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)
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: LUM-201.
- Who it may be relevant to
- Registry conditions: Growth Hormone Deficiency. Basic parameters: 5 years — 14 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States, Australia
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
An Extension Study to Monitor Long-Term Safety of LUM-201 Treatment in Children With Idiopathic Growth Hormone Deficiency
Overview
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.
Interventions
- Drug LUM-201
Administered orally once daily
Primary outcome measures
- Incidence of treatment-emergent adverse events [Time frame: Day 1 to Month 36]
Secondary outcome measures (6)
- Growth Hormone Concentrations in subjects [Time frame: Day 1 to Month 36]
- Insulin-like growth factor 1 concentration [Time frame: Day 1 to Month 36]
- Height standard deviation score (SDS) [Time frame: Day 1 to Month 36]
- Change in Weight SDS [Time frame: Day 1 to Month 36]
- Annualized Height Velocity (AHV) achieved by daily dosing 1.6mg/kg of LUM-201 [Time frame: Day 1 to Month 36]
- Change in Bone Age (BA) [Time frame: Day 1 to Month 36]
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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
United States · 6 centers
- University of Iowa — Iowa City
- UMass Memorial Medical Center — Worcester
- Children's Mercy Hospital — Kansas City
- University of South Carolina — Charleston
- Texas Tech University Health Sciences Center — Amarillo
- Seattle Children's Hospital — Seattle
Australia · 1 center
- Canberra Hospital — Garran
Identifiers
NCT: NCT05796440 · LUM-201-02