Trial · NCT07887347
A Study of the Safety and Efficacy of Prime Editing (PM647a) in Participants With Alpha-1 Antitrypsin Deficiency
NCT07887347, Phase 1, Phase 2, Not yet recruiting, Prime Medicine, Inc.
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. A Study of the Safety and Efficacy of Prime Editing (PM647a) in Participants With Alpha-1 Antitrypsin Deficiency. Trials, record NCT07887347. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT07887347
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study of the Safety and Efficacy of Prime Editing (PM647a) in Participants With Alpha-1 Antitrypsin Deficiency
- NCT number
- NCT07887347
- Status
- Not yet recruiting
- Phases
- Phase 1, Phase 2
- Enrollment
- 54 participants, estimated
- Lead sponsor
- Prime Medicine, Inc.
- Conditions
- Alpha-1 Antitrypsin Deficiency, Alpha-1 Antitrypsin Deficiency-associated Lung Disease, Alpha-1 Antitrypsin Deficiency (AATD), AATD
- Interventions
- PM647a
- First posted
- 2026-10-21
- Last updated
- 2026-10-21
Source ClinicalTrials.gov
Registered outcomes18
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Safety and tolerability of PM647a. Quantified by frequency and severity of treatment emergent adverse events (TEAEs) | Immediately post-infusion up to Week 48 | |
| Secondary | Frequency and severity of Dose Limiting Toxicities (DLTs) | Infusion up to and including 28 days | |
| Secondary | Percentage of participants with ≥ 11 µM total serum alpha-1 antitrypsin (AAT) levels | Week 12 post-infusion | For participants treated with a dose of PM647a at or above the OBD, central laboratory quantitative immunoturbidimetry |
| Secondary | Percentage of participants with ≥ 11 µM total serum AAT levels | Week 4, Week 6, Week 12, Week 24 and Week 48 post-infusion | Central laboratory quantitative immunoturbidimetry |
| Secondary | Percentage of participants with total serum AAT levels ≥ the lower limit of normal | Week 4, Week 6, Week 12, Week 24 and Week 48 post-infusion | Central laboratory quantitative immunoturbidimetry |
| Secondary | Change in total serum AAT levels, including changes in levels of pathogenic AAT (Z-AAT) and normal (M-AAT) | Baseline to Week 4, Week 6, Week 12, Week 24, and Week 48 post-infusion | Total AAT measured by immunoturbidimetry; M-AAT and Z-AAT isoforms by liquid chromatography/mass spectrometry (LC/MS) |
| Secondary | Time from PM647a infusion to total serum AAT levels ≥ 11 µM, ≥ 20 µM, or within the normal range | Measured over the course of the study (through Week 48) | |
| Secondary | Change in Forced Expiratory Volume (FEV1) | From baseline to Week 6 and to Week 48 post-infusion | Pulmonary function testing (PFT), using standardized pre- and post-bronchodilator spirometry |
| Secondary | Change in Forced Vital Capacity (FVC) | From baseline to Week 6 and to Week 48 post-infusion | Spirometry measuring the volume of air forcibly exhaled following maximal inspiration |
| Secondary | Change in FEV1/FVC | From baseline to Week 6 and to Week 48 post-infusion |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 registered outcomesOutcomes0
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/NCT07887347
GET https://conductscience.org/api/trials/v1/NCT07887347/registered