Overview
Sponsor-declared trial summary
Pyruvate kinase deficiency
• To evaluate long-term safety following infusion of autologous CD34+ cells transduced with the therapeutic LV. • To determine the long-term persistence of the transgene in mononuclear cells in the blood, and to evaluate potential correlations between transgene persistence and transfusion independence (when relevant), …
Key facts
- Sponsor
- Rocket Pharmaceuticals Inc.
- Participant type
- Pediatric, Patients
- Age range
- 0-17 years, 18-64 years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Hemic and Lymphatic Diseases [C15]
- Trial duration
- 6 Jun 2023 → ongoing
- Decision date (initial)
- 2023-01-09
- Transition trial
- No
- Low-intervention
- No
- Rare-disease indication
- Yes
- Vulnerable population
- No
- Funding sources
- Rocket Pharmaceuticals, Inc.
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Safety
• To evaluate long-term safety following infusion of autologous CD34+ cells transduced with the therapeutic LV.
• To determine the long-term persistence of the transgene in mononuclear cells in the blood, and to evaluate potential correlations between transgene persistence and transfusion independence (when relevant), reduction in transfusion requirements (when relevant), and clinically significant reduction of anemia.
• To determine long-term clonality patterns beyond the RP-L301 parent study (RP-L301-0119).
• To assess, when relevant, RCL in serum and peripheral blood cells (this will not be considered relevant for subjects in whom no evidence of RCL was identified during the initial year following investigational autologous cell infusion).
Conditions and MedDRA coding
Pyruvate kinase deficiency
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 21.1 | PT | 10037682 | Pyruvate kinase deficiency anaemia | 100000004850 |
Regulatory references
- Plan to share IPD
- No
| EU CT number | Title | Sponsor |
|---|---|---|
| 2019-001656-19 | Gene Therapy for Pyruvate Kinase Deficiency (PKD): A Phase I Clinical Trial to Evaluate the Safety of the Infusion of Autologous CD34+ Cells Transduced with a Lentiviral Vector Carrying the Codon Optimized Red Cell Pyruvate Kinase (coRPK) Gene in Adult and Pediatric Subjects with PKD., Terapia génica para la deficiencia de piruvato quinasa (PKD): un ensayo clínico de fase I para evaluar la seguridad de la infusión de células CD34 + autólogas transducidas con un vector lentiviral que contiene el gen de piruvato quinasa de glóbulos rojos (CoRPK) con optimización de codones en sujetos adultos y pediátricos con PKD |
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 4
- Patient was enrolled in the parent Study RP-L301-0119
- Patient received an autologous infusion of CD34+ enriched cells transduced ex vivo with a lentiviral vector carrying the coRPK gene
- Patient is willing and able to adhere to the study visit schedule and other protocol requirements
- Patient provided written informed consent and, as applicable, assent to participate in the current study in accordance with current regulatory requirements
Exclusion criteria 1
- There are no exclusion criteria for the current study
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 7
- Overall survival
- Peripheral blood genetic correction, as demonstrated by vector copy number (VCN)
- Any red blood cell transfusion
- Sustained and significant decrease in hemoglobin unrelated to infection or surgery
- Adverse events related to worsening iron overload (for example, cirrhosis, cardiomyopathy, etc.)
- Any adverse event considered at least possibly related to RP-L301
- Development of any malignancy
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 1
PRD7873153 · Product
- Active substance
- Autologous CD34 Hematopoietic Stem Cells Transduced Ex Vivo with a Lentiviral Vector Encoding the Codon-Optimized Version of Pklr Gene
- Pharmaceutical form
- INFUSION
- Route of administration
- INTRAVENIOUS INFUSION
- Authorisation status
- Not Authorised
- ATC code
- B05AX — -
- MA holder
- ROCKET PHARMACEUTICALS, INC
- Paediatric formulation
- No
- Orphan designation
- Yes
- Orphan designation number
- EU/3/14/1330
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Rocket Pharmaceuticals Inc.
- Sponsor organisation
- Rocket Pharmaceuticals Inc.
- Address
- 9 Cedarbrook Dr, East Windsor East Windsor
- City
- Cranbury
- Postcode
- 08512-3618
- Country
- United States
Scientific contact point
- Organisation
- Rocket Pharmaceuticals Inc.
- Contact name
- Patient Advocacy
Public contact point
- Organisation
- Rocket Pharmaceuticals Inc.
- Contact name
- Patient Advocacy
Third parties 6
| Organisation | City, country | Duties |
|---|---|---|
| Intrinsic Lifesciences LLC ORG-100044000
|
La Jolla, United States | Laboratory analysis |
| Premier Research Group S.L. ORG-100013963
|
Madrid, Spain | On site monitoring, Code 12, Code 5, Data management, E-data capture, Code 8 |
| Patient Primary - MDgroup ORL-000000903
|
Durham, United States | Other |
| PPD Global Central Labs ORG-100046496
|
Zaventem, Belgium | Laboratory analysis |
| Medizinische Hochschule Hannover ORG-100024473
|
Hanover, Germany | Laboratory analysis |
| Indiana University Gene Therapy Testing Laboratory ORL-000000975
|
Indianapolis, United States | Laboratory analysis |
Locations
1 EU/EEA country · 2 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Spain | Ongoing, recruitment ended | 4 | 2 |
| Rest of world
United States
|
— | 1 | — |
Investigational sites
Country notifications
Trial-start, recruitment-start, end and early-termination notifications submitted per Member State
| Country | Trial start | Trial end | Recruitment start | Recruitment end | Early termination |
|---|---|---|---|---|---|
| Spain | 2023-06-06 | 2023-09-11 | 2025-09-08 |
Application history
5 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2022-09-20 | Spain | Acceptable 2022-11-02
|
2023-01-09 |
| 2 | SUBSTANTIAL MODIFICATION | SM-1 | 2023-01-10 | Spain | Acceptable | 2023-01-13 |
| 3 | NON SUBSTANTIAL MODIFICATION | NSM-2 | 2023-04-13 | Spain | Acceptable | 2023-04-13 |
| 4 | SUBSTANTIAL MODIFICATION | SM-2 | 2024-01-10 | Spain | Acceptable 2024-02-23
|
2024-02-27 |
| 5 | SUBSTANTIAL MODIFICATION | SM-4 | 2025-02-21 | Spain | Acceptable 2025-04-21
|
2025-04-22 |