E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Children with autism spectrum disorder and gastrointestinal symptoms. |
Børn med autismespektrumforstyrrelser og mave-tarmproblemer. |
|
E.1.1.1 | Medical condition in easily understood language |
Children with autism spectrum disorder and gastrointestinal symptoms. |
Børn med autismespektrumforstyrrelser og mave-tarmproblemer. |
|
E.1.1.2 | Therapeutic area | Psychiatry and Psychology [F] - Mental Disorders [F03] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10008520 |
E.1.2 | Term | Childhood autism |
E.1.2 | System Organ Class | 100000004873 |
|
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10013225 |
E.1.2 | Term | Disorder gastrointestinal |
E.1.2 | System Organ Class | 100000004856 |
|
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 |
To investigate safety and treatment effect of an allogeneic adipose tissue derived mesenchymal stromal cell product (CSCC_ASC) in children with autism spectrum disorder and gastrointestinal symptoms. |
At vurdere sikkerhed og behandlingseffekt af et stamcelleprodukt (CSCC_ASC) hos børn med autismespektrumforstyrrelse og mave-tarmproblemer. |
|
E.2.2 | Secondary objectives of the trial |
Not applicate. |
Ingen andre formål. |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Diagnosis of autism spectrum disorder. Ongoing gastrointestinal symptoms or previous gastrointestinal symptoms which disappeared or were reduced after dietary changes. Age 6 – 14 years.
|
Diagnose af autismespektrumforstyrrelser. Igangværende mave-tarmproblemer eller forhenværende mave-tarmproblemer som forsvandt eller blev reduceret efter kostændringer. Alder 6 – 14 år.
|
|
E.4 | Principal exclusion criteria |
Known genetic syndrome or pathogenic mutation or copy number variation associated with autism spectrum disorder.
Known CNS-infection (now or previously) and/or HIV positivity.
Primary immunodeficiency disorder or autoimmune cytopenia.
Current treatment with cytotoxic drugs or systemic administered glucocorticoids and/or immunosuppressive therapy or other anti-inflammatory medication (except nonsteroidal anti-inflammatory drugs).
Epilepsy or known seizure disorder (now or previously).
|
Kendt genetisk syndrom eller patogen mutation eller kopiantalvariation associeret med autismespektrumforstyrrelser.
Kendt CNS-infektion (nu eller tidligere) og/eller HIV-positivitet.
Primær immundefekt eller autoimmun cytopeni.
Nuværende behandling med cytotoksiske lægemidler eller systemisk glukokortikoider og/eller immunsuppressiv terapi eller anden antiinflammatorisk medicin (undtagen non-steroide antiinflammatoriske midler).
Epilepsi eller kendt anfaldsforstyrrelse (nu eller tidligere).
|
|
E.5 End points |
E.5.1 | Primary end point(s) |
Difference from baseline to follow-up within the group treated with CSCC_ASC: Safety within the 12 weeks follow-up period: AE, SAE, SUSAR and DSUR.
|
Difference fra baseline til opfølgning indenfor gruppen behandlet med CSCC_ASC: Sikkerhed indenfor 12-ugers opfølgningsperiode: AE, SAE, SUSAR og DSUR.
|
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
|
E.5.2 | Secondary end point(s) |
Difference from baseline to follow-up within the group treated with CSCC_ASC: Changes in:
- Autism spectrum disorder score test. - The Infant Gastrointestinal Symptom Questionnaire. - Faecal microbiota and short-chain fatty acid. - Biomarkers for inflammation, donor tissue type HLA antibodies, biomarkers for assessment of intestinal permeability and for immune cell characterization.
|
Difference fra baseline til opfølgning indenfor gruppen behandlet med CSCC_ASC: Ændringer i:
- Autismespektrumforstyrrelser score test. - Spædbørnsspørgeskema om mave-tarmproblemer. - Fækal mikrobiota og kortkædede fedtsyrer. - Biomarkører for inflammation, donorspecifikke HLA-antistoffer, biomarkører til vurdering af tarmpermeabilitet og for karakterisering af immunceller.
|
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
|
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 | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | Yes |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | Information not present in EudraCT |
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) | 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 | Yes |
E.8.1.7.1 | Other trial design description |
|
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.8.2.4 | Number of treatment arms in the trial | 2 |
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.8.4.1 | Number of sites anticipated in Member State concerned | 1 |
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
|
The trial will end when the last participant has had the last visit. |
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 3 |
E.8.9.1 | In the Member State concerned months | |
E.8.9.1 | In the Member State concerned days | |