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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04614337




Registration number
NCT04614337
Ethics application status
Date submitted
26/10/2020
Date registered
4/11/2020

Titles & IDs
Public title
Phase 2 Study of LUM-201 in Children With Growth Hormone Deficiency (OraGrowtH210 Trial)
Scientific title
A Multicenter, 24-Month, Randomized, Open-Label, Active Control, Parallel Arm, Phase 2 Study of Daily Oral LUM-201 in Naïve-to-Treatment, Prepubertal Children With Idiopathic Growth Hormone Deficiency (GHD)
Secondary ID [1] 0 0
LUM-201-01
Universal Trial Number (UTN)
Trial acronym
OraGrowtH210
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
Treatment: Drugs - rhGH Norditropin® pen (34 µg/kg)

Experimental: LUM-201 (0.8 mg/kg/day) -

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

Experimental: LUM-201 (3.2 mg/kg/day) -

Active comparator: rhGH (34 µg/kg/day) -


Treatment: Drugs: LUM-201
Administered orally once daily

Treatment: Drugs: rhGH Norditropin® pen (34 µg/kg)
Administered subcutaneously (s.c., under the skin) once daily.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Percentage of subjects selected by PEM strategy who meet target growth
Timepoint [1] 0 0
Day 1 to Month 6
Primary outcome [2] 0 0
AHV after 6 months on LUM-201 compared to rhGH
Timepoint [2] 0 0
Day 1 to Month 6
Secondary outcome [1] 0 0
Degree of concordance between the first and second assessment with the PEM strategy.
Timepoint [1] 0 0
Screening to Day 1
Secondary outcome [2] 0 0
Incidence of adverse events in children with GHD
Timepoint [2] 0 0
Day 1 to Month 24
Secondary outcome [3] 0 0
Height standard deviation score (SDS)
Timepoint [3] 0 0
Day 1 to Month 6 and Month 12
Secondary outcome [4] 0 0
Height velocity standard deviation score (HV-SDS)
Timepoint [4] 0 0
Day 1 to Month 6, and Month 12
Secondary outcome [5] 0 0
Change in Weight
Timepoint [5] 0 0
Day 1 to Month 6, and Month 12
Secondary outcome [6] 0 0
Change in Weight SDS
Timepoint [6] 0 0
Day 1 to Month 6 and Month 12
Secondary outcome [7] 0 0
Change in BMI
Timepoint [7] 0 0
Day 1 to Month 6 and Month 12
Secondary outcome [8] 0 0
Change in BMI SDS
Timepoint [8] 0 0
Day 1 to Month 6 and Month 12
Secondary outcome [9] 0 0
Bone Age
Timepoint [9] 0 0
Day 1 to Month 6 and Month 18
Secondary outcome [10] 0 0
Pharmacokinetics of LUM-201
Timepoint [10] 0 0
Day 1 to Month 6 and 12
Secondary outcome [11] 0 0
GH Concentration on maintenance treatment
Timepoint [11] 0 0
Day 1 to Month 6 and 12
Secondary outcome [12] 0 0
Insulin-like growth factor 1 SDS
Timepoint [12] 0 0
Day 1 to Month 6 and 12

Eligibility
Key inclusion criteria
* Have an established diagnosis of idiopathic PGHD as determined by standard diagnostic criteria. Eligible subjects must be naïve-to-treatment and be prepubertal.
* Morning cortisol = 7 µg/dL or stimulated cortisol = 14 µg/dL.
* At Screening, be = 3.0 years and = 11.0 years for girls and = 12.0 years for boys.
* Have HT-SDS = -2.0 or HT-SDS = 2 SD below mean parental HT-SDS.
* Have a baseline height velocity < 5.5 cm/year based on at least 6 months of growth.
* Have a bone age delayed by = 6 months with respect to chronological age.
* Have prepubertal status as evidenced by Tanner Stage I breast development in girls and testicular volume < 4.0 mL in boys.
* In girls, have genetic testing results to rule out Turner syndrome. If SHOX genetic testing results are available, they need to be negative.
* Have normal thyroid function. Subjects diagnosed with hypothyroidism must have documented successful treatment for at least 30 days prior to Day 1.
Minimum age
3 Years
Maximum age
12 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Any medical or genetic condition which, in the opinion of the Investigator or Medical Monitor (MM), can be an independent cause of short stature and/or limit the response to exogenous growth factor treatment. (Examples: diabetes, idiopathic short stature).
* A medical or genetic condition that, in the opinion of the Investigator and/or MM, adds unwarranted risk to use of LUM-201 or rhGH.
* Use of any medication that, in the opinion of the Investigator and/or MM, can independently cause short stature or limit the response to exogenous growth factors (Example: glucocorticoids).
* Evidence or history of an intracranial mass (e.g., pituitary tumor, craniopharyngioma).
* Suspicion of absent pituitary function as evidenced by a maximal stimulated GH = 3 ng/mL on two prior standard of care GH stimulation tests, or pituitary deficiencies beyond GH and thyroid function.
* Malnutrition as evidenced by medical history or a body weight < 3rdth percentile for current height.
* BMI > 95th percentile.
* Gestational age-adjusted birth weight < 5th percentile (small for gestational age).
* History of spinal, cranial, or total body irradiation.
* Treatment with medications known to act as moderate or strong inhibitors or strong inducers of CYP3A/4, or with medications known to act as strong inhibitors of P-glycoprotein (P-gp) or potent substrates of P-gp or Multidrug and toxin extrusion protein 1 (MATE1).

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
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
31/12/2020
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/10/2024
Actual
Sample size
Target
80
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
ACT,VIC
Recruitment hospital [1] 0 0
Canberra Hospital - Garran
Recruitment hospital [2] 0 0
Department of Pediatrics and Endocrinology- Monash Health - Clayton
Recruitment hospital [3] 0 0
Royal Children's Hospital - Melbourne
Recruitment hospital [4] 0 0
Queensland Children's Hospital - South Brisbane
Recruitment postcode(s) [1] 0 0
2605 - Garran
Recruitment postcode(s) [2] 0 0
3168 - Clayton
Recruitment postcode(s) [3] 0 0
3052 - Melbourne
Recruitment postcode(s) [4] 0 0
- South Brisbane
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Colorado
Country [3] 0 0
United States of America
State/province [3] 0 0
District of Columbia
Country [4] 0 0
United States of America
State/province [4] 0 0
Georgia
Country [5] 0 0
United States of America
State/province [5] 0 0
Indiana
Country [6] 0 0
United States of America
State/province [6] 0 0
Iowa
Country [7] 0 0
United States of America
State/province [7] 0 0
Kentucky
Country [8] 0 0
United States of America
State/province [8] 0 0
Massachusetts
Country [9] 0 0
United States of America
State/province [9] 0 0
Minnesota
Country [10] 0 0
United States of America
State/province [10] 0 0
Missouri
Country [11] 0 0
United States of America
State/province [11] 0 0
New York
Country [12] 0 0
United States of America
State/province [12] 0 0
Ohio
Country [13] 0 0
United States of America
State/province [13] 0 0
Oklahoma
Country [14] 0 0
United States of America
State/province [14] 0 0
Pennsylvania
Country [15] 0 0
United States of America
State/province [15] 0 0
South Carolina
Country [16] 0 0
United States of America
State/province [16] 0 0
Texas
Country [17] 0 0
United States of America
State/province [17] 0 0
Virginia
Country [18] 0 0
United States of America
State/province [18] 0 0
Washington
Country [19] 0 0
Israel
State/province [19] 0 0
Tiqwa
Country [20] 0 0
New Zealand
State/province [20] 0 0
Wellington
Country [21] 0 0
New Zealand
State/province [21] 0 0
Auckland
Country [22] 0 0
Poland
State/province [22] 0 0
Bialystok
Country [23] 0 0
Poland
State/province [23] 0 0
Lodz
Country [24] 0 0
Poland
State/province [24] 0 0
Pomorskie
Country [25] 0 0
Poland
State/province [25] 0 0
Rzeszów
Country [26] 0 0
Poland
State/province [26] 0 0
Szczecin
Country [27] 0 0
Poland
State/province [27] 0 0
Warsaw
Country [28] 0 0
Poland
State/province [28] 0 0
Wroclaw
Country [29] 0 0
Ukraine
State/province [29] 0 0
Kyiv

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

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Undecided
No/undecided IPD sharing reason/comment


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/NCT04614337