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20/05/2026

Clinical trials at VHIR enable the approval of new therapies for patients

Infermeres d'assaigs clínics

Clinical trials nurses

Infant que participa en un assaig clínic

Child participating in a clinical trial

20/05/2026

In 2025, the institute had 688 active trials, with a special focus on rare diseases and pediatrics.

Clinical trials are an essential tool for developing new treatments, validating their safety and efficacy, and advancing toward more personalized medicine. The Campus Vall d'Hebron, of which the Vall d’Hebron Institut de Recerca (VHIR) is part, once again consolidates its position as an international reference center in clinical research, with sustained activity that directly contributes to the arrival of new therapies for patients.

Throughout 2025, VHIR has maintained intense clinical research activity, launching 244 new clinical trials. In total, it has participated in 1,088 active clinical studies, 688 of which are clinical trials. This activity includes studies across all phases of clinical development, with 71 phase I trials, 211 phase II, 368 phase III, and 38 phase IV. Early-phase studies represent more than 40% of the total, a particularly relevant fact as they are key to validating the safety and efficacy of new therapeutic strategies before clinical use.

VHIR also maintains a strong commitment to research in areas with a high unmet need for new therapeutic options, such as rare diseases and pediatrics. In this regard, there are currently 241 clinical trials linked to rare diseases and 224 studies focused on pediatric populations. At the same time, research in advanced therapies continues to be strengthened, with 32 active clinical trials based on these treatments.

This scientific progress would not be possible without the participation of volunteers and patients. Last year, 825 new patients joined VHIR clinical trials, contributing to the development of new treatments and facilitating access to innovative therapies.

The impact of VHIR’s clinical research is also reflected in the arrival of new medicines into clinical practice. In 2025, the European Medicines Agency (EMA) recommended the approval of 20 new active substances, 8 of which were developed through clinical trials in which VHIR participated. It has also been involved in one out of every three CAR-T therapies approved by the EMA and the Food and Drug Administration (FDA) in fields beyond oncology and hematology.

New treatments in pediatrics, rare diseases, and other conditions

VHIR actively participates in studies that help improve the treatment of diseases in both pediatric and adult populations.

In pediatrics, Vall d’Hebron has participated in the REACH5 study, which demonstrated the long-term benefits of ruxolitinib in children with graft-versus-host disease. Also noteworthy is an international clinical trial showing that the combination of chemotherapy and immunotherapy with dinutuximab beta improves the response to the treatment of neuroblastoma. In addition, VHIR has participated in the study that demonstrated for the first time the efficacy of a preventive migraine treatment in children and adolescents with fremanezumab.

In the field of rare diseases, Vall d’Hebron has played a key role in the development of a treatment for thymidine kinase 2 deficiency (TK2d), a severe rare muscular disorder. The clinical trial showed a significant improvement in patient survival and contributed to the approval of the treatment by the U.S. Food and Drug Administration (FDA).

VHIR clinical research also drives new therapeutic strategies for adults. Recently, an international study with Vall d’Hebron participation showed the benefits of a new oral obesity drug, l’orforglipron, which has been shown to reduce body weight and improve cardiovascular risk factors. This therapy has also recently been approved by the FDA.

In gynecology, a clinical trial using a novel cell therapy has shown promising results in women with Asherman syndrome who had not responded to previous treatments. Meanwhile, the European TRUSTroke project, coordinated by Vall d’Hebron, has launched a clinical study to develop an AI-based solution for monitoring strokek patients.

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