Long-Term Follow-Up (LTFU) for Gene Therapy of Leukocyte Adhesion Deficiency-I (LAD-I). Phase I/II clinical study to evaluate the safety and efficacy of the infusion of autologous hematopoietic stem cells transduced with a lentiviral vector encoding the ITGB2 gene

Overview

Sponsor-declared trial summary

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] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Trial duration

15 Sep 2023 → ongoing

Decision date (initial)

2023-07-06

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: Efficacy, Safety

To evaluate long-term safety following infusion of RP-L201
To evaluate long term efficacy following infusion of RP-L201

Conditions and MedDRA coding

Leukocyte Adhesion Deficiency-I (LAD-I)

Study design 1 period

Regulatory references

Scientific advice from competent authorities

European Medicines Agency

EMA paediatric investigation plan (PIP)

EMEA-002562-PIP01-19

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 4

1. Subject must have been enrolled in the Phase I/II parent study RP-L201-0318.
2. Subjects must have received RP-L201 in the parent Study RP-L201-0318.
3. Subjects must be willing and able to adhere to the study visit schedule and other protocol requirements.
4. Subjects must be willing and able to provide provided written informed consent and, as applicable, assent to participate in the current study in accordance with current regulatory requirements.

Exclusion criteria 1

1. There are not exclusion criteria in this study.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 15

1. Any significant infection, defined as those requiring hospitalization or intravenous antimicrobials
2. Any new skin or oral lesion potentially caused by underlying LAD-I disease.
3. Any late-occurring (more than 2 years post-infusion) SAE considered at least possibly related to the investigational RP-L201 study treatment.
4. Incidence of any malignancy
5. Any new incidence of secondary graft failure defined as a sustained decrease in PBMC VCN of <0.1 or PB neutrophil CD18 expression of <10% on 2 consecutive evaluations separated by an interval of at least 1 month, and not considered related to a concurrent infection or due to non-RP-L201 drug-related toxicity.
6. Any new concerns for GvHD based on GVHD clinical classifications defined
7. Allogeneic HSCT-free survival;
8. Event free survival defined as survival in the absence of graft failure or GvHD;
9. Reduction of significant infections, defined as those requiring hospitalization or intravenous antimicrobials
10. Reduction of infection-related hospitalizations, and infection-related prolonged hospitalization (lasting ≥7 days) beyond the initial 24 months of RP-L201-0318;
11. Improvement or resolution of LAD-I-related neutrophilia and leukocytosis
12. Resolution of LAD-I-related skin rash or periodontal abnormalities;
13. Persistence of transgene in PB cells as demonstrated by VCN of at least 0.1 in PBMCs and PB CD15+ granulocytes;
14. Persistence of CD18 neutrophil expression defined by PB neutrophil CD18 expression to at least 10% of normal;
15. Persistence of CD11 a/b neutrophil co-expression.

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

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

Locations

1 EU/EEA country · 1 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 11 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

4 submissions — initial application plus substantial / non-substantial modifications