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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
This is a multi-national trial. The trial aims to study the long-term safety of LUM-201 in subjects with Idiopathic Pediatric Growth Hormone Deficiency (iPGHD). This study will also assess pharmacodynamics and efficacy response to therapy with LUM-201.
Trial website
https://clinicaltrials.gov/study/NCT05796440
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



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT05796440