Overview
Sponsor-declared trial summary
Phase
Therapeutic exploratory (Phase II)
Status
Ongoing, recruiting
Participants planned
69
Countries
1
Sites
7
Idiopathic Growth Hormone Deficiency
Assess the long-term safety of LUM-201 administration in children with idiopathic growth hormone deficiency (GHD)
Key facts
- Sponsor
- Lumos Pharma Inc.
- Participant type
- Pediatric, Patients
- Age range
- 0-17 years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Hormonal diseases [C19]
- Trial duration
- 28 Dec 2023 → ongoing
- Decision date (initial)
- 2023-10-02
- Transition trial
- No
- Low-intervention
- No
- Rare-disease indication
- Yes
- Vulnerable population
- Yes
- Funding sources
- Lumos Pharma, Inc. 4200 Marathon Blvd. Suite 200 Austin, Texas 78756, USA
External identifiers
- EU CT number
- 2023-504020-25-00
- ClinicalTrials.gov
- NCT05796440
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Safety
Assess the long-term safety of LUM-201 administration in children with idiopathic growth hormone deficiency (GHD)
Secondary objectives 2
- Assess pharmacodynamic (PD) markers of LUM-201 treatment
- Assess long-term growth outcomes in response to LUM-201 treatment
Conditions and MedDRA coding
Idiopathic Growth Hormone Deficiency
Regulatory references
- EMA paediatric investigation plan (PIP)
- EMEA-003032-PIP01-21
- Plan to share IPD
- No
| EU CT number | Title | Sponsor |
|---|---|---|
| 2022-001695-34 | An Open-Label, Multicenter, Phase 2 Study to Evaluate Growth and Safety of LUM-201 Following 12 Months of Daily rhGH Treatment in Children with Idiopathic Growth Hormone Deficiency who have Previously Completed the LUM-201-01 Trial, Otwarte, wieloośrodkowe badanie fazy 2 oceniające wzrost i bezpieczeństwo stosowania LUM-201 po 12 miesiącach codziennego leczenia rhGH u dzieci z idiopatycznym niedoborem hormonu wzrostu, które wcześniej ukończyły badanie LUM-201-01 | |
| 2020-000874-92 | A Multicenter, 6-Month, Randomized, Open-Label, Active Control, Parallel Arm, Phase 2b Study of Daily Oral LUM-201 in Naïve-to-Treatment, Prepubertal Children with Growth Hormone Deficiency (GHD), Wieloośrodkowe, 6-miesięczne, Randomizowane, Otwarte, Kontrolowane, Równoramienne Badanie Fazy 2b z Podawanym Codziennie Doustnie LUM-201 Dzieciom Wcześniej Nieleczonym w Okresie Przedpokwitaniowym z Niedoborem Hormonu Wzrostu (GHD)., Wieloośrodkowe, 6-miesięczne, Randomizowane, Otwarte, Kontrolowane, Równoramienne Badanie Fazy 2b z Podawanym Codziennie Doustnie LUM-201 Dzieciom Wcześniej Nieleczonym w Okresie Przedpokwitaniowym z Niedoborem Hormonu Wzrostu (GHD)., Wieloośrodkowe, 24-miesięczne, Randomizowane, Otwarte, Kontrolowane, Równoramienne Badanie Fazy 2 z Podawanym Codziennie Doustnie LUM-201 Dzieciom Wcześniej Nieleczonym w Okresie Przedpokwitaniowym z Idiopatycznym Niedoborem Hormonu Wzrostu (GHD)., Wieloośrodkowe, 12-miesięczne, Randomizowane, Otwarte, Kontrolowane, Równoramienne Badanie Fazy 2 z Podawanym Codziennie Doustnie LUM-201 Dzieciom Wcześniej Nieleczonym w Okresie Przedpokwitaniowym z Niedoborem Hormonu Wzrostu (GHD)., Wieloośrodkowe, 6-miesięczne, Randomizowane, Otwarte, Kontrolowane, Równoramienne Badanie Fazy 2b z Podawanym Codziennie Doustnie LUM-201 Dzieciom Wcześniej Nieleczonym w Okresie Przedpokwitaniowym z Niedoborem Hormonu Wzrostu (GHD). |
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 4
- 1. Parent/caregiver must sign the informed consent, and the subject must sign the assent, as applicable.
- 2. 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. Specific enrollment criteria for subjects in each LUM-201 trial are listed below. Subjects transitioning from the LUM-201-01 trial will be considered eligible for enrollment pending successful participation in the LUM-201-01 trial, having met an AHV of ≥ 6.7 cm/year after 12 months of LUM-201 therapy. Subjects transitioning from the LUM-201-04 trial will be considered eligible for enrollment pending successful completion of 12 months of therapy with rhGH on the LUM-201-01 trial, followed by 12 months of treatment with LUM-201 in the LUM-201-04 trial and having met an AHV of ≥ 80% of first year growth on rhGH.
- 3. Subjects who are sexually active must use an acceptable form of contraception.
- 4. Is eligible for a Day 1 visit as confirmed by the principal investigator (PI).
Exclusion criteria 2
- 1. Has a medical or genetic condition that, in the opinion of the PI and/or MMs, adds unwarranted risk to use of LUM-201.
- 2. 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 (see Appendix 1 of protocol for list of example medications). 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.
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 1
- 1. Occurrence of AEs, SAEs; laboratory results, and physical examination findings.
Secondary endpoints 7
- 1. GH values: actual and change over time (every 6 months).
- 2. IGF-1 values: actual and change over time (every 6 months).
- 3. IGFBP-3 values: actual and change over time (every 6 months).
- 4. Change in height standard deviation score (HT-SDS) every 6 months.
- 5. Change in weight and weight SDS every 6 months.
- 6. Change in body mass index (BMI) and BMI SDS every 6 months.
- 7. Change in Bone Age (BA) compared to Chronological Age (CA) (e.g., BA/CA) ratio assessed annually.
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 1
PRD10426714 · Product
- Active substance
- Ibutamoren Mesilate
- Pharmaceutical form
- TABLET
- Route of administration
- ORAL
- Max daily dose
- 1.6 mg/kg milligram(s)/kilogram
- Max total dose
- 1.6 mg/kg milligram(s)/kilogram
- Max treatment duration
- 36 Month(s)
- Authorisation status
- Not Authorised
- MA holder
- LUMOS PHARMA, INC
- Paediatric formulation
- No
- Orphan designation
- Yes
- Orphan designation number
- EU/3/17/1882
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Lumos Pharma Inc.
- Sponsor organisation
- Lumos Pharma Inc.
- Address
- 2503 South Loop Drive Suite 5100
- City
- Ames
- Postcode
- 50010-8641
- Country
- United States
Scientific contact point
- Organisation
- Lumos Pharma Inc.
- Contact name
- Julianne Craeger
Public contact point
- Organisation
- Lumos Pharma Inc.
- Contact name
- Julianne Craeger
Locations
1 EU/EEA country · 7 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Poland | Ongoing, recruiting | 21 | 7 |
| Rest of world
United States, Israel, New Zealand, Australia
|
— | 48 | — |
Investigational sites
Wroclaw Medical University
Klinika Endokrynologii i Diabetologii Wieku Rozwojowego UM, Ul. Tytusa Chalubinskiego 5, 50-368, Wroclaw
Uniwersytecki Dzieciecy Szpital Kliniczny Im. L. Zamenhofa W Bialymstoku samodzielny publiczny zakład opieki zdrowotnej
Klinika Pediatrii, Endokrynologii, Diabetologii z Pododdziałem Kardiologii, Ul. Jerzego Waszyngtona 17, 15-269, Bialystok
Uniwersyteckie Centrum Kliniczne
Klinika Pediatrii, Diabetologii i Endokrynologii, Ul. Debinki 7, 80-211, Gdansk
Instytut Pomnik Centrum Zdrowia Dziecka
Klinika Endokrynologii i Diabetologii, Aleja Dzieci Polskich 20, 04-730, Warsaw
Uniwersyteckie Centrum Kliniczne Warszawskiego Uniwersytetu Medycznego
SP Dziecięcy Szpital Kliniczny w Warszawie, Klinika Pediatrii i Endokrynologii, Ul. Zwirki I Wigury 63a, 02-091, Warsaw
Sonomed Sp. z o.o.
Sonomed - Centrum Medyczne, Ul. Ks. Bp. Wladyslawa Bandurskiego 98/u12, 71-685, Szczecin
Instytut Centrum Zdrowia Matki Polki
Klinika Endokrynologii i Chorób Metabolicznych, Ul. Rzgowska 281/289, 93-338, Lodz
Country notifications
Trial-start, recruitment-start, end and early-termination notifications submitted per Member State
| Country | Trial start | Trial end | Recruitment start | Recruitment end | Early termination |
|---|---|---|---|---|---|
| Poland | 2023-12-28 | 2023-12-28 |
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 6 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Recruitment arrangements (for publication) | K1_Recruitment arrangements | 1 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements_v3_Polish | 3 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Assent_13-17 | 1.6 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Assent_7-12 | 1.5 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_DP ICF_v1.1_22Aug2023_public | 1.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Parental ICF_redacted | 1.4 |
Application history
5 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2023-06-05 | Poland | Acceptable with conditions 2023-09-25
|
2023-10-02 |
| 2 | SUBSTANTIAL MODIFICATION | SM-1 | 2023-10-03 | Poland | Acceptable 2023-12-04
|
2023-12-06 |
| 3 | SUBSTANTIAL MODIFICATION | SM-2 | 2023-12-07 | Poland | Acceptable | 2024-03-20 |
| 4 | NON SUBSTANTIAL MODIFICATION | NSM-1 | 2024-08-22 | Poland | Acceptable | 2024-08-22 |
| 5 | NON SUBSTANTIAL MODIFICATION | NSM-3 | 2025-08-20 | Poland | Acceptable | 2025-08-20 |