Clinical trials: Debility
Cardiac Diseases, Muscular Weakness
Biomarker for Pompe Disease (BioPompe)
Development of a new MS-based biomarker for the early and sensitive diagnosis of Pompe disease from blood (plasma)
- Conditions
- Cardiac Diseases, Muscular Weakness, Hepato-splenomegaly, Lung Disease
- Trial name
- BioPompe (NCT01457443)
- Where
- 4 sites in Germany, India and 1 more
- Sponsor
- CENTOGENE GmbH Rostock
Thymidine Kinase 2 Deficiency
An Open-Label Study of Continuation Treatment With Combination Pyrimidine Nucleosides in Patients With TK2 Deficiency
This is a Phase 2 prospective open-label treatment study of the safety and efficacy of doxecitine and doxribtimine in study participants with thymidine kinase 2 (TK2) deficiency who participated in the retrospective study…
- Trial name
- Continuation (NCT03845712)
- Testing
- doxecitine and doxribtimine
- Where
- 8 sites in United States, Israel and 1 more
- Volunteers
- 47 people
- Sponsor
- UCB BIOSCIENCES, Inc.
Thymidine Kinase 2 (TK2)
A RETROspective Study of Patients With TK2d
This is a medical chart review study to collect information for patients who have been taking pyrimidine nucleosides for treatment of TK2 deficiency.
- Trial name
- RETRO (NCT03701568)
- Testing
- dC/dT
- Where
- 8 sites in United States, Israel and 1 more
- Volunteers
- 38 people
- Sponsor
- Zogenix MDS, Inc.
Gait Impairment, Cerebral Palsy (CP)
Clinical Investigation to Validate the Safety and Performance of the ABLE Kids Device in Pediatric Patients With Neurological Gait Disorders in a Clinical Setting
The primary objective of the study is to validate the safety and clinical performance of the ABLE Kids device in pediatric patients with neurological gait disorders or muscular weakness during a 10-session gait training program…
- Conditions
- Gait Impairment, Cerebral Palsy (CP), Neuromuscular Diseases (NMD), Spinal Cord Injuries (SCI)
- Trial name
- NCT07745712
- Testing
- ABLE Kids
- Where
- Spain
- Volunteers
- 15 people
- Sponsor
- ABLE Human Motion S.L.
Increased Lordosis/Scoliosis, Hyporeflexia
Biomarker for Duchenne Muscular Dystrophy
International, multicenter, observational, longitudinal study to identify biomarker/s for Duchenne Muscular Dystropy (DMD) and to explore the clinical robustness, specificity, and long-term variability of these biomarker/s.
- Conditions
- Increased Lordosis/Scoliosis, Hyporeflexia, Duchenne Muscular Dystrophy, Red-Green Color Blindness
- Trial name
- BioDuchenne (NCT02994030)
- Where
- 12 sites in Albania, Egypt and 6 more
- Volunteers
- 103 people
- Sponsor
- CENTOGENE GmbH Rostock
Thymidine Kinase 2 Deficiency
A Retrospective Study of Subjects With Thymidine Kinase 2 Deficiency
This is a multicenter, multinational, retrospective chart-review study to evaluate survival in patients with Thymidine Kinase 2 deficiency (TK2d).
- Trial name
- NCT05017818
- Testing
- Observational retrospective data
- Where
- 12 sites in United States, Russia and 2 more
- Volunteers
- 61 people
- Sponsor
- Zogenix MDS, Inc.
Acute Myeloid Leukemia (AML)
Luveltamab Tazevibulin (STRO-002) in Infants and Children < 12 Years of Age With Relapsed/Refractory CBFA2T3::GLIS2 AML
This trial will evaluate whether luveltamab tazevibulin is well tolerated and active against a rare form of AML carrying a particular genetic abnormality called CBFA2T3::GLIS2 that arises in infants and children.
- Trial name
- NCT06679582
- Testing
- Luveltamab tazevibulin
- Where
- 36 sites in United States, Austria and 7 more
- Volunteers
- 1 people
- Sponsor
- Sutro Biopharma, Inc.
Cerebral Palsy
Study Assessing High-Frequency Chest Wall Oscillation (HFCWO) in Preventing Pulmonary Exacerbations
To asssess efficacy of airway clearance provided by Vest therapy (HFCWO) in the reduction of respiratory exacerbations requiring hospitalization or antibiotic utilization in patients with muscle weakness and restrictive lung…
- Trial name
- NCT00739310
- Testing
- Vest Treatment (high frequency chest wall oscillation)
- Where
- United States
- Volunteers
- 29 people
- Sponsor
- Hill-Rom
Duchenne Muscular Dystrophy With Mutations Amenable to PBGENE-DMD
PBGENE-DMD Phase 1/2a Safety and Preliminary Efficacy Study in Duchenne Muscular Dystrophy (FUNCTION-DMD)
The purpose of this Phase 1/2a trial is to evaluate the safety, tolerability, and preliminary efficacy of PBGENE-DMD in patients with DMD harboring mutations amenable to excision of exons 45-55.
- Trial name
- NCT07429240
- Testing
- PBGENE-DMD (IV)
- Where
- 2 sites in United States
- Volunteers
- 18 people
- Sponsor
- Precision BioSciences, Inc.
Thymidine Kinase 2 Deficiency
A Study of the Efficacy and Safety of MT1621 in Thymidine Kinase 2 (TK2) Deficiency (Treatment naïve)
This is a Phase 3b, prospective, single-arm, multicenter, open-label treatment study of the efficacy and safety of MT1621 in pediatric and adolescent patients with thymidine kinase 2 deficiency (TK2d).
- Trial name
- NCT04581733
- Testing
- MT1621
- Sponsor
- Zogenix MDS, Inc.
Peripheral Neuropathy, Muscle Weakness
Biomarker for Metachromatic Leukodystrophy (BioMeta) Disease
Development of a new MS-based biomarker for the early and sensitive diagnosis of Metachromatic Leu-kodystrophy disease from blood (plasma)
- Trial name
- BioMeta (NCT01536327)
- Where
- 5 sites in Egypt, Germany and 2 more
- Sponsor
- CENTOGENE GmbH Rostock