Pyruvate kinase deficiency anaemia

4 trials 1 recruiting MedDRA PT
4 Registered trials
1 Recruiting
2 Unique sponsors
5 Unique IMPs
3 Commercial trials
Drug classes (ATC)
B Haematology (ATC B) 2
L Oncology (ATC L) 1
— ATC code not specified 2
Mechanism of action (INN-stem analysis)
Unknown / small molecule 3 substances
Alkylating agent 1 substance
Colony-stimulating factor 1 substance
Phase III: 2 Phase I (Other): 2

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Show: 4 trials

Phase III

2 trials
  • Agios Pharmaceuticals Inc. Commercial
    MITAPIVAT
    2024-515024-37-00 4 countries 2022 n=11
    Ended
  • Agios Pharmaceuticals Inc. Commercial
    MITAPIVAT
    2024-515025-28-00 5 countries 2022 n=14
    Recruiting

Phase I (Other)

2 trials
  • Rocket Pharmaceuticals Inc. Commercial
    AUTOLOGOUS CD34+ HEMATOPOIETIC STEM CELLS TRANSDUCED EX VIVO WITH A LENTIVIRAL VECTOR ENCODING THE CODON-OPTIMIZED VERSION OF PKLR GENE; BUSULFAN; FILGRASTIM; PLERIXAFOR
    2024-511520-13-00 1 country 2019 n=3 Orphan
    Ended
  • Rocket Pharmaceuticals Inc. Academic
    AUTOLOGOUS CD34+ HEMATOPOIETIC STEM CELLS TRANSDUCED EX VIVO WITH A LENTIVIRAL VECTOR ENCODING THE CODON-OPTIMIZED VERSION OF PKLR GENE
    2022-501526-38-00 1 country 2023 n=4 Orphan
    Ended
Sponsor activity
SponsorTypeTrialsPhasesRecruiting
Rocket Pharmaceuticals Inc. Commercial 2 Phase I (Other) 0
Agios Pharmaceuticals Inc. Commercial 2 Phase III 1
New trials by year
2019
1
2022
2
2023
1
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