Systemic neuroscience
Neuromuscular studies
| Specialisation | Systemic neurosciences (neuromuscular diseases) |
| Indication | Hereditary transthyretin-mediated amyloid polyneuropathy |
| Short title | ION-682884-CS13 |
| Study title | Extension study to evaluate the long-term safety and efficacy of ION-682884 in patients with hereditary transthyretin-mediated amyloid polyneuropathy |
| Brief description | Evaluation of the long-term safety and efficacy of ION-682884 in patients with hereditary transthyretin-mediated amyloid polyneuropathy |
| Phase | IIIb |
| EudraCT-Number | 2021-001427-40 |
| Status | finalised |
| Contact | Study director: Prof Dr Markus Weiler Phone: 06221-567504 E-mail: markus.weiler(at)med.uni-heidelberg.de Study coordination: Sandra Schmich Tel: 06221 56-37194 Fax: 06221 56-5935 |
| Specialisation | Systemic neurosciences (neuromuscular diseases) |
| Indication | Hereditary transthyretin amyloidosis |
| Short title | ACT-EARLY (AG10-501) |
| Study title | A randomised, multicentre, double-blind, placebo-controlled phase III study for the prevention of transthyretin amyloidosis in young people with acoramidis |
| Brief description | A phase III, randomised, multicentre, double-blind, placebo-controlled trial for the prevention of transthyretin amyloidosis with acoramidis in people with a confirmed variant in the transthyretin gene. |
| Phase | PHASE III |
| EudraCT-Number | 2024-513547-82 |
| Status | Open, recruitment from Q2 / 2025 |
| Contact | Study management: Prof Dr Markus Weiler Phone: 06221-567504 E-mail: markus.weiler(at)med.uni-heidelberg.de Study coordination: Sandra Schmich Tel: 06221 56-37194 Fax: 06221 56-5935 |
| Specialisation | Systemic neurosciences (neuromuscular diseases) |
| Indication | 5q Spinal muscular atrophy |
| Short title | ASCEND |
| Study title | Study to evaluate higher dose nusinersen (BIIB058) in patients with spinal muscular atrophy previously treated with risdiplam |
| Brief description | Evaluation of higher dose nusinersen (BIIB058) in patients with spinal muscular atrophy previously treated with risdiplam |
| Phase | IIIb |
| EudraCT-Number | 2021-001294-23 |
| Status | Open, recruitment ongoing |
| Contact person | Study management: Prof Dr Markus Weiler Phone: 06221-567504 E-mail: markus.weiler(at)med.uni-heidelberg.de Study coordination: Sandra Schmich Tel: 06221 56-37194 Fax: 06221 56-5935 |
| Specialisation | Systemic neurosciences (neuromuscular diseases) |
| Indication | 5q Spinal muscular atrophy |
| Short title | SMArtCARE |
| Study title | Follow-up and therapy evaluation in spinal muscular atrophy |
| Short description | Follow-up and therapy evaluation in spinal muscular atrophy |
| Phase | n. a. |
| EudraCT-Number | n. a. |
| Status | Open, recruitment ongoing |
| Contact person | Study management: Prof Dr Markus Weiler Phone: 06221-567504 E-mail: markus.weiler(at)med.uni-heidelberg.de Study coordination: Sandra Schmich Tel: 06221 56-37194 Fax: 06221 56-5935 |
| Specialisation | Systemic neurosciences (neuromuscular diseases) |
| Indication | Amyotrophic lateral sclerosis (ALS) and other motor neurone diseases |
| Short title | EARLY-ALS |
| Study title | Study to investigate early symptoms in patients with amyotrophic lateral sclerosis and other motor neurone diseases |
| Short description | Investigation of early symptoms in patients with amyotrophic lateral sclerosis and other motor neurone diseases |
| Phase | n.a. |
| EudraCT-Number | n.a. |
| Status | Open, recruitment ongoing |
| Contact person | Study management: Prof Dr Markus Weiler Phone: 06221-567504 E-mail: markus.weiler(at)med.uni-heidelberg.de Study coordination: Sandra Schmich Tel: 06221 56-37194 Fax: 06221 56-5935 |
| Specialisation | Systemic neurosciences (neuromuscular diseases) |
| Indication | Amyotrophic lateral sclerosis (ALS) and other motor neurone diseases |
| Short title | TEAR-ALS |
| Study title | Study to analyse biomarkers from tear fluid in patients with amyotrophic lateral sclerosis and other motor neuron diseases |
| Short description | Analysis of biomarkers from tear fluid in patients with amyotrophic lateral sclerosis and other motor neurone diseases |
| Phase | n.a. |
| EudraCT-Number | n.a. |
| Status | Open, recruitment is ongoing. |
| Contact | Study director: Prof Dr Markus Weiler Tel: 06221-567504 E-mail: markus.weiler(at)med.uni-heidelberg.de Study coordination: Sandra Schmich Tel: 06221 56-37194 Fax: 06221 56-5935 |
| Specialisation | Systemic neurosciences (neuromuscular diseases) |
| Indication | Amyotrophic lateral sclerosis (ALS) |
| Study title | Deficits and potential for improvement in the psychosocial and palliative care of ALS patients in Germany |
| Brief description | Multicentre, questionnaire-based study to assess satisfaction with palliative, including psychosocial care for ALS patients in Germany |
| Phase | n.a. |
| EudraCT-Number | n.a. |
| Status | Open, recruitment is ongoing. |
| Contact | Study director: Prof Dr Markus Weiler Phone: 06221-567504 E-mail: markus.weiler(at)med.uni-heidelberg.de Study coordination: Sandra Schmich Tel: 06221 56-37194 Fax: 06221 56-5935 |