Search clinical trials for: Orphan drug

Total 260840 results

Bioprojet

Completed

Efficacy and Safety Study of BF2.649 in the Treatment of Excessive Daytime Sleepiness in Narcolepsy (Harmony1)

Sleep Disorders | Narcolepsy | Cataplexy | Excessive Daytime Sleepiness

Switzerland
Emphycorp
Cellular Sciences, inc.

Completed

Inhaled Sodium Pyruvate for the Treatment of Cystic Fibrosis.

Cystic Fibrosis

United States
Children's Hospital Medical Center, Cincinnati
FDA Office of Orphan Products Development

Completed

Oxandrolone for the Treatment of Bone Marrow Aplasia in Fanconi Anemia

Fanconi Anemia

United States
Medical University of South Carolina
Merck Sharp & Dohme LLC; FDA Office of Orphan Products Development

Completed

Safety and Efficacy of Alendronate (Fosamax) in Children With Osteoporosis

Osteoporosis

United States
University of Florence

Completed

High-dose Ibuprofen for Patent Ductus Arteriosus (PDA) in Preterm Infant

Ductus Arteriosus, Patent

Italy
AOP Orphan Pharmaceuticals AG

Completed

Anagrelide vs. Hydroxyurea - Efficacy and Tolerability Study in Patients With Essential Thrombocythaemia (ANAHYDRET)

Essential Thrombocythaemia

Austria, Czech Republic, France, Germany, Hungary, Italy, Lithuania, Poland, Singapore, Slovenia
Institut National de la Santé Et de la Recherche...

Completed

Interleukin-1 Receptor Antagonist (IL-1RA) (ANAKINRA) IN SEVERE SYSTEMIC-ONSET JUVENILE IDIOPATHIC ARTHRITIS

Systemic-Onset Juvenile Idiopathic Arthritis

France
Amgen

Completed

Evaluating Kineret® (Anakinra) in Rheumatoid Arthritis (RA) Subjects Using aSelf-Reported Questionnaire

Rheumatoid Arthritis
Amgen

Completed

Evaluation of the Efficacy of Combination Treatment With Anakinra and Pegsunercept in Improving Rheumatoid Arthritis

Rheumatoid Arthritis
Amgen

Terminated

The SPECTRA Study

Rheumatoid Arthritis
Amgen

Completed

Juvenile Rheumatoid Arthritis

Juvenile Chronic Arthritis
AOP Orphan Pharmaceuticals AG

Completed

Lancovutide (Moli1901) Inhalation Solution Study in Adolescents and Adults With Cystic Fibrosis

Cystic Fibrosis

Austria
Swedish Orphan Biovitrum
Amgen

Withdrawn

A Study of Palifermin for the Reduction of Oral Mucositis in Subjects With Locally Advanced Head and Neck Cancer Receiving Postoperative Radiotherapy

Head and Neck Cancer
Orphan Medical
Johns Hopkins University

Completed

Physiological, Behavioral and Subjective Effects of Drugs (GHB)

Sedative Abuse

United States
National Institute of Arthritis and Musculoskeletal...
FDA Office of Orphan Products Development

Completed

Etanercept for Wegener's Granulomatosis

Wegener's Granulomatosis

United States
Orphanetics Pharma Entwicklungs GmbH

Terminated

Long-Term Tetrahydrobiopterin Treatment in PKU Patients of 0-18 Years - Study on Phenylalanine Tolerance and Safety

Phenylalanine Hydroxylase Deficiencies
National Cancer Center, Korea

Unknown

Study of Genetic Polymorphisms of Drug Transporters and Orphan Nuclear Receptors on Treatment Effects of Irinotecan

Colorectal Neoplasms | Secondary

Korea, Republic of
The Avicena Group

Unknown

Study of Creatine Monohydrate in Patients With Amyotrophic Lateral Sclerosis

Amyotrophic Lateral Sclerosis (ALS)

United States
Orphan Medical

Completed

Safety and Efficacy of Xyrem Oral Solution (Sodium Oxybate) Compared With Placebo in Narcoleptic Patients

Narcolepsy

United States, Canada, Switzerland
Brigham and Women's Hospital
National Institute of General Medical Sciences (NIGMS)

Not yet recruiting

Understanding the Acute Pain Phenotype in Patients Undergoing Surgery

Acute Pain | Opioid Use | Chronic Post Operative Pain

United States
Sisli Hamidiye Etfal Training and Research Hospital

Not yet recruiting

Frequency of Adverse Events in Pediatric Patients Receiving Sedation for Magnetic Resonance Imaging

Pediatric Anesthesia | Magnetic Resonance Imaging | Sedation | Adverse Effect
Guy's and St Thomas' NHS Foundation Trust

Not yet recruiting

Mechanistic Insights From Temporary Pacing in HFpEF

Heart Failure Preserved Ejection Fraction | Pacing
Castilla-La Mancha Health Service

Not yet recruiting

Clinical Utility of Tight Objectives of Advanced Hybrid Closed-loop Systems Among Type 1 Diabetes Patients (TightT1AHCL) (TightT1AHCL)

Type 1 Diabetes

Spain
TC Erciyes University

Not yet recruiting

Comparing Inhalation and Total Intravenous Anesthesia Methods

Hernia, Inguinal | Children

Turkey
Samsung Medical Center

Recruiting

Systemic Treatment Alone Versus Systemic Treatment Plus Radiotherapy in Oligometastatic Renal Cell Carcinoma (SABLOR)

Kidney Neoplasm

Korea, Republic of
University of Aarhus

Recruiting

Strategy for Withdrawal of Pharmacological Treatment for Urinary Incontinence in Children (StayDry)

Urinary Incontinence in Children

Denmark
Kumho HT Inc.

Recruiting

Study of DNP002 in Patients With Solid Tumors

Advanced Solid Tumor

Korea, Republic of
University of Delaware

Recruiting

Daily Stress and Vascular Function in Midlife as a Risk Factor for Cognitive Decline

Middle-aged Adults

United States
Pfizer

Not yet recruiting

A Study to Learn How Different Forms of The Study Medicine Called Phenytoin Are Taken up Into the Blood in Healthy Adults

Healthy
Copernicus Memorial Hospital
Medical Research Agency, Poland

Not yet recruiting

Application of MET-PET in Fusion With MRI in the Treatment of Glioblastoma Multiforme

Glioblastoma Multiforme

Poland
University of Nottingham
Wellcome Trust

Not yet recruiting

Measuring and Mapping Cognitive Decline After Brain Radiosurgery (CoDeB-Rad)

Quality of Life | Cognitive Decline | Brain Metastases | SRS | Meningiomas | Stereotactic Radiosurgery

United Kingdom
Northeast Normal University

Recruiting

Metacognitive Therapy for Obsessive-Compulsive Disorder (MCTOCD)

Obsessive-Compulsive Disorder

China
Shanghai Bovax Biotechnology Co., Ltd.

Not yet recruiting

A Phase III Study to Evaluate the Efficacy, Immunogenicity and Safety of the 9-valent HPV Vaccine in Chinese Males

Anal Cancer | HPV-Related Carcinoma | Penile Cancer | Genital Wart | AIN3 | AIN2 | PIN-1 | AIN1 | PIN2 | PIN3

China
Second Affiliated Hospital, School of Medicine,...
Huashan Hospital; The Affiliated Nanjing Drum Tower Hospital of Nanjing University... and other collaborators

Not yet recruiting

Tract-based Artificial Intelligence (AI) Robot Guiding System in Basal Ganglion Hemorrhage Evacuation

Basal Ganglia Hemorrhage

China
University of Birmingham
Université d'Abomey-Calavi; Kigali University Teaching Hospital

Not yet recruiting

A Stratified, Multi-ARm, muLti-site Randomised Platform Trial Aiming to Reduce the INcidence of Post-operative SSI (MARLIN)

Surgical Site Infection

South Africa, Ghana, Benin, India, Mexico, Nigeria, Rwanda
Radboud University Medical Center

Not yet recruiting

Methylphenidate in KBG Syndrome: N-of-1 Series (KBGS_N_of_1)

ADHD - Combined Type | Kbg Syndrome
Second Affiliated Hospital, School of Medicine,...

Not yet recruiting

Efficacy and Safety of Dupilumab in Combination With Tofacitinib in Moderate to Severe Adult AD Patients

Atopic Dermatitis
Drexel University
University of Michigan; Bar Ilan University

Not yet recruiting

UnityPhilly Response App for Overdose Reversal

Opioid Overdose
Kerry Rogers

Not yet recruiting

Pirtobrutinib (LOXO-305) and Venetoclax for the Treatment of Patients With CLL or SLL Resistant to Covalent BTKi

Chronic Lymphocytic Leukemia | Small Lymphocytic Lymphoma

United States
Suzhou Suncadia Biopharmaceuticals Co., Ltd.

Not yet recruiting

Adebrelimab With or Without SHR-8068 in Combination With Cisplatin Plus Gemcitabine as First-line Treatment in Patients With Advanced Biliary Tract Cancer

Biliary Tract Cancer
Ferring Pharmaceuticals

Not yet recruiting

An Exploratory Study to Investigate the Effect of FE 999302 When Given Together With Follitropin Delta During Controlled Ovarian Stimulation (Celestial-1)

Fertility
The First Affiliated Hospital of Zhejiang Chinese...

Recruiting

Effect and Mechanism of Auricular Point Stimulation on Functional Dyspepsia With Sleep Disorder

Functional Dyspepsia | Sleep Disorder

China
M.D. Anderson Cancer Center

Not yet recruiting

Prebiotic Food-enriched Diet (PreFED) to Enhance the Microbiome and Response to First-line Immunotherapy in Unresectable Melanoma

Unresectable Melanoma

United States
Brigham and Women's Hospital
Gilead Sciences; Trutag Technologies

Not yet recruiting

Development of a Silica Microparticle Taggant System to Measure ART Adherence (TruTag)

Medication Adherence | Hiv | Compliance, Medication
Asociación Española para el Estudio del Hígado

Recruiting

Spanish Wilson Disease Registry

Wilson Disease

Spain
University of Reading
King's College London; University of Leeds

Not yet recruiting

The Impact of Faba Bean Rich Bread on Iron Status, Postprandial Lipaemia and Satiety (RtP)

Anemia, Iron Deficiency | Low Iron Stores | Cardiometabolic Disease

United Kingdom
Shanghai BDgene Co., Ltd.

Recruiting

A Phase 1 Study of the Safety and Efficacy of Gene Modified Autologous Hematopoietic Stem Cell in β-thalassemia Major

β-thalassemia

China
Tanta University

Not yet recruiting

Fexofenadine Versus Gabapentin for Uremic Pruritus in Patients on Regular Hemodialysis

Hemodialysis | ESRD | Renal Replacement Therapy | End-stage Renal Disease | Uremic Pruritus | Chronic Kidney Disease-associated Pruritus

Egypt
Sohag University

Recruiting

Immunohistopathologic Findings of Cesarean Scar Niche

Cesarean Scar Niche

Egypt
Genexine, Inc.

Recruiting

Efepoetin Alfa for Treatment of Anemia in Chronic Kidney Disease on Dialysis

Anemia of Chronic Kidney Disease

Korea, Republic of

Search clinical trials for: Orphan drug

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