Register a trial

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/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
Completed
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
Actual
4/09/2024
Sample size
Target
Accrual to date
Final
82
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