Overview
Sponsor-declared trial summary
Phase
Therapeutic exploratory (Phase II)
Status
Ongoing, recruiting
Participants planned
14
Countries
2
Sites
2
Danon Disease
The primary objective of this study is to evaluate whether RP-A501 (an AAV9 capsid containing the LAMP2B transgene; AAV9.LAMP2B) will result in significant improvement as assessed via the primary endpoint comprised of LAMP2 myocardial tissue expression and LVMI.
Key facts
- Sponsor
- Rocket Pharmaceuticals Inc.
- Participant type
- Pediatric, Patients
- Age range
- 0-17 years, 18-64 years, 65+ years
- Gender
- Male
- Therapeutic area
- Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
- Trial duration
- 26 Feb 2026 → ongoing
- Decision date (initial)
- 2024-01-19
- Transition trial
- No
- Low-intervention
- No
- Rare-disease indication
- Yes
- Vulnerable population
- Yes
External identifiers
- EU CT number
- 2023-506480-34-00
- ClinicalTrials.gov
- NCT06092034
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Efficacy, Therapy, Safety
The primary objective of this study is to evaluate whether RP-A501 (an AAV9 capsid containing the LAMP2B transgene; AAV9.LAMP2B) will result in significant improvement as assessed via the primary endpoint comprised of LAMP2 myocardial tissue expression and LVMI.
Secondary objectives 5
- To assess the impact of RP-A501 on the components of the primary endpoint
- To assess the impact of RP-A501 on biomarker evidence of myocardial injury
- To assess the impact of RP-A501 on quality of life and heart failure symptoms
- To assess the impact of RP-A501 on event-free survival
- To assess the impact of RP-A501 on safety
Conditions and MedDRA coding
Danon Disease
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 20.0 | LLT | 10071756 | Danon disease | 10010331 |
Regulatory references
- Scientific advice from competent authorities
- European Medicines Agency
- Plan to share IPD
- No
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 8
- Documentation of a pathogenic or likely pathogenic variant of the LAMP2 gene.
- Male gender.
- Age ≥8 years.
- Evidence of left ventricular hypertrophy with preserved systolic function phenotype as defined by each of the following: a. For subjects < 18 years, z-score of the left ventricular posterior wall or interventricular septum at end diastole ≥+ 2, and for subjects ≥18 years, left ventricular posterior wall or interventricular septum at end diastole >13 mm (>12 mm if family history of clinically significant Danon disease), b. Left ventricular ejection fraction (LVEF) ≥ 50%.
- New York Heart Association (NYHA) Class II to III.
- hsTnI ≥20% above the ULN
- Ability to comply with study procedures including investigational therapy and follow-up evaluations.
- Has received approved vaccination against Neisseria meningitidis ≥ 6 weeks before administration of RP-A501.
Exclusion criteria 8
- Anti-AAV9 neutralizing antibody titer >1:40.
- Intravenous inotropic, vasodilator, or diuretic therapy within the 30 days prior to enrollment.
- Presence or requirement for mechanical circulatory support (MCS).
- Presence or requirement for mechanical ventilation.
- History of intracardiac thrombosis or arterial thromboembolic events including stroke, transient ischemic attack (TIA), acute coronary syndrome, myocardial infarction or unstable angina.
- Prior cardiovascular (CV) surgery, percutaneous coronary intervention (PCI), or valvuloplasty.
- Greater than moderate valvular stenosis or regurgitation on most recent echocardiographic assessment.
- Prior cardiac or other organ (lung, liver, other) transplantation.
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 1
- Primary endpoint including myocardial tissue expression of LAMP2 protein and decrease in left ventricular mass index (LVMI). Evaluation of efficacy associated with RP-A501.
Secondary endpoints 3
- LAMP2 protein expression, LVMI, hsTnI, NT-proBNP, KCCQ, and NYHA class. Evaluation of efficacy associated with RP-A501.
- Event free survival with events defined as death, heart transplant, mechanical circulatory support (MCS) or heart failure hospitalization. Evaluation of efficacy associated with RP-A501.
- Incidence, severity and duration of treatment emergent safety events. Evaluation of safety.
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 1
PRD10873488 · Product
- Active substance
- Adeno-Associated Virus Serotype 9 Vector Containing the Human LAMP2 Isoform B Transgene
- Substance synonyms
- RP-A501, Recombinant adeno-associated virus serotype 9 vector containing the human-lysosome-associated membrane glycoprotein 2 isoform B transgene
- Other product name
- Recombinant Adeno-associated virus serotype 9 vector containing the human-lysosome-associated membrane glycoprotein 2 isoform B transgene
- Pharmaceutical form
- SOLUTION FOR INFUSION
- Route of administration
- INTRAVENOUS INFUSION
- Max daily dose
- 67000000000000 Other
- Max total dose
- 67000000000000 Other
- Max treatment duration
- 1 Day(s)
- Authorisation status
- Not Authorised
- ATC code
- A16AX — VARIOUS ALIMENTARY TRACT AND METABOLISM PRODUCTS
- MA holder
- ROCKET PHARMACEUTICALS, INC
- Paediatric formulation
- No
- Orphan designation
- Yes
- Orphan designation number
- EMA/OD/0000141142
Auxiliary 22
Epysqli 300 mg concentrate for solution for infusion
PRD10444544 · Product
- Active substance
- Eculizumab
- Substance synonyms
- ABP-959, H5G1.1
- Pharmaceutical form
- SOLUTION FOR INFUSION
- Route of administration
- INTRAVENOUS ADMINISTRATION
- Max daily dose
- 1200 mg milligram(s)
- Max total dose
- 1200 mg milligram(s)
- Max treatment duration
- 3 Month(s)
- Authorisation status
- Authorised
- ATC code
- L04AA25 — -
- Marketing authorisation
- EU/1/23/1735/001
- MA holder
- SAMSUNG BIOEPIS NL B.V.
- MA country
- EU
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
SUB12570MIG · Substance
- Active substance
- Rituximab
- Pharmaceutical form
- CONCENTRATE FOR SOLUTION FOR INFUSION
- Route of administration
- INTRAVENOUS INFUSION
- Max daily dose
- 750 mg/m2 milligram(s)/square meter
- Max total dose
- 750 mg/m2 milligram(s)/square meter
- Max treatment duration
- 2 Week(s)
- Authorisation status
- Authorised
- ATC code
- - — -
- Marketing authorisation
- -
- Paediatric formulation
- No
- Orphan designation
- Yes
- Orphan designation number
- EU/3/17/1869
- Modified vs. Marketing Authorisation
- No
BEKEMV 300 mg concentrate for solution for infusion
PRD10347676 · Product
- Active substance
- Eculizumab
- Substance synonyms
- ABP-959, H5G1.1
- Pharmaceutical form
- CONCENTRATE FOR SOLUTION FOR INFUSION
- Route of administration
- INTRAVENIOUS INFUSION
- Max daily dose
- 1200 mg milligram(s)
- Max total dose
- 1200 mg milligram(s)
- Max treatment duration
- 3 Month(s)
- Authorisation status
- Authorised
- ATC code
- L04AA25 — -
- Marketing authorisation
- EU/1/23/1727/001
- MA holder
- AMGEN TECHNOLOGY (IRELAND) UC
- MA country
- EU
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
SUB10537MIG · Substance
- Active substance
- Sirolimus
- Pharmaceutical form
- COATED TABLET
- Route of administration
- ORAL USE
- Max daily dose
- 2 mg milligram(s)
- Max total dose
- 2 mg milligram(s)
- Max treatment duration
- 3 Month(s)
- Authorisation status
- Authorised
- ATC code
- - — -
- Marketing authorisation
- -
- Paediatric formulation
- No
- Orphan designation
- Yes
- Orphan designation number
- EU/3/17/1910
- Modified vs. Marketing Authorisation
- No
SUB09611MIG · Substance
- Active substance
- Paracetamol
- Pharmaceutical form
- TABLET
- Route of administration
- ORAL USE
- Max daily dose
- 1 g gram(s)
- Max total dose
- 1 g gram(s)
- Max treatment duration
- 2 Week(s)
- Authorisation status
- Authorised
- ATC code
- - — -
- Marketing authorisation
- -
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Methylprednisolone Sodium Succinate
SUB14562MIG · Substance
- Active substance
- Methylprednisolone Sodium Succinate
- Pharmaceutical form
- POWDER FOR SOLUTION FOR INFUSION
- Route of administration
- INTRAVENOUS USE
- Max daily dose
- 0.8 mg/kg milligram(s)/kilogram
- Max total dose
- 0.8 mg/kg milligram(s)/kilogram
- Max treatment duration
- 4 Day(s)
- Authorisation status
- Authorised
- ATC code
- - — -
- Marketing authorisation
- -
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
SUB10020MIG · Substance
- Active substance
- Prednisone
- Pharmaceutical form
- TABLET
- Route of administration
- ORAL USE
- Max daily dose
- 60 mg milligram(s)
- Max total dose
- 60 mg milligram(s)
- Max treatment duration
- 3 Month(s)
- Authorisation status
- Authorised
- ATC code
- - — -
- Marketing authorisation
- -
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
SUB01769MIG · Substance
- Active substance
- Diphenhydramine Hydrochloride
- Pharmaceutical form
- TABLET
- Route of administration
- ORAL USE
- Max daily dose
- 50 mg milligram(s)
- Max total dose
- 50 mg milligram(s)
- Max treatment duration
- 2 Week(s)
- Authorisation status
- Authorised
- ATC code
- - — -
- Marketing authorisation
- -
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
SUB10020MIG · Substance
- Active substance
- Prednisone
- Pharmaceutical form
- TABLET
- Route of administration
- ORAL USE
- Max daily dose
- 60 mg milligram(s)
- Max total dose
- 60 mg milligram(s)
- Max treatment duration
- 3 Month(s)
- Authorisation status
- Authorised
- ATC code
- - — -
- Marketing authorisation
- -
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
SUB12570MIG · Substance
- Active substance
- Rituximab
- Pharmaceutical form
- CONCENTRATE FOR SOLUTION FOR INFUSION
- Route of administration
- INTRAVENOUS INFUSION
- Max daily dose
- 750 mg/m2 milligram(s)/square meter
- Max total dose
- 750 mg/m2 milligram(s)/square meter
- Max treatment duration
- 2 Week(s)
- Authorisation status
- Authorised
- ATC code
- - — -
- Marketing authorisation
- -
- Paediatric formulation
- No
- Orphan designation
- Yes
- Orphan designation number
- EU/3/17/1869
- Modified vs. Marketing Authorisation
- No
SUB01769MIG · Substance
- Active substance
- Diphenhydramine Hydrochloride
- Pharmaceutical form
- TABLET
- Route of administration
- ORAL USE
- Max daily dose
- 50 mg milligram(s)
- Max total dose
- 50 mg milligram(s)
- Max treatment duration
- 2 Week(s)
- Authorisation status
- Authorised
- ATC code
- - — -
- Marketing authorisation
- -
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
SUB10537MIG · Substance
- Active substance
- Sirolimus
- Pharmaceutical form
- COATED TABLET
- Route of administration
- ORAL USE
- Max daily dose
- 2 mg milligram(s)
- Max total dose
- 2 mg milligram(s)
- Max treatment duration
- 3 Month(s)
- Authorisation status
- Authorised
- ATC code
- - — -
- Marketing authorisation
- -
- Paediatric formulation
- No
- Orphan designation
- Yes
- Orphan designation number
- EU/3/17/1910
- Modified vs. Marketing Authorisation
- No
SUB10537MIG · Substance
- Active substance
- Sirolimus
- Pharmaceutical form
- COATED TABLET
- Route of administration
- ORAL USE
- Max daily dose
- 2 mg milligram(s)
- Max total dose
- 2 mg milligram(s)
- Max treatment duration
- 3 Month(s)
- Authorisation status
- Authorised
- ATC code
- - — -
- Marketing authorisation
- -
- Paediatric formulation
- No
- Orphan designation
- Yes
- Orphan designation number
- EU/3/17/1910
- Modified vs. Marketing Authorisation
- No
BEKEMV 300 mg concentrate for solution for infusion
PRD10458812 · Product
- Active substance
- Eculizumab
- Substance synonyms
- ABP-959, H5G1.1
- Pharmaceutical form
- SOLUTION FOR INFUSION
- Route of administration
- INTRAVENIOUS INFUSION
- Max daily dose
- 1200 mg milligram(s)
- Max total dose
- 1200 mg milligram(s)
- Max treatment duration
- 3 Month(s)
- Authorisation status
- Authorised
- ATC code
- L04AA25 — -
- Marketing authorisation
- EU/1/23/1727/001
- MA holder
- AMGEN TECHNOLOGY (IRELAND) UC
- MA country
- Norway
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Methylprednisolone Sodium Succinate
SUB14562MIG · Substance
- Active substance
- Methylprednisolone Sodium Succinate
- Pharmaceutical form
- POWDER FOR SOLUTION FOR INFUSION
- Route of administration
- INTRAVENOUS USE
- Max daily dose
- 0.8 mg/kg milligram(s)/kilogram
- Max total dose
- 0.8 mg/kg milligram(s)/kilogram
- Max treatment duration
- 4 Week(s)
- Authorisation status
- Authorised
- ATC code
- - — -
- Marketing authorisation
- -
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
BEKEMV 300 mg concentrate for solution for infusion
PRD10421748 · Product
- Active substance
- Eculizumab
- Substance synonyms
- ABP-959, H5G1.1
- Pharmaceutical form
- SOLUTION FOR INFUSION
- Route of administration
- INTRAVENIOUS INFUSION
- Max daily dose
- 1200 mg milligram(s)
- Max total dose
- 1200 mg milligram(s)
- Max treatment duration
- 3 Month(s)
- Authorisation status
- Authorised
- ATC code
- L04AA25 — -
- Marketing authorisation
- EU/1/23/1727/001
- MA holder
- AMGEN TECHNOLOGY (IRELAND) UC
- MA country
- Iceland
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
SUB10020MIG · Substance
- Active substance
- Prednisone
- Pharmaceutical form
- TABLET
- Route of administration
- ORAL USE
- Max daily dose
- 60 mg milligram(s)
- Max total dose
- 60 mg milligram(s)
- Max treatment duration
- 3 Month(s)
- Authorisation status
- Authorised
- ATC code
- - — -
- Marketing authorisation
- -
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
SUB10020MIG · Substance
- Active substance
- Prednisone
- Pharmaceutical form
- TABLET
- Route of administration
- ORAL USE
- Max daily dose
- 60 mg milligram(s)
- Max total dose
- 60 mg milligram(s)
- Max treatment duration
- 3 Month(s)
- Authorisation status
- Authorised
- ATC code
- - — -
- Marketing authorisation
- -
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
SUB10020MIG · Substance
- Active substance
- Prednisone
- Pharmaceutical form
- TABLET
- Route of administration
- ORAL USE
- Max daily dose
- 60 mg milligram(s)
- Max total dose
- 60 mg milligram(s)
- Max treatment duration
- 3 Month(s)
- Authorisation status
- Authorised
- ATC code
- - — -
- Marketing authorisation
- -
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
SUB25187 · Substance
- Active substance
- Eculizumab
- Pharmaceutical form
- SOLUTION FOR INFUSION
- Route of administration
- INTRAVENOUS INFUSION
- Max daily dose
- 1200 mg milligram(s)
- Max total dose
- 1200 mg milligram(s)
- Max treatment duration
- 3 Month(s)
- Authorisation status
- Authorised
- ATC code
- - — -
- Marketing authorisation
- -
- Paediatric formulation
- No
- Orphan designation
- Yes
- Orphan designation number
- EU/3/14/1304
- Modified vs. Marketing Authorisation
- No
SUB09611MIG · Substance
- Active substance
- Paracetamol
- Pharmaceutical form
- TABLET
- Route of administration
- ORAL USE
- Max daily dose
- 1 g gram(s)
- Max total dose
- 1 g gram(s)
- Max treatment duration
- 2 Week(s)
- Authorisation status
- Authorised
- ATC code
- - — -
- Marketing authorisation
- -
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
SUB10537MIG · Substance
- Active substance
- Sirolimus
- Pharmaceutical form
- ORAL SOLUTION
- Route of administration
- ORAL USE
- Max daily dose
- 2 mg milligram(s)
- Max total dose
- 2 mg milligram(s)
- Max treatment duration
- 3 Month(s)
- Authorisation status
- Authorised
- ATC code
- - — -
- Marketing authorisation
- -
- Paediatric formulation
- No
- Orphan designation
- Yes
- Orphan designation number
- EU/3/17/1910
- Modified vs. Marketing Authorisation
- No
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Rocket Pharmaceuticals Inc.
- Sponsor organisation
- Rocket Pharmaceuticals Inc.
- Address
- 9 Cedarbrook Drive
- City
- Cranbury
- Postcode
- 08512-3618
- Country
- United States
Scientific contact point
- Organisation
- Rocket Pharmaceuticals Inc.
- Contact name
- Kinnari Patel
Public contact point
- Organisation
- Rocket Pharmaceuticals Inc.
- Contact name
- Miriam Zeini Moreno
Third parties 7
| Organisation | City, country | Duties |
|---|---|---|
| Bioagilytix Labs LLC ORG-100013030
|
Durham, United States | Laboratory analysis |
| Labcorp Central Laboratory Services S.a.r.l. ORG-100011524
|
Meyrin, Switzerland | Laboratory analysis |
| CTI Clinical Trial and Consulting Services Europe GmbH ORG-100008276
|
Ulm, Germany | On site monitoring, Code 10, Code 12, Code 5, Data management |
| Eresearchtechnology Inc. ORG-100013039
|
Philadelphia, United States | Other |
| Premier Research s.r.o. ORG-100047985
|
Petrzalka, Slovakia | Code 8 |
| Fisher Bioservices Inc. ORG-100011655
|
Frederick, United States | Other |
| Labcorp Early Development Laboratories Inc. ORG-100012865
|
Greenfield, United States | Laboratory analysis |
Locations
2 EU/EEA countries · 2 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Germany | Ongoing, recruiting | 2 | 1 |
| Italy | Ongoing, recruiting | 2 | 1 |
| Rest of world
United States, United Kingdom
|
— | 10 | — |
Investigational sites
Deutsches Herzzentrum Muenchen Des Freistaates Bayern Klinik An Der Technischen Universitaet Muenchen
Department of Congenital Heart Defects and Pediatric Cardiology, Lazarettstrasse 36, Neuhausen-Nymphenburg, Munich
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 |
|---|---|---|---|---|---|
| Germany | 2024-06-18 | 2024-07-08 | |||
| Italy | 2024-10-29 | 2025-01-20 |
Oversight and notifications
Regulatory notifications under CTR Articles 38, 52, 53, 54 and 77
Temporary halts 2 · Art. 38 CTR
Temporary halt TH-84979
- Halt date
- 2025-05-25
- Member states concerned
- Italy
- Publication date
- 2025-06-02
- Reason
- Sponsor decision, Safety related (clinical or pre-clinical results)
- Explanation
- Per protocol, study enrollment was suspended and planned treatment of any new subjects with RP-A501 was halted on 07-May-2025. This was based on previously reported SAEs events (CIOMS submission dates of 15-May-2025 and 20-May-2025). Subsequently, the Sponsor received FDA notification of clinical hold. One subject developed bacteremia and sepsis which subsequently resulted in death on 25-May-2025.
- Follow-up measures
- An investigation is currently ongoing. Per protocol, study enrollment was suspended and planned treatment of any new subjects with RP-A501 was halted on 07-May-2025.
- Benefit-risk balance changed
- Yes
- Treatment stopped
- Yes
Temporary halt TH-84977
- Halt date
- 2025-05-25
- Member states concerned
- Germany
- Publication date
- 2025-06-02
- Reason
- Sponsor decision, Safety related (clinical or pre-clinical results)
- Explanation
- Per protocol, study enrollment was suspended and planned treatment of any new subjects with RP-A501 was halted on 07-May-2025. This was based on previously reported SAEs events (CIOMS submission dates of 15-May-2025 and 20-May-2025). Subsequently, the Sponsor received FDA notification of clinical hold. One subject developed bacteremia and sepsis which subsequently resulted in death on 25-May-2025.
- Follow-up measures
- An investigation is currently ongoing. Per protocol, study enrollment was suspended and planned treatment of any new subjects with RP-A501 was halted on 07-May-2025.
- Benefit-risk balance changed
- Yes
- Treatment stopped
- Yes
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 40 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | D1_RP-A501-0123_Clinical Risk Management Plan 2023-506480-34-00_Redacted | 1 |
| Protocol (for publication) | D1_RP-A501-0123_Laboratory Manual 2023-506480-34-00_Redacted | 1 |
| Protocol (for publication) | D1_RP-A501-0123_Pharmacy Manual 2023-506480-34-00_Redacted | 1 |
| Protocol (for publication) | D1_RP-A501-0123_Protocol 2023-506480-34-00_Redacted | 5.0 |
| Protocol (for publication) | D4_RP-A501-0123_Patient facing documents KCCQ12 SO_DE | 1.0 |
| Protocol (for publication) | D4_RP-A501-0123_Patient facing documents KCCQ12_DE_Redacted | na |
| Protocol (for publication) | D4_RP-A501-0123_Patient facing documents KCCQ12_IT_Redacted | na |
| Protocol (for publication) | D4_RP-A501-0123_PFD_Sponsor statement_Licensed PFD | na |
| Protocol (for publication) | D4_RP-A501-0123_PFD_Sponsor statement_Licensed PFD_IT | na |
| Recruitment arrangements (for publication) | K1_RP-A501-0123_DE_Recruitment arrangements_eng | 1 |
| Recruitment arrangements (for publication) | K1_RP-A5010123_IT_Recruitment arrangements_eng | 1 |
| Subject information and informed consent form (for publication) | L1_RP-A501-0123_DE_SIS and ICF_Adult_ger_Redacted | 4.0 |
| Subject information and informed consent form (for publication) | L1_RP-A501-0123_DE_SIS and ICF_Assent 12-17_ger_Redacted | 4.0 |
| Subject information and informed consent form (for publication) | L1_RP-A501-0123_DE_SIS and ICF_Assent 8-11_ger_Redacted | 4.0 |
| Subject information and informed consent form (for publication) | L1_RP-A501-0123_DE_SIS and ICF_Parent_ger_Redacted | 4.0 |
| Subject information and informed consent form (for publication) | L1_RP-A5010123_IT_SIS and ICF_Adult_ITA_Redacted | 3.0 |
| Subject information and informed consent form (for publication) | L1_RPA5010123_IT_SIS and ICF_Assent 12-17_ITA_Redacted | 3.0 |
| Subject information and informed consent form (for publication) | L1_RPA5010123_IT_SIS and ICF_Assent 8-11_ITA_Redacted | 3.0 |
| Subject information and informed consent form (for publication) | L1_RPA5010123_IT_SIS and ICF_Parent_ITA_Redacted | 3.0 |
| Subject information and informed consent form (for publication) | L2_RP-A501-0123_DE_Other subj inf material_ PatientPrimary_Payment card_ger | 1.0 |
| Subject information and informed consent form (for publication) | L2_RP-A501-0123_DE_Other subj inf material_ PatientPrimary_Personal Data Consent Form_ger | 1.0 |
| Subject information and informed consent form (for publication) | L2_RP-A501-0123_DE_Other subj inf material_ PatientPrimary_Welcome letter_ger | 1.0 |
| Subject information and informed consent form (for publication) | L2_RP-A501-0123_DE_Other subj inf material_PatientPrimary_Expense Claim Form_ger_Redacted | 1.0 |
| Subject information and informed consent form (for publication) | L2_RP-A501-0123_DE_Other subj inf material_PatientPrimary_Privacy Notice_ger | 2.0 |
| Subject information and informed consent form (for publication) | L2_RP-A501-0123_DE_Other subj inf material_Primarius_App Setup_ger | 3.0 |
| Subject information and informed consent form (for publication) | L2_RP-A501-0123_DE_Other subj inf material_Primarius_Screenshots_ger | 4.0 |
| Subject information and informed consent form (for publication) | L2_RP-A501-0123_IT_Other subj info mat_PatientPrimary Personal Data Consent Form_ita | 1.0 |
| Subject information and informed consent form (for publication) | L2_RP-A501-0123_IT_Other subj info mat_PatientPrimary_Patient Welcome Letter_ita | 1.0 |
| Subject information and informed consent form (for publication) | L2_RP-A501-0123_IT_Other subj info material_PatientPrimary_Expense Claim Form_ita_Redacted | 1.0 |
| Subject information and informed consent form (for publication) | L2_RP-A501-0123_IT_Other subject info material_ PatientPrimary_Privacy Notice_ita | 2 |
| Subject information and informed consent form (for publication) | L2_RP-A501-0123_IT_Other subject info material_GP Letter_ita | 1 |
| Subject information and informed consent form (for publication) | L2_RP-A501-0123_IT_Other subject info material_PatientPrimary_Payment Card Letter_ita | 1 |
| Subject information and informed consent form (for publication) | L2_RP-A501-0123_IT_Other subject info material_Primarius_ Screenshots_ita | 4.0 |
| Subject information and informed consent form (for publication) | L2_RP-A5010123_IT_Other subject info material_Primarius_App Setup_ita | 3 |
| Synopsis of the protocol (for publication) | D1_RP-A501-0123_Protocol synopsis layperson_DE 2023-506480-34-00 | 2.1 |
| Synopsis of the protocol (for publication) | D1_RP-A501-0123_Protocol synopsis layperson_ENG 2023-506480-34-00 | 2.1 |
| Synopsis of the protocol (for publication) | D1_RP-A501-0123_Protocol synopsis layperson_IT 2023-506480-34-00 | 2.3 |
| Synopsis of the protocol (for publication) | D1_RP-A501-0123_Protocol synopsis_DE 2023-506480-34-00_Redacted | 5.0 |
| Synopsis of the protocol (for publication) | D1_RP-A501-0123_Protocol synopsis_ENG 2023-506480-34-00_Redacted | 5.0 |
| Synopsis of the protocol (for publication) | D1_RP-A501-0123_Protocol synopsis_IT 2023-506480-34-00_Redacted | 5.0 |
Application history
5 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2023-10-20 | Germany | Acceptable 2024-01-18
|
2024-01-19 |
| 2 | SUBSTANTIAL MODIFICATION | SM-1 | 2024-04-15 | Germany | Acceptable 2024-05-15
|
2024-05-17 |
| 3 | SUBSEQUENT ADDITION OF MSC | APP-3 | 2024-06-10 | Acceptable 2024-05-15
|
2024-08-28 | |
| 4 | SUBSTANTIAL MODIFICATION | SM-2 | 2024-11-21 | Germany | Acceptable 2025-02-11
|
2025-02-11 |
| 5 | SUBSTANTIAL MODIFICATION | SM-4 | 2025-11-07 | Germany | Acceptable 2026-02-23
|
2026-02-25 |