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NCT04784052 · ClinicalTrials.gov registry record · Phase 1
Depleted Donor Stem Cell Transplant in Children and Adults With Fanconi Anemia After Being Conditioned With a Regimen Containing Briquilimab
A Phase 1 study of Fanconi Anemia, sponsored by Porteus, Matthew, MD.
- Recruiting
- Registry status
- Phase 1
- Development phase
- 18
- Enrollment target
- 1
- Study location
NCT04784052: Recruiting Phase 1 study of Fanconi Anemia, sponsored by Porteus, Matthew, MD.
NCT04784052 is a Phase 1 study of Fanconi Anemia that is actively recruiting participants, run by Porteus, Matthew, MD. The registered enrollment target is 18 participants, below the 335-participant average among 15 other Fanconi Anemia trials with a reported enrollment target (95% lower). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04784052, a Phase 1 study of Fanconi Anemia, is actively recruiting participants, sponsored by Porteus, Matthew, MD.
- RECRUITING
- Registry status
- Phase 1
- Development phase
- 18 participants
- Enrollment target
- 1
- Study location
Study Summary
The objective of this clinical trial is to develop a cell therapy for Fanconi Anemia which enables enhanced donor hematopoietic and immune reconstitution with decreased toxicity by transplanting depleted stem cells from a donor with and without using an experimental antibody treatment called JSP-191 as a part of conditioning. This experimental treatment will hopefully cause fewer side effects than chemotherapy (the current standard of care method). Participants will be administered the conditioning regimen, are assessed until they receive the depleted stem cell infusion, and will be followed for up to 2 years after the cell infusion.
Primary Outcome
Recorded and graded according to the Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0
Conditions Studied
Interventions
- DRUG Cyclophosphamide
- DEVICE CliniMACS Prodigy System
- DRUG JSP191
- BIOLOGICAL Depleted Stem Cell Transplant
- BIOLOGICAL Rabbit Anti-Thymoglobulin (rATG)
Study Locations (1)
California
- Stanford University - Stanford
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 18 participants |
| Start Date | 2021-12-07 |
| Est. Completion | 2028-12 |
| Phase | Phase 1 |
What NCT04784052 shows while recruiting
NCT04784052 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 18 participants, a relatively small participant target, below the 335-participant average among 15 other Fanconi Anemia trials with a reported enrollment target (95% lower).
The record links to 1 condition, with Fanconi Anemia appearing as the primary indexed condition, and to 5 interventions - of which Cyclophosphamide is the first listed.
NCT04784052 reports a single indexed study location in California.
Frequently Asked Questions
What is clinical trial NCT04784052 about?
NCT04784052 is a clinical study titled "Depleted Donor Stem Cell Transplant in Children and Adults With Fanconi Anemia After Being Conditioned With a Regimen Containing Briquilimab". The objective of this clinical trial is to develop a cell therapy for Fanconi Anemia which enables enhanced donor hematopoietic and immune reconstitution with decreased toxicity by transplanting depleted stem cells from a donor with and without using an experimental antibody treatment called JSP-191...
What is the current status of trial NCT04784052?
This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 18 participants. The study started on 2021-12-07. Estimated completion is 2028-12.
What conditions does trial NCT04784052 study?
This clinical trial studies the following conditions: Fanconi Anemia.
What interventions are being tested in trial NCT04784052?
The interventions under investigation include: Cyclophosphamide (DRUG), CliniMACS Prodigy System (DEVICE), JSP191 (DRUG), Depleted Stem Cell Transplant (BIOLOGICAL), Rabbit Anti-Thymoglobulin (rATG) (BIOLOGICAL).
Who is sponsoring clinical trial NCT04784052?
This trial is sponsored by Porteus, Matthew, MD, which has 2 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT04784052 being conducted?
This trial has 1 study location across California. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
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Where NCT04784052's enrollment target sits among peer trials
18 13th of 15 higher than 3 of 15 other Fanconi Anemia trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Fanconi Anemia trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
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