Trial Review

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Trial registered on ANZCTR


Registration number
ACTRN12623001112651
Ethics application status
Not required
Date submitted
19/09/2023
Date registered
25/10/2023
Date last updated
25/10/2023
Date data sharing statement initially provided
25/10/2023
Type of registration
Retrospectively registered

Titles & IDs
Public title
HEPATA- HEreditary Pancreatitis and Auto-islet transplant Trials Australia.
Scientific title
HEPATA- HEreditary Pancreatitis and Auto-islet transplant Trials Australia: Effect on analgesic requirement and glycemic control.
Secondary ID [1] 310564 0
MRF2014872
Universal Trial Number (UTN)
Trial acronym
HEPATA
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Hereditary Pancreatitis 331406 0
Condition category
Condition code
Human Genetics and Inherited Disorders 328148 328148 0 0
Other human genetics and inherited disorders
Cancer 328344 328344 0 0
Pancreatic
Oral and Gastrointestinal 328345 328345 0 0
Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon
Inflammatory and Immune System 328346 328346 0 0
Other inflammatory or immune system disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Total Pancreatectomy and Islet-Auto Transplantation (TP-IAT or TPIAT)
Admission: Patient admitted the day before the Pancreatectomy and Islet transplant, pre-operative bloods taken and reviewed by hepatobiliary surgical team.
Pancreatectomy: Surgery (3-5 hours duration)scheduled early morning allowing time to transport the organ to islet cell isolation facility in Melbourne. Hepatobiliary Surgeon to remove the pancreas and hand to renal surgeon who will perfuse and prepare the organ to be packed for transport. The patient then goes to recovery and then ICU until later returning to operating theatres/angiography suite for Islet transplant late evening on the same day.
Islet Transplant: Radiologist performs percutaneous puncture of the portal vein and positions the infusion catheter at the bifurcation of the vein to deliver cells to both lobes of the liver. Islet cells are infused into the liver via the catheter by gravity over a 15-30 min period. The patient then goes to recovery/ ICU. Once stable the patient is admitted to the renal transplant ward until hospital discharge 5-7 days after the TPIAT procedure. Total duration of procedure 1-2 hours. Currently, access to this treatment is only available through this study and is one of the primary reasons for this trial. The aim being to capture enough outcome data to support an application for activity based funding for the procedure through the Australian Medicare Benefits Schedule. This would ensure all Australians have access to this treatment in the future.
Intervention code [1] 326962 0
Treatment: Surgery
Comparator / control treatment
Patients will serve as their own control prior to TP-IAT intervention.
Control group
Active

Outcomes
Primary outcome [1] 336022 0
Glycaemic control
Instruments; Composite measure of Serum HbA1c, fasting c-peptide and average daily insulin dose.
Data will be collected during routine clinical follow-up appointments,
Timepoint [1] 336022 0
< 1 month pre-TPIAT and 3, 6, 12, 24 and 36 months post TP-IAT
Primary outcome [2] 336023 0
Analgesic requirement
Instrument: Composite measure of daily pain medication and dosage.
Data will be collected during routine clinical follow-up appointments.
Timepoint [2] 336023 0
< 1 month pre-TPIAT and 3, 6, 12, 24 and 36 months post TP-IAT
Secondary outcome [1] 426503 0
Quality of Life
Timepoint [1] 426503 0
< 1 month pre TP-IAT and 12, 24 and 36 months post TP-IAT
Instrument: SF-12 assessment tool

Eligibility
Key inclusion criteria
Individuals must have molecular genotype-confirmed Hereditary Pancreatitis (PRSS-1,SPINK-1,CFTR,CTRC ) AND symptoms of Pancreatitis fulfilling the Minnesota Criteria for TP-IAT
The Minnesota criteria:
(1) Diagnosis of pancreatitis with the features of chronic abdominal pain of > 6 months duration with evidence of; i), identified gene mutation (PRSS1, SPINK1, CRTR, CTRC); ii), pancreatic calcification on CT scan; iii), > 4/9 Endoscopic Ultrasound Criteria (EUS); iv), histological confirmed diagnosis of CP.
(2) At least one of the following: daily narcotic dependence, pain resulting in impaired quality of life which may include: inability to attend school, recurrent hospitalisations and inability to participate in usual, age appropriate activities.
(3) Complete evaluation with no reversible cause of pancreatitis present or untreated.
(4) Failure to respond to maximal medical therapy and endoscopic therapy.
(5) Adequate islet cell function (non-diabetic or C-peptide positive).
Minimum age
No limit
Maximum age
75 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Absolute contraindications: suspicion of pancreatic carcinoma complicating chronic pancreatitis, presence of intrapapillary mucinous neoplasia (IPMNs), ongoing significant alcohol consumption. Relative Contraindications: anticipated poor islet yields due to: age (>75 yrs), previous pancreatic surgery and insulin dependency, increased surgical risks due to hostile abdomen (previous abdominal catastrophies) and surgical comorbidities (morbid obesity, ongoing smoking) and pregnancy. Patients with acute pancreatitis with other readily identifiable non-genetic causes such as alcohol, gall stone, auto-immune pancreatitis, hyper-triglyceridaemia and anatomic variants e.g. pancreas divisum and without a family history that is suggestive of an inherited disorder.

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised 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
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Not Applicable
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Date of first participant enrolment
Anticipated
Actual
28/06/2022
Date of last participant enrolment
Anticipated
31/12/2025
Actual
Date of last data collection
Anticipated
31/12/2026
Actual
Sample size
Target
24
Accrual to date
9
Final
Recruitment in Australia
Recruitment state(s)
ACT,NSW,NT,QLD,SA,TAS,WA,VIC

Funding & Sponsors
Funding source category [1] 314769 0
Government body
Name [1] 314769 0
National Health and Medical Research Council, Medical Research Future Fund MRFF - Rare Cancer and Rare Diseases
Country [1] 314769 0
Australia
Primary sponsor type
Hospital
Name
Central Adelaide Local Health Network
Address
1 Port Road, Adelaide SA 5000
Country
Australia
Secondary sponsor category [1] 316753 0
None
Name [1] 316753 0
Address [1] 316753 0
Country [1] 316753 0

Ethics approval
Ethics application status
Not required
Ethics committee name [1] 313778 0
Ethics committee address [1] 313778 0
Ethics committee country [1] 313778 0
Date submitted for ethics approval [1] 313778 0
Approval date [1] 313778 0
Ethics approval number [1] 313778 0

Summary
Brief summary
The HEPATA Trial is designed to deliver comprehensive evidence for TP-IAT inclusion on the Australian Medicare Benefits Schedule. This will provide activity based funding enabling equitable access to this therapeutic intervention to all Australians and dramatically improve outcomes and prognosis for all HP patients. It will also inform the generation of clinical guidelines for treatment of hereditary pancreatitis with TP-IAT.


This study is designed to deliver comprehensive evidence for Total Pancreatectomy and Islet-Auto Transplantation (TP-IAT) inclusion on the Australian Medicare Benefits Schedule for patients with hereditary pancreatitis

Who is it for?
You may be eligible to join this study if you are less than 75 years old and have molecular genotype-confirmed hereditary pancreatitis (PRSS-1, SPINK-1, CFTR, CTRC) and symptoms of pancreatitis

Study details
All participants in this study will undergo a total pancreatectomy procedure performed by a hepatobiliary surgeon. The pancreas will then be prepared for transport to the islet cell isolation facility by a renal surgeon. Islet cells are isolated for transplantation the same day.
A radiologist transplants the isolated islet cells back (via infusion) into the patient.

Participants will then be followed-up and assessed regularly at 3, 6, 12, 24 and 36 months post-procedure to determine impact of TP-IAT on glycemic control, pain and quality of life.

It is hoped that this research project will provide evidence to support an application for activity based funding for the TP-IAT procedure through the Australian Medicare Benefits Schedule. This would ensure all Australians have access to this treatment in the future
Trial website
Trial related presentations / publications
Public notes
TP-IAT is an approved clinical procedure within Central Adelaide Local Health Network - Royal Adelaide Hospital and conforms to the NHMRC guidelines (https://www.nhmrc.gov.au/about-us/publications/national-statement-ethical-conduct-human-research-2007-updated-2018).

Contacts
Principal investigator
Name 129330 0
Prof Toby Coates
Address 129330 0
Level 7 Royal Adelaide Hospital, 1 Port Road Adelaide SA 5000
Country 129330 0
Australia
Phone 129330 0
+61 439901856
Fax 129330 0
Email 129330 0
Toby.Coates@sa.gov.au
Contact person for public queries
Name 129331 0
Prof Toby Coates
Address 129331 0
Level 7 Royal Adelaide Hospital, 1 Port Road Adelaide SA 5000
Country 129331 0
Australia
Phone 129331 0
+61 439901856
Fax 129331 0
Email 129331 0
Toby.Coates@sa.gov.au
Contact person for scientific queries
Name 129332 0
Prof Toby Coates
Address 129332 0
Level 7 Royal Adelaide Hospital, 1 Port Road Adelaide SA 5000
Country 129332 0
Australia
Phone 129332 0
+61 439901856
Fax 129332 0
Email 129332 0
Toby.Coates@sa.gov.au

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

Documents added manually
No documents have been uploaded by study researchers.

Documents added automatically
No additional documents have been identified.