working memory (fMRI). As a secondary outcome measure, the investigators will correlate the findings from neuroimaging with cognitive functioning. This protocol is based on the previous 5104 protocol, now includes children to evaluate the age and stage of disease on these indices in a cohort that is undergoing important developmental events against an age matched typically developing cohort.

Conditions: Ornithine Transcarbamylase Deficiency
Being tested: MRI scanning, Cognitive testing
Keywords: Neuroimaging, MRI, Urea cycle, hyperammonemia, cognitive function, ornithine transcarbamylase deficiency
Medical subject headings: Ornithine Carbamoyltransferase Deficiency Disease, Hyperammonemia

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

MRI scanningOther
Also called MRI
1H MRS, DTI, FMRI
Cognitive testingBehavioural
Behavioral testing

Study groups in NCT01569568
Study group	Role	What they receive
Subjects with OTCD		males and females ages 7-60 years with OTCD who are able to undergo MRI and cognitive testing MRI scanning 1H MRS, DTI, FMRI Cognitive testing Neuropsychological testing Receives: Other: MRI scanning; Behavioral: Cognitive testing
Healthy controls		males and females ages 7-60 years who are healthy controls who are able to undergo MRI and cognitive testing MRI scanning 1H MRS, DTI, FMRI Cognitive testing Neuropsychological testing Receives: Other: MRI scanning; Behavioral: Cognitive testing

What is being measured

The primary outcome is the main question the study is designed to answer.

Concentration of Glutamine and Myoinositol
Concentration based on area under curve on 1H Magnetic Resonance Spectroscopy(MRS) and quantitated by LCModel (a method that allows automatic quantitation of spectroscopy data). A metabolite's tissue concentration is related to the integrated amplitude, the area under the curve of the MRS signal, it produces. While MRS signals are usually acquired in the time domain as free induction decays or echoes, they are usually viewed and analyzed in the frequency domain. The frequency domain representation is derived from the acquired time domain data by the Fourier Transform. The protocol we use selects 257 averages. The machine summates the data at each time point to generate one value for the area under the curve. Therefore, we don't have the measurement at each time point.
Furthermore, we measured voxels in two different brain areas containing different kinds of brain matter: one voxel was located in posterior cingulate gray matter (PCGM) and the other in parietal white matter (PWM).
Measured: Baseline
Functional Connectivity of Assessed by Resting-state fMRI
Investigation of differences in functional connectivity of OTCD patients compared to healthy controls, particularly in the default-mode network (DMN) and the set-maintenance network (SMN). Participants underwent a resting-state scan using 3T fMRI. Combining independent component analysis (ICA) and region-of-interest (ROI) analyses, identified the nodes that comprised each network in each group, and assessed internodal connectivity. For each subject, this analysis generated a correlation value, which reflected the strength of functional connectivity between each ROI pair.The correlation r-values were normalized using Fisher's r-to-Z-transform, generating z-scores. The DMN was composed of 1) anterior cingulate/medial prefrontal cortex (ACC/mPFC), 2) posterior cingulate cortex (PCC), and 3) bilateral inferior parietal lobule (IPL). The SMN was composed of 1)ACC, 2) bilateral superior frontal gyrus (SFG), and 3) bilateral anterior insula/frontal operculum (aI/fO).
Measured: Baseline
Fractional Anisotropy Assessed Using DTI
Fractional Anisotropy (FA) is a measure of the diffusion asymmetry within a voxel as defined by its eigenvalues. In our study, FA is being used as a measure of white matter integrity, because FA is very sensitive to small microstructural changes.Fractional anisotropy (FA) is a scalar value between zero and one (0-1) that describe anisotropy of a diffusion process. A value of zero means that diffusion is isotropic, i.e. it is unrestricted (or equally restricted) in all directions. A value of one means that diffusion occurs only along one axis and is fully restricted along all other directions.
Measured: Baseline

Secondary outcomes (1)

Neuropsychological Assessment
Testing consisted of the Wechsler Abbreviated Scale of Intelligence (WASI), Comprehensive Trail Making Test (CTMT) (range 17-87), and the Behavioral Rating Inventory of Executive Function (BRIEF) (range GEC: 70-210; BRI:39-82 ; MI:41-92). The WASI includes three measures of intelligence; including, performance IQ (sum of block design and matrices sub scales; range: 40-160), verbal IQ (sum of vocabulary and similarities sub scales; range 40-160), and total IQ (sum of all four subscales; range: 80-320). The CTMT measures simple attention and executive function, it consists of five dot to dots that increase with complexity and difficulty. Higher values indicate better outcomes for all scales.
Measured: Baseline

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Sep 2010Aug 2014

First posted3 Apr 2012 (estimated)
Study startSep 2010
Primary completionAug 2014
Study completionAug 2014

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (7)Papers the sponsor has linked to this study.
Other trials for Ornithine Transcarbamylase DeficiencyStudies of the same condition, open and under way.
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: Observational
Observational model: Cohort
Time perspective: Prospective
Enrolment: 49 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01569568
Sponsor's protocol ID: UCRDC 5107
Status last verified: Feb 2024
First submitted: 30 Mar 2012
Last updated: 8 Mar 2024
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
Individual data sharing: Yes: Final data is on the UCDC website

Publications

Gropman AL, Shattuck K, Prust MJ, Seltzer RR, Breeden AL, Hailu A, Rigas A, Hussain R, VanMeter J. Altered neural activation in ornithine transcarbamylase deficiency during executive cognition: an fMRI study. Hum Brain Mapp. 2013 Apr;34(4):753-61. doi: 10.1002/hbm.21470. Epub 2011 Nov 23.
PubMed 22110002
Prust MJ, Gropman AL, Hauser N. New frontiers in neuroimaging applications to inborn errors of metabolism. Mol Genet Metab. 2011 Nov;104(3):195-205. doi: 10.1016/j.ymgme.2011.06.020. Epub 2011 Jun 30.
PubMed 21778100
Gropman AL, Gertz B, Shattuck K, Kahn IL, Seltzer R, Krivitsky L, Van Meter J. Diffusion tensor imaging detects areas of abnormal white matter microstructure in patients with partial ornithine transcarbamylase deficiency. AJNR Am J Neuroradiol. 2010 Oct;31(9):1719-23. doi: 10.3174/ajnr.A2122. Epub 2010 May 20.
PubMed 20488904
Gropman A. Brain imaging in urea cycle disorders. Mol Genet Metab. 2010;100 Suppl 1(Suppl 1):S20-30. doi: 10.1016/j.ymgme.2010.01.017. Epub 2010 Feb 13.
PubMed 20207564
Oldham MS, VanMeter JW, Shattuck KF, Cederbaum SD, Gropman AL. Diffusion tensor imaging in arginase deficiency reveals damage to corticospinal tracts. Pediatr Neurol. 2010 Jan;42(1):49-52. doi: 10.1016/j.pediatrneurol.2009.07.017.
PubMed 20004862
Gropman AL, Sailasuta N, Harris KC, Abulseoud O, Ross BD. Ornithine transcarbamylase deficiency with persistent abnormality in cerebral glutamate metabolism in adults. Radiology. 2009 Sep;252(3):833-41. doi: 10.1148/radiol.2523081878. Epub 2009 Jun 30.
PubMed 19567648
Gropman AL, Fricke ST, Seltzer RR, Hailu A, Adeyemo A, Sawyer A, van Meter J, Gaillard WD, McCarter R, Tuchman M, Batshaw M; Urea Cycle Disorders Consortium. 1H MRS identifies symptomatic and asymptomatic subjects with partial ornithine transcarbamylase deficiency. Mol Genet Metab. 2008 Sep-Oct;95(1-2):21-30. doi: 10.1016/j.ymgme.2008.06.003. Epub 2008 Jul 26.
PubMed 18662894

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

Official record

RecruitingObservational study
Long-term Follow-up (LTFU) Study of Participants in Any iECURE Protocol Using an Investigational Product (IP)
Ornithine Transcarbamylase Deficiency, Ornithine Transcarbamylase Deficiency Disease
RecruitingObservational study
Abbott Vascular Medical Device Registry
Acute Myocardial Infarction, Restenoses, Coronary
RecruitingPhase 1/2
Phase 1/2 Study of KRRO-121 in Healthy Volunteers and Patients With UCD
Urea Cycle Disorders, Inborn, Urea Cycle Disorders
Active, not recruitingPhase 3
An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency
Ornithine Transcarbamylase Deficiency, Ornithine Transcarbamylase Deficiency Disease
Active, not recruitingPhase 3
Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency
OTC Deficiency

All Ornithine Transcarbamylase Deficiency trials

Study at a glance

From the registry record, last updated by the sponsor on 8 Mar 2024. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Investigation of Brain Nitrogen in Partial Ornithine Transcarbamylase Deficiency (OTCD) Using 1 H MRS, DTI, and fMRI
ClinicalTrials.gov ID: NCT01569568
Acronym / sponsor ID: UCRDC 5107
Conditions: Ornithine Transcarbamylase Deficiency
Keywords: Neuroimaging, MRI, Urea cycle, hyperammonemia, cognitive function, ornithine transcarbamylase deficiency
Study type: Observational
Recruitment status: Completed
Enrolment: 49 participants (actual)
Lead sponsor: Andrea GropmanUniversity, hospital or other
Collaborators: Children's National Research Institute
Study start: Sep 2010
Primary completion: Aug 2014 (actual)
Study completion: Aug 2014 (actual)
First posted: 3 Apr 2012 (estimated)
Results: Posted 14 Apr 2017

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

Study design

Observational model: Cohort
Time perspective: Prospective
Number of arms: 2

Arm	Type	Receives
Subjects with OTCD	–	Other: MRI scanning, Behavioral: Cognitive testingmales and females ages 7-60 years with OTCD who are able to undergo MRI and cognitive testing MRI scanning 1H MRS, DTI, FMRI Cognitive testing Neuropsychological testing
Healthy controls	–	Other: MRI scanning, Behavioral: Cognitive testingmales and females ages 7-60 years who are healthy controls who are able to undergo MRI and cognitive testing MRI scanning 1H MRS, DTI, FMRI Cognitive testing Neuropsychological testing

Who may qualify

Ages: 7 to 60 years
Sex: All
Healthy volunteers: Accepted
Age groups: Child, Adult
Study population: Males and females, ages 7-60 years with ornithine transcarbamylase deficiency Males and females, ages 7-60 years who are healthy controls without ornithine transcarbamylase deficiency

Key inclusion criteria (8 listed)

Patients with OTCD;
Age range: 7-60 years
Able to undergo neuroimaging safely (i.e. without presence of ferromagnetic devices)
Subject has a documented full scale IQ > 70
Healthy males and females without metabolic disease aged 7-60 years

Key exclusion criteria (10 listed)

Mental retardation (i.e., Full Scale IQ< 70)
Age range <7 or >60 years
Presence of ferromagnetic device(s) that preclude safe imaging
Pregnant female
Subjects with a documented history of an intellectual deficit (i.e., Full Scale IQ< 70)

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
MRI scanningAlso: MRI1H MRS, DTI, FMRI	Other	Healthy controls, Subjects with OTCD
Cognitive testingBehavioral testing	Behavioural	Healthy controls, Subjects with OTCD

Primary outcome

Concentration of Glutamine and Myoinositol
Time frame: Baseline
Concentration based on area under curve on 1H Magnetic Resonance Spectroscopy(MRS) and quantitated by LCModel (a method that allows automatic quantitation of spectroscopy data). A metabolite's tissue concentration is related to the integrated amplitude, the area under the curve of the MRS signal, it produces. While MRS signals are usually acquired in the time domain as free induction decays or echoes, they are usually viewed and analyzed in the frequency domain. The frequency domain representation is derived from the acquired time domain data by the Fourier Transform. The protocol we use selects 257 averages. The machine summates the data at each time point to generate one value for the area under the curve. Therefore, we don't have the measurement at each time point. Furthermore, we measured voxels in two different brain areas containing different kinds of brain matter: one voxel was located in posterior cingulate gray matter (PCGM) and the other in parietal white matter (PWM).
Functional Connectivity of Assessed by Resting-state fMRI
Time frame: Baseline
Investigation of differences in functional connectivity of OTCD patients compared to healthy controls, particularly in the default-mode network (DMN) and the set-maintenance network (SMN). Participants underwent a resting-state scan using 3T fMRI. Combining independent component analysis (ICA) and region-of-interest (ROI) analyses, identified the nodes that comprised each network in each group, and assessed internodal connectivity. For each subject, this analysis generated a correlation value, which reflected the strength of functional connectivity between each ROI pair.The correlation r-values were normalized using Fisher's r-to-Z-transform, generating z-scores. The DMN was composed of 1) anterior cingulate/medial prefrontal cortex (ACC/mPFC), 2) posterior cingulate cortex (PCC), and 3) bilateral inferior parietal lobule (IPL). The SMN was composed of 1)ACC, 2) bilateral superior frontal gyrus (SFG), and 3) bilateral anterior insula/frontal operculum (aI/fO).
Fractional Anisotropy Assessed Using DTI
Time frame: Baseline
Fractional Anisotropy (FA) is a measure of the diffusion asymmetry within a voxel as defined by its eigenvalues. In our study, FA is being used as a measure of white matter integrity, because FA is very sensitive to small microstructural changes.Fractional anisotropy (FA) is a scalar value between zero and one (0-1) that describe anisotropy of a diffusion process. A value of zero means that diffusion is isotropic, i.e. it is unrestricted (or equally restricted) in all directions. A value of one means that diffusion occurs only along one axis and is fully restricted along all other directions.

1 secondary outcome

Neuropsychological Assessment
Time frame: Baseline

Results and safety

Results posted to the registry on 14 Apr 2017, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Subjects With OTCD	20	20
Healthy Controls	29	27

Primary outcome: Concentration of Glutamine and Myoinositol

Time frame: Baseline. Mean (Standard Deviation). Unit: mM

Measure	Subjects With OTCD n = 18	Healthy Controls n = 26
Concentration of glutamine in PWM	2.61 (1.07)	1.66 (0.93)
Concentration of myoinositol in PWM	2.23 (0.84)	2.69 (0.48)
Concentration of glutamine in PCGM	5.22 (1.70)	3.62 (0.91)
Concentration of myoinositol in PCGM	3.80 (0.80)	4.37 (0.46)

Subjects With OTCD vs Healthy Controls: p = 0.001, t-test, 2 sided
Subjects With OTCD vs Healthy Controls: p = 0.011, t-test, 2 sided
Subjects With OTCD vs Healthy Controls: p = 0.004, t-test, 2 sided

Population analysed: Two OTCD patients and one healthy control were excluded due to excessive head motion.

Primary outcome: Functional Connectivity of Assessed by Resting-state fMRI

Time frame: Baseline. Mean (Standard Deviation). Unit: z-scores

Measure	Subjects With OTCD n = 13	Healthy Controls n = 22
DMN: ACC/mPFC & left IPL connectivity	-0.86 (0.70)	0.18 (0.30)
DMN: ACC/mPFC & PCC connectivity	-0.10 (0.70)	0.12 (0.40)
DMN: ACC/mPFC & right IPL connectivity	-0.26 (0.53)	0.07 (0.34)
DMN: PCC & left IPL connectivity	0.69 (0.63)	0.69 (0.31)
DMN: PCC & right IPL connectivity	0.66 (0.42)	0.64 (0.30)
DMN: left IPL & right IPL connectivity	0.75 (0.69)	0.70 (0.34)
SMN: ACC & left aI/fO connectivity	0.19 (0.39)	0.28 (0.41)
SMN: ACC & left SFG connectivity	0.28 (0.35)	0.44 (0.29)

Subjects With OTCD vs Healthy Controls: p <0.001, ANOVA
Subjects With OTCD vs Healthy Controls: p = 0.024, ANOVA
Subjects With OTCD vs Healthy Controls: p = 0.040, ANOVA

Population analysed: Resting state data was not acquired for several of our participants (7 controls and 4 patients). Furthermore, 3 OTCD patients were excluded from the analyses due to excessive head motion.

Primary outcome: Fractional Anisotropy Assessed Using DTI

Time frame: Baseline. Mean (Standard Deviation). Unit: units on a scale

Measure	Subjects With OTCD n = 20	Healthy Controls n = 27
Value	0.25 (0.003)	0.3 (0.008)

1 secondary outcome is reported in the full results.

Adverse events

Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Subjects With OTCD	0 of 20 (0%)	0 of 20 (0%)	Not reported
Healthy Controls	0 of 29 (0%)	0 of 29 (0%)	Not reported

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 8 Mar 2024.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Background: PubMed 22110002Gropman AL, Shattuck K, Prust MJ, Seltzer RR, Breeden AL, Hailu A, Rigas A, Hussain R, VanMeter J. Altered neural activation in ornithine transcarbamylase deficiency during executive cognition: an fMRI study. Hum Brain Mapp. 2013 Apr;34(4):753-61. doi: 10.1002/hbm.21470. Epub 2011 Nov 23.
Background: PubMed 21778100Prust MJ, Gropman AL, Hauser N. New frontiers in neuroimaging applications to inborn errors of metabolism. Mol Genet Metab. 2011 Nov;104(3):195-205. doi: 10.1016/j.ymgme.2011.06.020. Epub 2011 Jun 30.
Background: PubMed 20488904Gropman AL, Gertz B, Shattuck K, Kahn IL, Seltzer R, Krivitsky L, Van Meter J. Diffusion tensor imaging detects areas of abnormal white matter microstructure in patients with partial ornithine transcarbamylase deficiency. AJNR Am J Neuroradiol. 2010 Oct;31(9):1719-23. doi: 10.3174/ajnr.A2122. Epub 2010 May 20.
Background: PubMed 20207564Gropman A. Brain imaging in urea cycle disorders. Mol Genet Metab. 2010;100 Suppl 1(Suppl 1):S20-30. doi: 10.1016/j.ymgme.2010.01.017. Epub 2010 Feb 13.
Background: PubMed 20004862Oldham MS, VanMeter JW, Shattuck KF, Cederbaum SD, Gropman AL. Diffusion tensor imaging in arginase deficiency reveals damage to corticospinal tracts. Pediatr Neurol. 2010 Jan;42(1):49-52. doi: 10.1016/j.pediatrneurol.2009.07.017.
Background: PubMed 19567648Gropman AL, Sailasuta N, Harris KC, Abulseoud O, Ross BD. Ornithine transcarbamylase deficiency with persistent abnormality in cerebral glutamate metabolism in adults. Radiology. 2009 Sep;252(3):833-41. doi: 10.1148/radiol.2523081878. Epub 2009 Jun 30.
Background: PubMed 18662894Gropman AL, Fricke ST, Seltzer RR, Hailu A, Adeyemo A, Sawyer A, van Meter J, Gaillard WD, McCarter R, Tuchman M, Batshaw M; Urea Cycle Disorders Consortium. 1H MRS identifies symptomatic and asymptomatic subjects with partial ornithine transcarbamylase deficiency. Mol Genet Metab. 2008 Sep-Oct;95(1-2):21-30. doi: 10.1016/j.ymgme.2008.06.003. Epub 2008 Jul 26.
Family support groupLink provided by the sponsor.
Urea Cycle rare disease consortiumLink provided by the sponsor.
Individual participant dataSharing planned: Yes. Final data is on the UCDC website

Related trials for Ornithine Transcarbamylase Deficiency

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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.

Andrea L Gropman, M.D. principal investigator

Children's National Research Institute

Questions about this trial

What is this clinical trial studying?

An observational study of Ornithine Transcarbamylase Deficiency, run by Andrea Gropman.

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 8 inclusion and 10 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: finished, as of the sponsor's last update on 8 Mar 2024. Status can change between updates, so confirm with the study contact before making plans.

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.

Are results available for this study?

Yes. Results are posted in the registry (first posted 14 Apr 2017). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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?

Concentration of Glutamine and Myoinositol (time frame: Baseline). The study lists 3 primary outcomes in all. 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 NCT01569568, which also lists 7 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 8 Mar 2024.