A Phase 1/2 interventional study of "Ex vivo" expanded autologous human oral epithelium containing stem cells in Hypospadias, sponsored by Holostem s.r.l.. Terminated at 1 site in Italy. Open to male participants aged 5 Years to 17 Years. Per ClinicalTrials.gov, last updated 2024-11-12.
Sponsored by Holostem s.r.l. · Phase 1/2, Interventional, and Treatment
The purpose of this trial is to assess the safety and efficacy of Holour in patients suffering from hypospadias treatment failure. Holour is intended for urethral replacement and is made from ex vivo expanded autologous oral mucosal cells including stem cells.
Holour is a prospective, open-label, uncontrolled pilot clinical trial phase i/ii. The aim of this clinical trial is to perform a tissue engineering therapy to assess the safety (at 3 and 12 months) and efficacy (at 12 months) of the autologous treatment.
Patients will be screened according to the study inclusion and exclusion criteria and will be candidate for the treatment if all eligibility criteria are met. Up to 8 patients will be treated by means of autologous cultured oral mucosa grafts containing oral mucosa stem cells. Holour is produced by ex vivo expansion of cells obtained from oral mucosa biopsy collected from patient. The biopsy will be processed in a laboratory of a regenerative medicine manufacturing site where the epithelial cells will be isolated, expanded and prepared as final graft to be implanted. Holour administration will be planned according to the procedures and the need of the patient.
The entire procedure envisages the following steps:
Penile urethroplasty in two stages:
The treatment includes a single administration of Holour; in case of "failure" of the first procedure eligible patients can undergo to a second implantation.
Three months after the transplantation, primary endpoint will be evaluated by the investigator. The study completion will be reached when 1 year (secondary endpoint) of follow-up after the last transplant in the last patient will be accomplished.
The end of the trial is defined as the last visit of the last patient after the last treatment if any.
123 studies on the registry are indexed under Hypospadias; 34 are open to participants now.
This study's enrollment of 1 is below the median of 70 across 97 interventional studies indexed under Hypospadias.
Browse Hypospadias studies →Holostem s.r.l. is the lead sponsor of 6 studies on the registry; none are open to participants now.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Allergy, sensitivity or intolerance to drugs or ( to any of the excipients listed in Investigator's brochure or in this protocol):
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
A scale of how much an illness limits everyday activity. For people being assessed to join a study, scores run from 0 (fully active) to 4; lower is more active.
Studies most often ask for 0–1 or 0–2. Your doctor gives the score; the WHO scale uses the same steps. The full scale also has 5, which records death.
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraindication
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ECOG performance status
A scale of how much an illness limits everyday activity. For people being assessed to join a study, scores run from 0 (fully active) to 4; lower is more active.
Studies most often ask for 0–1 or 0–2. Your doctor gives the score; the WHO scale uses the same steps. The full scale also has 5, which records death.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Excipients
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
The first step consists in a small oral mucosa biopsy collection by surgeon under general anaesthesia. The biopsy will be processed in a laboratory of a regenerative medicine manufacturing site where the epithelial cells will be isolated, expanded and prepared as final graft to be implanted. The treatment for urethra reconstruction required a two stage urethroplasty: 1. First stage: application of Holour on the wound bed prepared according to standard surgery The penis will be immobilized for some days after this operation. 2. Second stage: surgical procedure for urethral tubularization and penile reconstruction according to standard surgical procedure. The surgical procedures may be followed by a post-implantation treatment (if necessary) with corticosteroids and antibiotics.
Drug: "Ex vivo" expanded autologous human oral epithelium containing stem cells
The treatment includes a single administration of investigational product through a dedicated surgical procedure of penis wound bed preparation, epithelial surface adaption and product application followed by urethra tubularization and penis reconstruction.
Number and percentage of patients experiencing AESI
To demonstrate the safety of Holour in terms of AESI (persistent fever, infections)
Time frame: 3 months after treatment
Number and percentage of patients experiencing ADRs
To demonstrate the safety of Holour in terms of ADRs
Time frame: 3 months after treatment
Number and percentage of patients experiencing SAEs
To demonstrate the safety of Holour in terms of SAEs
Time frame: 3 months after treatment
Number and percentage of patients experiencing Serious ADRs
To demonstrate the safety of Holour in terms of Serious ADRs
Time frame: 3 months after treatment
Number and percentage of patients experiencing AESI
To demonstrate the safety of Holour in terms of AESI (persistent fever, infections)
Time frame: 12 months after treatment
Number and percentage of patients experiencing ADRs
To demonstrate the safety of Holour in terms of AESI ADRs
Time frame: 12 months after treatment
Number and percentage of patients experiencing SAEs
To demonstrate the safety of Holour in terms of SAEs
Time frame: 12 months after treatment
Number and percentage of patients experiencing Serious ADRs
To demonstrate the safety of Holour in terms of Serious ADRs
Time frame: 12 months after treatment
Number of patients with implantation success
Implantation success is defined on the basis of the degree of clinical (no lesions, no significant retractions) epithelial stability on the transplanted area. The degree of epithelial stability will be evaluated by visual inspection, cistouretrography, uretroscophy and checked for evaluating reepithelialization, erosions, infection, etc. and documented with photos with measures.
Time frame: 12 months after treatment
percentage of patients with implantation success
Implantation success is defined on the basis of the degree of clinical (no lesions, no significant retractions) epithelial stability on the transplanted area. The degree of epithelial stability will be evaluated by visual inspection, cistouretrography, uretroscophy and checked for evaluating reepithelialization, erosions, infection, etc. and documented with photos with measures.
Time frame: 12 months after treatment
Number of patients with the treatment efficacy after Holour implantation and surgical reconstruction
Regeneration of a clinically normal appearing urethra epithelium with absence of detectable erosions/retractions and Clinical functionality (uroflowmetry) after the second step (urethral reconstruction) of transplant.
Time frame: up to 1 year after implantation
Evaluation of the percentage of re-epithelialization
Evaluation of the percentage of re-epithelialization
Time frame: up to 1 year after implantation
Evaluation of the clinical epithelial stability on the transplanted area by visual inspections
Evaluation of the regeneration of a clinically normal appearing epithelium with absence of detectable erosions/retractions
Time frame: up to 1 year after implantation
Evaluation of scar retraction presence
Evaluation of the penile retraction due to presence/absence of scars
Time frame: up to 1 year after implantation
Evaluation of uroflowmetry rate
Evaluation of the change of uroflowmetry compared to the baseline value with change of the curve shape and significant increase of the qmax flow rate;
Time frame: From 3 to 12 months after the treatment
Evaluation of the post-void residual volume through uroflowmetry
Evaluation of the change of the post-void residual compared to the baseline
Time frame: From 3 to 12 months after the treatment
Evaluation of the presence and severity of surgical complications
Presence and severity of surgical complications using the Clavien Dindo scale with the classification into 5 grades (I, II, III, IV and V from no need for clinical intervention to the death of the patient)
Time frame: up to 1 year after implantation
Evaluation of the adeverse events in terms of SAEs, AESI, ADR Serious ADRs,
Evaluation of the adeverse events in terms of SAEs, AESI, ADR Serious ADRs,
Time frame: up to 1 year after implantation
Plan to share: No
No publications or documents are linked to this record.
Ran from Nov 2021 to primary completion in Dec 2022, 12 months.
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This study is terminated, as verified in Nov 2024. You cannot join it, but the record below documents what was studied.
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Holostem s.r.l.