Clinical trials: Multiple Anomalies
Brain and Central Nervous System Tumors, Chronic Myeloproliferative Disorders
Methadone, Morphine, or Oxycodone in Treating Pain in Patients With Cancer
RATIONALE: Methadone, morphine, or oxycodone may help relieve pain caused by cancer. It is not yet known whether methadone is more effective than morphine or oxycodone in treating pain in patients with cancer.
- Conditions
- Brain and Central Nervous System Tumors, Chronic Myeloproliferative Disorders, Leukemia, Lymphoma
- Trial name
- NCT00726830
- Testing
- methadone hydrochloride, morphine sulfate, oxycodone hydrochloride
- Where
- 2 sites in United States
- Volunteers
- 1 people
- Sponsor
- M.D. Anderson Cancer Center
Chronic Myeloproliferative Disorders, Graft Versus Host Disease
Fludarabine, Cyclophosphamide, and Total-Body Irradiation Followed by Cyclosporine and Mycophenolate Mofetil in Treating Patients Who Are Undergoing a Donor Umbilical Cord Blood Transplant for Hematologic Cancer
RATIONALE: Giving low doses of chemotherapy, such as fludarabine and cyclophosphamide, and radiation therapy before a donor umbilical cord blood stem cell transplant helps stop the growth of cancer cells.
- Conditions
- Chronic Myeloproliferative Disorders, Graft Versus Host Disease, Leukemia, Lymphoma
- Trial name
- NCT00255684
- Testing
- graft-versus-tumor induction therapy, cyclophosphamide, cyclosporine
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- University of Rochester
Primary Immunodeficiency Disease
An Extension Study of TAK-664 for Japanese People With Primary Immunodeficiency Disease
This study is an extension study for participants with primary immunodeficiency disorders who were previously treated with IGSC, 20% in the TAK-664-3001 study.
- Trial name
- NCT04842643
- Testing
- IGSC 20% infusion
- Where
- 8 sites in Japan
- Volunteers
- 12 people
- Sponsor
- Takeda
Atopic Dermatitis
A Phase 2 Study of CIM331 for Atopic Dermatitis Patients
To assess the safety, tolerability and efficacy of CIM331, compared to placebo, in atopic dermatitis patients who are inadequately controlled by or intolerant to topical therapy
- Trial name
- NCT01986933
- Testing
- nemolizumab (CIM331)
- Where
- 18 sites in United States
- Volunteers
- 264 people
- Sponsor
- Chugai Pharmaceutical
Pulmonary Hypertension, Familial Persistent, of the Newborn
A Study To Evaluate Safety And Efficacy Of IV Sildenafil In The Treatment Of Neonates With Persistent Pulmonary Hypertension Of The Newborn
This study will evaluate whether IV sildenafil can reduce the time on inhaled nitric oxide treatment and reduce the failure rate of available treatments for persistent pulmonary hypertension of the newborn.
- Trial name
- NCT01720524
- Testing
- iv sildenafil
- Where
- 42 sites in United States, Belgium and 10 more
- Volunteers
- 59 people
- Sponsor
- Pfizer's Upjohn has merged with Mylan to form Viatris Inc.
Pompe Disease
Drug-drug Interaction Study
This study evaluates drug-drug interactions between AT2220 (duvoglustat) and recombinant human alpha-glucosidase (rhGAA, also known as alglucosidase alfa) in participants with Pompe Disease.
- Trial name
- NCT01380743
- Testing
- duvoglustat, rhGAA
- Where
- 15 sites in United States, Canada and 2 more
- Volunteers
- 25 people
- Sponsor
- Amicus Therapeutics
Hereditary Angioedema (HAE)
A Double-Blind, Multiple Ascending Dose Study to Assess Safety, Tolerability and Pharmacokinetics of DX-2930 in Hereditary Angioedema Participants
The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK) profile of multiple subcutaneous administrations of DX-2930 across a range of doses in HAE participants.
- Trial name
- NCT02093923
- Testing
- DX-2930
- Where
- 14 sites in United States, Italy and 1 more
- Volunteers
- 38 people
- Sponsor
- Shire
Atopic Dermatitis
JAK1 Inhibitor With Medicated Topical Therapy in Adolescents With Atopic Dermatitis
This is a randomized, double blind, placebo controlled, parallel group, Phase 3 study to evaluate the efficacy and safety of PF 04965842 in adolescent participants 12 to <18 years of age with moderate to severe AD.
- Trial name
- JADE TEEN (NCT03796676)
- Testing
- PF-04965842, PF04965842
- Where
- 136 sites in United States, Australia and 12 more
- Volunteers
- 287 people
- Sponsor
- Pfizer
Kidney, Polycystic
HALT Progression of Polycystic Kidney Disease Study B
The efficacy of interruption of the renin-angiotensin-aldosterone system (RAAS) on the progression of cystic disease and on the decline in renal function in autosomal dominant kidney disease (ADPKD) will be assessed in two…
- Trial name
- HALT PKD B (NCT01885559)
- Testing
- Lisinopril, Telmisartan
- Where
- 7 sites in United States
- Volunteers
- 486 people
- Sponsor
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
CAH - Congenital Adrenal Hyperplasia
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NBI-74788 (Crinecerfont) in Pediatric Participants With Congenital Adrenal Hyperplasia
This is a Phase 2, open-label, multiple-dose, study to assess the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of NBI-74788 (crinecerfont) in pediatric participants (14 to 17 years of age) with a…
- Trial name
- NCT04045145
- Testing
- Crinecerfont
- Where
- 6 sites in United States
- Volunteers
- 8 people
- Sponsor
- Neurocrine Biosciences
Down Syndrome
Efficacy Assessment of Systematic Treatment With Folinic Acid and Thyroid Hormone on Psychomotor Development of Down Syndrome Young Children
Evaluation of the following in very young children with Down syndrome: the efficacy of systematic treatment with L-thyroxine at controlled doses (clinically and by ultrasensitive thyreostimulating hormone (TSH) assay), the…
- Trial name
- ACTHYF (NCT01576705)
- Testing
- thyroid hormone and folinic acid
- Where
- France
- Volunteers
- 175 people
- Sponsor
- Institut Jerome Lejeune
Phenylketonuria
A Study to Evaluate Subcutaneously Administered rAvPAL-PEG in Patients With Phenylketonuria for 24 Weeks
The primary objective of the study is to evaluate the effect of dosing regimens of multiple subcutaneous (SC) doses of rAvPAL-PEG to induce an early and sustained Phe reduction while decreasing the frequency and severity of…
- Trial name
- NCT01560286
- Testing
- BMN 165 (rAvPAL-PEG)
- Where
- 6 sites in United States
- Volunteers
- 24 people
- Sponsor
- BioMarin Pharmaceutical
Dermatitis, Atopic
An Ascending Multiple Dose Study of VTP-38543 in Adult Participants With Mild to Moderate Atopic Dermatitis
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and preliminary clinical efficacy of VTP-38543 administered as a cream, twice-daily, for 28 days in otherwise healthy adult…
- Trial name
- NCT02655679
- Testing
- VTP-38543, Vehicle with Transcutol®P, Vehicle without Transcutol®P
- Where
- 12 sites in United States, Canada
- Volunteers
- 104 people
- Sponsor
- Vitae Pharmaceuticals, Inc.
Von Hippel-Lindau Disease
Natural History and Management of Pancreatic Lesions in Von Hippel-Lindau Disease
Von Hippel-Lindau disease (VHL) is an inherited cancer syndrome. Patients are at risk for developing pancreatic cysts and tumors. These tumors are more aggressive in some people than in others.
- Trial name
- NCT00062166
- Where
- United States
- Volunteers
- 340 people
- Sponsor
- National Cancer Institute (NCI)
Congenital Adrenal Hyperplasia, CAH - Congenital Adrenal Hyperplasia
Study of SPR001 in Adults With Classic Congenital Adrenal Hyperplasia
This is a multicenter Phase 2, multiple dose, dose escalation study to evaluate the safety, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of SPR001 in adult patients with classic congenital adrenal hyperplasia (CAH).
- Trial name
- NCT03257462
- Testing
- SPR001
- Where
- 9 sites in United States
- Volunteers
- 24 people
- Sponsor
- Spruce Biosciences
Cystic Fibrosis
Effects of an Antioxidant-Enriched Multivitamin Supplement on Inflammation and Oxidative Stress in Cystic Fibrosis
The purpose of this study will be to evaluate the effects of a modified formulation of AquADEKs (AquADEKs-2) on markers of inflammation, antioxidant levels and oxidative stress.
- Trial name
- AquADEKs-2 (NCT01859390)
- Testing
- AquADEKs-2, control multivitamin
- Where
- 15 sites in United States
- Volunteers
- 73 people
- Sponsor
- University of Colorado, Denver
Chronic Hepatitis C Infection
Open-Labeled Study of PSI-7977 and RBV With and Without PEG-IFN in Treatment-Naïve Patients With HCV GT2 or GT3
This study is to assess the safety and tolerability of sofosbuvir (SOF) 400 mg with and without ribavirin (RBV) and/or with and without pegylated interferon alfa-2a (PEG) in subjects with genotype 1, 2 or 3 hepatitis C (HCV)…
- Trial name
- NCT01260350
- Testing
- SOF, RBV, PEG
- Where
- 2 sites in New Zealand
- Volunteers
- 292 people
- Sponsor
- Gilead Sciences
Tuberous Sclerosis Complex
Adjunctive GNX Treatment Compared With Placebo in Children and Adults With TSC-related Epilepsy
This is a Phase 3, global, double-blind, randomized, placebo-controlled study of adjunctive GNX treatment in children and adults with TSC-related epilepsy.
- Trial name
- NCT05323734
- Testing
- Ganaxolone
- Where
- 63 sites in United States, Australia and 8 more
- Volunteers
- 129 people
- Sponsor
- Marinus Pharmaceuticals
Tumors Characterized by Genetic Abnormalities of ALK
A Dose Escalation/Expansion Study of LDK378 in Patients With Tumors Characterized by Genetic Abnormalities in Anaplastic Lymphoma Kinase
This study assessed the safety and efficacy of LDK378 in adult patients with genetic abnormalities in anaplastic lymphoma kinase (ALK).
- Trial name
- NCT01283516
- Testing
- LDK378
- Where
- 21 sites in United States, Australia and 9 more
- Volunteers
- 304 people
- Sponsor
- Novartis Pharmaceuticals
Huntington's Disease
Study to Measure Cerebrospinal Fluid Mutant Huntingtin Protein in Participants With Early Manifest Stage I or Stage II Huntington's Disease
The study is designed as a multi-site, prospective, 15-month longitudinal, cohort study measuring CSF mHTT in participants with early manifest Stage I or Stage II Huntington's Disease (HD).
- Trial name
- NCT03664804
- Testing
- No Study Drug was Administered in this Study
- Where
- 15 sites in United States, Canada and 2 more
- Volunteers
- 95 people
- Sponsor
- Hoffmann-La Roche