Bijlage IIa: Overzicht van door de CCMO beoordeelde onderzoeksprotocollen met een positief besluit

Dossiernummer

Studietitel

CCMO-categorie

NL82318.000.22

A Phase 2, Multi-Center, Randomized, Placebo-Controlled, Dose-Finding Study Evaluating Efficacy, Safety and Tolerability of Different Doses and Regimens of Allocetra-OTS for the Treatment of Organ Failure in Adult Sepsis Patients

Celtherapie

NL83665.000.23

Adoptive TIL therapy in combination with chemoimmunotherapy in advanced NSCLC patients

Celtherapie

NL82545.000.22

A Phase 1/2 Study to Evaluate the Safety, Tolerability, and Efficacy of VX-880 in Subjects Who Have Type 1 Diabetes Mellitus With Impaired Hypoglycemic Awareness and Severe Hypoglycemia

Celtherapie

NL82815.000.22

Assess effect and safety of intra-arterial autologous mesoangioblasts administration to the upper arm of m.3243A>G mutation carriers

Celtherapie

NL83484.000.22

Safety and Efficacy of MCA-derived Mesenchymal Stromal Cell Therapy in Renal Transplant Recipients: The Nereid Study

Celtherapie

NL75563.000.22

Induction of neo-antigen specific cytotoxic T cells by autologous tumor lysate-loaded specialized crosspresenting dendritic cells in epithelial ovarian cancer patients treated with neoadjuvant chemotherapy, the NEODOC study

Celtherapie

2022-500266-10-00

A double-blind, randomized, placebo-controlled, interventional, multicenter, phase III clinical trial to investigate the safety and efficacy of ABCB5-positive mesenchymal stromal cells (ABCB5+ MSCs) on epidermolysis bullosa (EB)

Celtherapie

2023-508157-15-01 (herindiening)

An Open Label Study to Evaluate the Safety and Immunogenicity of 2 Doses of 100µg BG505 SOSIP.664 gp140 Vaccine, Adjuvanted, given to a Population of Adults in Good General Health Who have Received 3 doses of 300µg BG505 SOSIP. GT 1.1 gp140 Vaccine, Adjuvanted

Vaccin

NL83287.000.23

A Pilot, Multicenter, Randomized, Non Comparative, Double-Blind Study of Phage Therapy in Patients with Hip or Knee Prosthetic Joint Infection due to Staphylococcus aureus Treated with DAIR and Antibiotic Therapy

Product dat levende (micro-)organismen of virussen bevat

NL83488.000.23

A Phase 1b/2a, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate Nebulized Bacteriophage Treatment in Outpatient Adult Cystic Fibrosis (CF) Subjects with Chronic Pseudomonas aeruginosa (PsA) Pulmonary Infection

Product dat levende (micro-)organismen of virussen bevat

NL83210.000.22

ORACLE: A long-term follow-up study to evaluate the safety and durability of GT005 in participants with geographic atrophy, secondary to age-related macular degeneration treated in a Gyroscope-sponsored antecedent study.

Gentherapie / GGO

NL81647.000.22

A Phase 1 Open-label, Multicenter Study Evaluating the Safety and Efficacy of KITE-363, an Autologous Anti-CD19/CD20 CAR T-cell Therapy, in Subjects With Relapsed and/or Refractory B-cell Lymphoma

Gentherapie / GGO

NL82794.000.22

Long-Term Follow-Up of Patients who have received an Autologous Antigen-Specific Chimeric Antigen Receptor T Regulatory Cell Therapy (TX200-TR101) in a prior clinical study.

Gentherapie / GGO

NL83771.000.23

A Phase 1/2, First-in-Human, Open-Label, Accelerated-Titration, Two-Part Clinical Trial of TK-8001 (MAGE-A1-Directed TCR-Transduced Autologous CD8+ T-cells) in Patients with HLA-A*02:01 Genotype and Advanced-Stage/Metastatic, MAGE-A1+ Solid Tumors that Either Have No Further  Approved Therapeutic Alternative(s) or are not Eligible for them or are in a Non-Curable State and Have Received a Minimum of Two Lines of Systemic Therapy

Gentherapie / GGO

NL83142.000.22

A Phase 1/2 Dose-Escalation and Dose-Expansion Study of the Safety and Efficacy of Anti-CD7 Allogeneic CAR-T cells (WU-CART-007) in Patients with Relapsed or Refractory T-cell Acute Lymphoblastic Leukemia (T-ALL)/Lymphoblastic Lymphoma (LBL)

Gentherapie / GGO

NL82130.000.22

Coadministration of genetically attenuated Plasmodium falciparum mei2 (GA2) sporozoites with adjuvants - a proof of principle study

Gentherapie / GGO

NL83819.041.23

Assessing Frequency of HLA-Genotype and Tumor Antigen Expression in Subjects with Relapsed/Refractory, Advanced-Stage Solid Tumors that may Qualify for Novel T Cell Receptor Based  Therapies

Gentherapie / GGO

2023-505806-41-00

A phase I/II trial of MB-dNPM1-TCR.1 in HLA-A*02:01-positive patients with relapsed or refractory NPM1-mutated AML to determine safety and obtain first data on efficacy

Gentherapie / GGO

2022-500746-16-00

A Phase 2b, Randomized, Double-masked, Multicenter, Dose-ranging, Sham-controlled Clinical Trial to Evaluate Intravitreal JNJ-81201887 (AAVCAGsCD59) Compared to Sham Procedure for the Treatment of Geographic Atrophy (GA) Secondary to Age-related Macular Degeneration (AMD)

Gentherapie / GGO

2022-503140-41-00

A Phase 3, multicenter, randomized, open-label, parallel group, treatment study to assess the efficacy and safety of the lifileucel (LN-144, autologous tumor-infiltrating lymphocytes [TIL]) regimen in combination with pembrolizumab compared with pembrolizumab monotherapy in participants with untreated, unresectable or metastatic melanoma

Gentherapie / GGO

2023-504422-19-00

A non-randomized, open-label, uncontrolled, multicenter study to evaluate the safety, tolerability, and efficacy of GLPG5101 for up to 2 years, in adult subjects with treatment-refractory active systemic lupus erythematosus

Gentherapie / GGO

2022-501489-24-00

An Adaptive Phase 3, Randomized, Open-Label, Multicentre Study to Compare the Efficacy and Safety of Yescarta versus Standard of Care Therapy as First-Line Therapy in Subjects with High-Risk Large B-Cell Lymphoma (ZUMA-23)

Gentherapie / GGO

2022-501262-21-00

A Phase 2, Open-Label, Multicenter, Basket Study Evaluating the Safety and Efficacy of Brexucabtagene Autoleucel in Adults with Rare B-cell Malignancies (ZUMA-25) – Substudy D - Relapsed/Refractory Hairy Cell Leukemia (HCL)

Gentherapie / GGO

2022-501261-46-00

A Phase 2, Open-Label, Multicenter, Basket Study Evaluating the Safety and Efficacy of Brexucabtagene Autoleucel in Adults with Rare B-cell Malignancies (ZUMA-25) – Substudy C - Relapsed/Refractory Burkitt Lymphoma

Gentherapie / GGO

2022-501260-18-00

A Phase 2, Open-Label, Multicenter, Basket Study Evaluating the Safety and Efficacy of Brexucabtagene Autoleucel in Adults with Rare B-cell Malignancies (ZUMA 25) – Substudy B – Relapsed/Refractory Richter Transformation (RT)

Gentherapie / GGO

2022-501259-10-00

A Phase 2, Open-Label, Multicenter, Basket Study Evaluating the Safety and Efficacy of Brexucabtagene Autoleucel in Adults with Rare B-cell Malignancies (ZUMA-25) – Substudy A – Relapsed/Refractory Waldenstrom Macroglobulinemia

Gentherapie / GGO

2022-500782-27-00

A phase I/II open label, multicenter study evaluating the feasibility, safety and efficacy of point-of-care manufactured anti-BCMA CAR T cells (BCMACP03) in subjects with relapsed/refractory Multiple Myeloma (r/r MM)

Gentherapie / GGO

NL81984.000.22

A Randomized, Double-blind, Placebo-controlled, Parallel-group, Multiple-dose Phase 2 Study  to Evaluate the Efficacy and Safety of BMS-986263 in Adults with Compensated Cirrhosis from  Nonalcoholic Steatohepatitis (NASH)

Oligonucleotide / RNA therapie

NL82028.000.22

A Phase 2 Open-Label Extension Study to Evaluate the Long-Term Safety and Efficacy of ARO-APOC3 in Adults with Dyslipidemia

Oligonucleotide / RNA therapie

NL83022.000.23

Phase I/IIa, first-in-human, open-label, dose escalation trial with expansion cohorts to evaluate safety, pharmacokinetics,  pharmacodynamics, and preliminary efficacy of BNT141 as a monotherapy and in combination with other anti-cancer agents in  patients with CLDN18.2-positive solid tumors

Oligonucleotide / RNA therapie

NL82432.000.22

A Phase 2 Study to Evaluate Efficacy, Safety and Tolerability of VIR-2218 and VIR-3434 in Participants with Chronic Hepatitis D Virus Infection (SOLSTICE)

Oligonucleotide / RNA therapie

NL83219.000.23

First-in-human, open-label, multicenter, Phase I/IIa, dose escalation trial with expansion cohorts to evaluate safety and preliminary efficacy of BNT142 in patients with CLDN6-positive advanced solid tumors

Oligonucleotide / RNA therapie

NL82579.000.22

An open-label, single arm, multicenter extension study to evaluate long-term safety and tolerability of inclisiran in participants with heterozygous or homozygous familial hypercholesterolemia who have completed the adolescent ORION-16 or ORION-13 studies (VICTORION-PEDS-OLE)

Oligonucleotide / RNA therapie

NL81760.000.22*

A Global, Phase 1/2, Open-Label, Dose Optimization Study to Evaluate the Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of mRNA-3705 in Participants with Isolated Methylmalonic Acidemia Due to Methylmalonyl-CoA Mutase Deficiency

Oligonucleotide / RNA therapie

Minderjarige proefpersonen

2022-501420-20-00

ISIS 678354-CS6 A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study of Olezarsen (ISIS 678354) Administered Subcutaneously to Patients with Severe Hypertriglyceridemia

Oligonucleotide / RNA therapie

2022-500758-41-00

A Multi-Center, Randomized, Double-Blind, Placebo-Controlled Multiple Ascending Dose Study To Evaluate The Safety And Tolerability Of Qrl-201 In Amyotrophic Lateral Sclerosis

Oligonucleotide / RNA therapie

2022-502096-32-00

A Randomized, Double-blind, Placebo-controlled, Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of AOC 1020 Administered Intravenously to Adult Participants with Facioscapulohumeral Muscular Dystrophy (FSHD)

Oligonucleotide / RNA therapie

2022-501608-85-00

A Double-blind, Randomized, Placebo-controlled, Multicenter Study Assessing the Impact of Olpasiran on Major Cardiovascular Events in Patients with Atherosclerotic Cardiovascular Disease and Elevated Lipoprotein (a)

Oligonucleotide / RNA therapie

2022-501426-38-00

J3L-MC-EZEB: A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study to Investigate the Efficacy and Safety of LY3819469 in Adults with Elevated Lipoprotein(a)

Oligonucleotide / RNA therapie

2022-502404-73-00

A Phase II, Open-Label, Multicenter, Randomized Study of the Efficacy and Safety of Adjuvant Autogene Cevumeran Plus Atezolizumab and mFOLFIRINOX versus mFOLFIRINOX Alone in Patients with Resected Pancreatic Ductal Adenocarcinoma

Oligonucleotide / RNA therapie

2023-504786-23-00

A Phase 1/2 Dose-escalation Study Evaluating the Safety, Tolerability, and Efficacy of VX-522 in Subjects 18 Years of Age and Older With Cystic Fibrosis and a CFTR Genotype Not Responsive to CFTR Modulator Therapy

Oligonucleotide / RNA therapie

2022-502779-40-00

A randomized, double-blind, placebo-controlled multicenter study to evaluate the effect of inclisiran on preventing major adverse cardiovascular events in high-risk primary prevention patients (VICTORION-1 PREVENT)

Oligonucleotide / RNA therapie

2022-501423-25-00

A Phase 1 dose-finding study to evaluate safety and tolerability of CVGBM in patients with surgically resected glioblastoma (GBM) or astrocytoma with a molecular signature of unmethylated glioblastoma

Oligonucleotide / RNA therapie

2022-503022-13-00

ISIS 678354-CS9:  A Randomized, Double-blind, Placebo-Controlled, Phase 3 Study of Olezarsen (ISIS 678354) in Patients with Hypertriglyceridemia and Atherosclerotic Cardiovascular Disease (Established or at Increased Risk for), or with Severe Hypertriglyceridemia

Oligonucleotide / RNA therapie

2022-501644-15-01*

(herindiening)

A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Assess the Efficacy, Safety, and Tolerability of BIIB080 in Subjects with Mild Cognitive Impairment Due to Alzheimer’s Disease or Mild Alzheimer’s Disease Dementia

Oligonucleotide / RNA therapie
Wilsonbekwame proefpersonen

2022-502432-39-00*

ION356-CS1: A Phase 1b Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Patients with Pelizaeus-Merzbacher Disease

Oligonucleotide / RNA therapie

Minderjarige proefpersonen

2022-501997-20-00*

A Phase 1/2, Global, Open-Label, Extension Study to Evaluate the Long-Term Safety and Clinical Activity of mRNA-3705 in Participants Previously Enrolled in the mRNA-3705-P101 Study.

Oligonucleotide / RNA therapie

Minderjarige en wilsonbekwame proefpersonen

NL80728.000.23

Exploring the presence and characteristics of physical fatigability in children and adolescents with facioscapulohumeral dystrophy (FSHD)

Minderjarige proefpersonen

NL83362.000.23

Tracheomalacia in babies after correction of Esophageal Atresia - A First step to reduce respiratory morbidity

Minderjarige proefpersonen

NL83741.000.23

Evaluation of the outcome of clinical or home introduction of milk in children with Non-IgE-mediated cows Milk Allergy (ENIGMA trial)

Minderjarige proefpersonen

NL84027.000.23

The prevalence of Exercise Induced Laryngeal Obstruction (EILO) amongst children with asthma

Minderjarige proefpersonen

NL83069.000.23

Trial readiness and fitness for congenital myopathies: A 2-year prospective natural history study including a cross-sectional study on muscle fatigability

Minderjarigen

NL84098.000.23

The effect of standard or higher protein feeding on indicator amino acid oxidation in ICU patients

Wilsonbekwame proefpersonen

2022-502629-16-00

A Phase 3 Randomized, Placebo-Controlled, Double-Blind, Multicenter Study to Evaluate the Efficacy and Safety of Nipocalimab in Pregnancies at Risk for Severe Hemolytic Disease of the Fetus and Newborn (HDFN).

Onderzoek met zwangeren en/of borst-voedinggevende vrouwen

2023-503220-76-01

(herindiening)

Prednisolone administration in women with unexplained REcurrent Miscarriages

Onderzoek met zwangeren en/of borst-voedinggevende vrouwen

2022-501534-33-00

Luteal Phase Support in MOH-IUI treatment (LUMO study)

Onderzoek met zwangeren en/of borst-voedinggevende vrouwen

NL82714.000.22

The effect of antenatal acetaminophen administration on breathing effort of premature infants at birth: a pilot study

Onderzoek met zwangeren en/of borst-voedinggevende vrouwen

Embryowet
Minderjarigen

NL80866.000.22

Non-invasive preimplantation genetic testing (niPGT) - haplotyping by sequencing embryo spent culture medium

Embryowet

Minderjarigen

NL81942.000.22

Choice Preclinical Gametes Study - Sperm Survival 

Embryowet

NL82275.000.22*

Tina-quant Lp(a) RxDx Phase III Trial  Sample Measurement

IVDR
Geneesmiddelen-onderzoek

NL82339.000.23

Clinical validation of fetal RHD genotyping in gestational week 11

IVDR

NL79738.000.23

Motorized arm support augmenting upper extremity function of people with Duchenne Muscular Dystrophy

MDR

NL82767.000.22

A Phase 1/2 Study to Evaluate the Safety, Tolerability, and Efficacy of VX-264 in Subjects With Type 1 Diabetes Mellitus

MDR

NL84127.000.23

Proof of concept functional prototype INES full exoskelet

MDR