E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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E.1.1.1 | Medical condition in easily understood language |
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E.1.1.2 | Therapeutic area | Diseases [C] - Immune System Diseases [C20] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 16.1 |
E.1.2 | Level | HLGT |
E.1.2 | Classification code | 10003816 |
E.1.2 | Term | Autoimmune disorders |
E.1.2 | System Organ Class | 10021428 - Immune system disorders |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To evaluate efficacy of SST looking at the change in uptake on SRS in a subset of chronically active patients in which intensification of corticosteroid therapy is not indicated. |
Het evalueren van de effectiviteit van sandostatine door een verschil in uptake in SRS in een subset van sarcoïdose patienten bij wie geen noodzaak is tot intensivering van therapie met corticosteroïden. |
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E.2.2 | Secondary objectives of the trial |
To study the composite clinical score using the following parameters: blood test (ESR, CRP, full blood count, lysozyme, ACE, 25-hydroxyvitamin D, 1,25-dihydroxyvitamin D, sIL-2R), quality of life (RAND-36) and when applicable lung function test (FVC, DLCO) and skin evaluation. |
Bestuderen van een samengestelde klinische score gebaseerd op de volgende parameters: bloedtest ( BSE, CRP, bloedbeeld, lysozym, ACE, 25-hydroxyvitamine D, 1.25-dihydroxyvitamine D, sIL-2R), kwaliteit van leven (RAND-36) en waar mogelijk longfunctie test (FVC, DLCO) en huidbeoordeling. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
• Age above 18 years with obtained written consent
• Have biopsy-proven symptomatic, stable, chronic sarcoidosis for minimal three years.
• Have a positive SRS
• Involvement of skin, joint, lymph nodes or lung. Patients with pulmonary involvement have a diffusing capacity between 60 and 75 percent.
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Leeftijd boven 18 jaar
Histologisch bewezen, symptomatische, stabiele, chronische sarcoidose minstens 3 jaar.
Postieve Somatostatine receptor scan
Betrokkenheid van huid, gewricht, lymfeklieren, long ( diffusiecapaciteit tussen 60-75 %) |
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E.4 | Principal exclusion criteria |
• Corticosteroid use up to three months prior of trial
• Chronic renal failure defined as a GFR below 50%
• Liver disease
• Have an indication for intensifying immunosuppressive therapy; threatening organ damage
• Have received anti TNF-α therapy
• Have an underlying cardiac disease
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Corticosteroid gebruik korter dan drie maanden voor trial
Chronisch nierfalen met een GFR onder 50%
Leverziekten
Indicatie voor intensifering van therapie door dreigende orgaanschade
Eerdere behandeling met TNF blokkers
Onderliggende cardiale ziekte |
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E.5 End points |
E.5.1 | Primary end point(s) |
To evaluate efficacy of SST in a subset of patients that are refractory/intolerant for corticosteroid therapy by a change in uptake on SRS. Change in uptake in SRS, defined as improved, no change, worse. |
Verschill in uptake op de somatostatine receptor scan gedefinieerd als verbeterd, onveranderd en verslechterd |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Before start of study and at month 9. |
Voor de eerste gift van sandostatine en na 9 maanden. |
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E.5.2 | Secondary end point(s) |
To study the composite clinical score using the following parameters. Measurements of ESR, CRP, full blood count, lysozyme, ACE, 25-hydroxyvitamin D, 1,25-dihydroxyvitamin D3 sIL-2R. Quality of life score assessment with RAND-36 will be conducted. When applicable lung-function test with FVC and DLCO and skin evaluation will take place. |
Bloedtesten: BSE, CRP, Bloedbeeld, lysozym, ACE, 25-hydroxyvitamine D, 1.25-dihydroxyvitamine D, sIl-2R. Kwaliteit van leven beoordeling met de RAND-36. Wanneer mogelijk longfunctie test met FVC en DLCO en huidbeoordeling. |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Bloodtests, quality of life and skin assesment takes place before start of study and at 2 weeks and 3, 6, 9, 12 months. Lungfunction before trial and at 6 months. |
Bloedtesten, kwaliteit van leven vragenlijst en huidbeoordeling vinden plaats voor de eerste gift en bij 2 weken, 3, 6, 9 en 12 maanden. Longfunctie test voor de eerste gift en na 6 maanden. |
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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 | No |
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 | No |
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) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
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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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | |
E.8.9.1 | In the Member State concerned months | |
E.8.9.1 | In the Member State concerned days | |