A Multicenter, 12-Month, Randomized, Double Blind, Placebo-Controlled Phase 3 Efficacy and Safety Study of Daily Oral LUM 201 in Naïve-to-Treatment, Prepubertal Children with Growth Hormone Deficiency (GHD)

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Lumos Pharma LLC

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Hormonal diseases [C19]

Decision date (initial)

2026-02-22

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Lumos Pharma LLC

External identifiers

EU CT number

2025-523820-26-00

ClinicalTrials.gov

NCT06948214

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Pharmacodynamic, Efficacy, Pharmacokinetic, Therapy, Safety

To evaluate the effect of LUM 201 1.6 mg/kg/day on growth rates over 12 months in prepubertal children with GHD

Secondary objectives 1

1. Change from baseline in IGF-1 SDS, HT-SDS, and IGFBP-3 SDS.

Conditions and MedDRA coding

Growth Hormone Deficiency (GHD)

Study design 3 periods

Regulatory references

Scientific advice from competent authorities

European Medicines Agency

EMA paediatric investigation plan (PIP)

EMEA-003032-PIP01-21

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 12

1. • Written informed consent must be provided by the subject’s parent(s) or legally acceptable representative(s) prior to any study related procedures. Where applicable based on age and local regulations, the subject must also provide age-appropriate assent. The subject and the parent(s) or legally acceptable representative(s) must be willing and able to comply with all study procedures.
2. • Subjects must be naïve to treatment and prepubertal (Tanner stage I, which is determined by breast development in girls and testicular volume < 4.0 mL in boys).
3. • Subjects must have a maximal GH response of < 10 ng/mL from 2 prior GH stimulation tests conducted within the preceding 12 months. Acceptable GH stimulation tests include insulin, glucagon, arginine, clonidine and levodopa (L-DOPA). Additionally, 2 GH stimulation tests completed on the same day might be utilized, as well as other validated and approved GH stimulation tests at the discretion of the MM.
4. • Impaired height defined as ≥ 2.0 standard deviations (SDs) below the mean height for chronological age and sex at the Screening Visit according to the World Health Organization (WHO) Growth Charts.
5. • During the Screening Visit or within the preceding 3 months, subjects must exhibit a morning (prior to 9 a.m. local time) or random cortisol level of ≥ 7.0 µg/dL (193.0 nmol/L). If subjects have a morning or random cortisol response of < 7.0 µg/dL, they will need to undergo a stimulated cortisol test (adrenocorticotropic hormone [ACTH], insulin, glucagon) to qualify for the trial. The stimulated peak of cortisol response must be ≥ 18.0 µg/dL (500.0 nmol/L) for older polyclonal antibody assays. A stimulated peak less than 18 on newer-generation assays and considered normal (i.e. adrenally sufficient) will be acceptable after discussion with medical monitor group.
6. • At the Screening Visit, be age ≥ 3.0 years and age ≤ 10.0 years for girls and ≤ 11.0 years for boys.
7. • Have accurate baseline height velocity (HV) based on ≥ 6 months of growth assessments and confirmed by the MM establishing baseline HV < 25th percentile for age and sex. If only available prior assessments of growth were conducted 3 to 6 months prior to the Screening Visit, the MM should be contacted for consultation. Although it's preferable to conduct baseline height assessments using a wall-mounted stadiometer in the pediatric endocrinologist’s office, baseline AHV can be derived from data collected from the referral office.
8. • Undergo a BA assessment either during the Screening Visit or within 12 months prior. The assessment, interpreted using central reader methodology, must demonstrate a delay of ≥ 12 months compared to the chronological age.
9. • Girls should undergo genetic testing to eliminate the possibility of Turner syndrome. Additionally, if there are results from the short stature homeobox (SHOX) genetic test, they should indicate a negative outcome.
10. • Have normal thyroid function (TSH and free T4 in normal range). Subjects diagnosed with primary hypothyroidism must have documented euthyroid for ≥ 3 months prior to the Day 1 Visit.
11. • IGF-1 standard deviation score (SDS) ≤ -1.0 at the Screening Visit, compared to age and sex normalized range measured at central laboratory.
12. • IGF-1 concentration > 30.0 ng/mL (> 3.92 nmol/L) and a peak GH response of ≥ 5.0 ng/mL from the Screening Visit PEM test.

Exclusion criteria 29

1. • Any medical or genetic condition which, in the opinion of the Investigator or MM, can be an independent cause of short stature and/or limit the response to growth hormone treatment (e.g. diabetes, idiopathic short stature [ISS; except in countries where ISS diagnostic criteria overlap with pediatric GHD criteria in the US], SHOX-deficiency, any chondrodysplasia, other named syndromes).
2. • An arm span to height ratio > 2 SDs below the mean for age and sex to rule out subclinical hypochondroplasia as an etiology for short stature (mesomelia).
3. • A medical or genetic condition that, in the opinion of the Investigator and/or MM, adds unwarranted risk to use of LUM-201.
4. • 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.
5. • Current inflammatory diseases requiring systemic corticosteroid treatment for > 2 consecutive weeks within the last 3 months prior to the Screening Visit. Children requiring inhaled glucocorticoid therapy at a dose of > 400.0 µg/day of inhaled budesonide or equivalents for > 4 consecutive weeks within the last 12 months prior to the Screening Visit.
6. • Use of hormone replacement therapy for any hormone deficiency other than stable primary thyroid hormone deficiency.
7. • Any ECG at the Screening Visit noted to have a clinically significant abnormality, as confirmed by the MM.
8. • The presence of any circumstance likely to prevent successful completion of this trial.
9. • Any subjects suspected of having past or present intracranial tumor growth as confirmed by brain imaging prior to the Screening or Day 1 Visit. Nonspecific abnormalities should be discussed with the MM.
10. • Any subject suspected of having intracranial hypertension (IH) as confirmed by fundoscopy and other assessments. Signs and symptoms of IH should be discussed with the MM.
11. • Any subject with serum alanine transaminase (ALT), aspartate transaminase (AST), or total bilirubin > upper limit of normal (ULN).
12. • Multiple pituitary hormone deficiencies.
13. • Prior treatment with growth factors including, but not limited to, GH, IGF-1, and GH secretagogues. (These may be used for limited time as a diagnostic test.)
14. • Suspicion of absent pituitary function as evidenced by a maximal stimulated GH ≤ 3.0 ng/mL on any prior standard of care GH stimulation test completed within 12 months, or pituitary deficiencies beyond GH.
15. • Malnutrition as determined by the Investigator based on clinical findings, medical history and laboratory findings.
16. • Body weight ≤ 14.0 kg.
17. • BMI < -2 or > +2 SDs for age and sex based on WHO standards.
18. • Birth weight for gestational age < 3rd percentile based on WHO or local standards.
19. • Participation in any therapeutic trial of investigational drug(s) within 6 months prior to the Screening visit.
20. • Known or suspected allergy to LUM-201, placebo, or one of their excipients.
21. • Unwilling to accept randomization assignment.
22. • Treatment with medications known to be moderate or strong inhibitors or strong inducers of cytochrome P450 (CYP) 3A/4.
23. • Treatment with medications known to be predominantly metabolized (< 5% contribution by other isoforms) by either CYP3A/4, 2C8, 2C9, or 2C19.
24. • Treatment with medications known to act as strong inhibitors of P-glycoprotein (P-gp), potent substrates of P-gp, or multidrug and toxin extrusion protein 1.
25. • History of spinal, cranial, or total body irradiation.
26. • History or presence of malignant disease; any evidence of present tumor growth.
27. • Children with psychosocial short stature.
28. • Closed epiphyses.
29. • Attention deficit hyperactivity disorder (ADHD) diagnosis, regardless of treatment.>

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. AHV from Day 1 to Month 12 in the LUM-201 and placebo arms

Secondary endpoints 6

1. • Change from baseline in IGF-1 SDS
2. • Change from baseline in HT-SDS
3. • Change from baseline in IGFBP-3 SDS
4. • IGF-1 SDS
5. • HT SDS
6. • IGFBP-3 SDS

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 3

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Lumos Pharma LLC

Sponsor organisation

Lumos Pharma LLC

Address

2503 South Loop Drive Suite 5100

City

Ames

Postcode

50010-8641

Country

United States

Scientific contact point

Organisation

Lumos Pharma LLC

Contact name

Susan Thornton

Public contact point

Organisation

Lumos Pharma LLC

Contact name

Lumos Pharma

Third parties 1

Locations

5 EU/EEA countries · 17 investigational sites

By country

Investigational sites

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 59 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

1 submissions — initial application plus substantial / non-substantial modifications