Clinical trials: Rare Disease
Hereditary Tyrosinemia, Type I
A Non-interventional, Post-Marketing Study to Describe Outcome of Nitisinone Treatment in HT-1 Patients
This is a prospective, non-interventional, non-comparative, multicenter study to collect data on HT-1 patients in China treated with Nitisinone in a routine clinical setting. No tests or examinations are mandated in the study.
- Trial name
- NCT06227429
- Testing
- Nitisinone
- Where
- 4 sites in China
- Sponsor
- Swedish Orphan Biovitrum
Non-small Cell Lung Cancer
A Study to Evaluate the Efficacy and Safety of Sutetinib Maleate Capsule in Locally Advanced or Metastatic NSCLC
The main objective of the study will be to evaluate the efficacy of sutetinib maleate capsules in participants with locally advanced or metastatic non-small cell lung cancer NSCLC (uncommon EGFR mutations only).
- Trial name
- NCT06010329
- Testing
- Sutetinib Maleate Capsule
- Where
- 9 sites in United States
- Volunteers
- 66 people
- Sponsor
- Teligene US
Atrial Fibrillation With 2 or 3° AV or Bifascicular Bundle Branch (BBB) Block, Normal Sinus Rhythm With 2 or 3° AV or BBB Block
Evaluation of a New Cardiac Pacemaker
An evaluation of a safety and performance of a new cardiac pacemaker
- Conditions
- Atrial Fibrillation With 2 or 3° AV or Bifascicular Bundle Branch (BBB) Block, Normal Sinus Rhythm With 2 or 3° AV or BBB Block, Sinus Bradycardia With Infrequent Pauses or Unexplained Syncope With EP Findings
- Trial name
- NCT01700244
- Testing
- Pacemaker implant
- Where
- 8 sites in Czechia, Germany and 1 more
- Volunteers
- 36 people
- Sponsor
- Nanostim, Inc.
Solid Tumor
A Study to Evaluate Rucaparib in Participants With Solid Tumors and With Deleterious Mutations in HRR Genes
A Phase 2, open-label, single-arm trial to evaluate the response of rucaparib in participants with various solid tumors and with deleterious mutations in Homologous Recombination Repair (HRR) genes.
- Trial name
- LODESTAR (NCT04171700)
- Testing
- Rucaparib
- Where
- 19 sites in United States
- Volunteers
- 83 people
- Sponsor
- pharmaand GmbH
Rare Diseases, Neuromyelitis Optica Spectrum Disorder (NMOSD)
Epidemiological Study of Treatment Approaches on AQP4-IgG Positive NMOSD in Russia
This is a multi-centre, retrospective-prospective, single-arm, non-interventional (observational) cohort study with secondary data collection within real-world settings of participants with AQP4-IgG positive NMOSD.
- Trial name
- NCT07247292
- Where
- Russia
- Volunteers
- 3 people
- Sponsor
- AstraZeneca
Lung Cancer, Adjuvant
A Study to Assess Zipalertinib Versus Placebo in Participants With Early Stage NSCLC With Uncommon EGFR Mutations, Following Complete Tumor Resection
The purpose of this study is to compare the efficacy of zipalertinib versus placebo in participants with early stage resected non-small cell lung cancer (NSCLC) harboring uncommon epidermal growth factor receptor mutation…
- Conditions
- Lung Cancer, Adjuvant, Post-surgical, EGFR
- Trial name
- REZILIENT4 (NCT07128199)
- Testing
- TAS6417
- Where
- 253 sites in United States, Argentina and 22 more
- Volunteers
- 360 people
- Sponsor
- Taiho Oncology, Inc.
Nasopharyngeal Neoplasms
Safety and Efficacy of CC-486 in Previously Treated Patients With Locally Advanced or Metastatic Nasopharyngeal Carcinoma
The purpose of this study is to evaluate the safety and efficacy of CC-486 in previously treated patients with locally advanced or metastatic nasopharyngeal carcinoma having failed one to two previous regimens, including…
- Trial name
- NCT02269943
- Testing
- CC-486
- Where
- 25 sites in United States, Canada and 7 more
- Volunteers
- 36 people
- Sponsor
- Celgene
Occasional Hair Loss
Evaluation of the Dietary Supplement Effect on Improvement of Hair Condition in Subjects With Occasional Hair Loss.
The goal of this study is to learn if dietary supplement works to help with improving hair condition in subjects with occasional hair loss. It will also learn about its safety.
- Trial name
- NCT07835646
- Testing
- Dietary supplement for hair loss
- Where
- Spain
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Lacer S.A.
Hereditary Cystatin C Amyloid Angiopathy (HCCAA), Hemorrhage Brain
Safety, Tolerability and Efficacy of NPI-001 in Patients With Hereditary Cystatin C Amyloid Angiopathy (HCCAA)
The study aims to measure safety, tolerability and biomarker-based efficacy of NPI-001 (AT-001) in Subjects with Hereditary Cystatin C Amyloid Angiopathy (HCCAA).
- Conditions
- Hereditary Cystatin C Amyloid Angiopathy (HCCAA), Hemorrhage Brain, Hemorrhage Cerebral
- Trial name
- HCCAA (NCT07716865)
- Testing
- NACA
- Where
- Iceland
- Volunteers
- 15 people
- Sponsor
- Arctic Therapeutics
Primary Hyperoxaluria Type 1, PH1
Study of YOLT-203 in Children and Adults With Primary Hyperoxaluria Type 1 (PH1)
This study will be conducted to evaluate the efficacy and safety of YOLT-203 in children and adults with Primary Hyperoxaluria Type 1.
- Trial name
- NCT07587021
- Testing
- YOLT-203
- Where
- 4 sites in United States
- Volunteers
- 36 people
- Sponsor
- YolTech Therapeutics Co., Ltd
Friedreich's Ataxia (FA)
A Study of SGT-212 Gene Therapy in Friedreich's Ataxia
This is a phase 1b, first in-human, open-label, dose-finding study investigating the safety and tolerability of SGT-212 in participants with Friedreich's ataxia (FA).
- Trial name
- FALCON (NCT07180355)
- Testing
- SGT-212
- Where
- 3 sites in United States
- Volunteers
- 10 people
- Sponsor
- Solid Biosciences Inc.
Bullous Pemphigoid
A Study to Investigate the Use of Benralizumab in Patients With Bullous Pemphigoid.
The purpose of this study is to investigate the use of benralizumab is effective in the treatment of patients symptomatic Bullous Pemphigoid (BP).
- Trial name
- FJORD (NCT04612790)
- Testing
- Benralizumab
- Where
- 39 sites in United States, Australia and 9 more
- Volunteers
- 67 people
- Sponsor
- AstraZeneca
Autoimmune Hepatitis
Effect of JKB-122 on Prednisolone and Azathioprine Induced Remission in Autoimmune Hepatitis (AIH)
This is a Phase 2 study. All patients will receive prednisolone and AZA as standard of care (SOC) during the study. At the end of the study, all data collected will be analyzed the efficacy and safety of JKB-122 on SOC reduction…
- Trial name
- NCT04371718
- Testing
- JKB-122
- Volunteers
- 120 people
- Sponsor
- TaiwanJ Pharmaceuticals Co., Ltd
Hemoglobinopathies, Myelodysplastic Syndromes
Magnetic Resonance Imaging (MRI) Assessments of the Heart and Liver Iron Load in Patients With Transfusion Induced Iron Overload
This study will evaluate the change in cardiac iron load over a 53 week period measured by MRI in 2 cohorts of patients
- Conditions
- Hemoglobinopathies, Myelodysplastic Syndromes, Other Inherited or Acquired Anaemia, MPD Syndrome
- Trial name
- NCT00673608
- Testing
- deferasirox
- Where
- 4 sites in Australia
- Volunteers
- 118 people
- Sponsor
- Novartis
Healthy, Lupus
Direct to Patient Minimal Risk Biospecimen and Data Collection Research
This study aims to help researchers better understand health conditions and develop improved tests, treatments, and cures for diseases.
- Conditions
- Healthy, Lupus, Celiac, Kidney Disease
- Trial name
- JBMR (NCT07128966)
- Testing
- Observation
- Where
- United States
- Volunteers
- 100,000 people, healthy volunteers welcome
- Sponsor
- Joined Bio
Central Nervous System Neoplasms, Glioblastoma
Phase I Study of Oral ONC206 in Recurrent and Rare Primary Central Nervous System Neoplasms
The primary objective of this Phase 1, open-label, dose-escalation, and exploratory study is to evaluate the safety and tolerability profile (establish the maximum-tolerated dose) and evaluate the occurrence of dose-limiting…
- Conditions
- Central Nervous System Neoplasms, Glioblastoma, Gliosarcoma, Adult, Anaplastic Oligodendroglioma
- Trial name
- NCT04541082
- Testing
- ONC206
- Where
- United States
- Volunteers
- 102 people
- Sponsor
- Jazz Pharmaceuticals
Huntington Disease
An Extension Study to Evaluate the Long-Term Safety and Efficacy of Votoplam in Participants With Huntington's Disease (HD)
The primary goal of this study is to evaluate the long-term safety and pharmacodynamic effects of votoplam in participants with HD.
- Trial name
- NCT06254482
- Testing
- Votoplam
- Where
- 24 sites in Australia, Austria and 8 more
- Volunteers
- 144 people
- Sponsor
- Novartis Pharmaceuticals
Pulmonary Arterial Hypertension
Safety and Efficacy of BIA 5-1058 in PAH
This study aims to assess the safety and tolerability of the individual highest tolerated zamicastat doses, achieved in the study BIA-51058-201, during long-term treatment in Pulmonary Arterial Hypertension (PAH) disease.
- Trial name
- NCT06009185
- Testing
- Oral zamicastat
- Where
- 11 sites in Austria, Germany and 4 more
- Volunteers
- 20 people
- Sponsor
- Bial - Portela C S.A.
Amyotrophic Lateral Sclerosis
GM604 Phase 2A Randomized Double-blind Placebo Controlled Pilot Trial in Amyotrophic Lateral Disease (ALS)
GM604 is an endogenous human embryonic stage neural regulatory and signaling peptide that controls the development, monitoring and correction of the human nervous system.
- Trial name
- GALS (NCT01854294)
- Testing
- GM604
- Where
- 2 sites in United States
- Volunteers
- 12 people
- Sponsor
- Genervon Biopharmaceuticals, LLC
Solid Advanced Tumor, Lymphoma
Dose-finding and Dose Expansion Study of OSE-279 in Subjects With Advanced Solid Tumors or Lymphomas
This is a phase 1/2, multicenter, dose-finding and dose expansion study of OSE-279, a PD-1 blocking monoclonal antibody, in subjects with advanced solid tumors or lymphomas.
- Conditions
- Solid Advanced Tumor, Lymphoma, NSCLC (Non-small Cell Lung Cancer)
- Trial name
- NCT05751798
- Testing
- Part A: OSE-279 100mg, Part A: OSE-279 300mg, Part A: OSE-279 600mg
- Where
- 11 sites in Belgium, France and 1 more
- Volunteers
- 41 people
- Sponsor
- OSE Immunotherapeutics