Clinical trials: ATC
Rare Disorders, Undiagnosed Disorders
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
The Coordination of Rare Diseases at Sanford (CoRDS) is an international patient registry that serves as a centralized resource for rare disease research.
- Conditions
- Rare Disorders, Undiagnosed Disorders, Disorders of Unknown Prevalence, Cornelia De Lange Syndrome
- Trial name
- CoRDS (NCT01793168)
- Where
- United States
- Volunteers
- 20,000 people
- Sponsor
- Sanford Health
Ataxia-Telangiectasia (A-T)
Expanded Access Program (EAP) of Levacetylleucine for Ataxia-Telangiectasia (A-T)
This expanded access program provides investigational levacetylleucine to patients with Ataxia-Telangiectasia who are not eligible for clinical trials and have no satisfactory alternative therapies.
- Trial name
- NCT07380165
- Testing
- Levacetylleucine
- Sponsor
- IntraBio Inc
Ataxia Telangiectasia (AT)
Conjugate Pneumococcal Vaccine in Ataxia Telangiectasia (AT)
Ataxia Telangiectasia (AT) is an autosomal recessive inherited condition caused by mutations in the ATM gene1. Patients suffer from neuro-degenerative problems, usually commencing in the second year of life, and affecting…
- Trial name
- NCT00656409
- Testing
- Conjugated pneumococcal vaccine (Prevenar)
- Where
- United Kingdom
- Volunteers
- 30 people
- Sponsor
- Institute of Child Health
Ataxia Telangiectasia, Growth Failure
Status of Growth Hormone/ Insulin-like Growth Factor-1 (GH/IGF-1) Axis and Growth Failure in Ataxia Telangiectasia (AT)
This study will evaluate the status of the growth hormone/ insulin-like growth factor-1 (GH/IGF-1) axis in relation to growth failure, body weight and composition and neuroprotection in children with Ataxia telangiectasia (AT).
- Trial name
- GHAT (NCT01052623)
- Testing
- Somatropin, Clonidine, L-Arginin-Hydrochloride, Estradiol valerate
- Where
- Germany
- Volunteers
- 24 people
- Sponsor
- Johann Wolfgang Goethe University Hospital
ATM Gene Mutation, ATR Gene Mutation
Lung-MAP: Talazoparib in Treating Patients With HRRD Positive Recurrent Stage IV Squamous Cell Lung Cancer
This phase II trial studies how well talazoparib works in treating patients with homologous recombination repair deficiency (HRRD) positive stage IV squamous cell lung cancer that has come back after previous treatment.
- Conditions
- ATM Gene Mutation, ATR Gene Mutation, BARD1 Gene Mutation, BRCA1 Gene Mutation
- Trial name
- NCT03377556
- Testing
- Laboratory Biomarker Analysis, Pharmacological Study, Talazoparib
- Where
- 1,157 sites in United States, Canada
- Volunteers
- 51 people
- Sponsor
- SWOG Cancer Research Network
Ataxia Telangiectasia, Infections
Susceptibility to Infections in Ataxia Telangiectasia
Death in Ataxia telangiectasia (A-T) is usually due to cancer or chronic lung failure around 20 years of age. Despite low lymphocyte counts (CD3, CD4, CD8 and CD19), IgA and IgG subclass deficiency opportunistic and acute severe…
- Conditions
- Ataxia Telangiectasia, Infections, Immunodeficiency, Lung Disease
- Trial name
- NCT02345135
- Testing
- Blood collection, Lung function measurement, Symptom diary
- Volunteers
- 41 people, healthy volunteers welcome
- Sponsor
- Johann Wolfgang Goethe University Hospital
Ataxia Telangiectasia
Immunogenicity of Pneumococcal Vaccines in Ataxia-telangiectasia Patients
Ataxia-telangiectasia (AT) is a rare genetic disorder characterized by gait disorders, neuromotor dysfunction, eye abnormalities and immune deficiency.
- Trial name
- NCT01075438
- Testing
- Prevenar® (7-valent pneumococcal conjugate vaccine), Pneumovax® (pneumococcal polysaccharide vaccine)
- Where
- Israel
- Volunteers
- 20 people
- Sponsor
- Sheba Medical Center
Ataxia Telangiectasia in Children, Ataxia Telangiectasia
Home-based Complex Intervention for Children With Ataxia Telangiectasia
Ataxia telangiectasia is a rare, genetic and progressive condition with no known cure. Therapies present a mainstream management option and have the potential to offer optimisation of fitness and general health.
- Trial name
- NCT05692622
- Testing
- Whole-body exercise and respiratory exercise
- Where
- United Kingdom
- Volunteers
- 40 people
- Sponsor
- University of Plymouth
Advanced Stage Solid Tumors
ATRi Transition Rollover Study
The main purpose of the study was to monitor the safety of participant receiving long-term treatment of Berzosertib as monotherapy.
- Trial name
- NCT03309150
- Testing
- Berzosertib
- Where
- United Kingdom
- Volunteers
- 1 people
- Sponsor
- Merck KGaA, Darmstadt, Germany
Ataxia Telangiectasia
International Ataxia Rating Scale in Younger Patients
The project will collect information on the mapping of clinical ratings on a number of scales that are used in the assessment of patients with ataxias.
- Trial name
- NCT01942850
- Where
- 5 sites in United States, India and 2 more
- Volunteers
- 63 people
- Sponsor
- Quince Therapeutics S.p.A.
Ataxia Telangiectasia
Susceptibility to Infections, Tumor Risk and Liver Disease in Patients With Ataxia Telangiectasia
Ataxia telangiectasia (A-T) is a rare devastating human recessive disorder characterized by progressive cerebellar ataxia, immunodeficiency, chromosomal instability and cancer susceptibility.
- Trial name
- NCT03357978
- Testing
- bioelectrical impedance analysis, blood draw, transient elastography (FibroScan)
- Where
- Germany
- Volunteers
- 30 people
- Sponsor
- Johann Wolfgang Goethe University Hospital
Nervous System Disease, Genetic Syndrome
Intra-Erythrocyte Dexamethasone Sodium Phosphate in Ataxia Telangiectasia Patients
Objectives: The objective of study was to evaluate the safety and the efficacy of EryDex (Dexamethasone sodium phosphate encapsulated in autologous erythrocytes, using the EryDex System - EDS) at two dose levels (low dose and…
- Trial name
- ATTeST (NCT02770807)
- Testing
- EryDex Low dose DSP, EryDex High dose DSP
- Where
- 22 sites in United States, Australia and 10 more
- Volunteers
- 176 people
- Sponsor
- Quince Therapeutics S.p.A.
Ataxia Telangiectasia
Ataxia-telangiectasia: Treating Mitochondrial Dysfunction With a Novel Form of Anaplerosis
Study design: Parallel group, placebo-controlled, dose-escalation each 2 months for 12 months. Dose based on percent (%) of calculated caloric intake.
- Trial name
- A-TC7 (NCT04513002)
- Testing
- Triheptanoin
- Where
- Australia
- Volunteers
- 30 people
- Sponsor
- The University of Queensland
Ataxia Telangiectasia
Safety and Efficacy of Mutation-targeted Precision Genetic Therapy for Ataxia-Telangiectasia (A-T)
This project aims to evaluate the safety and efficacy of precision genetic therapy for patients with Ataxia-telangiectasia (A-T), a rare neurodegenerative disease caused by mutations in the ATM gene.
- Trial name
- NCT07215416
- Testing
- Antisense oligonucleotide targeting the ATM gene
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Timothy Yu
Ataxia Telangiectasia
Open-Label Extension of EryDex Study IEDAT-04-2022
This is an international, multi-center, prospective, open-label, non-comparative study aiming to provide access to treatment with EryDex to ataxia telangiectasia (A-T) patients who completed the IEDAT-04-2022 trial which studied…
- Trial name
- OLE_NEAT (NCT06664853)
- Testing
- Dexamethasone sodium phosphate
- Where
- 21 sites in United States, Denmark and 7 more
- Volunteers
- 101 people
- Sponsor
- Quince Therapeutics S.p.A.
Ataxia-Telangiectasia (A-T)
Cell-Based Approaches For Modeling and Treating Ataxia-Telangiectasia
This research is being done to better understand the causes of the disease Ataxia-Telangiectasia and, in the longer-term, develop new therapies for the disease using stem cells.
- Trial name
- NCT02246491
- Testing
- A-T iPS cell line, Carrier patients iPS cell line
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Johns Hopkins University
Ataxia Telangiectasia Louis-Bar, Ataxia Telangiectasia in Children
MBM-01 (Tempol) for the Treatment of Ataxia Telangiectasia
Ataxia Telangiectasia (A-T) is an autosomal recessively inherited neurodegenerative disorder that also has dramatic effects on the immune and endocrine systems.
- Conditions
- Ataxia Telangiectasia Louis-Bar, Ataxia Telangiectasia in Children, Ataxia Telangiectasia
- Trial name
- NCT04887311
- Testing
- MBM-01
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Matrix Biomed, Inc.
Ataxia-Telangiectasia
Study for Treatment of Cancer in Children With Ataxia-telangiectasia
This is a pilot/feasibility study designed to investigate the feasibility of treating children with Ataxia-Telangiectasia (A-T) and cancer with regimens nearly as intense as non-A-T patients with cancer would receive.
- Trial name
- NCT00187057
- Testing
- vinblastine, vincristine, prednisone, daunorubicin, doxorubicin, methotrexate, cyclophosphamide, L-asparaginase, etoposide, cytarabine, mercaptopurine
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- St. Jude Children's Research Hospital
Ataxia Telangiectasia
N-Acetyl-L-Leucine for Ataxia-Telangiectasia (A-T)
This is a multinational, multicenter, open-label, rater-blinded prospective Phase II study which will assess the safety and efficacy of N-Acetyl-L-Leucine (IB1001) for the treatment of Ataxia-Telangiectasia (A-T).
- Trial name
- NCT03759678
- Testing
- IB1001
- Where
- 4 sites in United States, Germany and 2 more
- Volunteers
- 17 people
- Sponsor
- IntraBio Inc
Ataxia Telangiectasia
NAD Supplementation to Prevent Progressive Neurological Disease in Ataxia Telangiectasia
The study investigates the effect of dietary supplementation of nicotinamide ribonucleoside (NR) in children with ataxia telangiectasia (AT), with main focus on neurological symptoms.
- Trial name
- NCT04870866
- Testing
- Nicotinamide ribonucleoside
- Where
- 2 sites in Norway
- Volunteers
- 13 people
- Sponsor
- University Hospital, Akershus