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Trial registered on ANZCTR


Registration number
ACTRN12612000355875
Ethics application status
Approved
Date submitted
27/03/2012
Date registered
28/03/2012
Date last updated
22/03/2017
Type of registration
Prospectively registered

Titles & IDs
Public title
A Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study to Evaluate the Safety and Tolerability in Patients With Celiac Disease
Scientific title
A Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study to Evaluate the Safety and Tolerability in Patients With Celiac Disease
Secondary ID [1] 280228 0
Nil
Universal Trial Number (UTN)
Nil
Trial acronym
Nil
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Celiac Disease 286175 0
Condition category
Condition code
Inflammatory and Immune System 286370 286370 0 0
Other inflammatory or immune system disorders
Oral and Gastrointestinal 286382 286382 0 0
Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
The study will consist of a Screening Period, Treatment Period, and a Follow-up Period. Escalating dose cohorts will receive active (12 patients per cohort) or placebo given intradermally in a 2:1 ratio. Patients will receive the same dose for the whole Treatment period. During the Screening period medical history, physical examination, vital sign measurements, ECG, and laboratory assessment will be performed as safety assessments. These assessments will also be performed during the study. Adverse events and concomitant medications will be assessed from the Screening visit until the end of the study.
Intervention code [1] 284561 0
Treatment: Drugs
Comparator / control treatment
Placebo: Sodium chloride 0.9% USP (same as vehicle/diluent), dosed at same time as active treatment.
Control group
Placebo

Outcomes
Primary outcome [1] 286825 0
The number and percentage of Adverse Events collected during the Treatment Period. At each visit, patients will be asked standard questions: Have you had any health problems since the previous visit or when you were last asked? and Have you had any new symptoms? to elicit any medically related changes in their well-being. Responses and details will be recorded in the database. Any clinically significant results from any safety assessments may be recorded as an Adverse Event, based on the investigator's medical opinion. Expected Adverse Events are headache and abdominal pain.
Timepoint [1] 286825 0
Adverse Events will be monitored from the time of signing the Informed consent Form until the end of the study.
Secondary outcome [1] 296743 0
Results of safety and tolerability assessments collected during the Treatment Period.
Timepoint [1] 296743 0
Safety and tolerability assessments performed at each visit.
Secondary outcome [2] 296744 0
The PK endpoints are plasma drug concentrations and PK parameters in patients who received active drug.
Timepoint [2] 296744 0
Pharmacokinetic blood samples will be drawn at different timepoints during the study.

Eligibility
Key inclusion criteria
Diagnosis of Celiac Disease
Minimum age
18 Years
Maximum age
70 Years
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Patient has not been prescribed and/or has not followed a GFD for at least 12 months.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Patients will be allocated to active or placebo using an IVRS system.
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Randomisation generated by IVRS/IWRS system.
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?



Intervention assignment
Other
Other design features
Dose escalation study.
Phase
Phase 1
Type of endpoint(s)
Safety/efficacy
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
QLD,SA,WA,VIC
Recruitment postcode(s) [1] 5158 0
5000

Funding & Sponsors
Funding source category [1] 284984 0
Commercial sector/Industry
Name [1] 284984 0
ImmusanT, Inc
Address [1] 284984 0
One Broadway, 14th Floor
Cambridge, MA 02142
Country [1] 284984 0
United States of America
Primary sponsor type
Commercial sector/Industry
Name
ImmusanT, Inc
Address
One Broadway, 14th Floor
Cambridge, MA 02142
Country
United States of America
Secondary sponsor category [1] 283848 0
None
Name [1] 283848 0
Address [1] 283848 0
Country [1] 283848 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 286993 0
Bellberry Limited
Ethics committee address [1] 286993 0
229 Greenhill Road Dulwich SA 5065
Ethics committee country [1] 286993 0
Australia
Date submitted for ethics approval [1] 286993 0
28/03/2012
Approval date [1] 286993 0
21/05/2012
Ethics approval number [1] 286993 0
2012-04-735-AA

Summary
Brief summary
This a randomized, double-blind, placebo-controlled, multiple ascending dose study to evaluate the safety and tolerability in patients with celiac disease who are on a gluten-free diet.
Trial website
Nil
Trial related presentations / publications
Nil
Public notes

Contacts
Principal investigator
Name 33986 0
Dr James Daveson
Address 33986 0
Q-Pharm Pty Ltd
PO Box 78
Royal Brisbane Hospital
Herston QLD 4029
Country 33986 0
Australia
Phone 33986 0
+61 7 33671065
Fax 33986 0
Email 33986 0
jamesdaveson@uq.edu.au
Contact person for public queries
Name 17233 0
Ms Sandrien Louwaars
Address 17233 0
CPR Pharma Services
Suite C, 32 West Thebarton Road
THEBARTON SA 5031
Country 17233 0
Australia
Phone 17233 0
+61 8 8125 1907
Fax 17233 0
Email 17233 0
sandrien.louwaars@cprservices.com.au
Contact person for scientific queries
Name 8161 0
Dr Patrick Griffin
Address 8161 0
ImmusanT, Inc
One Broadway
14th Floor
Cambridge, MA 02142
Country 8161 0
United States of America
Phone 8161 0
+1 646-541-4588
Fax 8161 0
Email 8161 0
pgriff@pol.net

No information has been provided regarding IPD availability
Summary results
Have study results been published in a peer-reviewed journal?
Other publications
Have study results been made publicly available in another format?
Results – basic reporting
Results – plain English summary