Trial · NCT04041232
PBA Use for Treatment of ATF6-/- Patients
NCT04041232, Early phase 1, Suspended, Columbia University
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. PBA Use for Treatment of ATF6-/- Patients. Trials, record NCT04041232. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04041232
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- PBA Use for Treatment of ATF6-/- Patients
- NCT number
- NCT04041232
- Status
- Suspended
- Phases
- Early phase 1
- Enrollment
- 2 participants, actual
- Lead sponsor
- Columbia University
- Conditions
- ACHROMATOPSIA 7, Achromatopsia
- Interventions
- PBA
- First posted
- 2019-08-01
- Last updated
- 2026-09-11
Source ClinicalTrials.gov
Registered outcomes9
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Changes in best corrected visual acuity (BCVA) | Baseline, 1 month, 3 months, 6 months post-PBA use | to measure changes in vision at each time point |
| Primary | Changes in contrast sensitivity | Baseline, 1 month, 3 months, 6 months post-PBA use | using Pelli Robson charts |
| Primary | Changes in color vision | Baseline, 1 month, 3 months, 6 months post-PBA use | using D50 |
| Primary | Changes in macular sensitivity | Baseline, 1 month, 3 months, 6 months post-PBA use | using microperimetry (Nidek) |
| Primary | Changes in retinal imaging | After 1 and 3 months of PBA use. If changes in retinal function is observed, an additional ophthalmic evaluation will be conducted after 6 months of PBA use | including optical coherence tomography (OCT), short wavelength autofluorescence (SW-AF), and near-infrared autofluorescence (NIR-AF) |
| Primary | Changes in Full-field Electroretinogram (ffERG) X | After 1 and 3 months of PBA use. If changes in retinal function is observed, an additional ophthalmic evaluation will be conducted after 6 months of PBA use | to measure changes in rod and cone traces |
| Secondary | Changes in intraocular pressure | Baseline, 1 month, 3 months, 6 months post-PBA use | part of full ophthalmic evaluation |
| Secondary | Changes in anterior segment | After 1 and 3 months of PBA use. If changes in retinal function is observed, an additional ophthalmic evaluation will be conducted after 6 months of PBA use | part of full ophthalmic evaluation |
| Secondary | Changes observed in posterior segment (slit lamp and binocular fundus examination) | After 1 and 3 months of PBA use. If changes in retinal function is observed, an additional ophthalmic evaluation will be conducted after 6 months of PBA use | part of full ophthalmic evaluation |
Source ClinicalTrials.gov
Outcomes0
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
None in this release.
Results by group0
Each measurement the trial posted, group by group, in the words and numbers it posted.
None in this release.
Participant flow0
How many started, completed or left each group, period by period.
None in this release.
Adverse events0
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
None in this release.
Adverse event totals0
The totals the trial posted for each group.
None in this release.
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT04041232
GET https://conductscience.org/api/trials/v1/NCT04041232/registered