Évaluation de suivi des événements indésirables retardés liés à la thérapie génique après la participation aux essais cliniques de la Direction de l'oncologie pédiatrique
Background:
- Gene therapy involves changing the genes inside the body s cells to stop disease. It is very closely regulated. People who have had this therapy may have problems months or even years later. Researchers do not know the long-term side effects, so they want to study people who have had the therapy. They want the study to continue over the next 15 years.
Objective:
- To study over time the negative side effects from genetically engineered cellular therapy. This will be studied in people who have been in Pediatric Oncology Branch (POB) gene therapy trials.
Eligibility:
- People who are currently or were previously in a research study with gene therapy in the National Cancer Institute POB.
Design:
- Participants blood will be tested right before they get the genetically changed cells. They will get the cells as part of another study.
- For the next year, they will come back to the clinic or see their doctor at home at least every 3 months. They will answer questions about their health and blood will be drawn.
- For the next 5 years, they will go to the clinic or see their own doctor once a year. They will have physical exam and blood will be drawn.
- For 10 years after that, they will be asked every year for health information.
- Participants will keep their contact information up to date with researchers. They may be phoned for more health information.
- If the participant was under 18 years old when given the gene therapy and turns 18 during this follow-up, they will be asked to sign a new consent form when they turn 18.
Aperçu de l'étude
Statut
Les conditions
Intervention / Traitement
Description détaillée
Background:
- Study subjects exposed to gene therapy interventions may be at risk for delayed or longterm adverse consequences. The U.S. Food and Drug Administration (FDA) has issued Long Term Follow-up After Administration of Human Gene Therapy Products: Guidance for Industry and Testing of Retroviral Vector-Based Human Gene Therapy Products for Replication Competent Retrovirus During Product Manufacture and Patient Follow-up: Guidance for Industry, which outlines a framework to assess the risk of gene therapy-related delayed adverse events and the essential elements appropriate for the conduct of long-term follow up observations.
- Accordingly, some vector designs (i.e. use of an adenovirus), based on duration of persistence accompanied by cumulative preclinical and clinical evidence, may require shorter periods of observation than other vector designs (i.e. use of a lentivirus).
Objective:
-To conduct long term safety evaluations for adverse events associated with genetically engineered cellular therapy in subjects who have participated in Pediatric Oncology Branch (POB) clinical trials.
Eligibility:
-Subjects who are planned to receive or have received at least one dose of a genetically engineered cellular therapy on a POB gene therapy clinical trial are eligible to participate.
Design:
-
Subjects will be evaluated for long term safety and occurrence of adverse events according to the requirements established by the FDA guidance and the NIH Guidelines for Research Involving Recombinant or Synthetic Nucleic Acid Molecules (NIH Guidelines), to include:
-
Long term follow-up after genetically engineered cellular therapy may include:
- Post-cell infusion evaluations for T-Cell persistence (if applicable)
- Monitoring for replication-competent retrovirus (RCR) or replication-competent lentivirus (RCL)
- Physical examination
- Questionnaire to detect delayed adverse events
- Medical record review related to long term adverse events
-
To ensure adequate follow up for participants in POB receiving genetically engineered cellular therapy, a maximum accrual of 500 participants will be allowed on this study. To accommodate up to 15-year follow-up of participants, this study will remain open until 2050.
Type d'étude
Inscription (Estimé)
Contacts et emplacements
Lieux d'étude
-
-
Maryland
-
Bethesda, Maryland, États-Unis, 20892
- National Institutes of Health Clinical Center
-
-
Critères de participation
Critère d'éligibilité
Âges éligibles pour étudier
Accepte les volontaires sains
Sexes éligibles pour l'étude
Méthode d'échantillonnage
Population étudiée
Participants screening for, participating in, or have participated in a POB gene therapy clinical trial and have received/or be scheduled to receive a genetically engineered cellular therapy.
La description
- INCLUSION CRITERIA:
- Participants must be identified eligible for, participating in, or have participated in a POB genetically engineered cellular therapy clinical trial and have received/or be scheduled to receive a genetically engineered cellular therapy.
- All monitoring and testing described in this protocol will pertain ONLY to genetically engineered cellular therapy received at the National Institutes of Health (NIH). RCR/RCL monitoring and persistence will NOT be followed for cells administered at another institution.
Plan d'étude
Comment l'étude est-elle conçue ?
Détails de conception
- Modèles d'observation: COHORT
- Perspectives temporelles: PROSPECTIVE
Cohortes et interventions
Groupe / Cohorte |
Intervention / Traitement |
|---|---|
|
1
Patients screening for, participating in, or have participated in a POB gene therapy clinical trial and have received/or be scheduled to receive a genetically engineered cellular therapy. |
Que mesure l'étude ?
Principaux critères de jugement
Mesure des résultats |
Description de la mesure |
Délai |
|---|---|---|
|
Conduct long term safety evaluations after gene therapy
Délai: Every 3 months X 1 year then annually X 15 years
|
PCR results (for RCR/RCL, gene transduced cells) |
Every 3 months X 1 year then annually X 15 years
|
Collaborateurs et enquêteurs
Parrainer
Les enquêteurs
- Chercheur principal: John W Glod, M.D., National Cancer Institute (NCI)
Publications et liens utiles
Liens utiles
Dates d'enregistrement des études
Dates principales de l'étude
Début de l'étude (Réel)
Achèvement primaire (Estimé)
Achèvement de l'étude (Estimé)
Dates d'inscription aux études
Première soumission
Première soumission répondant aux critères de contrôle qualité
Première publication (Estimé)
Mises à jour des dossiers d'étude
Dernière mise à jour publiée (Réel)
Dernière mise à jour soumise répondant aux critères de contrôle qualité
Dernière vérification
Plus d'information
Termes liés à cette étude
Mots clés
Termes MeSH pertinents supplémentaires
Autres numéros d'identification d'étude
- 150028
- 15-C-0028
Plan pour les données individuelles des participants (IPD)
Prévoyez-vous de partager les données individuelles des participants (DPI) ?
Description du régime IPD
All IPD recorded in the medical record will be shared with intramural investigators upon request.
Délai de partage IPD
Clinical data available during the study and indefinitely.
Critères d'accès au partage IPD
Clinical data will be made available via subscription to BTRIS and with the permission of the study PI.
Type d'informations de prise en charge du partage d'IPD
- PROTOCOLE D'ÉTUDE
- SÈVE
- CIF
Informations sur les médicaments et les dispositifs, documents d'étude
Étudie un produit pharmaceutique réglementé par la FDA américaine
Étudie un produit d'appareil réglementé par la FDA américaine
Ces informations ont été extraites directement du site Web clinicaltrials.gov sans aucune modification. Si vous avez des demandes de modification, de suppression ou de mise à jour des détails de votre étude, veuillez contacter register@clinicaltrials.gov. Dès qu'un changement est mis en œuvre sur clinicaltrials.gov, il sera également mis à jour automatiquement sur notre site Web .