Clinical trials: Autosomal Recessive Disorder
Hereditary Spastic Paraplegia, Spastic Paraplegia Type 11
Trehalose Administration in Subjects With Spastic Paraplegia 11 (3AL-SPG11)
Hereditary spastic paraparesis type 11 (SPG11) is caused by mutations in the SPG11 gene that produces spatacsin, a protein involved in lysosomal function.
- Trial name
- 3AL-SPG11 (NCT04912609)
- Where
- Italy
- Volunteers
- 13 people
- Sponsor
- IRCCS Fondazione Stella Maris
Congenital Adrenal Hyperplasia
Efficacy, Safety, Tolerability, and Pharmacokinetics of NBIP-01435 in Adult Participants With Classic Congenital Adrenal Hyperplasia (CAH)
The main objective of this study is to evaluate the efficacy of different dose levels of NBIP-01435 on reducing elevated androstenedione (A4) levels in adults with classic CAH.
- Trial name
- NCT07850739
- Testing
- NBIP-01435
- Volunteers
- 36 people
- Sponsor
- Neurocrine Biosciences
Spinal Cord Injuries
Phase I/II Study of KP-100IT in Acute Spinal Cord Injury
This study is randomized, double-blind, placebo-controlled Phase I/II study designed to evaluate safety and efficacy of KP-100IT, code of Hepatocyte Growth Factor (HGF) formulation for intrathecal injection, as a treatment for…
- Trial name
- NCT02193334
- Testing
- KP-100IT
- Where
- 2 sites in Japan
- Volunteers
- 45 people
- Sponsor
- Kringle Pharma, Inc.
Antibody-mediated Rejection, Hyperacute Rejection of Cardiac Transplant
The De-novo Use of Eculizumab in Presensitized Patients Receiving Cardiac Transplantation
All individuals who receive a heart transplant are at risk for developing antibody-mediated rejection (AMR). An antibody is a protein produced by the body's immune system when it detects a foreign substance, called an antigen.
- Conditions
- Antibody-mediated Rejection, Hyperacute Rejection of Cardiac Transplant, Left Ventricular Dysfunction, Cardiac Allograft Vasculopathy
- Trial name
- DUET (NCT02013037)
- Testing
- Eculizumab
- Where
- United States
- Volunteers
- 36 people
- Sponsor
- Cedars-Sinai Medical Center
Acute Leukemia, Severe Aplastic Anemia
AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion
Children, adolescents, and young adults with malignant and non-malignant conditionsundergoing an allogeneic stem cell transplantation (AlloSCT) will have the stem cells selected utilizing α/β CD3+/CD19+ cell depletion.
- Conditions
- Acute Leukemia, Severe Aplastic Anemia, Non-hodgkin Lymphoma, Hodgkin Lymphoma
- Trial name
- NCT04099966
- Testing
- alpha beta depletion
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Mitchell Cairo
Respiratory Failure, Sedation
Effects of NIV and CPAP on Ventilation Distribution, Measured by EIT, During Deep Sedation in Paediatric Patients
In patients undergoing spontaneous breathing (SB) deep sedation there is a re-distribution of ventilation towards lungs non-dependant areas (ventral areas in supine position).
- Conditions
- Respiratory Failure, Sedation, Mechanical Ventilation Complication
- Trial name
- NIVEIT-ped (NCT05495477)
- Testing
- EIT
- Where
- Italy
- Volunteers
- 20 people
- Sponsor
- Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico
Molybdenum Cofactor Deficiency (MoCD), Rare Autosomal Recessive Disorder
Phase 1 Single Dose Study of ALXN1101 in Healthy Volunteers
Phase 1 single dose study of ALXN1101 in healthy volunteers.
- Conditions
- Molybdenum Cofactor Deficiency (MoCD), Rare Autosomal Recessive Disorder, Deficiency of Activity of Molybdenum-dependent Enzymes (Sulfite Oxidase [SOX], Xanthine Dehydrogenase, and Aldehyde Oxidase)
- Trial name
- NCT01894165
- Testing
- ALXN1101
- Where
- United States
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Origin Biosciences
Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Lymphoblastic Leukemia in Remission
Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies
This phase II trial studies how well giving fludarabine phosphate, melphalan, and low-dose total-body irradiation (TBI) followed by donor peripheral blood stem cell transplant (PBSCT) works in treating patients with hematologic…
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities
- Trial name
- NCT01529827
- Testing
- fludarabine phosphate, melphalan, total-body irradiation
- Where
- United States
- Volunteers
- 94 people
- Sponsor
- Roswell Park Cancer Institute