Bijlage IIIa: onderzoeksprotocollen beoordeeld door de CCMO met een positief besluit

Dossier Nr.

Beoordelings-rol Nederland

Studie titel

Categorie

2022-500306-17-00

MSC-multi      

A multi-center, randomized, active controlled clinical trial to evaluate the efficacy and safety of OTL-203 in subjects with mucopolysaccharidosis type I, Hurler syndrome (MPS-IH) compared to standard of care with allogeneic hematopoietic stem cell transplantation (allo-HSCT)

1,2

2022-500747-21-00

RMS-multi

Long-term Extension Study for Participants with Geographic Atrophy (GA) Secondary to Age-related Macular Degeneration (AMD) in JNJ-81201887 Parent Clinical Studies

1

2022-501694-39-01

RMS-nat  

A Prospective Study Comparing [18F]mFBG PET-CT to [123I]mIBG Scanning in Neuroblastoma

6

2022-502135-19-00

MSC-multi      

A randomized double-blind, placebo-controlled, multicenter trial assessing the impact of lipoprotein(a) lowering with pelacarsen (TQJ230) on the progression of calcific aortic valve stenosis [Lp(a)FRONTIERS CAVS]

1

2022-502629-16-00

RMS-multi

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).

4

2022-502910-10-00

MSC-multi      

A Global, Phase 1/2, Open-label, Dose Optimization Study to Evaluate the Safety, Pharmacodynamics, and Pharmacokinetics of mRNA-3927 in Participants with Propionic Acidemia

1,6

2022-502963-39-00

MSC-multi      

A Phase 1/2, adaptive, open-label, single ascending dose to multiple ascending dose study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of mRNA-3745 in participants with glycogen storage disease type 1a (GSD1a), followed by an open-label extension

1,6

2022-502972-22-00

MSC-multi      

A Randomized, Double-Blind, Placebo-Controlled Phase 3 Study of VE303 for Prevention of Recurrent Clostridioides difficile Infection: The RestoratiVE303 Study

1,3,7

2023-503281-23-00

RMS-multi

A seamless Phase I/II trial with an initial open-label dose escalation part and a subsequent randomised, double-blind, placebo-controlled expansion part to evaluate the safety, tolerability, and efficacy of a single dose of BI 3720931, an inhaled lentiviral vector gene therapy, in adult people with cystic fibrosis who are ineligible for CFTR modulators (LenticlairTM 1)

1

2023-503765-37-00

RMS-multi

An Extension Study Assessing the Long-term Safety and Efficacy of Etranacogene Dezaparvovec (CSL222) Previously Administered to Adult Male Subjects with Hemophilia B

1

2023-504909-37-00

RMS-multi

A clinical trial to evaluate the long-term safety and durability of efficacy of BI 3720931, an inhaled lentiviral vector gene therapy, after single dose administration in a previous clinical trial in people with cystic fibrosis rolled-over from a previous clinical trial with BI 3720931 (LenticlairTM-ON).

1,4

2023-505262-28-00

RMS-multi

A Phase 1 Trial of the Menin Inhibitor Ziftomenib in Combination with Chemotherapy for Children with Relapsed/Refractory KMT2A-rearranged, NUP98-rearranged, or NPM1-mutant Acute Leukemia

6

2023-505704-30-00

RMS-nat  

A Phase 1b/2 Open-label Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Exploratory Efficacy Following ABBV-CLS-7262 Administration in Adult and Pediatric Subjects with Vanishing White Matter Disease

6

2023-506028-10-00

RMS-multi

A Phase 1/2, Randomized, Double-blind, Placebo‑controlled Single- and Multiple‑dose Escalation Study Evaluating the Safety, Tolerability,
 Pharmacokinetics, and Pharmacodynamics of VX‑670 in Adult Subjects with Myotonic Dystrophy Type 1

1

2023-506240-16-00

RMS-multi

A Phase 1/2, Open-label Study Evaluating the Efficacy, Safety, and Pharmacokinetics (PK) of Luveltamab Tazevibulin (STRO-002) in Infants and Children < 12 years of age with CBFA2T3::GLIS2 Acute Myeloid Leukemia (AML)

6

2023-506267-33-00

MSC-multi      

A Phase 1b Multicenter, Open-label, Study of JNJ-90014496, an Autologous CD19/CD20 Bi-specific CAR-T Cell Therapy in Adult Participants with Relapsed or Refractory B-cell Non-Hodgkin Lymphoma

1,2

2023-506301-20-00

MSC-multi      

A Multicenter, Open-label Study to Assess the Safety, Tolerability, Pharmacokinetics, and Effect on Seizures and Behavioral Symptoms of Radiprodil in Patients with Tuberous Sclerosis Complex (TSC) or Focal Cortical Dysplasia (FCD) Type II.

6

2023-506327-29-00

MSC-multi      

A Phase 3 Randomized Double-blind Study of Adjuvant Pembrolizumab With or Without V940 in Participants With Resectable Stage II to IIIB (N2) NSCLC not Achieving pCR After Receiving Neoadjuvant Pembrolizumab With Platinum-based Doublet Chemotherapy (INTerpath-009)

1

2023-506348-17-00

MSC-multi      

A single-arm, multi-center, open-label Phase II study to determine the safety and efficacy of MB-CART2019.1 in pediatric subjects with relapsed/refractory (r/r) mature B-cell neoplasms who have relapsed after one or more prior therapies, including subjects with primary refractory disease

1,2

2023-506394-35-02

RMS-nat  

Phase Ia/b double-blind, placebo-controlled, dose escalating safety study of detoxified Shigella flexneri 2a Artificial Invasin Complex (InvaplexAR-Detox) vaccine formulated with and without dmLT adjuvant given intramuscularly to healthy adults in the Netherlands and Zambia

3

2023-506400-99-00

MSC-multi      

Phase II randomized study evaluating a Pragmatic approach to Adoptive Cell Therapy (ACT) using an IL2 analog (ANV419) vs High dose IL2 after Tumor Infiltrating Lymphocytes (TIL) Therapy in patients with melanoma, NSCLC and cervical cancer. The PragmaTIL

2

2023-507178-41-00

RMS-multi

LIGHTBEAM-U01-Substudy 01A: A Phase 1/2 Substudy to Evaluate the Safety and Efficacy of Zilovertamab Vedotin in Pediatric and Young Adult Participants with Hematologic Malignancies or Solid Tumors.

1,6

2023-507311-35-00

RMS-multi

A Study to Evaluate the Pharmacokinetics of Afamelanotide in Patients with Erythropoietic Protoporphyria (EPP).

6

2023-507717-94-00

RMS-multi

A multicentre, intra-patient randomised controlled Phase III study to confirm the efficacy and safety of denovoSkin™, a bilayer engineered collagen-based skin graft composed of autologous fibroblasts and keratinocytes, for the treatment of patients with deep partial and full-thickness burns

2

2023-507763-18-01

RMS-nat  

Progesterone for prEventing Preterm births in women with PlacEnta pRevia (PEPPER-trial)

4,5

2023-508139-29-00

MSC-multi      

An Open-Label, Multicenter, First-in-Human, Phase 1 Dose Escalation and Multicohort Expansion Study of INBRX-109 in Subjects with Locally Advanced or Metastatic Solid Tumors, Including Sarcomas.

6

2023-508157-15-01

RMS-nat  

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

3

2023-508558-25-01

RMS-nat  

D-Sense 2 - Immune intervention with proinsulin-peptide pulsed tolerogenic dendritic cells in type 1 diabetes
 A phase 1b safety and immunological effectivity study.

2

2023-509023-40-00

MSC-multi      

A RANDOMIZED PHASE II, DOUBLE-BLIND, MULTICENTER STUDY EVALUATING THE EFFICACY AND SAFETY OF AUTOGENE CEVUMERAN PLUS NIVOLUMAB VERSUS NIVOLUMAB AS ADJUVANT THERAPY IN PATIENTS WITH HIGH-RISK MUSCLE- INVASIVE UROTHELIAL CARCINOMA

1

2023-509190-23-00

MSC-multi      

J3L-MC-EZEF:A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Investigate the Effect of Lepodisiran on the Reduction of Major Adverse Cardiovascular Events in Adults with Elevated Lipoprotein(a) who have Established Atherosclerotic Cardiovascular Disease or Are at Risk for a First Cardiovascular Event – ACCLAIM-Lp(a)

1

2023-509256-34-00

RMS-multi

A Phase 1/2 Dose-Exploration and Dose-Expansion Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients with Alpha-1 Antitrypsin Deficiency (AATD)-Associated Lung Disease and/or Liver Disease

1

2023-509673-22-00

RMS-nat  

Phase Ib study combining dinutuximab beta with induction chemotherapy regimens in patients with newly diagnosed high-risk neuroblastoma: SIOPEN-Pilot01

6

2023-509748-89-00

RMS-multi

A Phase 1/2a Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ARO-DUX4 in Adult Patients with Facioscapulohumeral Muscular Dystrophy Type 1

1

2023-510150-17-00

MSC-multi      

A Phase 2, adaptive, randomized, open-label, assessor-blinded active-controlled study to evaluate the efficacy and safety of rapcabtagene autoleucel versus Standard of Care in patients suffering from systemic lupus erythematosus (SLE) with active, refractory lupus nephritis (LN).

1,2

2023-510173-34-00

RMS-multi

A long-term follow-up study for patients treated with Galapagos CAR T-cell therapies

2

2023-510196-59-00

MSC-multi      

A Phase 3, open-label, randomized, controlled study to evaluate the immune response, safety and reactogenicity of RSVPreF3 OA investigational vaccine when co-administered with a COVID-19 mRNA vaccine (Omicron XBB.1.5) in adults aged 50 years and above.

1,3

2023-510219-20-00

MSC-multi      

GSDIa Disease Monitoring Program

1,4

2023-510380-34-00

MSC-multi      

A Phase II, multi-part, three-year, randomized, open-label, assessor-blinded, active-controlled, multicenter study to evaluate the efficacy and safety of rapcabtagene autoleucel versus rituximab treatment in participants with severe refractory diffuse cutaneous systemic sclerosis

1,2

2023-510382-10-00

RMS-nat  

Metformin for the prolongation of pregnancy in preterm preeclampsia: the PI-NL trial

4

2024-510581-17-00

RMS-multi

(ASK-CHF2-CS201) A Phase 2, adaptive, double-blinded, placebo-controlled, randomized, multi-center trial to evaluate the efficacy, safety and tolerability of intracoronary infusion of AB-1002 in adult subjects with New York Heart Association (NYHA) Class III heart failure and non-ischemic cardiomyopathy

1

2024-511184-28-00

RMS-multi

A Phase 2a, Randomized, Placebo-Controlled, Double Blind Multiple Ascending Dose Study in Patients with Cystic Fibrosis Carrying the 3849 +10 Kb C->T Mutation to Evaluate the Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of SPL84‎

1

2024-511188-26-00

MSC-multi      

A Phase 3, Randomized, Open-Label Study to Compare the Efficacy and Safety of Anitocabtagene Autoleucel Versus Standard of Care Therapy in Participants With Relapsed/Refractory Multiple Myeloma

1,2

2024-511378-60-00

RMS-multi

A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Global Study to Evaluate the Efficacy and Safety of Intravenous AOC 1001 for the Treatment of Myotonic Dystrophy Type 1

1

2024-512169-15-00

RMS-multi

A Phase 1/2, Multicenter Study Evaluating the Safety, Tolerability, and Biodistribution of RCT2100 with Single-Ascending Doses in Healthy Participants and Multiple-Ascending Doses and Proof-of-Concept in Participants with Cystic Fibrosis

1

2024-512651-20-00

RMS-multi

A Phase 2, Multicenter, Open-label Study to Evaluate the Pharmacokinetics and Safety of RLYB212 in Pregnant Women at Higher Risk for HPA-1a Alloimmunization

4

2024-512720-11-00

MSC-multi      

A PIVOTAL, RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE-BLIND, MULTICENTER, INTERNATIONAL PHASE III CLINICAL TRIAL TO INVESTIGATE THE EFFICACY AND SAFETY OF ALLO-APZ2-CVU ON WOUND HEALING OF THERAPY-RESISTANT NON-HEALING CHRONIC VENOUS ULCERS (CVU)

2

2024-513516-99-00

RMS-nat  

(CoGa-Re) Durability of protection after single immunisation with genetically attenuated Plasmodium falciparum ∆mei2 (GA2) sporozoites – a follow-up, controlled human malaria rechallenge study

1,3,7

2024-514137-38-00

MSC-multi      

A Phase 2, randomized, open-label, controlled study to evaluate the efficacy and safety of rapcabtagene autoleucelversus comparator in participants with severe refractory idiopathic inflammatory myopathies

1,2

2024-514871-17-00

RMS-nat  

A DOUBLE-BLIND, RANDOMIZED, PLACEBO-CONTROLLED, SINGLE AND MULTIPLE ASCENDING DOSE PHASE 1 STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETIC, AND PHARMACODYNAMIC PROFILE OF RES-010 IN HEALTHY, OVERWEIGHT, AND MODERATELY OBESE SUBJECTS

1

NL83125.000.23

Long-term follow-up of patients with spinal muscular atrophy Treated with OAV101 IT or OAV101 IV in Clinical Trials

1

NL83517.000.23

Moses II – Measuring Elecsys β-Amyloid (1-42) II and Phospho-Tau (181P) in CSF Samples supporting Phase 2 Clinical Trial of 247AD201 (BIIB080)

6

NL83678.000.24

A Pilot Study into the Use of Intraoperative Indocyanine Green Fluorescence Angiography in Young Infants & Neonates

6

NL84591.000.24

Short term variation analysis versus visual evaluation of cardiotocography in fetal growth restriction

4

NL84835.000.23

Utilization of the Navigate Anti-AAV9 Antibody Assay in Support of the Novartis’ Clinical Studies: COAV101B12301 “STEER” and COAV101B12302 “STRENGTH”

1,8

NL85436.000.24

Validating and realising a clinically tested A-EMDR application for postpartum traumas

4

NL85463.000.24

Determination of the Claudin 6 Status Using the BioNTech Diagnostics CLAUDENTIFY® 6 IHC-Assay for the BNT142-01 study (Clinical Trial)

1,8

NL85712.000.23

MASTER: A study on the effects of a parent training for toddlers with emotion dysregulation and an increased likelihood of developing ADHD

6

NL85997.000.24

Assessment of muscle properties in neuromuscular disorders: The muscle toolbox

6

NL86484.091.24

Optimizing parasite transmission to laboratory-reared mosquitoes following controlled human malaria infection with Plasmodium vivax asexual blood stage parasites in malaria-naïve healthy volunteers in the Netherlands

3

NL86724.000.24

Reference values for the Muscle Power Sprint Test (MPST) in children from the age of 5 up to and including 12 years

6

NL86996.000.24

A Multi-center, Clinical Performance Study for FNAIT Laboratory Screening Tests for RLYB212 Phase 2 Study (IPA2202)

4,8

NL87384.000.24

Is Rescue ICSI a useful tool in the routine IVF lab for patients undergoing total IVF fertilization failure?’

5

NL87518.000.24

Dietary protein provides building blocks for all human tissues: the metabolic fate of milk protein an explorative proof-of-principle study

6

2023-510063-35-00

MSC-multi 

Phase 1/1b Study of the Safety, Pharmacokinetics, Pharmacodynamics and Preliminary Clinical Activity of RP-6306 Alone or in Combination with RP-3500 or Debio 0123 in Patients with Advanced Solid Tumors (MYTHIC Study)

6

2023-507220-23-00

MSC-multi 

MAGNITUDE: A Phase 3, Multinational, Multicenter, Randomized, Double‑Blind, Placebo‑Controlled Study to Evaluate the Efficacy and Safety of NTLA‑2001 in Participants with Transthyretin Amyloidosis with Cardiomyopathy (ATTR‑CM).

1

2023-507956-56-00

MSC-multi 

Long-Term Follow-Up of Subjects Treated With NTLA-2002

1