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    Summary
    EudraCT Number:2020-000874-92
    Sponsor's Protocol Code Number:LUM-201-01
    National Competent Authority:Poland - Office for Medicinal Products
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2020-10-26
    Trial results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedPoland - Office for Medicinal Products
    A.2EudraCT number2020-000874-92
    A.3Full title of the trial
    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).
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    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).
    A.3.2Name or abbreviated title of the trial where available
    LUM-201-01
    A.4.1Sponsor's protocol code numberLUM-201-01
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT04614337
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorLumos Pharma, Inc.
    B.1.3.4CountryUnited States
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportLumos Pharma
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationLumos Pharma
    B.5.2Functional name of contact pointRegulatory Affairs, Lumos Pharma
    B.5.3 Address:
    B.5.3.1Street Address4200 Marathon Blvd. Suite 200
    B.5.3.2Town/ cityAustin, TX
    B.5.3.3Post code78756
    B.5.3.4CountryUnited States
    B.5.6E-mailjcreager@lumos-pharma.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/17/1882
    D.3 Description of the IMP
    D.3.1Product nameLUM-201
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNIbutamoren mesilate
    D.3.9.1CAS number 159752-10-0
    D.3.9.2Current sponsor codeLUM-201
    D.3.9.3Other descriptive nameIBUTAMOREN MESILATE
    D.3.9.4EV Substance CodeSUB189341
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number9
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.IMP: 2
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/17/1882
    D.3 Description of the IMP
    D.3.1Product nameLUM-201
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNIbutamoren mesilate
    D.3.9.1CAS number 159752-10-0
    D.3.9.2Current sponsor codeLUM-201
    D.3.9.3Other descriptive nameIBUTAMOREN MESILATE
    D.3.9.4EV Substance CodeSUB189341
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number18
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.IMP: 3
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/17/1882
    D.3 Description of the IMP
    D.3.1Product nameLUM-201
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNIbutamoren mesilate
    D.3.9.1CAS number 159752-10-0
    D.3.9.2Current sponsor codeLUM-201
    D.3.9.3Other descriptive nameIBUTAMOREN MESILATE
    D.3.9.4EV Substance CodeSUB189341
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number36
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.IMP: 4
    D.1.2 and D.1.3IMP RoleComparator
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation Yes
    D.2.1.1.1Trade name Norditropin
    D.2.1.1.2Name of the Marketing Authorisation holderNovo Nordisk
    D.2.1.2Country which granted the Marketing AuthorisationDenmark
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.4Pharmaceutical form Injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNrecombinant human growth hormone
    D.3.9.1CAS number 12629-01-5
    D.3.9.3Other descriptive namerecombinant human growth hormone
    D.3.9.4EV Substance CodeSUB10584MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number3.3
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.IMP: 5
    D.1.2 and D.1.3IMP RoleComparator
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation Yes
    D.2.1.1.1Trade name Norditropin
    D.2.1.1.2Name of the Marketing Authorisation holderNovo Nordisk
    D.2.1.2Country which granted the Marketing AuthorisationDenmark
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.4Pharmaceutical form Injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNrecombinant human growth hormone
    D.3.9.1CAS number 12629-01-5
    D.3.9.3Other descriptive namerecombinant human growth hormone
    D.3.9.4EV Substance CodeSUB10584MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number6.7
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Growth hormone deficiency
    E.1.1.1Medical condition in easily understood language
    Growth hormone deficiency
    E.1.1.2Therapeutic area Diseases [C] - Hormonal diseases [C19]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10056438
    E.1.2Term Growth hormone deficiency
    E.1.2System Organ Class 10014698 - Endocrine disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Preliminary clinical validation of predictive enrichment marker (PEM) strategy intended to select subjects likely to respond to therapy with LUM-201 based upon the percentage of subjects defined as PEM test positive (GH ≥ 5 ng/mL and IGF-1 > 30 ng/mL) who have a positive growth response (“responders” defined as ≥ 6.85 cm/yr annualized height velocity (AHV) from Baseline (Day 1) to Month 6)
    E.2.2Secondary objectives of the trial
    Select a pediatric dose of LUM-201 for use in future studies including Phase 3 trial(s) based on comparison of 6-month, AHVs achieved by daily rhGH and LUM-201 treatment in subjects with pediatric growth hormone deficiency selected by predictive enrichment markers (PEMs)
    Assess the safety and tolerability of oral LUM-201
    Assess the repeatability of the LUM-201 strategy in the classification of subjects as PEM positive or PEM negative
    Assess additional clinical parameters of efficacy including change in height standard deviation score (HT-SDS) from Day 1 to Month 6, change in weight and weight SDS from Day 1 to Month 6, change in body mass index and BMI SDS from Day 1 to Month 6, change in Bone Age
    Further explore pharmacokinetics of LUM-201 based upon plasma LUM-201 concentrations
    Determine short- and long-term pharmacodynamic (GH, IGF-1) responses to daily LUM-201
    Characterize variance in AHVs of rhGH and LUM-201 to enable sample size estimates for future trials
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Have an established diagnosis of PGHD made prior to screening for entry into this study. This diagnosis is to be made in standard of care fashion incorporating biochemical, radiographic and auxological data. Specific inclusion criteria listed in this section for inclusion into this study protocol which are also used to diagnose PGHD in general are not intended to replace, supplant, or be an all-inclusive substitute for a standard of care diagnosis. Eligible subjects must be naïve-to-treatment and be prepubertal.
    2. The diagnostic work up must include a maximal GH response < 10 ng/mL from two prior GH stimulation tests within the past 12 months (eligible tests include: glucagon, insulin, arginine, clonidine, and L-DOPA).
    3. At Screening Visit 1 or in the prior 12 months have a morning or random cortisol ≥ 7 µg /dL (193 nmol/L). Subjects with morning or random cortisol response < 7 µg /dL require a stimulated cortisol test (ACTH, insulin, glucagon) to be eligible for the study. Stimulated cortisol response should be ≥ 14 µg /dL (386 nmol/L).
    4. Be willing to provide written informed consent (and assent where applicable) to participate in this trial.
    5. At Screening Visit 1, be age ≥ 3.0 years and age ≤ 11.0 years for girls and ≤ 12.0 years for boys.
    6. Have HT-SDS ≤ -2.0, or the combination of a HT-SDS 2 SD below mean parental HT-SDS and a 6-month height velocity < 25th percentile for age and gender.
    7. Have a height-arm span difference < 7.0 cm.
    8. Have a bone age examination at Screening or within the 12 months prior to Screening that, in the interpretation of the Investigator, is delayed by ≥ 6 months with respect to chronological age for subjects ≤ 9.0 years old and by at least 1.0 year for subjects older than 9.0 years of age.
    9. Have prepubertal status as evidenced by Tanner Stage I breast development in girls and testicular volume < 4.0 mL in boys.
    10. In girls, have genetic testing results to rule out Turner syndrome. If SHOX genetic testing results are available, they need to be negative.
    11. Have normal thyroid function. Subjects diagnosed with hypothyroidism must have documented successful treatment for at least 30 days prior to Day 1.
    12. Additional inclusion criteria for participation in the Treatment portion of the study are baseline IGF-1 concentration > 30 ng/mL and a peak GH response of ≥ 5 ng/mL from the first of the two Screening portion PEM tests.
    E.4Principal exclusion criteria
    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 exogenous growth factor treatment. (Examples: diabetes, named syndromes).
    2. 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.
    3. 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).
    4. The presence of any circumstance likely to prevent successful completion of this trial.
    5. Evidence or history of an intracranial mass (e.g., pituitary tumor, craniopharyngioma).
    6. Prior treatment with growth factors including, but not limited to, GH, IGF-1, and GH secretagogues. (These may be used for limited times as a diagnostic test.)
    7. 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. Note: If a maximal stimulated GH response is > 3 ng/mL on one of the prior standard of care GH stimulation tests, subject is potentially eligible for participation in the study.
    8. Malnutrition as evidenced by medical history or a body weight < 5th percentile for current height.
    9. BMI > 95th percentile.
    10. Gestational age-adjusted birth weight < 5th percentile (small for gestational age), as noted in Appendix 1.
    11. Participation in any therapeutic trial of investigational drug(s) within the prior 6 months.
    12. Known or suspected allergy to LUM-201, rhGH, or one of their excipients.
    13. Unwilling to accept randomization assignment.
    14. Treatment with medications known to be moderate or strong inhibitors or strong inducers of CYP3A/4 as noted in Appendix 2.
    15. Treatment with medications known to be predominantly metabolized (< 5% contribution by other isoforms) by either CYP3A/4, CYP2C8, CYP2C9 or CYP2C19 as noted in Appendix 2.
    16. Treatment with methylphenidate or medications known to act as strong inhibitors of P-gp or potent substrates of P-gp or MATE1 as noted in Appendix 2.
    17. History of spinal, cranial, or total body irradiation.
    18. Recent commencement (within three months of the Screening Visit 1) of non-stimulant therapy to treat ADHD. MM should be contacted prior to screening of any potential subjects diagnosed with ADHD.
    E.5 End points
    E.5.1Primary end point(s)
    Clinical Outcome Measurements:
    • AHV from Baseline (Day 1) to Month 6
    • Change in HT-SDS from Day 1 to Month 6
    • Change in height velocity standard deviation score (HV-SDS)
    • Change in weight and weight SDS from Day 1 to Month 6
    • Change in body mass index (BMI) and BMI SDS from Day 1 to Month 6
    • Change in Bone Age
    PK/PD Endpoints
    • Plasma LUM-201 concentrations (in LUM-201-treated subjects)
    • Serum GH (LUM-201 subjects only) and IGF-1
    Safety Endpoints: AEs and SAEs, routine laboratory tests (hematology, chemistry panel including liver enzymes and urinalysis), HbA1c, blood glucose, free thyroxine (T4), triiodothyronine (T3), thyroid-stimulating hormone (TSH), lipids, physical examinations, interval history, vital signs, and ECGs.
    E.5.1.1Timepoint(s) of evaluation of this end point
    End of study
    E.5.2Secondary end point(s)
    Assessment of repeatability of the PEM classification (positive / negative) in subjects undergoing paired LUM-201 stimulation tests and paired IGF-1 baseline blood draws.
    Selection of a pediatric dose of LUM-201 for use in future studies including Phase 3 trial(s) based on comparison of 6-month, AHVs achieved by daily rhGH and LUM-201 treatment in subjects with PGHD selected by PEMs
    E.5.2.1Timepoint(s) of evaluation of this end point
    End of study
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group Yes
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) Yes
    E.8.2.2Placebo No
    E.8.2.3Other Yes
    E.8.2.3.1Comparator description
    3 different strenghts of LUM-201 compared with rhGH
    E.8.2.4Number of treatment arms in the trial4
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned9
    E.8.5The trial involves multiple Member States No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Australia
    Israel
    New Zealand
    Russian Federation
    Ukraine
    United States
    E.8.7Trial has a data monitoring committee Yes
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months0
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 80
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 80
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception No
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others Yes
    F.3.3.7.1Details of other specific vulnerable populations
    Minors between 3 and 11 years of age.
    F.4 Planned number of subjects to be included
    F.4.1In the member state50
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 50
    F.4.2.2In the whole clinical trial 80
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    Subjects completing the 6-month study will be eligible for inclusion in the Long-Term Extension Study. Subjects not completing the 6-months study will be treated at the discretion of the investigator / treating physician.
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2021-03-22
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-12-03
    P. End of Trial
    P.End of Trial StatusOngoing
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