Clinical trials: Hypoadrenalism
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)
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
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, 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
Lysosomal Storage Disease, Peroxisomal Disorder
Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorders
This study is designed to test the ability to achieve donor hematopoietic engraftment while maintaining low rates of transplant-related mortality (TRM) in patients with high-risk lysosomal and peroxisomal disorders using a novel…
- Trial name
- NCT01626092
- Testing
- Campath-1H, Clofarabine, Melphalan
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Cerebral Adrenoleukodystrophy (CALD)
A Clinical Study to Assess the Efficacy and Safety of Gene Therapy for the Treatment of Cerebral Adrenoleukodystrophy (CALD)
The purpose of this study is to evaluate the efficacy and safety of Lenti-D Drug Product (also known as elivaldogene autotemcel or Skysona, hereafter referred to as eli-cel) after myeloablative conditioning with busulfan and…
- Trial name
- NCT03852498
- Testing
- Lenti-D
- Where
- 8 sites in United States, France and 4 more
- Volunteers
- 35 people
- Sponsor
- Genetix Biotherapeutics 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.
Adrenal Insufficiency
Hydrocortisone Replacement in Patients With Secondary Adrenal Insufficiency (SUPREME CORT)
The aim of this study is to investigate whether a physiologically low hydrocortisone (HC) dose is better for cognition as compared to a physiologically high HC dose.
- Trial name
- SUPREME CORT (NCT01546922)
- Testing
- Hydrocortisone
- Where
- Netherlands
- Volunteers
- 63 people
- Sponsor
- University Medical Center Groningen
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
Shock
Steroid Use in Pediatric Fluid and Vasoactive Infusion Dependent Shock - Pilot Study
Approximately 20,000 children per year in North America present to the hospital with severe shock. Children who develop this condition have very low blood pressures and as a result may suffer damage to their internal organs and…
- Trial name
- STRIPES (NCT02044159)
- Testing
- Hydrocortisone
- Where
- 7 sites in Canada
- Volunteers
- 57 people
- Sponsor
- Children's Hospital of Eastern Ontario
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.
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
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
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
AMN, AMN Gene Mutation
A Study to Evaluate Administration of SBT101 Gene Therapy in Adult Patients With Adrenomyeloneuropathy (AMN)
This is a Phase 1/2 randomized, blinded, dose-escalation study to evaluate the safety and efficacy of intrathecal (IT) administration of SBT101, a recombinant adeno-associated virus serotype 9 (AAV9) containing a functional copy…
- Conditions
- AMN, AMN Gene Mutation, X-ALD
- Trial name
- PROPEL (NCT05394064)
- Testing
- SBT101 Treatment
- Where
- 2 sites in United States, Netherlands
- Volunteers
- 9 people
- Sponsor
- SwanBio Therapeutics, Inc.
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
Hereditary Spastic Paraplegia, Adrenomyeloneuropathy
Repetitive Transcranial Magnetic Stimulation as Therapy in Hereditary Spastic Paraplegia and Adrenomyeloneuropathy
Hereditary spastic paraplegia (HSP) is the group of inherited disorders, characterized by progressive gait disturbance. There is no established therapy. Adrenoleukodystrophy (AMN) is an x-linked hereditary disease.
- Trial name
- NCT03627416
- Testing
- rTMS
- Where
- Poland
- Volunteers
- 15 people
- Sponsor
- Jakub Antczak
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
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
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