Trial Review

Please note the ANZCTR will be unattended from Friday 20 December 2024 for the holidays. The Registry will re-open on Tuesday 7 January 2025. Submissions and updates will not be processed during that time.

Registering a new trial?

To achieve prospective registration, we recommend submitting your trial for registration at the same time as ethics submission.

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 details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05796440




Registration number
NCT05796440
Ethics application status
Date submitted
19/03/2023
Date registered
3/04/2023
Date last updated
15/02/2024

Titles & IDs
Public title
A Long-Term Safety Trial of LUM-201 in Children With Idiopathic Growth Hormone Deficiency Who Have Previously Completed a LUM-201 Clinical Trial (OraGrowtH211)
Scientific title
An Extension Study to Monitor Long-Term Safety of LUM-201 Treatment in Children With Idiopathic Growth Hormone Deficiency
Secondary ID [1] 0 0
LUM-201-02
Universal Trial Number (UTN)
Trial acronym
OraGrowtH211
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Growth Hormone Deficiency 0 0
Condition category
Condition code
Metabolic and Endocrine 0 0 0 0
Other endocrine disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - LUM-201

Experimental: LUM-201 (1.6 mg/kg/day) -


Treatment: Drugs: LUM-201
Administered orally once daily
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Incidence of treatment-emergent adverse events
Timepoint [1] 0 0
Day 1 to Month 36
Secondary outcome [1] 0 0
Growth Hormone Concentrations in subjects
Timepoint [1] 0 0
Day 1 to Month 36
Secondary outcome [2] 0 0
Insulin-like growth factor 1 concentration
Timepoint [2] 0 0
Day 1 to Month 36
Secondary outcome [3] 0 0
Height standard deviation score (SDS)
Timepoint [3] 0 0
Day 1 to Month 36
Secondary outcome [4] 0 0
Change in Weight SDS
Timepoint [4] 0 0
Day 1 to Month 36
Secondary outcome [5] 0 0
Annualized Height Velocity (AHV) achieved by daily dosing 1.6mg/kg of LUM-201
Timepoint [5] 0 0
Day 1 to Month 36
Secondary outcome [6] 0 0
Change in Bone Age (BA)
Timepoint [6] 0 0
Day 1 to Month 36

Eligibility
Key inclusion criteria
* Parent/caregiver must sign the informed consent, and the subject must sign the assent, as applicable.
* Must have successfully participated in a pediatric LUM-201 GHD study through at least the 12-month visit, and be eligible for continuation of treatment, pending all other enrollment criteria are met.
Minimum age
5 Years
Maximum age
14 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Medical or genetic condition that, in the opinion of the PI and/or MMs, adds unwarranted risk to the use of LUM-201
* Has planned or is receiving current long-term treatment with medications known to act as substrates, inducers, or inhibitors of the cytochrome system CYP3A4 that metabolizes LUM-201. Subjects receiving shorter-term (two weeks or less) treatment with these medications should be evaluated on case-by-case basis by the PI in consultation with the MMs.

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
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
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/08/2023
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/04/2028
Actual
Sample size
Target
60
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
ACT
Recruitment hospital [1] 0 0
Canberra Hospital - Garran
Recruitment postcode(s) [1] 0 0
2605 - Garran
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Iowa
Country [2] 0 0
United States of America
State/province [2] 0 0
Massachusetts
Country [3] 0 0
United States of America
State/province [3] 0 0
Missouri
Country [4] 0 0
United States of America
State/province [4] 0 0
South Carolina
Country [5] 0 0
United States of America
State/province [5] 0 0
Texas
Country [6] 0 0
United States of America
State/province [6] 0 0
Washington

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Lumos Pharma
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

No information has been provided regarding IPD availability


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results not provided in https://clinicaltrials.gov/study/NCT05796440