Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID

Model scores of the 29,894 trials active now.

Model score 23% for any early termination, 14% for termination due to slow enrollment. Past trials like this one (phase, sponsor type and disease area, started 2008–2016) ended terminated 18% of the time (283 trials).

Finished trial that started in 2015 or later: the model never saw it during training.

Cumulative chance of ending terminated (red) or completed (green) by each year from its start; it actually ended on 2019-09-26. 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: ulnevidence ofcardiacmarrowserum
  Phrases that lowered it: and safetysingleefficacy andsafety ofsubject
• Accepts healthy volunteers: No (raises risk) · Who can join
• Drug's FAERS reports with a death: 42.6% (raises risk) · Drug's FDA history
• Design: single group (raises risk) · Design
• Phase: 1/2 (raises risk) · Design
• Sponsor's past trials: 281 (lowers risk) · Sponsor's track record
• Trials the sponsor started in the prior 2 years: 67 (lowers risk) · Sponsor's track record
• Collaborators: 2 (lowers risk) · Design
• Drug's FAERS reports marked serious: 97.0% (raises risk) · Drug's FDA history
• Has a data monitoring committee: Yes (raises risk) · Design
• Minimum age: 0.08 (lowers risk) · Who can join
• Sponsor type: academic or other (raises risk) · Sponsor's track record
• Disease area, cancer: No (lowers risk) · Disease area
• Primary purpose: treatment (raises risk) · Design
• Sponsor's past termination rate: 7.5% (raises risk) · Sponsor's track record
• Maximum age: 17 (raises risk) · Who can join
• Number of arms: 1 (raises risk) · Design
• Drug's FAERS reports before start: 4046 (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 NCT03765632 · started 2018 · scored riskier than 88% 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
• MND-ADA Transduction of CD34+ Cells From Children With ADA-SCID NCT00794508 · started 2008 · scored riskier than 78% of active trials completed
• Study of Gene Therapy Using a Lentiviral Vector to Treat X-linked Chronic Granulomatous Disease NCT02234934 · started 2015 · scored riskier than 46% 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.