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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03790865




Registration number
NCT03790865
Ethics application status
Date submitted
28/12/2018
Date registered
2/01/2019

Titles & IDs
Public title
Effects of Livoletide (AZP-531) on Food-related Behaviors in Patients With Prader-Willi Syndrome
Scientific title
A Phase 2b/3 Study to Evaluate the Safety, Tolerability, and Effects of Livoletide (AZP-531), an Unacylated Ghrelin Analogue, on Food-related Behaviors in Patients With Prader-Willi Syndrome
Secondary ID [1] 0 0
AZP01-CLI-003
Universal Trial Number (UTN)
Trial acronym
ZEPHYR
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Prader-Willi Syndrome 0 0
Hyperphagia 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Mental Health 0 0 0 0
Learning disabilities
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above
Metabolic and Endocrine 0 0 0 0
Other endocrine disorders
Mental Health 0 0 0 0
Eating disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Livoletide
Treatment: Drugs - Placebo

Experimental: Low-Dose Livoletide - Daily subcutaneous injection of \~ 60 mcg/kg for 3 month double-blind core period and 9 month open label extension period.

Experimental: High-Dose Livoletide - Daily subcutaneous injection of \~120 mcg/kg for 3 month double-blind core period and 9 month open label extension period.

Placebo comparator: Placebo - Daily subcutaneous injection of 0.9% NaCl for the 3 month double-blind core period and then low-dose or high-dose livoletide for 9 month open label extension period.


Treatment: Drugs: Livoletide
Daily subcutaneous injection

Treatment: Drugs: Placebo
Daily subcutaneous injection
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change in Hyperphagia and Food-related Behaviors (Hyperphagia Questionnaire for Clinical Trials; HQ-CT)
Timepoint [1] 0 0
Baseline to month 3
Secondary outcome [1] 0 0
Percentage Change From Baseline to Month 3 in Total Body Fat Mass in Patients Eight to 65 Years of Age Defined as Overweight/Obese
Timepoint [1] 0 0
Baseline to month 3
Secondary outcome [2] 0 0
Change From Baseline to Month 3 in Waist Circumference in Patients Eight to 65 Years of Age Defined as Overweight/Obese
Timepoint [2] 0 0
Baseline to month 3
Secondary outcome [3] 0 0
Percentage Change From Baseline to Month 3 in Body Weight in Patients Eight to 65 Years of Age Defined as Overweight/Obese
Timepoint [3] 0 0
Baseline to month 3

Eligibility
Key inclusion criteria
* Confirmed genetic diagnosis of PWS
* Evidence of increased appetite or hyperphagia
* Patient must have a single primary caregiver who should be available for certain durations of the study
* BMI = 65 kg/m2
* Growth hormone treatment permitted if doses have been stable for at least 1 month prior to screening
Minimum age
4 Years
Maximum age
65 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* History of chronic liver disease
* Type 1 diabetes mellitus
* HbA1c > 10%
* Body weight <20 kg

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)
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
Stopped early
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
25/03/2019
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
25/05/2020
Sample size
Target
Accrual to date
Final
158
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Royal Prince Alfred Hospital - Camperdown
Recruitment hospital [2] 0 0
Austin Health - Melbourne
Recruitment hospital [3] 0 0
Perth Children's Hospital - Nedlands
Recruitment hospital [4] 0 0
The Childrens Hospital at Westmead - Westmead
Recruitment postcode(s) [1] 0 0
2050 - Camperdown
Recruitment postcode(s) [2] 0 0
3084 - Melbourne
Recruitment postcode(s) [3] 0 0
6009 - Nedlands
Recruitment postcode(s) [4] 0 0
2145 - Westmead
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Alabama
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Colorado
Country [4] 0 0
United States of America
State/province [4] 0 0
Kansas
Country [5] 0 0
United States of America
State/province [5] 0 0
Maryland
Country [6] 0 0
United States of America
State/province [6] 0 0
Massachusetts
Country [7] 0 0
United States of America
State/province [7] 0 0
Michigan
Country [8] 0 0
United States of America
State/province [8] 0 0
Minnesota
Country [9] 0 0
United States of America
State/province [9] 0 0
New York
Country [10] 0 0
United States of America
State/province [10] 0 0
Tennessee
Country [11] 0 0
United States of America
State/province [11] 0 0
Texas
Country [12] 0 0
United States of America
State/province [12] 0 0
Washington
Country [13] 0 0
Belgium
State/province [13] 0 0
Brussels
Country [14] 0 0
France
State/province [14] 0 0
Angers
Country [15] 0 0
France
State/province [15] 0 0
Bron
Country [16] 0 0
France
State/province [16] 0 0
Paris
Country [17] 0 0
France
State/province [17] 0 0
Toulouse
Country [18] 0 0
Italy
State/province [18] 0 0
Napoli
Country [19] 0 0
Italy
State/province [19] 0 0
Roma
Country [20] 0 0
Netherlands
State/province [20] 0 0
Rotterdam
Country [21] 0 0
Spain
State/province [21] 0 0
Alicante
Country [22] 0 0
Spain
State/province [22] 0 0
Barakaldo
Country [23] 0 0
Spain
State/province [23] 0 0
Barcelona
Country [24] 0 0
Spain
State/province [24] 0 0
Sabadell
Country [25] 0 0
United Kingdom
State/province [25] 0 0
Dundee
Country [26] 0 0
United Kingdom
State/province [26] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Millendo Therapeutics SAS
Address
Country

Ethics approval
Ethics application status

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

Contacts
Principal investigator
Name 0 0
Address 0 0
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?




Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results are available at https://clinicaltrials.gov/study/NCT03790865