Clinical trials: Adrenal Insufficiency
Adrenoleukodystrophy, Metachromatic Leukodystrophy
Stem Cell Transplant for Inborn Errors of Metabolism
The purpose of this study is to determine the safety and engraftment of donor hematopoietic cells using this conditioning regimen in patients undergoing a hematopoietic (blood forming) cell transplant for an inherited metabolic…
- Conditions
- Adrenoleukodystrophy, Metachromatic Leukodystrophy, Globoid Cell Leukodystrophy, Gaucher's Disease
- Trial name
- NCT00176904
- Testing
- Stem Cell Transplant, Busulfan, Cyclophosphamide, Antithymocyte Globulin
- Where
- United States
- Volunteers
- 135 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Cerebral Adrenoleukodystrophy (CALD)
A Study of the Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Cerebral Adrenoleukodystrophy (CALD)
This trial assessed the efficacy and safety of autologous cluster of differentiation 34 (CD34+) hematopoietic stem cells, transduced ex-vivo with Lenti-D lentiviral vector (also called elivaldogene autotemcel or eli-cel), for the…
- Trial name
- NCT01896102
- Testing
- Lenti-D Drug Product (eli-cel)
- Where
- 8 sites in United States, Argentina and 4 more
- Volunteers
- 32 people
- Sponsor
- Genetix Biotherapeutics Inc.
Adrenal Insufficiency
Once-daily Oral Modified Release Hydrocortisone in Patients With Adrenal Insufficiency
This is a randomised, controlled, open, two-armed, two-period cross-over, multi-centre phase II/III study to assess the safety, tolerability and pharmacokinetics of once-daily oral modified-release hydrocortisone in comparison to…
- Trial name
- NCT00915343
- Testing
- hydrocortisone (modified release), oral tablet 20 and 5 mg, Hydrocortisone, oral tablet, 10 mg
- Volunteers
- 64 people
- Sponsor
- Shire
Adrenoleukodystrophy, Metachromatic Leukodystrophy
HSCT for High Risk Inherited Inborn Errors
Hematopoietic stem cell transplantation has proven effective therapy for individuals with adrenoleukodystrophy (ALD), metachromatic leukodystrophy (MLD) or globoid cell leukodystrophy (GLD, or Krabbe disease).
- Conditions
- Adrenoleukodystrophy, Metachromatic Leukodystrophy, Globoid Cell Leukodystrophy, Tay Sachs Disease
- Trial name
- NCT00383448
- Testing
- Clofarabine, Total body Irradiation, Melphalan
- Where
- United States
- Volunteers
- 38 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Central Adrenal Insufficiency, Mifepristone
Mifepristone Dynamic Testing for Diagnosis for Central Adrenal Insufficiency
The researchers propose the use of mifepristone as an alternative way to test for Central Adrenal Insufficiency (CAI). They want to assess the feasibility of recruitment and the efficacy of the purposed method.
- Trial name
- NCT04588688
- Testing
- Mifepristone
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Tobias Else
Adrenoleukodystrophy
Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy
OBJECTIVES: I. Evaluate the clinical efficacy of combination glyceryl trierucate and glyceryl trioleate (Lorenzo's Oil) therapy in boys with X-linked adrenoleukodystrophy. II.
- Trial name
- NCT00004418
- Testing
- glyceryl trierucate/glyceryl trioleate
- Where
- 2 sites in United States
- Volunteers
- 126 people
- Sponsor
- Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
X-linked Adrenoleukodystrophy
Exercise Study of Function and Pathology for Women With X-linked Adrenoleukodystrophy
The purpose is to see how X-linked adrenoleukodystrophy (X-ALD) is associated with strength and sensation using MRI, in women with X-ALD.
- Trial name
- NCT01594853
- Testing
- exercise training
- Where
- United States
- Volunteers
- 31 people, healthy volunteers welcome
- Sponsor
- Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Bone Marrow Failure Syndrome, Thalassemia
Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation
In this study, the investigators test 2 dose levels of thiotepa (5 mg/kg and 10 mg/kg) added to the backbone of targeted reduced dose IV busulfan, fludarabine and rabbit anti-thymocyte globulin (rATG) to determine the minimum…
- Conditions
- Bone Marrow Failure Syndrome, Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia
- Trial name
- NCT03513328
- Testing
- Thiotepa--single daily dose, Thiotepa--escalated dose
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- University of Florida
Adrenal Insufficiency
Value of 25 mcg Cortrosyn Stimulation Test
The diagnosis of secondary AI is vital to prevent catastrophic events in patients. An optimal test should have a very low margin of error with high sensitivity and specificity, be easy and safe to administer, and have distinct…
- Trial name
- 25CST (NCT01428336)
- Testing
- 1 ug ACTH stimulation test, 250 ug ACTH stimulation test, 25 ug Cortrosyn stimulation test
- Where
- United States
- Volunteers
- 22 people, healthy volunteers welcome
- Sponsor
- The Cleveland Clinic
Adrenal Insufficiency
Cortisol Response to Moderate and Deep Sedation in Children
Currently, it is unknown whether sedation itself induces a rise in serum cortisol levels or if cortisol levels rise under only the most invasive of procedures, regardless of the type of anesthetic agent used.
- Trial name
- NCT01265966
- Where
- United States
- Volunteers
- 149 people
- Sponsor
- Tripler Army Medical Center
Adrenal Insufficiency
Treatment of Adrenal Insufficiency in Children
The study will be conducted in a total of 24 subjects before their 6th birthday, requiring replacement therapy for adrenal insufficiency due to either CAH, primary adrenal failure or hypopituitarism.
- Trial name
- NCT02720952
- Testing
- Infacort®
- Where
- Germany
- Volunteers
- 24 people
- Sponsor
- Neurocrine UK Limited
Adrenal Insufficiency, Excess Androgen
Comparison of Cortisol Pump With Standard Treatment for Congenital Adrenal Hyperplasia
Background: Congenital adrenal hyperplasia (CAH) is a genetic disorder of the adrenal gland. The adrenal gland is located in the abdomen and produces small amounts of hormones such as cortisol, aldosterone, and androgen.
- Conditions
- Adrenal Insufficiency, Excess Androgen, Congenital Adrenal Hyperplasia (CAH)
- Trial name
- NCT01859312
- Testing
- Hydrocortisone (Solucortef), Insulin pump (Medtronic)
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- National Institutes of Health Clinical Center (CC)
Leukodystrophy, White Matter Disease
LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies
Leukodystrophies, and other heritable disorders of the white matter of the brain, were previously resistant to genetic characterization, largely due to the extreme genetic heterogeneity of molecular causes.
- Conditions
- Leukodystrophy, White Matter Disease, 4H Syndrome, Adrenoleukodystrophy
- Trial name
- NCT02699190
- Where
- United States
- Volunteers
- 236 people
- Sponsor
- Children's Hospital of Philadelphia
Adrenal Insufficiency, Cirrhosis
Is Adrenal Insufficiency Under-diagnosed in Hospitalized Cirrhosis Patients?
The hepatoadrenal syndrome has been well described in the literature and is known to be associated with poorer outcomes in both stable and critically ill cirrhotic patients.
- Conditions
- Adrenal Insufficiency, Cirrhosis, Spur Cell Anemia, Lecithin Acyltransferase Deficiency
- Trial name
- NCT03368066
- Testing
- Cosyntropin
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- University of Virginia
Adrenal Insufficiency, Critical Illness
Pilot Study Assessing Oxidative Stress in Children
Role fo oxidative stress in adrenal insufficiency has not been studied. The degree of oxidative stress and it's role in pediatric critical illness is unknown.
- Trial name
- OxStress (NCT01052207)
- Where
- United States
- Volunteers
- 102 people
- Sponsor
- Emory University
Adrenal Insufficiency
Adrenal Suppression and Adrenal Recovery Induced by Megestrol Acetate
Megestrol Acetate (MA) is a progesterone-like hormone that has been utilized as a birth control agent, chemotherapeutic drug, and more recently, to induce appetite and weight gain in patients malnourished as a result of radiation…
- Trial name
- NCT00575029
- Testing
- megestrol acetate
- Where
- United States
- Volunteers
- 7 people, healthy volunteers welcome
- Sponsor
- University of Arkansas
Infantile Refsum's Disease, Zellweger Syndrome
Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic Acid
OBJECTIVES: I. To Evaluate the therapeutic efficacy of cholic acid during provision of compassionate treatment to patients with identified inborn errors of bile acid synthesis and metabolism II.
- Conditions
- Infantile Refsum's Disease, Zellweger Syndrome, Adrenoleukodystrophy, Peroxisomal Disorders
- Trial name
- NCT00007020
- Testing
- Cholic Acids
- Where
- United States
- Volunteers
- 85 people
- Sponsor
- Mirum Pharmaceuticals, Inc.
Mucopolysaccharidosis, Hurler Syndrome
Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders
Rationale: Chemotherapy administration before a donor stem cell transplant is necessary to stop the patient's immune system from rejecting the donor's stem cells.
- Conditions
- Mucopolysaccharidosis, Hurler Syndrome, Hunter Syndrome, Maroteaux-Lamy Syndrome
- Trial name
- NCT01043640
- Testing
- Campath-1H, Cyclophosphamide, Busulfan
- Where
- United States
- Volunteers
- 46 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Adrenal Insufficiency
Evaluation of Etomidate on Adrenal Function in Trauma Patients
Trauma patients are at increased risk for adrenal function insufficiency. A commonly used agent for rapid sequence intubation (RSI) is known to decrease adrenal function.
- Trial name
- NCT00462644
- Testing
- RSI sedation with etomidate/succinylcholine, RSI sedation with fentanyl/midazolam/succinylcholine
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- University of Tennessee
Adrenal Insufficiency
Extension Study for Patients Entered Into Study Infacort 003
A Phase 3, open-label, single-group, non-randomised, observational study of the safety and biochemical disease control of Infacort® in neonates, infants and children with adrenal insufficiency and congenital adrenal hyperplasia…
- Trial name
- NCT02733367
- Testing
- Infacort®
- Where
- Germany
- Volunteers
- 18 people
- Sponsor
- Neurocrine UK Limited