E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Operated craniopharyngioma with hypothalamic involvement, Craniopharyngioma is a pituitary tumor that often affects the hypothalamus. These patients suffer from an intractable weight gain. This is named hypothalamic obesity. There is presently no drug available to treat this obesity. |
Hypothalamisk övervikt. |
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E.1.1.1 | Medical condition in easily understood language |
Craniopharyngioma with Hypothalamic obesity |
Patienter som är operarade för hypofystumören kraniopharyngiom. Denna tumör drabbar ofta inte bara hypofysen med bred hormonbrist utan även hypothalamus med strörd energiomsättning. |
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E.1.1.2 | Therapeutic area | Diseases [C] - Hormonal diseases [C19] |
MedDRA Classification |
E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
Effects of Exenatid on Weigth during Caloric Restriction and physical excercise in patients with Craniopharyngioma |
Att undersöka om exenatid kan reducera vikten hos pt med kraniopharyngiom. |
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E.2.2 | Secondary objectives of the trial |
The secondary endpoints are improved insulin sensitivity, reduced leptin resistance and improvement in lipid balance. |
Att undersöka om patienternas höga kardiovakulära riskprofil förbättras av behandlingen. Dessutom att undersöka om såväl vischeralt som sub cutant fett reduceras. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Male, non-fertile women or female of childbearing potential using medically approved birth control method aged > 18 years.
All have the criteria of hypothalmic obesity, where the MRI of the brain clearly shows an affected hypothalamus. All patients have stable pituitary hormone substitutions when needed, including GH therapy. All patients have a BMI > 27 kg/m2.
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Patienter med hypofystumörer som påverkar hypothalamus och orsakar hypothalamisk obesitas.Män och kvinnor som använder contraception och är mer 18 år. Alla pt har stabil hormonbehandling för sin hypofyssvikt. Alla pt har ett BMI > 27 kg/m2. |
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E.4 | Principal exclusion criteria |
b) Exclusion criteria.
Pregnant or lactating female. Women of childbearing potential with no effective contraceptive method.
Type I Diabetes mellitus (Including LADA)
Treatment with antihyperglycemic agents apart from insulin and metformin.
History of unexplained pancreatitis, chronic pancreatitis, Stomach or gastric surgery.
Allergic reaction to any GLP-1 receptor agonist.
Clinical relevant history of gastrointestinal disease associated with nausea and vomiting.
Cardiovascular, hepatic, neurological, active malignant tumor or other major systemic disease or patients with short life expectancy making implementation of the protocol or interpretation of the study results difficult.
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Gravida kvinnor, eller kvinnor utan contraception. Pat med Typ I DM (inkluderande LADA).
Behandling med anti-hyperglylkemi medicinering, utom insulin och metformin.
Sjukhistoria med oförklarlig pancreatit, kronisk pancreatit eller som genomgått mag- eller tarm-operation.
Allergi mot GLP-1 agonist.
Relevant sjukhistoria med mag-tarm problem associerat med illamående och kräkningar.
Kardiovaskulär, hepatisk, neurologisk sjukdom, aktiv malignitet eller eller annan sjukdom med kort överlevnad som försvårar bedömningen av studieresultaten. |
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E.5 End points |
E.5.1 | Primary end point(s) |
The primary endpoint is weight reduction. |
Viktnedgång. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
The study is planned for 1 years. If successful the patients may contimue in the trial for 2 years. |
Studien är planerad till 1 år. Varje studie patient har möjlighet till 2 års behandling om resultaten är goda. |
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E.5.2 | Secondary end point(s) |
Cardiovascualr risk profile is evaluated including: Blood pressure, BMI, wais/hip ratio, body composition and lipidprofile, and HOMA-index (insulin sensitivity). |
Kardiovaskulär risk profil följes vilket inkluderar Bt, BMI, midja/höftmått, kroppssammansättning. Blodfetter och Homa index (insulinkänslighet). |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
After 2 years treatment for each patient. |
Efter 2 års behandling för varje pt. |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | Yes |
E.6.13.1 | Other scope of the trial description |
Weight reduction. |
Viktnedgång. |
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E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | Yes |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.3 |
The trial involves single site in the Member State concerned
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.5 | The trial involves multiple Member States | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.7 | Trial has a data monitoring committee | No |
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
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LVLS |
Sista besöket för sista pt. |
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 2 |
E.8.9.1 | In the Member State concerned months | |
E.8.9.1 | In the Member State concerned days | |