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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04513002




Registration number
NCT04513002
Ethics application status
Date submitted
26/07/2020
Date registered
14/08/2020

Titles & IDs
Public title
Ataxia-telangiectasia: Treating Mitochondrial Dysfunction With a Novel Form of Anaplerosis
Scientific title
A Phase 2A/2B Placebo-controlled Randomised Clinical Trial to Test the Ability of Triheptanoin to Protect Primary Airway Epithelial Cells Obtained From Participants With Ataxia-telangiectasia Against Death Induced by Glucose Deprivation
Secondary ID [1] 0 0
A-T2020/01
Universal Trial Number (UTN)
Trial acronym
A-TC7
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Ataxia Telangiectasia 0 0
Condition category
Condition code
Neurological 0 0 0 0
Other neurological disorders
Neurological 0 0 0 0
Neurodegenerative diseases
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Cardiovascular 0 0 0 0
Diseases of the vasculature and circulation including the lymphatic system

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Other - Triheptanoin

Other: Group 1: Triheptanoin and no Placebo - Parallel group, placebo-controlled, dose-escalation each 2 months for 12 months. Dose based on percent (%) of calculated caloric intake.

Thirty participants will be randomised in blocks on a 1:1:1 ratio into one of three groups.

Group 1: 10%, 20%, 35%, 35%, 35% (no placebo). Group 2: placebo, 10%, 20%, 35%, 35% Group 3: placebo, placebo, 10%, 20%, 35%

Other: Group 2: Placebo and Triheptanoin - Parallel group, placebo-controlled, dose-escalation each 2 months for 12 months. Dose based on percent (%) of calculated caloric intake.

Thirty participants will be randomised in blocks on a 1:1:1 ratio into one of three groups.

Group 1: 10%, 20%, 35%, 35%, 35% (no placebo). Group 2: placebo, 10%, 20%, 35%, 35% Group 3: placebo, placebo, 10%, 20%, 35%

Other: Group 3: Placebo, Placebo and Triheptanoin - Parallel group, placebo-controlled, dose-escalation each 2 months for 12 months. Dose based on percent (%) of calculated caloric intake.

Thirty participants will be randomised in blocks on a 1:1:1 ratio into one of three groups.

Group 1: 10%, 20%, 35%, 35%, 35% (no placebo). Group 2: placebo, 10%, 20%, 35%, 35% Group 3: placebo, placebo, 10%, 20%, 35%


Treatment: Other: Triheptanoin
Triheptanoin is a highly purified, synthetic medium odd-chain triglyceride that is catabolized to heptanoate.
Intervention code [1] 0 0
Treatment: Other
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Nasal epithelial cell survival under conditions of glucose deprivation.
Timepoint [1] 0 0
Day 1, baseline measurement
Primary outcome [2] 0 0
Nasal epithelial cell survival under conditions of glucose deprivation.
Timepoint [2] 0 0
Day 60, assessment of effects/changes from Day 1 baseline measurement
Primary outcome [3] 0 0
Nasal epithelial cell survival under conditions of glucose deprivation.
Timepoint [3] 0 0
Day 120, assessment of effects/changes from Day 60 baseline measurement
Primary outcome [4] 0 0
Nasal epithelial cell survival under conditions of glucose deprivation.
Timepoint [4] 0 0
Day 180, assessment of effects/changes from Day 120 baseline measurement
Primary outcome [5] 0 0
Nasal epithelial cell survival under conditions of glucose deprivation.
Timepoint [5] 0 0
Day 240, assessment of effects/changes from Day 180 baseline measurement
Primary outcome [6] 0 0
Nasal epithelial cell survival under conditions of glucose deprivation.
Timepoint [6] 0 0
Day 300, assessment of effects/changes from Day 240 baseline measurement
Primary outcome [7] 0 0
Nasal epithelial cell survival under conditions of glucose deprivation.
Timepoint [7] 0 0
Day 360, assessment of effects/changes from Day 300 baseline measurement
Secondary outcome [1] 0 0
Scales for assessment and rating of ataxia
Timepoint [1] 0 0
Day 1, Day 120, Day 240, Day 360
Secondary outcome [2] 0 0
International Cooperative Ataxia Rating Scale
Timepoint [2] 0 0
Day 1, Day 120, Day 240, Day 360
Secondary outcome [3] 0 0
Speech Pathology Assessments
Timepoint [3] 0 0
Day 1, Day 120, Day 240, Day 360
Secondary outcome [4] 0 0
Ophthalmology assessments
Timepoint [4] 0 0
Day 1, Day 120, Day 240, Day 360
Secondary outcome [5] 0 0
MRI lung imaging
Timepoint [5] 0 0
Performed at Day 1 and Day 360 in suitable participants
Secondary outcome [6] 0 0
Spirometry Vital capacity (litres)
Timepoint [6] 0 0
Day 1, Day 120, Day 240, Day 360
Secondary outcome [7] 0 0
Spirometry Forced vital capacity (litres)
Timepoint [7] 0 0
Day 1, Day 120, Day 240, Day 360
Secondary outcome [8] 0 0
Spirometry Forced expiratory volume in one second (litres)
Timepoint [8] 0 0
Day 1, Day 120, Day 240, Day 360
Secondary outcome [9] 0 0
Spirometry Peak expiratory flow (L.min-1)
Timepoint [9] 0 0
Day 1, Day 120, Day 240, Day 360
Secondary outcome [10] 0 0
Upper respiratory microbiome
Timepoint [10] 0 0
Day 1, Day 60, Day 120, Day 180, Day 240, Day 300, Day 360
Secondary outcome [11] 0 0
Faecal microbiome
Timepoint [11] 0 0
Day 1, Day 60, Day 120, Day 180, Day 240, Day 300, Day 360
Secondary outcome [12] 0 0
Intestinal permeability
Timepoint [12] 0 0
Day 1, Day 60, Day 120, Day 180, Day 240, Day 300, Day 360

Eligibility
Key inclusion criteria
* Patients of either sex, of any age, with a confirmed diagnosis of A-T,
* Patients who are able to undertake the study procedures,
* Families who are able to comply with the protocol for its duration and who provide informed patient assent and consent signed and dated by parent/legal guardian or adult participant according to local regulations.
Minimum age
No limit
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Patients whose parents/legal guardians are not able to provide consent
* Patients who have been in another randomised clinical intervention trial where the use of investigational medicinal product within 3 months or 5 half-lives, whichever is longer, before study enrolment
* Taking off label mediations or nutritional supplements that the PI consider would impact participant's safe participation.
* Patients who are pregnant and/or lactating, planning a pregnancy during the study. Contraception must be used for sexually active male and female participants
* Intestinal Malabsorption secondary to Pancreatic Insufficiency
* Liver enzymes (alanine aminotransferase [ALT]/aspartate aminotransferase [AST]) or total bilirubin > 2 x the upper limit of normal at the time of screening.
* Renal insufficiency as defined by estimated glomerular filtration rate (eGFR) < 30 mL/min/1.73m2 at the screening visit.
* Any comorbid medical condition that in the assessment of the PI that would impact participant's safe participation (e.g. active cancer requiring treatment)
* Evidence of dysphagia that places subject at risk of aspiration if orally fed.

Study design
Purpose of the study
Other
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
15/03/2022
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
10/07/2023
Sample size
Target
Accrual to date
Final
30
Recruitment in Australia
Recruitment state(s)
QLD
Recruitment hospital [1] 0 0
Queensland Children's Hospital - Brisbane
Recruitment postcode(s) [1] 0 0
4001 - Brisbane

Funding & Sponsors
Primary sponsor type
Other
Name
The University of Queensland
Address
Country
Other collaborator category [1] 0 0
Other
Name [1] 0 0
National Health and Medical Research Council, Australia
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
David Coman, MBBS FRACP
Address 0 0
Queensland Children's Hospital
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

TypeCitations or Other Details
Journal Zannolli R, Buoni S, Betti G, Salvucci S, Plebani ... [More Details]
Journal Gueven N, Luff J, Peng C, Hosokawa K, Bottle SE, L... [More Details]
Journal Valentin-Vega YA, Maclean KH, Tait-Mulder J, Milas... [More Details]
Journal Gillingham MB, Heitner SB, Martin J, Rose S, Golds... [More Details]
Journal Roe CR, Brunengraber H. Anaplerotic treatment of l... [More Details]
Journal Roe CR, Mochel F. Anaplerotic diet therapy in inhe... [More Details]
Journal Schiffmann R, Wallace ME, Rinaldi D, Ledoux I, Lut... [More Details]
Journal Hainque E, Caillet S, Leroy S, Flamand-Roze C, Ada... [More Details]
Journal Hainque E, Gras D, Meneret A, Atencio M, Luton MP,... [More Details]
Journal Hadera MG, Smeland OB, McDonald TS, Tan KN, Sonnew... [More Details]
Journal Hadera MG, McDonald T, Smeland OB, Meisingset TW, ... [More Details]
Journal Yeo AJ, Henningham A, Fantino E, Galbraith S, Krau... [More Details]
Journal McGrath-Morrow SA, Collaco JM, Detrick B, Lederman... [More Details]
Journal McGrath-Morrow SA, Ndeh R, Collaco JM, Rothblum-Ov... [More Details]
Journal Ross LJ, Capra S, Baguley B, Sinclair K, Munro K, ... [More Details]
Journal Morita DA, Glauser TA, Modi AC. Development and va... [More Details]
Journal Yeo AJ, Chong KL, Gatei M, Zou D, Stewart R, Withe... [More Details]


Results not provided in https://clinicaltrials.gov/study/NCT04513002