- Trials with a EudraCT protocol (15)
- Paediatric studies in scope of Art45 of the Paediatric Regulation (0)
15 result(s) found for: Capsid protein.
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EudraCT Number: 2016-000925-38 | Sponsor Protocol Number: UCL13/0076 | Start Date*: 2017-01-04 | ||||||||||||||||
Sponsor Name:University College London (UCL) | ||||||||||||||||||
Full Title: GO-8: Gene therapy for haemophilia A using a novel serotype 8 capsid pseudotyped adeno-associated viral vector encoding Factor VIII-V3 | ||||||||||||||||||
Medical condition: Severe Haemophilia A | ||||||||||||||||||
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Population Age: Adults, Elderly | Gender: Male | |||||||||||||||||
Trial protocol: GB (GB - no longer in EU/EEA) | ||||||||||||||||||
Trial results: (No results available) |
EudraCT Number: 2014-003880-38 | Sponsor Protocol Number: 270-201 | Start Date*: 2015-06-01 |
Sponsor Name:BioMarin Pharmaceutical Inc. | ||
Full Title: A Phase 1/2, Dose-Escalation Safety, Tolerability and Efficacy Study of BMN 270, an Adenovirus-Associated Virus Vector–Mediated Gene Transfer of Human Factor VIII in Patients with Severe Haemophilia A | ||
Medical condition: Haemophilia A | ||
Disease: | ||
Population Age: Adults | Gender: Male | |
Trial protocol: GB (GB - no longer in EU/EEA) | ||
Trial results: View results |
EudraCT Number: 2014-001874-32 | Sponsor Protocol Number: RDC-CNGA3-01 | Start Date*: 2015-09-09 | |||||||||||
Sponsor Name:Universitätsklinikum Tübingen | |||||||||||||
Full Title: Safety and efficacy of a bilateral single subretinal injection of rAAV.hCNGA3 in adult and minor patients with CNGA3-linked achromatopsia investigated in a randomized, wait list controlled, observe... | |||||||||||||
Medical condition: CNGA3-linked achromatopsia | |||||||||||||
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Population Age: Children, Under 18, Adults | Gender: Male, Female | ||||||||||||
Trial protocol: DE (Trial now transitioned) | |||||||||||||
Trial results: (No results available) |
EudraCT Number: 2005-005711-17 | Sponsor Protocol Number: AGT4HB | Start Date*: 2009-09-25 | ||||||||||||||||
Sponsor Name:St. Jude Children’s Research Hospital | ||||||||||||||||||
Full Title: AN OPEN LABEL DOSE-ESCALATION STUDY OF A SELF COMPLEMENTARY ADENO-ASSOCIATED VIRAL VECTOR (scAAV2/8-LP1-hFIXco) FOR GENE TRANSFER IN SUBJECTS WITH HEMOPHILIA B | ||||||||||||||||||
Medical condition: Gene therapy for Haemophilia B | ||||||||||||||||||
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Population Age: Adults | Gender: Male | |||||||||||||||||
Trial protocol: GB (Temporarily Halted) | ||||||||||||||||||
Trial results: (No results available) |
EudraCT Number: 2013-005579-42 | Sponsor Protocol Number: CT-AMT-060-01 | Start Date*: 2015-03-20 | |||||||||||
Sponsor Name:uniQure biopharma B.V. | |||||||||||||
Full Title: A phase I/II, open-label, uncontrolled, single-dose, dose-ascending, multi-centre trial investigating an adeno-associated viral vector containing a codon-optimized human factor IX gene (AAV5-hFIX) ... | |||||||||||||
Medical condition: Haemophilia B | |||||||||||||
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Population Age: Adults, Elderly | Gender: Male | ||||||||||||
Trial protocol: DE (Completed) DK (Completed) NL (Completed) | |||||||||||||
Trial results: View results |
EudraCT Number: 2017-004305-40 | Sponsor Protocol Number: CSL222-3001 | Start Date*: 2018-10-12 | |||||||||||
Sponsor Name:CSL Behring LLC | |||||||||||||
Full Title: Phase III, open-label, single-dose, multi-center multinational trial investigating a serotype 5 adeno-associated viral vector containing the Padua variant of a codon-optimized human factor IX gene ... | |||||||||||||
Medical condition: Hemophilia B | |||||||||||||
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Population Age: Adults, Elderly | Gender: Male | ||||||||||||
Trial protocol: DK (Trial now transitioned) GB (GB - no longer in EU/EEA) IE (Trial now transitioned) NL (Trial now transitioned) DE (Trial now transitioned) BE (Trial now transitioned) SE (Trial now transitioned) IT (Completed) | |||||||||||||
Trial results: (No results available) |
EudraCT Number: 2018-004024-11 | Sponsor Protocol Number: SHP648-101 | Start Date*: 2020-05-04 | |||||||||||
Sponsor Name:Baxalta Innovations GmbH | |||||||||||||
Full Title: An Open-Label, Multinational, Phase 1/2 Study of the Safety and Dose Escalation of SHP648, an Adeno-Associated Virus Serotype 8 (AAV8) Vector Expressing FIX Padua in Hemophilia B Subjects | |||||||||||||
Medical condition: Hemophilia B is a X-linked recessive bleeding disorder caused by mutations in the gene encoding clotting factor IX (FIX) that result in disruption of the normal clotting pathway. Hemophilia B affec... | |||||||||||||
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Population Age: Adults, Elderly | Gender: Male | ||||||||||||
Trial protocol: DE (Prematurely Ended) HU (Prematurely Ended) ES (Prematurely Ended) AT (Prematurely Ended) FR (Prematurely Ended) | |||||||||||||
Trial results: View results |
EudraCT Number: 2021-001079-18 | Sponsor Protocol Number: FLT180a-06 | Start Date*: 2022-04-27 | |||||||||||
Sponsor Name:Freeline Therapeutics Ltd | |||||||||||||
Full Title: A dose confirmation study of FLT180a (adeno-associated viral vector containing the Padua variant of a codon-optimized human Factor IX gene) in adult subjects with hemophilia B. | |||||||||||||
Medical condition: Haemophilia B. | |||||||||||||
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Population Age: Adults, Elderly | Gender: Male | ||||||||||||
Trial protocol: AT (Prematurely Ended) | |||||||||||||
Trial results: View results |
EudraCT Number: 2019-004451-37 | Sponsor Protocol Number: C3731003 | Start Date*: 2021-09-24 | |||||||||||
Sponsor Name:Pfizer Inc., 235 East 42nd Street, New York, NY 10017 | |||||||||||||
Full Title: Phase 3, Open-Label, Single-Arm Study to Evaluate the Efficacy and Safety of PF-07055480 (Recombinant AAV2/6 Human Factor VIII Gene Therapy) in Adult Male Participants with Moderately Severe to Sev... | |||||||||||||
Medical condition: hemophilia A | |||||||||||||
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Population Age: Adults | Gender: Male | ||||||||||||
Trial protocol: DE (Trial now transitioned) FR (Trial now transitioned) IT (Completed) ES (Restarted) | |||||||||||||
Trial results: (No results available) |
EudraCT Number: 2013-002090-21 | Sponsor Protocol Number: IM103177 | Start Date*: 2016-02-02 | |||||||||||
Sponsor Name:Bristol-Myers Squibb International Corporation | |||||||||||||
Full Title: Evaluation of Acute Rejection Rates in de novo Renal Transplant Recipients Following Thymoglobulin Induction, CNI-free, Nulojix (belatacept) -based Immunosuppression | |||||||||||||
Medical condition: Maintenance of renal transplant recipients. | |||||||||||||
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Population Age: Adults, Elderly | Gender: Male, Female | ||||||||||||
Trial protocol: DE (Completed) AT (Completed) | |||||||||||||
Trial results: View results |
EudraCT Number: 2019-001283-30 | Sponsor Protocol Number: SPK-3006-101 | Start Date*: 2020-06-16 | |||||||||||
Sponsor Name:Spark Therapeutics | |||||||||||||
Full Title: Phase 1/2, dose-escalation study to evaluate the safety, tolerability and efficacy of a single intravenous infusion of SPK-3006 in adults with late-onset Pompe disease | |||||||||||||
Medical condition: Pompe Disease (also known as glycogen storage disease type II) | |||||||||||||
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Population Age: Adults | Gender: Male, Female | ||||||||||||
Trial protocol: FR (Prematurely Ended) DE (Trial now transitioned) NL (Ongoing) DK (Prematurely Ended) | |||||||||||||
Trial results: (No results available) |
EudraCT Number: 2022-001157-23 | Sponsor Protocol Number: GO44096 | Start Date*: 2023-01-03 | |||||||||||
Sponsor Name:F.Hoffmann-La Roche Ltd | |||||||||||||
Full Title: A PHASE II, SINGLE-ARM, OPEN-LABEL STUDY EVALUATING THE SAFETY AND PHARMACOKINETICS OF THE INTRAVENOUS FIXED-DOSE COMBINATION (IV FDC) OF TIRAGOLUMAB AND ATEZOLIZUMAB IN PARTICIPANTS WITH LOCALLY A... | |||||||||||||
Medical condition: Solid Tumors | |||||||||||||
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Population Age: Adults, Elderly | Gender: Male, Female | ||||||||||||
Trial protocol: GR (Trial now transitioned) CY (Trial now transitioned) ES (Ongoing) HR (Trial now transitioned) | |||||||||||||
Trial results: (No results available) |
EudraCT Number: 2020-005229-95 | Sponsor Protocol Number: PBKR03-001 | Start Date*: 2021-08-23 | |||||||||||
Sponsor Name:Passage Bio, Inc. | |||||||||||||
Full Title: A Phase 1/2 Open-Label, Multicenter, Dose Ranging and Confirmatory Study to Assess the Safety, Tolerability and Efficacy of PBKR03 Administered to Pediatric Subjects with Early Infantile Krabbe Dis... | |||||||||||||
Medical condition: Krabbe disease (globoid cell leukodystrophy) is an autosomal recessive lysosomal storage disease (LSD) caused by mutations in the gene encoding the hydrolytic enzyme galactosylceramidase (galactoce... | |||||||||||||
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Population Age: Infants and toddlers, Under 18 | Gender: Male, Female | ||||||||||||
Trial protocol: NL (Prematurely Ended) | |||||||||||||
Trial results: (No results available) |
EudraCT Number: 2015-005576-22 | Sponsor Protocol Number: 201501 | Start Date*: 2018-05-22 | |||||||||||
Sponsor Name:BAXALTA INNOVATIONS GMBH | |||||||||||||
Full Title: A Global, Open-Label, Multicenter, Phase 1/2 Study of the Safety and Dose Escalation of BAX 888, an Adeno-Associated Virus Serotype 8 (AAV8) Vector Expressing B-Domain Deleted Factor VIII (BDD-FVII... | |||||||||||||
Medical condition: Hemophilia A is a rare congenital disease characterized by reduced or absent levels of the coagulation FVIII. It occurs in approximately 1 in 5,000 live male births. Mutations of the FVIII gene res... | |||||||||||||
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Population Age: Adults, Elderly | Gender: Male | ||||||||||||
Trial protocol: IT (Prematurely Ended) | |||||||||||||
Trial results: (No results available) |
EudraCT Number: 2017-001271-23 | Sponsor Protocol Number: C0371004 | Start Date*: 2019-02-20 | ||||||||||||||||
Sponsor Name:Pfizer Inc. | ||||||||||||||||||
Full Title: AN OPEN-LABEL, NON-INVESTIGATIONAL PRODUCT, MULTI-CENTER, LEAD-IN STUDY TO EVALUATE PROSPECTIVE EFFICACY AND SELECTED SAFETY DATA OF CURRENT FACTOR IX (FIX) OR FACTOR VIII (FVIII) PROPHYLAXIS REPLA... | ||||||||||||||||||
Medical condition: Moderately Severe To Severe Adult Hemophilia B Participants (FIX:C≤2%) Who Are Negative For Neutralizing Antibodies (NAb) To Adeno-Associated Virus Vector (AAV) Spark100. Moderately severe to sever... | ||||||||||||||||||
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Population Age: Adults | Gender: Male | |||||||||||||||||
Trial protocol: SE (Completed) GB (GB - no longer in EU/EEA) IE (Completed) DE (Completed) FR (Completed) ES (Ongoing) NL (Ongoing) | ||||||||||||||||||
Trial results: (No results available) |
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