Clinical trials: in Israel
Refractory or Recurrent Hypermutated Malignancies, Biallelic Mismatch Repair Deficiency (bMMRD) Positive Patients
Pilot Study of Nivolumab in Pediatric Patients With Hypermutant Cancers
This is an open-label, single arm, multi-center, pilot study of Nivolumab in pediatric patients with recurrent or refractory hypermutant malignancies aged 12 months to 18 years of age.
- Trial name
- NCT02992964
- Testing
- Nivolumab
- Where
- 10 sites in United States, Australia and 3 more
- Volunteers
- 11 people
- Sponsor
- The Hospital for Sick Children
Thrombotic Microangiopathy
Ravulizumab in Thrombotic Microangiopathy After Hematopoietic Stem Cell Transplant
This study will evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of ravulizumab in adult and adolescent participants with hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA).
- Trial name
- NCT04543591
- Testing
- Ravulizumab, Best supportive care
- Where
- 66 sites in United States, Australia and 15 more
- Volunteers
- 148 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Pulmonary Arterial Hypertension
A Clinical Study of to Confirm the Doses of Selexipag in Children With Pulmonary Arterial Hypertension
The purpose of this study to confirm the selexipag starting dose(s), selected based on pharmacokinetic (PK) extrapolation from adults, that leads to similar exposure as adults doses in children from greater than or equal to (>=)…
- Trial name
- NCT03492177
- Testing
- selexipag (Uptravi)
- Where
- 34 sites in United States, Belarus and 14 more
- Volunteers
- 63 people
- Sponsor
- Actelion
Hypertension, Pulmonary
A Study of Selexipag as Add-On Treatment to Standard of Care in Children With Pulmonary Arterial Hypertension
The purpose of this study is to evaluate whether the addition of selexipag to standard of care treatment delays disease progression in children with Pulmonary Arterial Hypertension (PAH) in comparison to placebo.
- Trial name
- SALTO (NCT04175600)
- Testing
- Selexipag, Standard of Care (SOC): Endothelin receptor antagonist, SOC: Phosphodiesterase type 5 (PDE-5) inhibitor
- Where
- 116 sites in United States, Australia and 30 more
- Volunteers
- 138 people
- Sponsor
- Actelion
Muscular Atrophy, Spinal
A Study to Learn About the Effect of Nusinersen (BIIB058) Given as Injections to Children With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Onasemnogene Abeparvovec (RESPOND)
In this study, researchers will learn more about the use of nusinersen (BIIB058) in participants with spinal muscular atrophy (SMA).
- Trial name
- RESPOND (NCT04488133)
- Testing
- Nusinersen
- Where
- 15 sites in United States, Germany and 3 more
- Volunteers
- 46 people
- Sponsor
- Biogen
Atypical Hemolytic Uremic Syndrome
A Study Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Crovalimab in Adult and Adolescent Participants With Atypical Hemolytic Uremic Syndrome (aHUS)
This study aims to evaluate the efficacy and safety of crovalimab in adult and adolescent participants with aHUS.
- Trial name
- COMMUTE-a (NCT04861259)
- Testing
- Crovalimab
- Where
- 41 sites in United States, Belgium and 14 more
- Volunteers
- 83 people
- Sponsor
- Hoffmann-La Roche
Growth Hormone Deficiency in Children
A Research Study in Children With a Low Level of Hormone to Grow. Treatment is Somapacitan Once a Week Compared to Norditropin® Once a Day (REAL4)
The study compares 2 medicines for children who do not have enough hormone to grow: somapacitan given once a week (a new medicine) and Norditropin® given once a day (the medicine doctors can already prescribe).
- Trial name
- REAL4 (NCT03811535)
- Testing
- Somapacitan, Norditropin®
- Where
- 139 sites in United States, Algeria and 24 more
- Volunteers
- 200 people
- Sponsor
- Novo Nordisk A/S
Progressive Familial Intrahepatic Cholestasis
Long Term Safety & Efficacy Study Evaluating The Effect of A4250 in Children With PFIC
Open-label extension study to evaluate long-term safety and persistence of effect of A4250 in children with progressive familial intrahepatic cholestasis (PFIC).
- Trial name
- PEDFIC 2 (NCT03659916)
- Testing
- A4250 (odevixibat)
- Where
- 41 sites in United States, Australia and 13 more
- Volunteers
- 116 people
- Sponsor
- Albireo, an Ipsen Company
Differentiated Thyroid Cancer
A Study of Cabozantinib Compared With Placebo in Subjects With Radioiodine-refractory Differentiated Thyroid Cancer Who Have Progressed After Prior Vascular Endothelial Growth Factor Receptor (VEGFR) -Targeted Therapy
The objective of this study is to evaluate the effect of cabozantinib compared with placebo on progression free survival (PFS) and objective response rate (ORR) in subjects with Radioiodine-Refractory Differentiated Thyroid…
- Trial name
- NCT03690388
- Testing
- Cabozantinib
- Where
- 164 sites in United States, Argentina and 23 more
- Volunteers
- 187 people
- Sponsor
- Exelixis
Colitis, Ulcerative
A Study of Ustekinumab in Pediatric Participants With Moderately to Severely Active Ulcerative Colitis (UC)
The purpose of this study is to evaluate: a) the efficacy of ustekinumab dosing in inducing clinical remission, b) safety profile of ustekinumab, and c) ustekinumab exposure (pharmacokinetics [PK]) in pediatric participants with…
- Trial name
- UNIFI Jr (NCT04630028)
- Testing
- Ustekinumab Dose Based on BSA and Body Weight
- Where
- 58 sites in United States, Belgium and 7 more
- Volunteers
- 112 people
- Sponsor
- Janssen Research & Development, LLC
Immune Thrombocytopenia
Study to Evaluate Rilzabrutinib in Adults and Adolescents With Persistent or Chronic Immune Thrombocytopenia (ITP)
This was a randomized, double-blind study of rilzabrutinib in patients with persistent or chronic ITP, with an average platelet count of <30,000/μL (and no single platelet count >35,000/μL) on two counts at least 5 days apart in…
- Trial name
- LUNA 3 (NCT04562766)
- Testing
- Rilzabrutinib
- Where
- 155 sites in United States, Argentina and 24 more
- Volunteers
- 232 people
- Sponsor
- Principia Biopharma, a Sanofi Company
Short Stature Children Born Small for Gestational Age (SGA)
A Research Study in Children Born Small and Who Stayed Small. Treatment is Somapacitan Once a Week Compared to Norditropin® Once a Day
The study compares 2 medicines used for the treatment of children who are born small and who stayed small: somapacitan given once a week (a new medicine) and Norditropin® given once a day (the medicine doctors can already…
- Trial name
- NCT03878446
- Testing
- Somapacitan, Norditropin®
- Where
- 95 sites in United States, Algeria and 21 more
- Volunteers
- 62 people
- Sponsor
- Novo Nordisk A/S
Spinal Deformity
Spinal Deformity Intraoperative Monitoring.
A multicenter, international prospectively collected patient cohort undergoing high-risk spinal cord level surgery or spinal osteotomy procedures will be enrolled to establish the incidence of intraoperative alerts in high-risk…
- Trial name
- NCT03880292
- Testing
- Document intraoperative Maneuvers
- Where
- 24 sites in United States, Australia and 12 more
- Volunteers
- 592 people
- Sponsor
- AO Foundation, AO Spine
Severe Hemophilia A
A Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Subcutaneous Emicizumab in Participants From Birth to 12 Months of Age With Hemophilia A Without Inhibitors
This is a Phase IIIb, multicenter, open-label, single-arm study of prophylactic emicizumab in previously untreated and minimally treated patients at study enrollment from birth to ≤12 months of age with severe hemophilia A…
- Trial name
- HAVEN 7 (NCT04431726)
- Testing
- Emicizumab
- Where
- 32 sites in United States, Australia and 12 more
- Volunteers
- 55 people
- Sponsor
- Hoffmann-La Roche
Colitis, Ulcerative
A Study to Assess the Efficacy and Safety of Golimumab in Pediatric Participants With Moderately to Severely Active Ulcerative Colitis
The purpose of this study is to evaluate efficacy of golimumab in inducing clinical remission as assessed by the Mayo score, in pediatric participants with moderately to severely active ulcerative colitis (UC).
- Trial name
- PURSUIT 2 (NCT03596645)
- Testing
- Golimumab, Infliximab
- Where
- 58 sites in United States, Belgium and 9 more
- Volunteers
- 84 people
- Sponsor
- Janssen Research & Development, LLC
Haemophilia A Without Inhibitors, Haemophilia B Without Inhibitors
Research Study to Look at How Well the Drug Concizumab Works in Your Body if You Have Haemophilia Without Inhibitors
This study will test how well a new medicine called concizumab works in the body of people with haemophilia A or B without inhibitors. The purpose is to show that concizumab can prevent bleeds in the body and is safe to use.
- Trial name
- explorer8 (NCT04082429)
- Testing
- Concizumab
- Where
- 113 sites in United States, Algeria and 31 more
- Volunteers
- 156 people
- Sponsor
- Novo Nordisk A/S
Epilepsy, Dravet Syndrome
A Phase 2, Multicenter, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy, Safety, and Tolerability of TAK-935 (OV935) as an Adjunctive Therapy in Pediatric Participants With Developmental and/or Epileptic Encephalopathies
The purpose of this study is to investigate the effect on the frequency of all seizures (convulsive and drop) in participants treated with TAK-935 compared to placebo.
- Conditions
- Epilepsy, Dravet Syndrome, Lennox-Gastaut Syndrome
- Trial name
- ELEKTRA (NCT03650452)
- Testing
- TAK-935
- Where
- 44 sites in United States, Australia and 6 more
- Volunteers
- 141 people
- Sponsor
- Takeda
Melanoma
A Study of Adjuvant Pembrolizumab/Vibostolimab (MK-7684A) Versus Pembrolizumab for Resected High-Risk Melanoma in Participants With High-Risk Stage II-IV Melanoma (MK-7684A-010/KEYVIBE-010)
The primary purpose of this study is to compare pembrolizumab/vibostolimab to pembrolizumab with respect to recurrence-free survival (RFS).
- Trial name
- NCT05665595
- Testing
- Pembrolizumab/Vibostolimab, Pembrolizumab
- Where
- 205 sites in United States, Argentina and 24 more
- Volunteers
- 1,594 people
- Sponsor
- Merck Sharp & Dohme LLC
Ulcerative Colitis
ABTECT-2 - ABX464 Treatment Evaluation for Ulcerative Colitis Therapy -2
This is a multicenter, randomized, placebo controlled study to evaluate the efficacy and safety of ABX464 given at 25 or 50 mg QD in inducing clinical remission in subjects with moderately to severely active ulcerative colitis…
- Trial name
- NCT05507216
- Testing
- ABX464
- Where
- 262 sites in United States, Argentina and 25 more
- Volunteers
- 636 people
- Sponsor
- Abivax S.A.
Epilepsy, Dravet Syndrome (DS)
A Study of Soticlestat in Adults and Children With Rare Epilepsies
The main aim is to assess the long-term safety and tolerability of soticlestat when used along with other anti-seizure treatment. Participants will receive soticlestat twice a day.
- Conditions
- Epilepsy, Dravet Syndrome (DS), Lennox-Gastaut Syndrome (LGS)
- Trial name
- Endymion 1 (NCT03635073)
- Testing
- Soticlestat
- Where
- 59 sites in United States, Australia and 6 more
- Volunteers
- 156 people
- Sponsor
- Takeda