Clinical trials: Severe Pain Syndromes, active
Trisomy 13 Syndrome, Arthrogryposis Congenita Multiplex With Intestinal Atresia
Palliative Care Needs of Children With Rare Diseases and Their Families
The palliative care needs of family caregivers of children with rare diseases and their children are largely unmet, including the need for support to prepare for future medical decision making.
- Conditions
- Trisomy 13 Syndrome, Arthrogryposis Congenita Multiplex With Intestinal Atresia, Asparagine Synthetase Deficiency, CHARGE Syndrome
- Trial name
- FACE-Rare (NCT06938542)
- Testing
- Family Centered pediatric palliative care for family caregivers of children with rare diseases.
- Where
- United States
- Volunteers
- 480 people, healthy volunteers welcome
- Sponsor
- Children's National Research Institute
Shwachman-Diamond Syndrome, SDS
Shwachman-Diamond Syndrome Global Patient Survey and Partnering Platform
The Shwachman-Diamond Syndrome Global Patient Survey and Collaboration Program (SDS-GPS) is an opportunity for patients and their families - from anywhere in the world - to share their experience living with SDS via a safe…
- Conditions
- Shwachman-Diamond Syndrome, SDS, IBMF, Congenital Neutropenia
- Trial name
- SDS-GPS (NCT06999954)
- Where
- United States
- Volunteers
- 8,000 people
- Sponsor
- Shwachman-Diamond Syndrome Alliance Inc
Severe Aplastic Anemia, Hypo-Plastic MDS
Unrelated Umbilical Cord Blood Transplantation for Severe Aplastic Anemia and Hypo-plastic MDS Using CordIn(TM), Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells to Expedite Engraftment and Improve Transplant Outcome
Background: Severe aplastic anemia (SAA) and myelodysplastic syndrome (MDS) are bone marrow diseases. People with these diseases usually need a bone marrow transplant.
- Conditions
- Severe Aplastic Anemia, Hypo-Plastic MDS, Myelodysplastic Syndrome (MDS)
- Trial name
- NCT03173937
- Testing
- Omidubicel (former CordIn), Miltenyi CliniMACS(R) CD34 Reagent System
- Where
- United States
- Volunteers
- 37 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Acute Leukemia, Myelodysplastic Syndromes
Sirolimus+Ruxolitinib+Mycophenolate Mofetil for Prophylaxis of aGVHD in Patients Receiving Haplo-HSCT Who Are Intolerant to CNI
Graft-versus-host disease (GVHD) is an important complication after transplantation, with an incidence of 40-60%, which can increase non-relapse mortality if poorly controlled.
- Conditions
- Acute Leukemia, Myelodysplastic Syndromes, Severe Aplastic Anemia (SAA)
- Trial name
- NCT07796620
- Testing
- Sirolimus, Ruxolitinib, MMF
- Volunteers
- 40 people
- Sponsor
- Peking University People's Hospital
Takayasu Arteritis, Large Vessel Vasculitis
Phase II Interventional Study Evaluating Efficacy and Safety of Secukinumab in Active Severe Takayasu Patients
Takayasu's arteritis (TAK) is a large vessel vasculitis preferentially affecting the aorta and its main branches, leading to wall vessels thickening, fibrosis, stenosis, and occlusion.
- Trial name
- STARS (NCT07477795)
- Testing
- Secukinumab, Standard of Care (SOC)
- Volunteers
- 52 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Bone Marrow Failure Syndrome, Severe Aplastic Anemia
CD34+ (Non-Malignant) Stem Cell Selection for Patients Receiving Allogeneic Stem Cell Transplantation
This study's goal is to determine the frequency and severity of acute graft versus host disease, to evaluate incidence of primary and secondary graft rejection, to assess event free survival and overall survival, to determine the…
- Conditions
- Bone Marrow Failure Syndrome, Severe Aplastic Anemia, Severe Congenital Neutropenia, Amegakaryocytic Thrombocytopenia
- Trial name
- NCT01966367
- Testing
- CD34 Stem Cell Selection Therapy
- Where
- United States
- Volunteers
- 37 people
- Sponsor
- Diane George
Severe Aplastic Anemia, MDS (Myelodysplastic Syndrome)
Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused With a Reduced Dose of Non-Mobilized Donor T-cells
Background: Stem cell transplants from related donors (allogenic stem cell transplants) can be used to treat individuals with certain kinds of severe blood diseases or cancers, such as severe anemia.
- Trial name
- NCT01174108
- Testing
- Miltenyi CD34 Reagent System, Donor derived G-CSF mobilized PBC
- Where
- 2 sites in United States
- Volunteers
- 120 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Severe Combined Immunodeficiencies (SCID)
Follow-up of the Cohort of Newborns Screened at Birth Using TREC Analysis
Since September 2025, neonatal screening for severe combined immunodeficiency (SCID) has been generalized in France. These genetic disorders, which are asymptomatic at birth, cause severe immunodeficiency, exposing infants to…
- Trial name
- DépisTrec (NCT07704281)
- Testing
- Data collection
- Where
- France
- Volunteers
- 450 people
- Sponsor
- Nantes University Hospital
Dry Eye Syndromes, Dry Eye Disease (DED)
Clinical Investigation Between the Topical Administration of a Tear Substitute With Ancillary Medicinal Substance and Hyaluronic Acid 0.3% (FBV_01) vs Hyaluronic Acid 0.3% in Subjects in the Treatment From Moderate to Severe DED
The goal of this clinical investigation plan is to evaluate the performance, tolerability, safety, and compliance of an ophthalmic solution containing an ancillary medicinal substance and hyaluronic acid 0.3% (FBV_01) compared…
- Trial name
- NCT07412860
- Testing
- FBV_01, HYLO-VISION® SafeDrop® Gel
- Where
- 2 sites in Italy
- Volunteers
- 74 people
- Sponsor
- FB Vision S.p.A
Dry Eye, Neurotrophic Keratitis
DOMPE -MT Neurotrophic Keratitis
TITLE: Toyos Clinic / A Phase 4 Study to Assess Longer Duration of Treatment with Cenegermin in Moderate to Severe Dry Eye-Associated Neurotrophic Keratitis
- Trial name
- NCT06947850
- Testing
- Cenegermin Ophthalmic Solution [Oxervate]
- Where
- United States
- Volunteers
- 10 people, healthy volunteers welcome
- Sponsor
- Toyos Clinic
Dry Eye
A Study to Evaluate the Performance and Safety of Artelac® Complete MDO® Versus VISMED® MULTI Eye Drops in the Management of Moderate to Severe Dry Eye Disease
A Double-Masked, Randomized, Parallel Group, Non-Inferiority Study to Evaluate the Performance and Safety of Artelac® Complete MDO® Versus VISMED® MULTI Eye Drops in the Management of Moderate to Severe Dry Eye Disease
- Trial name
- NCT07593885
- Testing
- Artelac® Complete MDO® Eye Drops, VISMED® MULTI Eye Drops
- Where
- 12 sites in Germany
- Volunteers
- 160 people
- Sponsor
- Dr. Gerhard Mann chem.-pharm. Fabrik GmbH
Severe Combined Immunodeficiency (SCID), Leaky SCID
Natural History Study of SCID Disorders
This study is a prospective evaluation of children with Severe Combined Immune Deficiency (SCID) who are treated under a variety of protocols used by participating institutions.
- Conditions
- Severe Combined Immunodeficiency (SCID), Leaky SCID, Omenn Syndrome, Reticular Dysgenesis
- Trial name
- NCT01186913
- Where
- 44 sites in United States, Canada
- Volunteers
- 690 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Severe Combined Immunodeficiency
The Experience of Screening for SCID
This project will evaluate the impact of including Severe Combined Immunodeficiency into the newborn bloodspot screening panel. It will recruit parents and health professionals primarily from the sites where this new form of…
- Trial name
- NCT05651113
- Testing
- SCID Screening
- Where
- United Kingdom
- Volunteers
- 90 people, healthy volunteers welcome
- Sponsor
- King's College London
Severe Combined Immunodeficiency
Autologous Gene Therapy for Artemis-Deficient SCID
This study aims to determine if a new method can be used to treat Artemis-deficient Severe Combined Immunodeficiency (ART-SCID), a severe form of primary immunodeficiency caused by mutations in the DCLRE1C gene.
- Trial name
- NCT03538899
- Testing
- AProArt-CD34, CliniMACS® CD34 Reagent System cell sorter device, Busulfan
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- University of California, San Francisco
Generalized Myasthenia Gravis
Study to Assess the Efficacy and Safety of IMVT-1402 in Participants With Mild to Severe Generalized Myasthenia Gravis
The purpose of the study is to assess the efficacy, safety and tolerability of IMVT-1402 in adult participants with mild to severe generalized myasthenia gravis.
- Trial name
- NCT07039916
- Testing
- IMVT-1402
- Where
- 88 sites in United States, Argentina and 13 more
- Volunteers
- 231 people
- Sponsor
- Immunovant Sciences GmbH
Anti-N-Methyl-D-Aspartate Receptor Encephalitis
Safety and Efficacy of Combined B Cell Depleting theRapy And Daratumumab In Autoimmune Encephalitis
Autoimmune encephalitis is an autoimmune disease of the central nervous system that targets neuronal autoantigens. Anti-neuronal autoantibodies are produced in patients, with anti-NMDAR antibody being the most common.Anti-NMDAR…
- Trial name
- RADIA (NCT06867991)
- Testing
- Ofatumumab combined with daratumumab, Ofatumumab, Repeated intravenous immunoglobulin/plasma exchange therapy
- Where
- 2 sites in China
- Volunteers
- 200 people
- Sponsor
- The First People's Hospital of Changzhou
Acute Coronary Syndrome Without ST Elevation on Electrocardiogram, Acute Coronary Syndromes (ACS)
Occurrence of Severe Cardiac Rhythm and Conduction Disturbances in Emergency Department Patients With Non-ST Elevation Acute Coronary Syndrome
Cardiovascular diseases are the leading cause of death worldwide. Among them, coronary artery disease-especially in its acute form, known as acute coronary syndrome (ACS)-is the most frequent cause of cardiovascular death.
- Conditions
- Acute Coronary Syndrome Without ST Elevation on Electrocardiogram, Acute Coronary Syndromes (ACS), Myocardial Infarction, Unstable Angina Pectoris, Sudden Cardiac Death, Stroke, Peripheral Artery Disease
- Trial name
- SurvSCA (NCT07112547)
- Where
- 10 sites in France
- Volunteers
- 500 people
- Sponsor
- University Hospital, Strasbourg, France
Dry Eye Disease, Dry Eye
Phase III Study of Topical Fibrinogen-Depleted Human Platelet Lysate Compared to Placebo for Moderate to Severe Dry Eye
Prospective, multicenter, randomized, vehicle-controlled, double-masked, study of CAM-101 topical ophthalmic solution compared to vehicle control followed by crossover to long-term follow-up open-label treatment with CAM-101 for…
- Conditions
- Dry Eye Disease, Dry Eye, Dry Eyes Chronic, Dry Eye Syndromes
- Trial name
- CAMOMILE-2 (NCT06903611)
- Testing
- CAM-101, Vehicle Control
- Volunteers
- 475 people, healthy volunteers welcome
- Sponsor
- Cambium Bio Limited
Generalized Myasthenia Gravis (gMG)
Zilucoplan for Severe gMG Exacerbations
This is an open-label, multicenter, interventional phase 3b study in participants with AChR+ gMG and severe exacerbation that require hospitalization. Patients will receive subcutaneous zilucoplan injections daily for 12 weeks.
- Trial name
- NCT07215949
- Testing
- Zilucoplan®
- Where
- 3 sites in United States
- Volunteers
- 15 people
- Sponsor
- Miriam Freimer
Dry Eye Disease (DED), Tear Break-Up Time
Efficacy of 0.28% Sodium Hyaluronate Eye Drops in Patient With Moderate to Severe Dry Eye
This study aimed to evaluate the efficacy and safety of 0.28% preservative-free SH and 0.18% SH eye drops in patients with dry eye disease.
- Conditions
- Dry Eye Disease (DED), Tear Break-Up Time, Tear Disorder, Tear Film Insufficiency
- Trial name
- NCT06860659
- Testing
- 0.28% Sodium Hyaluronate , preservative free eye drops, 0.18% Sodium Hyaluronate , preservative free eye drops
- Where
- Thailand
- Volunteers
- 84 people, healthy volunteers welcome
- Sponsor
- Department of Medical Services Ministry of Public Health of Thailand