Pilot and Feasibility Study of Hematopoietic Stem Cell Gene Transfer for the Wiskott-Aldrich Syndrome

Model scores of the 29,894 trials active now.

Model score 24% for any early termination, 9% for termination due to slow enrollment. Past trials like this one (phase, sponsor type and disease area, started 2008–2016) ended terminated 30% of the time (37 trials).

Finished trial that started 2008–2014: scored by a model trained without any trial from its start year, so its own outcome was never used.

Cumulative chance of ending terminated (red) or completed (green) by each year from its start; it actually ended on 2024-09-30. The rest of the way to 100% is still running.

From the competing-risks survival model (termination vs completion, still-running trials censored), fit to trials that started 2008–2014 and recalibrated to termination rates over 2022–2024, since termination has become more common. Checked on past years, it still ran a little low when the rise continued.

• Wording of the registration (title, summary, criteria) (raises risk) · Wording Phrases that raised it: donormarrowcellspatienthla
  Phrases that lowered it: bleedinghealthybloodabnormalclinical
• Accepts healthy volunteers: No (raises risk) · Who can join
• Phase: 1/2 (raises risk) · Design
• Sponsor's past termination rate: no earlier finished trials (lowers risk) · Sponsor's track record
• Design: single group (raises risk) · Design
• Disease area, cancer: No (lowers risk) · Disease area
• Sponsor type: academic or other (raises risk) · Sponsor's track record
• Responsible party: sponsor investigator (raises risk) · Design
• Maximum age: 35 (lowers risk) · Who can join
• Number of arms: 1 (raises risk) · Design
• Minimum age: 0.25 (lowers risk) · Who can join
• Trials the sponsor started in the prior 2 years: 1 (raises risk) · Sponsor's track record
• Primary purpose: treatment (raises risk) · Design
• Has a data monitoring committee: Yes (raises risk) · Design
• Blinding: none (raises risk) · Design
• Collaborators: 0 (raises risk) · Design
• Sponsor's past trials: 0 (raises risk) · Sponsor's track record
• Drug's FAERS reports marked serious: no FAERS history (raises risk) · Drug's FDA history

Every input that moved this score, largest first: SHAP contributions in log-odds, red raising risk and green lowering it. They describe the model, not causes of termination. Phrases are traced from the text features back to this registration's words, approximately.

• Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID NCT02999984 · started 2016 · scored riskier than 82% of active trials completed
• Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1 Alpha Shortened (EFS) Lentiviral Vector Encoding for the Human ADA Gene NCT01852071 · started 2013 · scored riskier than 62% of active trials completed
• Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID NCT03765632 · started 2018 · scored riskier than 88% of active trials completed
• Gene Transfer for Severe Combined Immunodeficiency, X-linked (SCID-X1) Using a Self-inactivating (SIN) Gammaretroviral Vector NCT01129544 · started 2010 · scored riskier than 79% of active trials completed
• Stem Cell Transplant Using Peripheral and Cord Blood Stem Cells to Treat Severe Aplastic Anemia and Myelodysplastic Syndrome NCT00604201 · started 2008 · scored riskier than 58% of active trials completed

Closest by meaning of the registration text (PubMedBERT embeddings), among finished trials. How they ended is context, not an input to this trial's score.