Table 2. Results from RPE65-LCA Clinical Trials.
Visual outcomes of RPE65-LCA patients following treatment. *NCT#01208389 is a Phase I follow-up study treating the contralateral eye to patients enrolled in NCT#00516477. **All clinical trial information was obtained from the Clinical Trials online registry, https://clinicaltrials.gov [165].
| NCT# | Sponsor | Cohort | Subjects | Reported improvements in visual outcomes | Ref **[165] |
|||||
|---|---|---|---|---|---|---|---|---|---|---|
| VA | MP | FST | TPLR | Motility Studies | New or additional pseudofovea formation |
|||||
| 00643747 | UCL | 1 | N=3 | 0/3 patients |
1/3 patients |
n/a | n/a | 1/3 patients |
n/a | [2] |
| 00481546 | U Penn - Scheie | 1 | N=3 | 0/3 patients |
n/a | 3/3 patients |
2/3 patients |
2/3 patients |
1/3 patients |
[3,7,34,35] |
| 2 | N=3 | 1/3 patients |
n/a | 3/3 patients |
3/3 patients |
0/3 patients |
2/3 patients |
[7,35] | ||
| 3 | N=3 | 1/3 patients |
n/a | 3/3 patients |
3/3 patients |
1/3 patients |
0/3 patients |
[7] | ||
| 4 | N=2 | 1/2 patients |
n/a | 2/2 patients |
1/2 patients |
1/2 patients |
0/2 patients |
[7] | ||
| 5 | N=4 | 0/4 patients |
n/a | 4/4 patients |
2/4 patients |
1/4 patients |
1/4 patients |
[7,35] | ||
| 00516477 | U Penn - CHOP | 1 | N=3 | 3/3 patients |
n/a | 3/3 patients |
3/3 patients |
3/3 patients |
n/a | [4,151] |
| 2 | N=6 | 3/6 patients |
n/a | 4/5 patients (1 n/a) |
4/5 patients (1 n/a) |
3/5 patients |
n/a | [151] | ||
| 3 | N=3 | 1/3 patients |
n/a | 1/2 patients (1 n/a) |
3/3 patients |
1/3 patients |
n/a | [151] | ||
| *01208389 | Spark Therapeutics |
1 | N=3 (N=12) |
3/3 patients |
n/a | 2/3 patients |
3/3 patients |
2/3 patients |
n/a | [154] |
Abbreviations: NCT#, ClinicalTrials.gov identifier number; UCL, University College London; U Penn – Scheie, University of Pennsylvania, Scheie Eye Institute; U Penn – CHOP, University of Pennsylvania – The Children’s Hospital of Philadelphia. VA, visual acuity; MP, microperimetry; FST, full-field sensitivity testing; TPLR, transient pupillary light response sensitivity; n/a, non-applicable or not-tested. Ref, references.