Clinical trials: Rare Disease
Skin Ulcers, Sickle Cell Anemia
Phase II Randomized Trial:Arginine Butyrate Plus Standard Local Therapy in Patients With Refractory Sickle Cell Ulcers
OBJECTIVES: I. Compare the efficacy of local care alone vs local care plus arginine butyrate in terms of healing rate in patients with refractory sickle cell ulcers. II.
- Trial name
- NCT00004412
- Testing
- Arginine Butyrate, Standard local care dressing
- Where
- 4 sites in United States
- Volunteers
- 23 people
- Sponsor
- Susan P. Perrine
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
Kohlmeier Degos Disease With Neurologic Involvement, Kohlmeier-Degos Disease
JAK/STAT Inhibition in CNS Kohlmeier-Degos Disease
Background: Kohlmeier-Degos (K-D) is a rare disease that leads to the inflammation and/or blockage of small blood vessels in many organs; these can include the skin, eyes (rare), small bowels, lungs, heart, and the brain and…
- Trial name
- NCT05998395
- Testing
- Ruxolitinib
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Wilms Tumor, Fibrosarcoma
Study of High-Dose Chemotherapy With Bone Marrow or Stem Cell Transplant for Rare Poor-Prognosis Cancers
The purpose of this study is to determine whether very high dosages of chemotherapy will improve the chance of surviving cancer.
- Conditions
- Wilms Tumor, Fibrosarcoma, Carcinoma, Round Cell, Nasopharyngeal Cancer
- Trial name
- NCT00141765
- Testing
- Myeloablative Chemotherapy, Stem Cell Rescue
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- University of Michigan Rogel Cancer Center
Neuroblastoma, Medulloblastoma
Molecular-Guided Therapy for Relapsed and Refractory Childhood Cancer
The purpose of this study is to test the feasibility (ability to be done) of experimental technologies to determine a tumor's molecular makeup (gene expression profile) and mutations.
- Conditions
- Neuroblastoma, Medulloblastoma, Brain Tumors, Rare Tumors
- Trial name
- NCT01802567
- Testing
- Guided Therapy- Pediatric Gene Analysis Platform
- Where
- 13 sites in United States
- Volunteers
- 52 people
- Sponsor
- Giselle Sholler
Lung Cancer
Osimertinib Plus Chemotherapy in Uncommon EGFRm NSCLC
This is an open-label, single-arm, multicenter, exploratory Phase II study sponsored by Astrazeneca Investment (China) Co., LTD.
- Trial name
- MINOVA (NCT05215951)
- Testing
- osimertinib
- Where
- 6 sites in China
- Volunteers
- 4 people
- Sponsor
- AstraZeneca
Dravet Syndrome, Lennox Gastaut Syndrome
A Study to Investigate the Long-Term Safety of ZX008 (Fenfluramine Hydrochloride) Oral Solution in Children and Adults With Epileptic Encephalopathy Including Dravet Syndrome and Lennox-Gastaut Syndrome
This is an international, multicenter, open-label, long-term safety study of ZX008 in subjects with Dravet syndrome, Lennox-Gastaut syndrome or epileptic encephalopathy
- Conditions
- Dravet Syndrome, Lennox Gastaut Syndrome, Epileptic Encephalopathy
- Trial name
- NCT03936777
- Testing
- ZX008 (Fenfluramine Hydrochloride)
- Where
- 71 sites in United States, Australia and 12 more
- Volunteers
- 412 people
- Sponsor
- Zogenix, Inc.
Calciphylaxis, Calcific Uremic Arteriolopathy
Phase 2 Study With SNF472 in Calciphylaxis Patients
To evaluate the effect of SNF472 on top of standard of care on promoting wound healing and other parameters of therapeutic response in haemodialysis patients with calciphylaxis (calcific uraemic arteriolopathy, CUA).
- Trial name
- NCT02790073
- Testing
- SNF472
- Where
- 3 sites in United States, United Kingdom
- Volunteers
- 14 people
- Sponsor
- Sanifit Therapeutics S. A.
Meconium Aspiration
Phase III Randomized Study of Lucinactant in Full Term Newborn Infants With Meconium Aspiration Syndrome
OBJECTIVES: Evaluate the safety and efficacy of lucinactant administered by bronchoalveolar lavage (BAL) in the treatment of meconium aspiration syndrome (MAS) in newborn infants.
- Trial name
- NCT00004500
- Testing
- Lucinactant, Standard Care
- Where
- United States
- Volunteers
- 69 people
- Sponsor
- Windtree Therapeutics
Alpha-1 Antitrypsin Deficiency (AATD), Pi*ZZ, Pi*SZ, Pi*Null, Another Rare Phenotype/Genotype Known to be Associated With Either Low or Functionally Impaired AAT Including F or I Mutations
Alvelestat (MPH966) for the Treatment of ALpha-1 ANTitrypsin Deficiency
This is a Phase 2, multicenter, double-blind, randomized (1:1), placebo-controlled, 12-week, proof-of-concept study to evaluate the safety and tolerability as well as the mechanistic effect of oral administration of alvelestat…
- Conditions
- Alpha-1 Antitrypsin Deficiency (AATD), Pi*ZZ, Pi*SZ, Pi*Null, Another Rare Phenotype/Genotype Known to be Associated With Either Low or Functionally Impaired AAT Including F or I Mutations, Emphysema or COPD
- Trial name
- ATALANTa (NCT03679598)
- Testing
- Alvelestat (MPH966)
- Where
- 10 sites in United States
- Volunteers
- 63 people
- Sponsor
- University of Alabama at Birmingham
Anemia, Kidney Failure
Dynepo Infrequent Dosing Study
The purpose of this study is to demonstrate non-inferiority of efficacy between twice weekly and once weekly dose schedule of Dynepo in previously erythropoietin (EPO)-naive patients, as measured by haemoglobin at week 24 and…
- Trial name
- NCT00450333
- Testing
- Dynepo (Epoetin delta), Dynepo
- Where
- 53 sites in Austria, Belgium and 5 more
- Volunteers
- 407 people
- Sponsor
- Shire
Rare Diseases
A Study to Evaluate Emapalumab in Japanese Healthy Volunteers.
This is a randomized, placebo controlled and double-blinded study to evaluate the pharmacokinetics (PK), pharmacodynamics (PD) and safety of a single dose (1 mg/kg) of emapalumab in adult healthy Japanese subjects.
- Trial name
- NCT04765553
- Testing
- NI-0501, Saline
- Where
- Japan
- Volunteers
- 8 people, healthy volunteers welcome
- Sponsor
- Swedish Orphan Biovitrum
Genetic Obesity, Obesity
Setmelanotide Phase 2 Treatment Trial in Participants With Rare Genetic Disorders of Obesity
The purpose of the study was to determine the effect of setmelanotide (RM-493) on weight, hunger assessments, and other factors in participants with rare genetic disorders of obesity.
- Conditions
- Genetic Obesity, Obesity, Obesity Due to Melanocortin 4 Receptor Deficiency
- Trial name
- NCT03013543
- Testing
- Setmelanotide
- Where
- 58 sites in United States, Canada and 7 more
- Volunteers
- 213 people
- Sponsor
- Rhythm Pharmaceuticals, Inc.
Pemphigus Vulgaris
Efficacy and Safety of Ofatumumab in Treatment of Pemphigus Vulgaris
Pemphigus vulgaris (PV) is a rare, chronic, debilitating, and potentially life-threatening autoimmune disorder that is characterized by mucocutaneous blisters.
- Trial name
- NCT01920477
- Testing
- Ofatumumab
- Where
- 16 sites in United States, Australia and 6 more
- Volunteers
- 35 people
- Sponsor
- Novartis Pharmaceuticals
Adrenocortical Carcinoma, Peritoneal Carcinomatosis
Surgery and Heated Chemotherapy for Adrenocortical Carcinoma
Background: Adrenocortical carcinoma (ACC) is a rare tumor of the adrenal gland. Few people who develop this disease live more than 5 years after being diagnosed.
- Trial name
- NCT01833832
- Testing
- Cisplatin, Cytoreductive surgery, sodium thiosulfate
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- National Cancer Institute (NCI)
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