Evidence map›Paper›PMID 41968461›Full record

Trial reportHaemophilia : the official journal of the World Federation of Hemophilia

Final Analysis of the Phase 1/2 Trial of Valoctocogene Roxaparvovec for Severe Haemophilia A.

Priyanka Raheja, Savita Rangarajan, Will Lester, Bella Madan, Glenn F Pierce, Emily Symington, Carolyn Millar, Dane Osmond, Mingjin Li, Konstantia-Maria Chavele

Registry-linked trialAbstract readClinical Trial, Phase IClinical Trial, Phase IIMulticenter Study
PubMed Full text at PMC Publisher
In one paragraph

Trial report in Haemophilia : the official journal of the World Federation of Hemophilia. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT02576795 (A Phase 1/2, Dose-Escalation, Safety, Tolerability and Efficacy Study of Valoctocogene Roxaparvovec, an Adenovirus-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Patients With Severe Haemophilia A), which is not on this map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

NCT02576795 phase1 / phase2completednot on this map

A Phase 1/2, Dose-Escalation, Safety, Tolerability and Efficacy Study of Valoctocogene Roxaparvovec, an Adenovirus-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Patients With Severe Haemophilia A

TypeinterventionalSponsorBioMarin PharmaceuticalRan2015 to 2024Enrolled15ConditionsSevere Haemophilia AArmsvaloctocogene roxaparvovec
3 · Its place in the literature

Who cites it

0 citing papers in PubMed.

No citing paper in PubMed yet.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

10 authors.

Priyanka RahejaHaemophilia Centre, Barts Health NHS Trust, The Royal London Hospital, London, UK.ORCID https://orcid.org/0000-0001-6365-2899
Savita RangarajanFaculty of Medicine, University of Southampton, Southampton, UK.
Will LesterUniversity Hospitals Birmingham NHS Foundation Trust, Birmingham, UK.ORCID https://orcid.org/0000-0001-8790-7112
Bella MadanGuy's and St Thomas' NHS Foundation Trust, London, UK.
Glenn F PierceIndependent Consultant, La Jolla, San Diego, California, USA.ORCID https://orcid.org/0000-0002-3310-328X
Emily SymingtonHaemophilia Centre, Cambridge University Hospitals NHS Foundation Trust, Cambridge, UK.ORCID https://orcid.org/0000-0003-2529-8168
Carolyn MillarImperial College Healthcare NHS Trust and Centre for Haematology, Department of Immunology and Inflammation, Imperial College London, London, UK.
Dane OsmondBioMarin Pharmaceutical Inc., Novato, California, USA.
Mingjin LiBioMarin Pharmaceutical Inc., Novato, California, USA.
Konstantia-Maria ChaveleBioMarin Pharmaceutical Inc., Novato, California, USA.

Funding

BioMarin Pharmaceutical
6 · The paper itself

Abstract

introductionValoctocogene roxaparvovec is an adeno-associated virus vector serotype 5 (AAV5)-mediated gene therapy for severe haemophilia A (HA).

aimReport the final safety and efficacy results of the phase 1/2 trial of valoctocogene roxaparvovec.

methodsAn open-label phase 1/2 trial (NCT02576795) enrolled adult males with severe HA (factor VIII [FVIII] ≤1 IU/dL) without FVIII inhibitors, anti-AAV5 antibodies, or liver dysfunction to receive 6 × 10

resultsFive of six 4 × 10

conclusionOver 7 years, valoctocogene roxaparvovec increased FVIII activity from baseline and improved haemostasis compared with FVIII prophylaxis for most participants. The 6 × 10

Indexed as

DependovirusGenetic TherapyGenetic VectorsHemophilia AAdultFactor VIIIGene Therapy AgentsHumansMaleMiddle AgedQuality of LifeRecombinant Fusion ProteinsTreatment OutcomeYoung AdultFactor VIIIRecombinant Fusion ProteinsValoctocogene Roxaparvovecadeno‐associated virusclinical trialfactor VIIIgene therapyHaemophilia

Identifiers

PMID41968461
PMCPMC13378670

What OpenQuestion holds

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Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.