Table 1.
List of Currently Available Registries for Cell and Gene Therapies
| Resource Name | Primary Scope | Funding | Accessibility | Website |
|---|---|---|---|---|
| Patient Outcomes Registries | ||||
| The European Society for Blood and Marrow Transplantation (EBMT) cell therapy registry | cell and cell-based gene therapies used for treating conditions other than hematopoietic stem cell transplantation (HSCT) | private (membership fees and corporate sponsorships) | member centers of the EBMT or upon request | https://www.ebmt.org/; updated regularly |
| The Center for International Blood and Marrow Transplantation Research (CIBMTR) cellular therapy registry | cell and cell-based gene therapies used for treating conditions other than hematopoietic stem cell transplantation (HSCT) | public (NIH, USA) and corporate sponsorships | member centers of the CIBMTR or upon request | https://www.cibmtr.org/Pages/index.aspx; under further development & expansion |
| Registry of Cell Therapy in Non-Ischemic Dilated Cardiomyopathy (RECORD) | evaluation of cell therapy in patients with non-ischemic dilated cardiomyopathy | public (University Medical Centre Ljubljana, Slovenia) | currently recruiting patients (ClinicalTrials.gov Identifier: NCT02445534) | N/A |
| Registry of Biological Cell Therapy for the Treatment of Patients with Thoracic & Cardiovascular Disease (ClinicalCellRegistry.com) | cell therapy evaluation for the treatment of thoracic and cardiovascular diseases | private (Amit N. Patel, University of Utah) | researchers who provide an IRB approval to the coordinating site at the University of Utah | http://clinicalcellregistry.com/; non- functioning link |
| Long-Term Follow-Up of Recipient of Gene Transfer Research | evaluation of safety in gene transfer therapy | M.D. Anderson Cancer Center, USA | currently recruiting patients (ClinicalTrials.gov Identifier: NCT01492036) | N/A |
| GENIALL Lipoprotein Lipase Deficiency (LPLD) Disease Registry (GENIALL) | long-term follow up of alipogene tiparvovec and registration of any genetically confirmed LPLD patients | private (uniQure biopharma and Chiesi Farmaceutici) | restricted | https://www.geniallregistry.com/login.jsp; updated regularly |
| Strimvelis Clinical Study Registry | evaluation of the long-term safety and effectiveness outcomes of subjects receiving Strimvelis | private (GlaxoSmithKline) | restricted | https://www.gsk-clinicalstudyregister.com/study/200195?search=study&#ps; updated regularly |
| Clinical Trials Registries | ||||
| NIH Genetic Modification Clinical Research Information System (GeMCRIS) | information about human gene transfer trials registered with the NIH | NIH | publicly accessible | https://www.gemcris.od.nih.gov/Contents/GC_HOME.asp?id=8434; last available data December 2016 |
| Gene Therapy Clinical Trials Worldwide | searchable information about approved, ongoing, or completed gene therapy clinical trials available on the internet | Journal of Gene Medicine | publicly accessible | http://www.abedia.com/wiley/; updated regularly |
| Gene Therapy Net | web-based information repository acting as a gene therapy trials portal (recruiting or not recruiting yet) by using the clinicaltrials.gov database | private | publicly accessible | http://www.genetherapynet.com; updated regularly |
| The European Network for the Advancement of Clinical Gene Transfer and Therapy (CLINIGENE) | gene therapy trials library | European Commission | publicly accessible | http://www.clinigene.org; last available data Dec 2010 |
| Cell and Gene Therapy Catapult | database for preclinical and clinical cell and gene therapy trials that take place in the UK | Department for Business, Energy and Industrial Strategy, U.K. government | publicly accessible | https://ct.catapult.org.uk; updated annually |
| DeReG: German Registry for Somatic Gene-Transfer Trials | somatic gene transfer trials | public (German Federal Ministry of Education and Research) | N/A | http://ww38.dereg.de/; non-functioning link |
| Belgian Biosafety Server | gene therapy trials in Belgium | public (Belgian federal web sites) | publicly accessible | http://www.biosafety.be/; updated regularly |
PubMed, clinicaltrials.gov, the Registry of Patient Registries (RoPR), the Patient Registry of Europe (PARENT), the WHO international clinical trials registry platform, as well as Google search engine were searched using several keyword combinations, including “cell therapy”, “gene therapy”, “regenerative therapies”, or “advanced therapy medicinal products (ATMPs)” combined with “registry,” “clinical trials registry,” “database,” “observational study”, or “long-term follow-up”. Five publications were identified as related to two cell and gene therapy registries (n = 2). A ClinicalTrials.gov search yielded 41 trials, of which four were identified as patient outcomes registries (n = 4). The AHRQ registry of patient registry (RoPR) search yielded 81 registries, of which one was identified as a relevant registry (n = 1). The search of the patient registry of Europe (PARENT) yielded one registry (n = 1). The WHO international clinical trials registry platform search identified one registry (n = 1). The web-based search identified 9 relevant registries. After removal of duplicates (n = 2) and the exclusion of registries with no clear information about their purpose retrievable through literature and/or their website (n = 1) or language other than English (n = 1), the final relevant registries numbered fourteen (n = 14). These registries were then split into two categories: patient outcomes registries and clinical trials registries. This table summarizes these search results with specific details on each registry characteristics.