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    Clinical Trial Results:
    Title: A Phase 2, Open-Label 20-Week Study to Evaluate the Safety and Efficacy of Setmelanotide in Subjects with Hypothalamic Obesity. Trial design: This was a Phase 2, multicentre, open-label, proof of concept study designed to assess the effect of setmelanotide on weight in individuals affected by hypothalamic obesity (HO). It was planned to enrol approximately 15 patients with HO, aged 6 to 40 years, across 3-5 clinical sites in the US. HO is caused by hypothalamic insults (eg, tumours, tumour resections, radiotherapy treatment) that may result in defective leptin signalling (the MC3/4 receptor pathway). The study consisted of a 2-8 week screening period and a 16-week treatment period. After enrolment, patients entered the screening period when they completed a daily hunger questionnaire. During the treatment period, all patients initiated treatment with subcutaneous (SC) setmelanotide and the dose was to be escalated to a final dose of 3.0 mg once daily (QD) (the therapeutic dose). Patients were to continue dosing at 3.0 mg QD and return to the clinic every 4 weeks (at Visits 3-5) to complete all study assessments. Note, doses of <3.0 mg QD could be administered if needed due to an adverse event or other safety or tolerability concern. After 16 weeks, at Visit 6, the patient received the last setmelanotide injection and participation in the study concluded in one of the following 2 ways: • Completed Visit 6 and enrolled in a separate extension study, Rhythm Study RM-493-022. • Decided not to participate in the extension study and proceeded to the End-of-Study (EOS) visit (Visit 7) at Week 20 for a final safety review. The median duration of treatment was 16.14 weeks (range: 7.43 to 22.57 weeks). The primary endpoint was defined as the proportion of patients who achieved at least 5% body mass index (BMI) reduction from baseline at ~16 weeks of treatment with setmelanotide.

    Summary
    EudraCT number
    2022-004107-32
    Trial protocol
    Outside EU/EEA  
    Global end of trial date
    28 Jun 2022

    Results information
    Results version number
    v1(current)
    This version publication date
    27 Apr 2023
    First version publication date
    27 Apr 2023
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    RM-493-030
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT04725240
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    Rhythm Pharmaceuticals, Inc,
    Sponsor organisation address
    222 Berkeley Street, 12th Floor, Boston, United States, MA 02116
    Public contact
    Clinical Trial Information Desk, Rhythm Pharmaceuticals, Inc., +1 857-264-4280, clinicaltrials@rhythmtx.com
    Scientific contact
    Clinical Trial Information Desk, Rhythm Pharmaceuticals, Inc., +1 857-264-4280, clinicaltrials@rhythmtx.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    No
    Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?
    Yes
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    31 Aug 2022
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    28 Jun 2022
    Global end of trial reached?
    Yes
    Global end of trial date
    28 Jun 2022
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The primary objective was to evaluate the change in body weight in response to setmelanotide administered subcutaneously (SC) daily in patients with HO.
    Protection of trial subjects
    The Institutional Review Board (IRB) reviewed all appropriate study documentation in order to safeguard the rights, safety, and well-being of the patients. The study was only conducted at sites where IRB/IEC approval had been obtained. This study was conducted in accordance with: • Consensus ethics principles derived from international ethics guidelines, including the Declaration of Helsinki and Council for International Organizations of Medical Sciences International Ethical Guidelines • The International Council for Harmonisation (ICH) Good Clinical Practices (GCP) Guideline [E6] • Applicable laws and regulatory requirements. After the study had been fully explained, written informed consent was obtained from either the patient or his/her guardian or legal representative prior to study participation. The method of obtaining and documenting the informed consent and the contents of the consent complied with ICH-GCP and all applicable regulatory requirement(s).
    Background therapy
    Any medication or vaccine (including over-the-counter or prescription medicines, vitamins, and/or herbal supplements) received at the time of enrolment or received during the study had to be recorded. The Medical Monitor was to be contacted with any questions regarding concomitant or prior therapy. However, patients were not permitted to enter the study if they have had weight gain >5% during the previous 3 months or >2% weight loss during the prior 3 months. Dietary and/or exercise regimens, with or without the use of medications, supplements or herbal treatments associated with weight loss (eg, orlistat, lorcaserin, phentermine, topiramate, naltrexone, bupropion, GLP1 receptor agonists, etc.) were allowed if: • the regimen and/or dose had been stable for at least 3 months prior to randomisation • the patient had not experienced weight loss ≥2% during the previous 3 months, AND • the patient intended to keep the regimen and/or dose stable throughout the course of the study. GLP 1 receptor agonists could be used up to the dose approved for the treatment of diabetes mellitus (eg, liraglutide up to a daily dose of 1.8 mg) as long as (1) it was not being prescribed for the treatment of obesity, (2) the dose had been stable for at least 3 months prior to enrolment, (3) the patient had not experienced >3% weight loss during the previous 3 months, AND (4) the patient intended to keep the dose stable throughout the course of the study. Other medications that could have caused weight loss were allowed as long as the patient (1) had used a stable dose for at least 3 months prior to enrolment, (2) had not lost weight during the previous 3 months, and (3) intended to keep the dose stable through the course of the study. All concomitant medications had to be kept at a stable dose throughout the course of the study, unless a dose change was necessary to treat an AE.
    Evidence for comparator
    Not applicable.
    Actual start date of recruitment
    06 Jun 2021
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    United States: 18
    Worldwide total number of subjects
    18
    EEA total number of subjects
    0
    Number of subjects enrolled per age group
    In utero
    0
    Preterm newborn - gestational age < 37 wk
    0
    Newborns (0-27 days)
    0
    Infants and toddlers (28 days-23 months)
    0
    Children (2-11 years)
    5
    Adolescents (12-17 years)
    8
    Adults (18-64 years)
    5
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    This study recruited 18 patients with HO in 5 clinical sites in the United States with the first patient enrolled on 06 June 2021 (first dosed 12 July 2021) and the last patient visit on 28 June 2022. Patients were included if they had documented evidence of HO.

    Pre-assignment
    Screening details
    Screening assessments included medical history, physical exam, comprehensive skin examination, laboratory tests, blood pressure, hunger scale, body composition, Columbia-Suicide Severity Rating Scale (C-SSRS) form, and energy expenditure evaluation.

    Period 1
    Period 1 title
    Baseline (overall period)
    Is this the baseline period?
    Yes
    Allocation method
    Not applicable
    Blinding used
    Not blinded
    Blinding implementation details
    This was an open-label, proof-of-concept study.

    Arms
    Arm title
    All patients
    Arm description
    All patients with HO included in the study.
    Arm type
    Experimental

    Investigational medicinal product name
    Setmelanotide
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Setmelanotide was administered once daily (QD) via SC injection. The therapeutic dose was 3.0 mg QD, which was achieved after dose titration. No dose >3.0 mg QD was to be administered. However, doses <3.0 mg QD could be administered, after consultation with the Sponsor, if necessary due to an AE or other safety or tolerability concern. The standard dose escalation was: Patients aged 6 to <16 years: 1.0 mg in Weeks 1-2, 2.0 mg in Weeks 3-4, and 3.0 mg in Weeks 5-16. Patients aged >=16 years: 2.0 mg in Weeks 1-2, and 3.0 mg in Weeks 3-16.

    Number of subjects in period 1
    All patients
    Started
    18
    Completed
    16
    Not completed
    2
         Adverse event, non-fatal
    2

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    All patients
    Reporting group description
    All patients with HO included in the study.

    Reporting group values
    All patients Total
    Number of subjects
    18 18
    Age categorical
    Units: Subjects
        < 18
    13 13
        >= 18
    5 5
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    15.0 ± 5.30 -
    Gender categorical
    Units: Subjects
        Female
    7 7
        Male
    11 11
    Weight at baseline
    The most recent weight measurement prior to the first administration of study drug.
    Units: kilogram(s)
        arithmetic mean (standard deviation)
    102.76 ± 30.120 -
    BMI at baseline
    The most recent body mass index (BMI) measurement prior to the first administration of study drug.
    Units: kg/m2
        arithmetic mean (standard deviation)
    37.95 ± 6.532 -
    Waist circumference at baseline
    The most recent waist circumference measurement prior to the first administration of study drug.
    Units: centimetre
        arithmetic mean (standard deviation)
    114.07 ± 16.354 -

    End points

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    End points reporting groups
    Reporting group title
    All patients
    Reporting group description
    All patients with HO included in the study.

    Subject analysis set title
    Patients aged ≥6 to <18 years
    Subject analysis set type
    Full analysis
    Subject analysis set description
    Patients aged ≥6 to <18 years

    Subject analysis set title
    Patients aged ≥18 years
    Subject analysis set type
    Full analysis
    Subject analysis set description
    Patients aged ≥18 years

    Subject analysis set title
    Patients aged <12 years
    Subject analysis set type
    Full analysis
    Subject analysis set description
    Patients aged <12 years

    Subject analysis set title
    Patients aged ≥12 years
    Subject analysis set type
    Full analysis
    Subject analysis set description
    Patients aged ≥12 years

    Primary: Patients with ≥5% BMI reduction from baseline

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    End point title
    Patients with ≥5% BMI reduction from baseline [1]
    End point description
    The proportion of patients with HO who achieved at least 5% reduction from baseline in BMI after 16 weekss of treatment with setmelanotide. The summary of the primary endpoint and the associated 2-sided 90% Clopper-Pearson confidence interval is provided. A p-value of <0.0001 was calculated from a one-sided exact binomial test comparing to the null hypothesis proportion of 0.05 (which comes from a historical control rate of 5%).
    End point type
    Primary
    End point timeframe
    From baseline to Week 16.
    Notes
    [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: No statistical analysis of the primary endpoint was planned or performed because this was an open-label study with no comparator group. A comparison with a historical control group is described above.
    End point values
    All patients
    Number of subjects analysed
    18
    Units: percent
        number (confidence interval 90%)
    88.9 (69.0 to 98.0)
    No statistical analyses for this end point

    Secondary: Composite reduction in BMI Z-score and change in body weight

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    End point title
    Composite reduction in BMI Z-score and change in body weight
    End point description
    Composite proportion of patients aged ≥6 to <18 years with ≥0.2 reduction of BMI Z-score and patients aged ≥18 years with ≥5% reduction of body weight from baseline after 16 weeks of setmelanotide. The summary of the endpoint and the associated 2-sided 90% Clopper-Pearson confidence interval is provided. A p-value of <0.0001 was calculated from a one-sided exact binomial test comparing to the null hypothesis proportion of 0.05 (which comes from a historical control rate of 5%).
    End point type
    Secondary
    End point timeframe
    From baseline to Week 16.
    End point values
    All patients
    Number of subjects analysed
    18
    Units: percent
        number (confidence interval 90%)
    88.9 (69.0 to 98.0)
    No statistical analyses for this end point

    Secondary: Reduction in BMI Z-score in patients aged ≥6 to <18 years

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    End point title
    Reduction in BMI Z-score in patients aged ≥6 to <18 years
    End point description
    The proportion of patients aged ≥6 to <18 years with HO with ≥0.2 reduction of BMI Z-score from baseline at 16 weeks of treatment with setmelanotide. The summary of the endpoint and the associated 2-sided 90% Clopper-Pearson confidence interval is provided. A p-value of <0.0001 was calculated from a one-sided exact binomial test comparing to the null hypothesis proportion of 0.05 (which comes from a historical control rate of 5%).
    End point type
    Secondary
    End point timeframe
    From baseline to Week 16.
    End point values
    Patients aged ≥6 to <18 years
    Number of subjects analysed
    13
    Units: percent
        number (confidence interval 90%)
    92.3 (68.4 to 99.6)
    No statistical analyses for this end point

    Secondary: Reduction in body weight in patients aged ≥18 years

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    End point title
    Reduction in body weight in patients aged ≥18 years
    End point description
    The proportion of patients aged ≥18 years with HO with ≥5% reduction of body weight from baseline at 16 weeks of treatment with setmelanotide. The summary of the endpoint and the associated 2-sided 90% Clopper-Pearson confidence interval is provided. A p-value of <0.0001 was calculated from a one-sided exact binomial test comparing to the null hypothesis proportion of 0.05 (which comes from a historical control rate of 5%).
    End point type
    Secondary
    End point timeframe
    From baseline to Week 16.
    End point values
    Patients aged ≥18 years
    Number of subjects analysed
    5
    Units: percent
        number (confidence interval 90%)
    80.0 (34.3 to 99.0)
    No statistical analyses for this end point

    Secondary: Change in waist circumference - ≥18 years of age

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    End point title
    Change in waist circumference - ≥18 years of age
    End point description
    The change in waist circumference from baseline in patients aged ≥18 years at 16 weeks of treatment with setmelanotide. The summary of the primary endpoint and the associated 2-sided 90% Clopper-Pearson confidence interval is provided.
    End point type
    Secondary
    End point timeframe
    From baseline to Week 16.
    End point values
    Patients aged ≥18 years
    Number of subjects analysed
    4
    Units: percent
        arithmetic mean (confidence interval 90%)
    -8.13 (-11.832 to -4.419)
    No statistical analyses for this end point

    Secondary: Change in waist circumference - <18 years of age

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    End point title
    Change in waist circumference - <18 years of age
    End point description
    The change in waist circumference from baseline in patients aged <18 years at 16 weeks of treatment with setmelanotide. The summary of the primary endpoint and the associated 2-sided 90% Clopper-Pearson confidence interval is provided.
    End point type
    Secondary
    End point timeframe
    From baseline to Week 16.
    End point values
    Patients aged ≥6 to <18 years
    Number of subjects analysed
    12
    Units: percent
        arithmetic mean (confidence interval 90%)
    -11.14 (-14.050 to -8.233)
    No statistical analyses for this end point

    Secondary: Change in hunger score - <12 years of age

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    End point title
    Change in hunger score - <12 years of age
    End point description
    The change in daily hunger score from baseline in patients aged <12 years at 16 weeks of treatment with setmelanotide. The summary of the primary endpoint and the associated 2-sided 90% Clopper-Pearson confidence interval is provided.
    End point type
    Secondary
    End point timeframe
    From baseline to Week 16.
    End point values
    Patients aged <12 years
    Number of subjects analysed
    5
    Units: percent
        arithmetic mean (confidence interval 90%)
    -62.02 (-84.741 to -39.307)
    No statistical analyses for this end point

    Secondary: Change in hunger score - ≥12 years of age

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    End point title
    Change in hunger score - ≥12 years of age
    End point description
    The change in daily hunger score from baseline in patients aged ≥12 years at 16 weeks of treatment with setmelanotide. The summary of the primary endpoint and the associated 2-sided 90% Clopper-Pearson confidence interval is provided.
    End point type
    Secondary
    End point timeframe
    From baseline to Week 16.
    End point values
    Patients aged ≥12 years
    Number of subjects analysed
    11
    Units: percent
        arithmetic mean (confidence interval 90%)
    -65.26 (-84.512 to -46.017)
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    From baseline to Week 16 of treatment.
    Assessment type
    Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    24.0
    Reporting groups
    Reporting group title
    All patients
    Reporting group description
    All patients with HO included in the study.

    Serious adverse events
    All patients
    Total subjects affected by serious adverse events
         subjects affected / exposed
    1 / 18 (5.56%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    0
    Infections and infestations
    Clostridium difficile colitis
         subjects affected / exposed
    1 / 18 (5.56%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    All patients
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    18 / 18 (100.00%)
    General disorders and administration site conditions
    Fatigue
         subjects affected / exposed
    3 / 18 (16.67%)
         occurrences all number
    4
    Injection site pain
         subjects affected / exposed
    3 / 18 (16.67%)
         occurrences all number
    3
    Injection site pruritus
         subjects affected / exposed
    2 / 18 (11.11%)
         occurrences all number
    2
    Pain
         subjects affected / exposed
    2 / 18 (11.11%)
         occurrences all number
    2
    Pyrexia
         subjects affected / exposed
    2 / 18 (11.11%)
         occurrences all number
    2
    Injection site erythema
         subjects affected / exposed
    1 / 18 (5.56%)
         occurrences all number
    1
    Injection site induration
         subjects affected / exposed
    1 / 18 (5.56%)
         occurrences all number
    1
    Injection site swelling
         subjects affected / exposed
    1 / 18 (5.56%)
         occurrences all number
    1
    Reproductive system and breast disorders
    Erection increased
         subjects affected / exposed [1]
    3 / 11 (27.27%)
         occurrences all number
    3
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    1 / 18 (5.56%)
         occurrences all number
    1
    Epistaxis
         subjects affected / exposed
    1 / 18 (5.56%)
         occurrences all number
    1
    Nasal congestion
         subjects affected / exposed
    1 / 18 (5.56%)
         occurrences all number
    1
    Psychiatric disorders
    Conversion disorder
         subjects affected / exposed
    1 / 18 (5.56%)
         occurrences all number
    1
    Investigations
    Alanine aminotransferase increased
         subjects affected / exposed
    1 / 18 (5.56%)
         occurrences all number
    1
    Blood creatine phosphokinase increased
         subjects affected / exposed
    1 / 18 (5.56%)
         occurrences all number
    1
    Blood uric acid increased
         subjects affected / exposed
    1 / 18 (5.56%)
         occurrences all number
    1
    Hepatic enzyme increased
         subjects affected / exposed
    1 / 18 (5.56%)
         occurrences all number
    2
    Weight decreased
         subjects affected / exposed
    1 / 18 (5.56%)
         occurrences all number
    1
    Injury, poisoning and procedural complications
    Contusion
         subjects affected / exposed
    2 / 18 (11.11%)
         occurrences all number
    2
    Radius fracture
         subjects affected / exposed
    1 / 18 (5.56%)
         occurrences all number
    1
    Congenital, familial and genetic disorders
    Type V hyperlipidaemia
         subjects affected / exposed
    1 / 18 (5.56%)
         occurrences all number
    1
    Nervous system disorders
    Headache
         subjects affected / exposed
    2 / 18 (11.11%)
         occurrences all number
    2
    Balance disorder
         subjects affected / exposed
    1 / 18 (5.56%)
         occurrences all number
    1
    Seizure
         subjects affected / exposed
    1 / 18 (5.56%)
         occurrences all number
    1
    Speech disorder
         subjects affected / exposed
    1 / 18 (5.56%)
         occurrences all number
    1
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    1 / 18 (5.56%)
         occurrences all number
    1
    Gastrointestinal disorders
    Nausea
         subjects affected / exposed
    11 / 18 (61.11%)
         occurrences all number
    15
    Vomiting
         subjects affected / exposed
    6 / 18 (33.33%)
         occurrences all number
    8
    Diarrhoea
         subjects affected / exposed
    4 / 18 (22.22%)
         occurrences all number
    4
    Abdominal pain
         subjects affected / exposed
    3 / 18 (16.67%)
         occurrences all number
    3
    Abdominal pain upper
         subjects affected / exposed
    1 / 18 (5.56%)
         occurrences all number
    2
    Gastritis
         subjects affected / exposed
    1 / 18 (5.56%)
         occurrences all number
    1
    Portal hypertensive gastropathy
         subjects affected / exposed
    1 / 18 (5.56%)
         occurrences all number
    1
    Hepatobiliary disorders
    Hypertransaminasaemia
         subjects affected / exposed
    2 / 18 (11.11%)
         occurrences all number
    2
    Hepatic steatosis
         subjects affected / exposed
    1 / 18 (5.56%)
         occurrences all number
    1
    Skin and subcutaneous tissue disorders
    Skin hyperpigmentation
         subjects affected / exposed
    6 / 18 (33.33%)
         occurrences all number
    6
    Acne
         subjects affected / exposed
    1 / 18 (5.56%)
         occurrences all number
    1
    Rash
         subjects affected / exposed
    1 / 18 (5.56%)
         occurrences all number
    1
    Rash erythematous
         subjects affected / exposed
    1 / 18 (5.56%)
         occurrences all number
    1
    Musculoskeletal and connective tissue disorders
    Back pain
         subjects affected / exposed
    2 / 18 (11.11%)
         occurrences all number
    2
    Myalgia
         subjects affected / exposed
    1 / 18 (5.56%)
         occurrences all number
    1
    Pain in extremity
         subjects affected / exposed
    1 / 18 (5.56%)
         occurrences all number
    1
    Scoliosis
         subjects affected / exposed
    1 / 18 (5.56%)
         occurrences all number
    1
    Infections and infestations
    COVID-19
         subjects affected / exposed
    4 / 18 (22.22%)
         occurrences all number
    5
    Upper respiratory tract infection
         subjects affected / exposed
    2 / 18 (11.11%)
         occurrences all number
    2
    Administration site cellulitis
         subjects affected / exposed
    1 / 18 (5.56%)
         occurrences all number
    1
    Gastroenteritis
         subjects affected / exposed
    1 / 18 (5.56%)
         occurrences all number
    1
    Sinusitis
         subjects affected / exposed
    1 / 18 (5.56%)
         occurrences all number
    1
    Staphylococcal abscess
         subjects affected / exposed
    1 / 18 (5.56%)
         occurrences all number
    1
    Metabolism and nutrition disorders
    Dehydration
         subjects affected / exposed
    1 / 18 (5.56%)
         occurrences all number
    1
    Notes
    [1] - The number of subjects exposed to this adverse event is less than the total number of subjects exposed for the reporting group. These numbers are expected to be equal.
    Justification: This denominator includes only the male subjects in the group (N = 11).

    More information

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    09 Sep 2021
    There was one protocol amendment. In this amendment, in addition to some clarifications or minor changes to inclusion and exclusion criteria, and administrative updates, the patient population was revised to allow older patients to be included (due to the rare nature of the disease under investigation) and the number of patients aged 6-10 years was restricted to a maximum of 10. Additionally, the primary endpoint was changed from an evaluation of weight loss to a reduction in BMI. Also, secondary endpoints were revised to define key secondary endpoints.

    Interruptions (globally)

    Were there any global interruptions to the trial? No

    Limitations and caveats

    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    None reported
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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