Clinical trials: Osteopetrosis
Osteopetrosis
Gene-Modified Stem Cell Therapy for Children With Autosomal Recessive Osteopetrosis (ARO)
This is a non-randomized, one-arm, open label, single-center, phase I/II, prospective study, to assess safety, feasibility and efficacy of FT024 in 8 children (Age: ≥ 28 days and ≤ 2 years old, Body weight: ≥ 4 kg) affected by…
- Trial name
- ARO (NCT07665021)
- Testing
- FT024
- Where
- Italy
- Volunteers
- 8 people
- Sponsor
- Fondazione Telethon
MLD, Krabbe Disease
Longitudinal Study of Neurodegenerative Disorders
The purpose of this study is to understand the course of rare genetic disorders that affect the brain. This data is being analyzed to gain a better understanding of the progression of the rare neurodegenerative disorders and the…
- Conditions
- MLD, Krabbe Disease, ALD, MPS I
- Trial name
- NCT03333200
- Testing
- Palliative Care, Hematopoetic Stem Cell Transplantation
- Where
- United States
- Volunteers
- 1,500 people
- Sponsor
- University of Pittsburgh
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes
Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC
This is a data collection study that will examine the general diagnostic and treatment data associated with the reduced-intensity chemotherapy-based regimen paired with simple alemtuzumab dosing strata designed to prevented graft…
- Conditions
- Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias
- Trial name
- PRO-RIC (NCT04528355)
- Testing
- data collection
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Paul Szabolcs
Mucopolysaccharidosis Disorders, Hurler Syndrome
MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
This single-institution, phase II study is designed to test the ability to achieve donor hematopoietic engraftment while maintaining low rates of transplant-related mortality (TRM) using busulfan- and fludarabine-based…
- Conditions
- Mucopolysaccharidosis Disorders, Hurler Syndrome, Hunter Syndrome, Maroteaux Lamy Syndrome
- Trial name
- NCT02171104
- Testing
- Stem Cell Transplantation, IMD Preparative Regimen, Osteopetrosis Only Preparative Regimen
- Where
- United States
- Volunteers
- 149 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes
Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT
The objective of this study is to evaluate the efficacy of using a reduced-intensity condition (RIC) regimen with umbilical cord blood transplant (UCBT), double cord UCBT, matched unrelated donor (MUD) bone marrow transplant…
- Conditions
- Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias
- Trial name
- HSCT+RIC (NCT01962415)
- Testing
- Hydroxyurea, Alemtuzumab, Fludarabine
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- Paul Szabolcs
Disorder; Trance, Anxiety
Hypnosis-Based Machine Learning Biomarker Study
This study seeks to contribute to the growing body of literature on hypnosis by providing robust, data-driven insights into the physiological mechanisms underlying trance states.
- Trial name
- NCT07102810
- Testing
- Standardized Hypnotic Susceptibility Testing
- Where
- United States
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- Icahn School of Medicine at Mount Sinai
Osteopenia, Bone Metabolism Disorders
Effects of Aerobic Versus Resistance Training on Bone Metabolism in Stage 3-4 Chronic Kidney Disease
This randomized controlled trial investigates the comparative effects of aerobic versus resistance training on bone mineral density (BMD) and bone metabolism markers in patients with stage 3 or 4 chronic kidney disease (CKD).
- Conditions
- Osteopenia, Bone Metabolism Disorders, Chronic Kidney Disease (Stage 3-4), Mineral and Bone Disorder (CKD-MBD)
- Trial name
- CKD-BONE-EX (NCT07133256)
- Testing
- Resistance Exercise, Aerobic Training
- Where
- Egypt
- Volunteers
- 60 people
- Sponsor
- Sinai University
Bone Marrow Failure, Osteopetrosis
Risk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Non-Malignant Diseases
This study proposes the use of a reduced intensity chemotherapy/radiation therapy regimen followed by stem cell transplantation, as compared to standard ablative chemotherapy regimens associated with stem cell transplantation, in…
- Conditions
- Bone Marrow Failure, Osteopetrosis, Fanconi Anemia, Severe Combined Immunodeficiency
- Trial name
- NCT01019876
- Testing
- Fludarabine, Cyclophosphamide, Busulfan
- Where
- United States
- Volunteers
- 38 people
- Sponsor
- Columbia University
Osteopetrosis
ACTIMMUNE in Intermediate Osteopetrosis
This study evaluates the effects of ACTIMMUNE (IFN-γ1b) in children and adults with intermediate osteoporosis. All participants will receive treatment with ACTIMMUNE for 12 months.
- Trial name
- NCT02666768
- Testing
- Interferon gamma-1b
- Where
- 2 sites in United States
- Volunteers
- 5 people
- Sponsor
- Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
Osteopetrosis
Outcomes of Patients With Osteopetrosis Weight-bearing Bone Fractures
Osteopetrosis is a rare genetic disorder characterized by the abnormal hardening and density of bones, leading to increased brittleness and susceptibility to fractures.
- Trial name
- NCT06521580
- Testing
- Open reduction and internal fixation
- Where
- Egypt
- Volunteers
- 6 people
- Sponsor
- Kasr El Aini Hospital
Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child
Safety Study of Gene Modified Donor T-cells Following TCRαβ+ Depleted Stem Cell Transplant
This study will evaluate pediatric patients with malignant or non-malignant blood cell disorders who are having a blood stem cell transplant depleted of T cell receptor (TCR) alfa and beta cells that comes from a partially…
- Conditions
- Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child, Lymphoma, Non-Hodgkin, Myelodysplastic Syndrome
- Trial name
- NCT02065869
- Testing
- BPX-501 T cells, Rimiducid
- Where
- 4 sites in Italy, United Kingdom
- Volunteers
- 187 people
- Sponsor
- Bellicum Pharmaceuticals
Chronic Periodontitis
Evaluation Efficacy of Melatonin Gel as Adjunctive Therapy in Chronic Periodontitis
This study was conducted to assess the benefit of locally delivered melatonin gel as adjunctive to scaling and root planning (SRP) in the treatment of chronic periodontitis.
- Trial name
- NCT05759416
- Testing
- Melatonin2%gel
- Where
- Egypt
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Al-Azhar University
Infantile Malignant Osteopetrosis
A Trial to Evaluate Safety and Efficacy of RP-L401-0120 in Subjects With Infantile Malignant Osteopetrosis
The primary objective of this Phase 1 study is to evaluate the therapeutic safety and feasibility of the investigational product (IP), RP-L401.
- Trial name
- NCT04525352
- Testing
- RP-L401
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Rocket Pharmaceuticals Inc.
Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child
Study of Gene Modified Donor T-cells Following TCR Alpha Beta Positive Depleted Stem Cell Transplant
This study will evaluate pediatric patients with malignant or non-malignant blood cell disorders who are having a blood stem cell transplant depleted of T cell receptor (TCR) alfa and beta cells that comes from a partially…
- Conditions
- Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child, Lymphoma, Non-Hodgkin, Myelodysplastic Syndromes
- Trial name
- NCT03301168
- Testing
- BPX-501 T cells, Rimiducid
- Where
- 10 sites in United States
- Volunteers
- 120 people
- Sponsor
- Bellicum Pharmaceuticals
Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia
T-cell Depleted Alternative Donor Transplantation
The primary purpose is to determine the ability of CD34+ selection and T cell depletion using the CliniMACS® device to prevent severe acute graft-versus-host disease (GVHD) in patients receiving a stem cell transplant from an…
- Conditions
- Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia, Chronic Myeloid Leukemia, Myelodysplastic Syndrome
- Trial name
- NCT00968864
- Testing
- CliniMACS® (T cell depletion)
- Where
- United States
- Volunteers
- 53 people
- Sponsor
- Wake Forest University Health Sciences
Breast Neoplasm Female, Stage I Breast Cancer
Biomarker Study of the Antitumoral Activity of Denosumab in the Pre- Operative Setting of Early Breast Cancer
This is a biomarker study designed to test the preclinically generated hypothesis of anti-tumoral activity of denosumab in patients with early breast cancer candidates a tumour excision
- Conditions
- Breast Neoplasm Female, Stage I Breast Cancer, Stage II Breast Cancer, Hormone Receptor Negative Neoplasm
- Trial name
- D-BIOMARK (NCT03691311)
- Testing
- Denosumab 120 MG/1.7 ML Subcutaneous Solution [XGEVA]
- Where
- Spain
- Volunteers
- 60 people
- Sponsor
- Institut Català d'Oncologia
Osteopenia, Arterial Hypertension
Comparative Effects of Moxonidine on Bone Metabolism, Vascular and Cellular Aging in Hypertensive Postmenopausal Women
This study evaluates the effect of moxonidine versus bisoprolol on collagen type 1 C-telopeptide in postmenopausal female patients with arterial hypertension and osteopenia.
- Trial name
- COMPASS (NCT02355821)
- Testing
- Moxonidine, Bisoprolol
- Where
- Russia
- Volunteers
- 114 people
- Sponsor
- National Research Center for Preventive Medicine
Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia
Expanded Access Protocol (EAP) Using the CliniMACS® Device for Pediatric Haplocompatible Donor Stem Cell Transplant
This protocol provides expanded access to bone marrow transplants for children who lack a histocompatible (tissue matched) stem cell or bone marrow donor when an alternative donor (unrelated donor or half-matched related donor)…
- Conditions
- Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia, Chronic Myeloid Leukemia, Myelodysplastic Syndrome
- Trial name
- NCT01200017
- Testing
- CD34+ enriched, T Cell Depleted donor stem cell product
- Where
- United States
- Sponsor
- Christopher Dvorak
Autosomal Dominant Osteopetrosis Type 2
Use of ACTIMMUNE in Patients With ADO2
This study is an open label use of ACTIMMUNE for patients with Autosomal Dominant Osteopetrosis Type 2(ADO2). Effects of treatment will be evaluated after 14 weeks on ACTIMMUNE by bone resorption markers.
- Trial name
- NCT02584608
- Testing
- ACTIMMUNE
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Indiana University
Severe Osteopetrosis
Allogeneic Transplantation For Severe Osteopetrosis
The purpose of this research is to explore what we believe may be a safer and more effective means of performing stem cell transplantation in patients with Osteopetrosis, using chemotherapy and radiation designed to bring about…
- Trial name
- NCT00775931
- Testing
- umbilical cord blood transplantation, Campath-1H, Total Lymphoid Irradiation
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- Masonic Cancer Center, University of Minnesota