Clinical trials: Leukocyte Adhesion Deficiency 1 (LAD)
Leukocyte Adhesion Deficiency
A Study to Evaluate Efficacy and Safety of AVTX-803 in Patients With Leukocyte Adhesion Deficiency Type II
The primary objective of this study is to evaluate the efficacy and safety of AVTX-803 compared to withdrawal in patients with Leukocyte Adhesion Deficiency, Type II (LAD II).
- Trial name
- NCT05462587
- Testing
- AVTX-803 (L-Fucose)
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- AUG Therapeutics
Leukocyte Adhesion Defect - Type I
A Gene Therapy Trial to Evaluate the Safety and Efficacy of RP-L201 in Subjects With Leukocyte Adhesion Deficiency-I
The primary purpose of the Phase I portion of the study is to determine the safety profile and preliminary evidence of efficacy associated with infusion of autologous gene-corrected hematopoietic stem cells.
- Trial name
- NCT03825783
- Testing
- RP-L201
- Where
- Spain
- Sponsor
- Rocket Pharmaceuticals Inc.
Leukocyte Adhesion Defect - Type I
A Clinical Trial to Evaluate the Safety and Efficacy of RP-L201 in Subjects With Leukocyte Adhesion Deficiency-I
The primary purpose of the Phase I portion of the study is to assess the therapeutic safety and preliminary efficacy of a hematopoietic cell-based gene therapy consisting of autologous CD34+ enriched cells transduced with the…
- Trial name
- NCT03812263
- Testing
- RP-L201
- Where
- 3 sites in United States, Spain and 1 more
- Volunteers
- 9 people
- Sponsor
- Rocket Pharmaceuticals Inc.
Leukocyte Adhesion Deficiency
An Extension Study Assessing the Safety and Efficacy of AVTX-803 in Subjects With Leukocyte Adhesion Deficiency Type II
The primary objective of this extension study is to assess the long-term safety and efficacy of AVTX-803 in subjects with LAD II (SLC35C1-CDG).
- Trial name
- NCT05754450
- Testing
- AVTX-803
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- AUG Therapeutics
Leukocyte Adhesion Deficiency
Long-Term Follow-Up (LTFU) for Gene Therapy of Leukocyte Adhesion Deficiency-I (LAD-I)
This Long-Term Follow-Up (LTFU) for Gene Therapy of Leukocyte Adhesion Deficiency-I (LAD-I) is a continuation of a Phase 1/2 clinical study to evaluate the safety and efficacy of the infusion of autologous hematopoietic stem…
- Trial name
- NCT06282432
- Where
- 3 sites in United States, Spain and 1 more
- Volunteers
- 9 people
- Sponsor
- Rocket Pharmaceuticals Inc.