E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Complications gastro-intestinales chroniques sévères post radiothérapie abdomino-pelvienne. |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 19.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10061171 |
E.1.2 | Term | Gastrointestinal disorder postoperative |
E.1.2 | System Organ Class | 10022117 - Injury, poisoning and procedural complications |
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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 evaluate the efficacy on rectorrhagia and/or hematuria of a treatment consisting of three intravenous allogeneic bone marrow MSC injections on patients affected by PRD and resistant to conventional treatments (wash-in steroids or plasma argon, laser YAG, formalin application). |
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E.2.2 | Secondary objectives of the trial |
1) To assess the safety of the evaluated treatment. 2) To assess the impact of the evaluated treatment on analgesic drug consumption (analgesic, opiates), frequency of diarrheas, quality of life and pain.
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
For patients, - More than 18 years of age; - Less than 80 years of age; - Any pelvic cancerous pathology irradiated with a history of pelvic complications of grade > 2 LENT SOMA scale); - Absence of tumor and metastasis objectified by bone scintigraphy; - After failure following at least three lines of conventional treatment; - Good physical condition (WHO-performance status 0-1); - Written informed consent; - Covered by health insurance.
For donors, - Between 18 years and 60 years (the upper age limit is justified by the viability of stem cells), - Establishment of physical and cognitive ability by a physician - Covered by health insurance - Written informed consent
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E.4 | Principal exclusion criteria |
For patients, - Pregnant or breast feeding women. Pregnancy test realized in the inclusion examination; Women with an intact uterus (unless amenorrhoeic for the last 24 months) not using effective means of contraception - Patients under “Tutelle” or “curatelle” - Unable to comply with the administration of the study - Contre-indication to the removal of bone marrow iliac / sternum
For Donors, - Risk of infection (see Appendix): 1. Infection known by human immunodeficiency virus (HIV), HTLV virus, hepatitis B or C virus. 2. Behavior considered at risk of infection: travel (in the 3 preceding months) except in Germany, Austria, Belgium, Spain, Finland, Ireland, Italy, Luxembourg, Netherlands, Portugal, Greece, Slovenia, Cyprus, Malta, Slovakia, Estonia, Bulgaria, Croatia, Hungary, Latvia, Lithuania , Poland, Romania , Czech Republic, United Kingdom, Denmark, Norway, Sweden, Switzerland, USA or Canada, risky sexual behavior (defined as unprotected sexual contact with a new partner in the previous six months), blood transfusion, piercing or tattoo in the six months prior. 3. Positive result at one of the testing for contagious diseases (Appendix 3). An old infection shall not constitute a criterion for exclusion. If repeated donations (many receivers), the balance of the donor screening will be renewed for each gift (unless the last balance sheet date is four weeks or less). 4. Known contagious disease 5. Episode of infectious character within seven days prior to screening 6. Risk factors for Creutzfeldt-Jakob disease (treatment by growth hormone extractive, of a family case of spongiform encephalopathy transmissible subacute of surgical intervention with opening of the Dura or neurosurgery or invasive brain exploration before 1st January 1995, presence of clinical neurological and neuropsychiatric signs recently evolutionary).
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E.5 End points |
E.5.1 | Primary end point(s) |
Primary endpoint : Success of the treatment is defined by a decrease of one grade on the LENT SOMA scale, four months after the first injection of MSCs. - Eligible patients must display a grade of more than a grade 2 for rectorrhagia or hematuria at the baseline. - Grade 3 on the LENT SOMA scale being defined by rectorrhagia or daily hematuria. - Grade 2 by 2 - 3 rectorrhagias or hematurias per week.
Secondary endpoints : 1. From the inclusion to the 4 months after the first injection of MSCs: i. Frequency of diarrhea; ii. Frequency of dysuria and painful mictions iii. Frequency of drug consumption (analgesic, opiate); iv. Quality of life evaluated by SF36 questionnaire and the Hospital Anxiety and depression Scale (HADS) questionnaire; v. Mean pain intensity evaluated by the numerical pain scale.
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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 | No |
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 | Information not present in EudraCT |
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 | 2 |
E.8.9.1 | In the Member State concerned months | 6 |
E.8.9.1 | In the Member State concerned days | 0 |