Genetic Diseases, Inborn

Forecast of the full-year count, planned starts included, not rows of this table: about 1,100 trials starting in 2026 and about 990 in 2027, the average of the 24 months through September, counted as registered and carried forward, with about 0.7 times the starts already registered for the rest of 2026 added. The rows of this table count trials registered so far with a start date in the year, planned starts included; the forecast looks ahead to the count for the whole year, so it is not the same measure. In backtests on conditions of this size, forecasts made part-way through the year were typically off by about 10 percent and ran about 4 percent high against the count later registered; forecasts made before the year began were off by about 25 percent and ran about 15 percent low against the count later registered, and had about 7 percent more error than repeating last year's count. The part-way figures are from forecasts that used a multiple fitted only on earlier years. A stricter check that trains only on older years did not support adding planned starts for conditions of this size: it ran about 6 percent high against the count later registered. These figures rest on a few years of history (target years from 2018 to 2025, varying by figure), place each condition as it is mapped today rather than as it was then, and score against a count for the newest year that was still being revised. Only condition series were scored.

Prediction, not a registry value: median of 36 months from start to primary completion across 1,450 trials still running at the 2026-10-09 snapshot; registered median of 23 months across 10,228 trials with a primary completion typed Actual. Source: ClinicalTrials.gov / AACT (CTTI), public domain.

Status as registered on ClinicalTrials.gov. Each trial is counted once.

These are the names sponsors entered on ClinicalTrials.gov that we have not matched to a single molecule. Some are drugs under a brand or code name; others are procedures, doses or arm labels.