Inadequate reporting quality of registered genome editing trials: an observational study
Abstract Background To assess registration completeness and safety data of trials on human genome editing (HGE) reported in primary registries and published in journals, as HGE has safety and ethical problems, including the risk of undesirable and unpredictable outcomes. Registration transparency ha...
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| Format: | Article |
| Language: | English |
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BMC
2022-05-01
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| Series: | BMC Medical Research Methodology |
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| Online Access: | https://doi.org/10.1186/s12874-022-01574-0 |
| _version_ | 1828786415303917568 |
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| author | Diana Jurić Michael Zlatin Ana Marušić |
| author_facet | Diana Jurić Michael Zlatin Ana Marušić |
| author_sort | Diana Jurić |
| collection | DOAJ |
| description | Abstract Background To assess registration completeness and safety data of trials on human genome editing (HGE) reported in primary registries and published in journals, as HGE has safety and ethical problems, including the risk of undesirable and unpredictable outcomes. Registration transparency has not been evaluated for clinical trials using these novel and revolutionary techniques in human participants. Methods Observational study of trials involving engineered site-specific nucleases and long-term follow-up observations, identified from the WHO ICTRP HGE Registry in November 2020 and two comprehensive reviews published in the same year. Registration and adverse events (AEs) information were collected from public registries and matching publications. Published data were extracted in May 2021. Results Among 81 eligible trials, most were recruiting (51.9%) phase 1 trials (45.7%). Five trials were withdrawn. Most trials investigated CAR T cells therapies (45.7%) and used CRISPR/Cas9 (35.8%) ex vivo (88.9%). Among 12 trials with protocols both registered and published, eligibility criteria, sample size, and secondary outcome measures were consistently reported for less than a half. Three trials posted results in ClinicalTrials.gov, and one reported serious AEs. Conclusions Incomplete registration and published data give emphasis to the need to increase the transparency of HGE trials. Further improvements in registration requirements, including phase 1 trials, and a more controlled publication procedure, are needed to augment the implementation of this promising technology. |
| first_indexed | 2024-12-12T00:10:59Z |
| format | Article |
| id | doaj.art-d5235a0142034a8aa7a317076daa3c52 |
| institution | Directory Open Access Journal |
| issn | 1471-2288 |
| language | English |
| last_indexed | 2024-12-12T00:10:59Z |
| publishDate | 2022-05-01 |
| publisher | BMC |
| record_format | Article |
| series | BMC Medical Research Methodology |
| spelling | doaj.art-d5235a0142034a8aa7a317076daa3c522022-12-22T00:44:58ZengBMCBMC Medical Research Methodology1471-22882022-05-0122111110.1186/s12874-022-01574-0Inadequate reporting quality of registered genome editing trials: an observational studyDiana Jurić0Michael Zlatin1Ana Marušić2Department of Pharmacology, School of Medicine, University of SplitSchool of Medicine, University of SplitDepartment of Research in Biomedicine and Health, School of Medicine, University of SplitAbstract Background To assess registration completeness and safety data of trials on human genome editing (HGE) reported in primary registries and published in journals, as HGE has safety and ethical problems, including the risk of undesirable and unpredictable outcomes. Registration transparency has not been evaluated for clinical trials using these novel and revolutionary techniques in human participants. Methods Observational study of trials involving engineered site-specific nucleases and long-term follow-up observations, identified from the WHO ICTRP HGE Registry in November 2020 and two comprehensive reviews published in the same year. Registration and adverse events (AEs) information were collected from public registries and matching publications. Published data were extracted in May 2021. Results Among 81 eligible trials, most were recruiting (51.9%) phase 1 trials (45.7%). Five trials were withdrawn. Most trials investigated CAR T cells therapies (45.7%) and used CRISPR/Cas9 (35.8%) ex vivo (88.9%). Among 12 trials with protocols both registered and published, eligibility criteria, sample size, and secondary outcome measures were consistently reported for less than a half. Three trials posted results in ClinicalTrials.gov, and one reported serious AEs. Conclusions Incomplete registration and published data give emphasis to the need to increase the transparency of HGE trials. Further improvements in registration requirements, including phase 1 trials, and a more controlled publication procedure, are needed to augment the implementation of this promising technology.https://doi.org/10.1186/s12874-022-01574-0Clinical trials on genome editing as topicGenome editingDatabasesReporting |
| spellingShingle | Diana Jurić Michael Zlatin Ana Marušić Inadequate reporting quality of registered genome editing trials: an observational study BMC Medical Research Methodology Clinical trials on genome editing as topic Genome editing Databases Reporting |
| title | Inadequate reporting quality of registered genome editing trials: an observational study |
| title_full | Inadequate reporting quality of registered genome editing trials: an observational study |
| title_fullStr | Inadequate reporting quality of registered genome editing trials: an observational study |
| title_full_unstemmed | Inadequate reporting quality of registered genome editing trials: an observational study |
| title_short | Inadequate reporting quality of registered genome editing trials: an observational study |
| title_sort | inadequate reporting quality of registered genome editing trials an observational study |
| topic | Clinical trials on genome editing as topic Genome editing Databases Reporting |
| url | https://doi.org/10.1186/s12874-022-01574-0 |
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