Clinical trial NCT05796440: 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)
Official title: An Extension Study to Monitor Long-Term Safety of LUM-201 Treatment in Children With Idiopathic Growth Hormone Deficiency
Study type: INTERVENTIONAL
Overall status: ENROLLING_BY_INVITATION
Phase: PHASE2
Conditions: Growth Hormone Deficiency
Keywords: LUM-201
PGHD
GHD
Growth hormone secretagogue
Oral
Height
Lead sponsor: Lumos Pharma
Sponsor class: INDUSTRY
First posted: 2023-04-03
Enrollment: 60
Sex: ALL
Minimum age (years): 5
Maximum age (years): 14
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: Open Label
Primary purpose: TREATMENT

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.

Detailed description: An Extension Study to Monitor Long-Term Safety of LUM-201 Treatment in Children with Idiopathic Growth Hormone Deficiency. This study will last for up to 36 months to allow collection of additional long-term safety and efficacy data related to LUM-201 treatment in the pediatric patient population. Subjects will enter into this trial after successful participation in a prior LUM-201 study. This study will consist of visits every 6 months through three years.

There are a total of 7 in-person visits with a follow-up phone call between visits. At the clinic visits, subjects will have a physical exam and blood collection as well as efficacy assessments.

Eligibility criteria: 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.

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.

Primary outcomes:
measure: Incidence of treatment-emergent adverse events
timeFrame: Day 1 to Month 36
Secondary outcomes:
measure: Growth Hormone Concentrations in subjects
timeFrame: Day 1 to Month 36
measure: Insulin-like growth factor 1 concentration
timeFrame: Day 1 to Month 36
measure: Height standard deviation score (SDS)
timeFrame: Day 1 to Month 36
measure: Change in Weight SDS
timeFrame: Day 1 to Month 36
measure: Annualized Height Velocity (AHV) achieved by daily dosing 1.6mg/kg of LUM-201
timeFrame: Day 1 to Month 36
measure: Change in Bone Age (BA)
timeFrame: Day 1 to Month 36
Other outcomes:
Locations:
city: Iowa City
country: United States
facility: University of Iowa
state: Iowa
city: Worcester
country: United States
facility: UMass Memorial Medical Center
state: Massachusetts
city: Kansas City
country: United States
facility: Children's Mercy Hospital
state: Missouri
city: Charleston
country: United States
facility: University of South Carolina
state: South Carolina
city: Amarillo
country: United States
facility: Texas Tech University Health Sciences Center
state: Texas
city: Seattle
country: United States
facility: Seattle Children's Hospital
state: Washington
city: Garran
country: Australia
facility: Canberra Hospital
state: Australian Capital Territory
Study officials:
References: