NCT04105166 is a Phase 1 clinical trial of RP-L301 in Pyruvate Kinase Deficiency, sponsored by Rocket Pharmaceuticals Inc..

Who is sponsoring NCT04105166?

NCT04105166 is sponsored by Rocket Pharmaceuticals Inc..

What drugs are being tested in NCT04105166?

Interventions in NCT04105166: RP-L301.

What condition does NCT04105166 study?

NCT04105166 studies Pyruvate Kinase Deficiency.

Is NCT04105166 still recruiting?

NCT04105166 is currently completed. Last updated 5 September 2025.

Last reviewed 2025-09-05 · How we verify

NCT04105166

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

Gene Therapy for Pyruvate Kinase Deficiency (PKD)

Completed Phase 1 Last updated 5 September 2025

What this trial tests

Phase 1 trial testing RP-L301 in Pyruvate Kinase Deficiency in 4 participants. Completed in 9 June 2025.

Timeline

6 July 2020

Primary endpoint
9 June 2025

9 June 2025

Quick facts

Lead sponsor	Rocket Pharmaceuticals Inc.
Phase	Phase 1
Status	Completed
Study type	INTERVENTIONAL
Allocation	na
Design	sequential
Masking	none
Primary purpose	treatment
Enrollment	4
Start date	6 July 2020
Primary completion	9 June 2025
Estimated completion	9 June 2025
Sites	3 locations across United States, Spain

Drugs / interventions tested

RP-L301 — full drug profile →

Conditions studied

Pyruvate Kinase Deficiency — all drugs for Pyruvate Kinase Deficiency →

Sponsor

Rocket Pharmaceuticals Inc. — full company profile →

Who can join

Adults 8 to 50, any sex, with Pyruvate Kinase Deficiency. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

This is an open-label Phase I trial to evaluate the safety of a hematopoietic cell-based gene therapy for patients with Pyruvate Kinase Deficiency (PKD).

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

Large-Scale Production of Lentiviral Vectors: Current Perspectives and Challenges.
Martínez-Molina E, Chocarro-Wrona C, Martínez-Moreno D, Marchal JA, et al · · 2020 · cited 55× · PMID 33153183 · DOI 10.3390/pharmaceutics12111051
Immunological barriers to haematopoietic stem cell gene therapy.
Charlesworth CT, Hsu I, Wilkinson AC, Nakauchi H. · · 2022 · cited 47× · PMID 35301483 · DOI 10.1038/s41577-022-00698-0
Molecular heterogeneity of pyruvate kinase deficiency.
Bianchi P, Fermo E. · · 2020 · cited 30× · PMID 33054047 · DOI 10.3324/haematol.2019.241141
Pyruvate Kinase Deficiency: Current Challenges and Future Prospects.
Fattizzo B, Cavallaro F, Marcello APML, Vercellati C, et al · · 2022 · cited 20× · PMID 36072510 · DOI 10.2147/jbm.s353907
Curative gene therapies for rare diseases.
Maldonado R, Jalil S, Wartiovaara K. · · 2021 · cited 19× · PMID 32803721 · DOI 10.1007/s12687-020-00480-6
Updates and advances in pyruvate kinase deficiency.
Luke N, Hillier K, Al-Samkari H, Grace RF. · · 2023 · cited 18× · PMID 36935283 · DOI 10.1016/j.molmed.2023.02.005
Innovative Treatments for Rare Anemias.
Cappellini MD, Marcon A, Fattizzo B, Motta I. · · 2021 · cited 18× · PMID 34095760 · DOI 10.1097/hs9.0000000000000576
Clinically relevant gene editing in hematopoietic stem cells for the treatment of pyruvate kinase deficiency.
Fañanas-Baquero S, Quintana-Bustamante O, Dever DP, Alberquilla O, et al · · 2021 · cited 14× · PMID 34485608 · DOI 10.1016/j.omtm.2021.05.001

Verify or expand the search:

Other recruiting trials for Pyruvate Kinase Deficiency

Currently open trials in the same condition.

NCT04902833 — Acquired Pyruvate Kinase Deficiency In Clonal Myeloid Neoplasms · active not recruiting
NCT03481738 — Pyruvate Kinase Deficiency Global Longitudinal Registry · active not recruiting

Other Rocket Pharmaceuticals Inc. trials

Trials by the same sponsor.

NCT05548855 — Natural History of Danon Disease · completed
NCT06282432 — Long-Term Follow-Up (LTFU) for Gene Therapy of Leukocyte Adhesion Deficiency-I (LAD-I) · active not recruiting
NCT04525352 — A Trial to Evaluate Safety and Efficacy of RP-L401-0120 in Subjects With Infantile Malignant Osteopetrosis · Phase 1 · terminated
NCT04248439 — Gene Therapy for Fanconi Anemia, Complementation Group A · Phase 2 · active not recruiting
NCT04437771 — Long-Term Follow-up of Subjects With Fanconi Anaemia Subtype A Treated With ex Vivo Gene Therapy · active not recruiting

Verify against primary sources

ClinicalTrials.gov — authoritative US registry record
WHO ICTRP — international registry index
EU Clinical Trials Register
Sponsor press releases (Google)

Data sources for this page

Trial protocol + status: ClinicalTrials.gov NCT04105166 (US National Library of Medicine, public domain)
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Rocket Pharmaceuticals Inc.
Last refreshed: 5 September 2025

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT04105166.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing