The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this information for consumers
Trial registered on ANZCTR


Registration number
ACTRN12615000289516
Ethics application status
Approved
Date submitted
17/03/2015
Date registered
27/03/2015
Date last updated
4/04/2017
Type of registration
Prospectively registered

Titles & IDs
Public title
A Phase II Open-Label Study of the Safety, Tolerability, Pharmacokinetics and Efficacy of ATL1103 300mg in Adult Patients with Acromegaly.
Scientific title
A Phase II Open-Label Study of the Safety, Tolerability, Pharmacokinetics and Efficacy of ATL1103 300mg in Adult Patients with Acromegaly.
Secondary ID [1] 285876 0
Protocol 1103-CT03
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Acromegaly 293795 0
Condition category
Condition code
Metabolic and Endocrine 294098 294098 0 0
Other metabolic disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Subjects will receive ATL1103 (growth hormone (GH) receptor antisense oligonucleotide) as a sterile aqueous solution administered by subcutaneous injection

Dose regimen 300mg twice a week for 13 weeks
Intervention code [1] 290857 0
Treatment: Drugs
Comparator / control treatment
None
Control group
Uncontrolled

Outcomes
Primary outcome [1] 293889 0
Safety and tolerability will be assessed by serum chemistry, haematology, urinalysis, physical exam, vital signs, electrocardiogram, MRI, and monitoring and recording adverse events
Timepoint [1] 293889 0
Over 13 weeks of treatment and 8 weeks of follow up
Primary outcome [2] 293890 0
Single and multiple dose pharmacokinetic parameters
Timepoint [2] 293890 0
Plasma will be collected for ATL1103 assays pre-dose and at 1, 2, 3, 4 and 6 hours post first and last doses, and pre-dose only on Day 4 of Weeks 1, 2, 4, 6, 8, 10, and 12.
Secondary outcome [1] 312099 0
Efficacy of ATL1103 will be assessed by measurement of serum insulin-like growth factor (IGF)-I levels.
Timepoint [1] 312099 0
Week 14 compared to Baseline.
Secondary outcome [2] 312100 0
To explore pharmacodynamic effects of ATL1103 on serum laboratory parameters (GH, GHBP, IGFBP-3, ALS, and IGF-II)
Timepoint [2] 312100 0
Week 14 compared to Baseline
Secondary outcome [3] 312101 0
To explore pharmacodynamic effects of ATL1103 on ring size assessment using standard set of jewellers rings
Timepoint [3] 312101 0
Week 14 compared to Baseline
Secondary outcome [4] 312102 0
To explore pharmacodynamic effects of ATL1103 on Signs and Symptoms Scale
Timepoint [4] 312102 0
Week 14 compared to Baseline

Eligibility
Key inclusion criteria
- Provide written informed consent in accordance with local regulations.
- Are 18 to 80 years of age inclusive.
- Have acromegaly due to pituitary adenoma (micro or macro adenoma) identified by Magnetic Resonance Imaging (MRI).
- Have serum IGF-I level at Screening >1.3 times the upper limit of normal (ULN).
- Have nadir serum GH levels > 1ng/mL at all test time points within the 2 hours post oral glucose load for an oral glucose tolerance test (OGTT).
- Are acromegaly treatment naive, or who have not taken other acromegaly medications for a period of 6 weeks to 4 months, depending on the medication.
Minimum age
18 Years
Maximum age
80 Years
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- Have acromegaly due to reasons other than pituitary adenoma.
- Have participated in any clinical investigation with an investigational drug within 3 months (4 months if the drug is a new chemical entity) preceding the Baseline visit or during the washout period.
- Have a history of clinically relevant gastrointestinal, hepatic, renal, endocrine (other than acromegaly), haematological, metabolic, neurologic or psychiatric disease that in the investigator’s opinion may compromise their safety or effect results from this study.
- Have any other medical condition which, in the judgement of the Investigator, might interfere with the objectives of the study, or are otherwise unsuitable for participation.

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
Who is / are masked / blinded?



Intervention assignment
Other design features
Phase
Phase 2
Type of endpoint(s)
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)
SA

Funding & Sponsors
Funding source category [1] 290457 0
Commercial sector/Industry
Name [1] 290457 0
Antisense Therapeutics Limited
Address [1] 290457 0
6 Wallace Avenue
Toorak Vic 3142
Country [1] 290457 0
Australia
Primary sponsor type
Commercial sector/Industry
Name
Antisense Therapeutics Limited
Address
6 Wallace Avenue
Toorak Vic 3142
Country
Australia
Secondary sponsor category [1] 289159 0
None
Name [1] 289159 0
Address [1] 289159 0
Country [1] 289159 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 292130 0
Royal Adelaide Hospital REC
Ethics committee address [1] 292130 0
North Tce
Adelaide 5000 SA
Ethics committee country [1] 292130 0
Australia
Date submitted for ethics approval [1] 292130 0
05/11/2014
Approval date [1] 292130 0
22/12/2014
Ethics approval number [1] 292130 0
HREC/14/RAH/499

Summary
Brief summary
ATL1103 is being developed as a potential treatment for acromegaly, a disease of excessive growth hormone and insulin-like growth hormone (IGF-I) action. Approximately four acromegaly patients will receive 300mg ATL1103 twice a week for 13 weeks. All treatments will be administered by subcutaneous injection.

The primary objectives of the study are to evaluate the safety and tolerability of a high dose of ATL1103, and to investigate the pharmacokinetic profiles of ATL1103. The effect of ATL1103 on serum IGF-I levels and on other pharmacodynamic markers will also be measured.
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 53646 0
Dr David Torpy
Address 53646 0
Royal Adelaide Hospital
North Tce
Adelaide 5000 SA
Country 53646 0
Australia
Phone 53646 0
+61 8 8222 5520
Fax 53646 0
Email 53646 0
david.torpy@health.sa.gov.au
Contact person for public queries
Name 53647 0
Ms Sue Turner
Address 53647 0
Antisense Therapeutics
6 Wallace Avenue
Toorak VIC 3142
Country 53647 0
Australia
Phone 53647 0
+61 3 9827 8999
Fax 53647 0
Email 53647 0
info@antisense.com.au
Contact person for scientific queries
Name 53648 0
Dr Lynne Atley
Address 53648 0
Antisense Therapeutics
6 Wallace Avenue
Toorak VIC 3142
Country 53648 0
Australia
Phone 53648 0
+61 3 9827 8999
Fax 53648 0
Email 53648 0
lynne.atley@antisense.com.au

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