Discontinued development programmes as evidence — included and excluded studies
The included-studies table with extracted data, the exclusions with reasons, and the risk-of-bias judgements.
§3Included and excluded studies
Every study contributing to this review is listed below with the data extracted from it. The extraction table is published at the level of the individual outcome and study so that the synthesis can be checked without the reader having to retrieve each source.
§3.1Included studies
Table 4. Included studies with the characteristics extracted from each.
| Study | Design | Randomised | Duration | Anchor outcome as reported | Year |
|---|---|---|---|---|---|
| DANU-PH2B-OBESITYDanuglipron | Randomised, double-blind, placebo-controlled | 605 | 32 weeks | Dose-dependent weight reduction accompanied by discontinuation rates above 50 % in some arms; development in obesity was subsequently discontinued | 2023 |
| DANU-PH2B-T2DDanuglipron | Randomised, double-blind, placebo-controlled | 411 | 16 weeks | Glycaemic effect demonstrated; the programme was discontinued for tolerability and hepatic-safety reasons | 2023 |
| CELIAC-PH3-CEDLARALarazotide acetate | Randomised, double-blind, placebo-controlled | — | 12 weeks | Terminated at a pre-planned interim analysis for futility. The Institute records the termination as the result | 2022 |
| MMPOWER-3SS-31 (elamipretide) | Randomised, double-blind, placebo-controlled | 218 | 24 weeks | Neither primary endpoint was met. The Institute records MMPOWER-3 as a negative phase 3 result and treats it as informative evidence rather than as an absence… | 2022 |
| ELAM-PH2-HFPEFSS-31 (elamipretide) | Randomised, double-blind, placebo-controlled | 71 | 4 weeks | No significant difference from placebo | 2019 |
| MMPOWER-2SS-31 (elamipretide) | Randomised crossover | 30 | 4 weeks per period | An increase in six-minute walk distance in a crossover design with a small sample | 2018 |
| AOD-OA-PH2AOD-9604 | Randomised, double-blind, placebo-controlled | — | 12 weeks | No consistent benefit demonstrated | 2015 |
| CELIAC-PH2BLarazotide acetate | Randomised, double-blind, placebo-controlled | 342 | 12 weeks | A symptom effect at the lowest dose only, with no effect at higher doses | 2015 |
| AOD-PH2B-OBESITYAOD-9604 | Randomised, double-blind, placebo-controlled | 536 | 12 weeks | No separation from placebo. Development in obesity was discontinued | 2007 |
| 9 included studies. Each links to its structured abstract, which carries a field-by-field provenance table distinguishing extracted figures from Institute reconstructions. | |||||
Contributing participants across studies reporting a randomised total: 2,213. Studies for which the Institute does not hold a randomised total contribute to the qualitative synthesis and not to any pooled figure.
§3.2Excluded at full text, with reasons
Table 5. Reports excluded at full text, by reason. A review that reports an exclusion count without reasons cannot be checked against its own protocol.
| Reason for exclusion | Reports |
|---|---|
| Not a randomised comparison | 6 |
| Population outside the review question | 5 |
| Intervention outside the review question | 5 |
| Comparator not eligible | 4 |
| No eligible outcome reported | 3 |
| Duplicate report of an included study | 3 |
| Conference abstract without extractable data | 5 |
| Retracted or subject to an expression of concern | 4 |
| 31 reports excluded at full text in total. | |
§3.3Risk of bias across included studies
Table 6. Risk-of-bias judgement recorded for each included study on the review's anchor outcome.
| Study | Risk of bias | Inconsistency | Indirectness | Imprecision | Publication bias |
|---|---|---|---|---|---|
| DANU-PH2B-OBESITY | Low | Low | Some | Some | Low |
| DANU-PH2B-T2D | Low | Low | Low | Low | High |
| CELIAC-PH3-CEDLARA | Low | Some | Low | Low | Some |
| MMPOWER-3 | Low | Low | Low | Some | Low |
| ELAM-PH2-HFPEF | Some | Low | Some | Low | Low |
| MMPOWER-2 | Low | Low | Low | Some | Low |
| AOD-OA-PH2 | Low | High | Low | Low | Low |
| CELIAC-PH2B | Some | Low | Low | Low | Low |
| AOD-PH2B-OBESITY | Low | Some | Low | Low | Some |
| Judgements are the Institute's own and are recorded per outcome. A study may carry a different judgement in a different review that assesses a different outcome from it. | |||||