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Summary
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| EudraCT Number: | 2015-000359-26 |
| Sponsor's Protocol Code Number: | ESTEVE-SANF-201 |
| National Competent Authority: | Spain - AEMPS |
| Clinical Trial Type: | EEA CTA |
| Trial Status: | Ongoing |
| Date on which this record was first entered in the EudraCT database: | 2016-02-03 |
| Trial results | |
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A. Protocol Information
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| A.1 | Member State Concerned | Spain - AEMPS | ||
| A.2 | EudraCT number | 2015-000359-26 | ||
| A.3 | Full title of the trial |
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| A.3.1 | Title of the trial for lay people, in easily understood, i.e. non-technical, language |
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| A.4.1 | Sponsor's protocol code number | ESTEVE-SANF-201 | ||
| A.7 | Trial is part of a Paediatric Investigation Plan | Yes | ||
| A.8 | EMA Decision number of Paediatric Investigation Plan | P/149/2014 | ||
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B. Sponsor Information
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| B.Sponsor: 1 | ||
| B.1.1 | Name of Sponsor | Laboratorios del Dr. Esteve, S.A. |
| B.1.3.4 | Country | Spain |
| B.3.1 and B.3.2 | Status of the sponsor | Commercial |
| B.4 Source(s) of Monetary or Material Support for the clinical trial: | ||
| B.4.1 | Name of organisation providing support | Laboratorios del Dr. Esteve,S.A. |
| B.4.2 | Country | Spain |
| B.5 Contact point designated by the sponsor for further information on the trial | ||
| B.5.1 | Name of organisation | Laboratorios del Dr. Esteve,S.A. |
| B.5.2 | Functional name of contact point | Adelaida Morte |
| B.5.3 | Address: | |
| B.5.3.1 | Street Address | Av. Mare de Déu de Montserrat,221 |
| B.5.3.2 | Town/ city | Barcelona |
| B.5.3.3 | Post code | 08041 |
| B.5.3.4 | Country | Spain |
| B.5.4 | Telephone number | 349344660006581 |
| B.5.6 | amorte@esteve.es | |
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D. IMP Identification
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| D.IMP: 1 | ||
| D.1.2 and D.1.3 | IMP Role | Test |
| D.2 | Status of the IMP to be used in the clinical trial | |
| D.2.1 | IMP to be used in the trial has a marketing authorisation | No |
| D.2.5 | The IMP has been designated in this indication as an orphan drug in the Community | Yes |
| D.2.5.1 | Orphan drug designation number | EU/3/11/877 |
| D.3 Description of the IMP | ||
| D.3.1 | Product name | Vector viral adenoasociado de serotipo 9 que contiene el gen de la sulfamidasa humana |
| D.3.2 | Product code | AAV9-CAG-coh-SGSH |
| D.3.4 | Pharmaceutical form | Suspension for injection |
| D.3.4.1 | Specific paediatric formulation | No |
| D.3.7 | Routes of administration for this IMP | Other use (Noncurrent) |
| D.3.8 to D.3.10 IMP Identification Details (Active Substances) | ||
| D.3.8 | INN - Proposed INN | Vector viral adenoasociado de serotipo 9 que contiene el gen de la sulfamidasa humana |
| D.3.9.1 | CAS number | 1352588-20-5 |
| D.3.9.2 | Current sponsor code | AAV9- CAG-coh-SGSH |
| D.3.9.3 | Other descriptive name | Vector viral adenoasociado de serotipo 9 que contiene el gen de la sulfamidasa humana |
| D.3.9.4 | EV Substance Code | SUB178857 |
| D.3.10 | Strength | |
| D.3.10.1 | Concentration unit | Other |
| D.3.10.2 | Concentration type | range |
| D.3.10.3 | Concentration number | 1000000000000 to 1000000000000000 |
| D.3.11 The IMP contains an: | ||
| D.3.11.1 | Active substance of chemical origin | No |
| D.3.11.2 | Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) | No |
| The IMP is a: | ||
| D.3.11.3 | Advanced Therapy IMP (ATIMP) | Yes |
| D.3.11.3.1 | Somatic cell therapy medicinal product | No |
| D.3.11.3.2 | Gene therapy medical product | Yes |
| D.3.11.3.3 | Tissue Engineered Product | No |
| D.3.11.3.4 | Combination ATIMP (i.e. one involving a medical device) | No |
| D.3.11.3.5 | Committee on Advanced therapies (CAT) has issued a classification for this product | No |
| D.3.11.4 | Combination product that includes a device, but does not involve an Advanced Therapy | No |
| D.3.11.5 | Radiopharmaceutical medicinal product | No |
| D.3.11.6 | Immunological medicinal product (such as vaccine, allergen, immune serum) | No |
| D.3.11.7 | Plasma derived medicinal product | No |
| D.3.11.8 | Extractive medicinal product | No |
| D.3.11.9 | Recombinant medicinal product | No |
| D.3.11.10 | Medicinal product containing genetically modified organisms | No |
| D.3.11.11 | Herbal medicinal product | No |
| D.3.11.12 | Homeopathic medicinal product | No |
| D.3.11.13 | Another type of medicinal product | No |
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D.8 Information on Placebo
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E. General Information on the Trial
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| 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] - Nutritional and Metabolic Diseases [C18] | ||
| MedDRA Classification | ||||
| 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 |
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| E.2.2 | Secondary objectives of the trial |
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| E.2.3 | Trial contains a sub-study | No | ||
| E.3 | Principal inclusion criteria |
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| E.4 | Principal exclusion criteria |
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| E.5 End points | ||||
| E.5.1 | Primary end point(s) |
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| E.5.1.1 | Timepoint(s) of evaluation of this end point |
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| E.5.2 | Secondary end point(s) |
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| E.5.2.1 | Timepoint(s) of evaluation of this end point |
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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 | Yes | ||
| E.6.8 | Bioequivalence | No | ||
| E.6.9 | Dose response | Yes | ||
| 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) | Yes | ||
| E.7.1.1 | First administration to humans | Yes | ||
| 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 | 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 |
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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 | 5 | ||
| E.8.9.1 | In the Member State concerned months | |||
| E.8.9.1 | In the Member State concerned days | |||
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F. Population of Trial Subjects
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| F.1 Age Range | |||
| F.1.1 | Trial has subjects under 18 | Yes | |
| F.1.1 | Number of subjects for this age range: | 6 | |
| F.1.1.1 | In Utero | No | |
| F.1.1.2 | Preterm newborn infants (up to gestational age < 37 weeks) | No | |
| F.1.1.3 | Newborns (0-27 days) | No | |
| F.1.1.4 | Infants and toddlers (28 days-23 months) | No | |
| F.1.1.5 | Children (2-11years) | Yes | |
| F.1.1.5.1 | Number of subjects for this age range: | 3 | |
| F.1.1.6 | Adolescents (12-17 years) | Yes | |
| F.1.1.6.1 | Number of subjects for this age range: | 3 | |
| F.1.2 | Adults (18-64 years) | No | |
| F.1.3 | Elderly (>=65 years) | No | |
| F.2 Gender | |||
| F.2.1 | Female | Yes | |
| F.2.2 | Male | Yes | |
| F.3 Group of trial subjects | |||
| F.3.1 | Healthy volunteers | No | |
| F.3.2 | Patients | Yes | |
| F.3.3 | Specific vulnerable populations | Yes | |
| F.3.3.1 | Women of childbearing potential not using contraception | No | |
| F.3.3.2 | Women of child-bearing potential using contraception | No | |
| F.3.3.3 | Pregnant women | No | |
| F.3.3.4 | Nursing women | No | |
| F.3.3.5 | Emergency situation | No | |
| F.3.3.6 | Subjects incapable of giving consent personally | Yes | |
| F.3.3.6.1 | Details of subjects incapable of giving consent |
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| F.3.3.7 | Others | No | |
| F.4 Planned number of subjects to be included | |||
| F.4.1 | In the member state | 6 | |
| 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) |
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G. Investigator Networks to be involved in the Trial
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N. Review by the Competent Authority or Ethics Committee in the country concerned
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| N. | Competent Authority Decision | Authorised |
| N. | Date of Competent Authority Decision | 2016-04-29 |
| N. | Ethics Committee Opinion of the trial application | Favourable |
| N. | Ethics Committee Opinion: Reason(s) for unfavourable opinion |
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| N. | Date of Ethics Committee Opinion | 2015-10-15 |
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P. End of Trial
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| P. | End of Trial Status | Ongoing |
