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NCT04007536
A Study of Potential Treatment-Responsive Biomarkers and Clinical Outcomes in Hunter Syndrome
trial testing No Intervention in Mucopolysaccharidosis II in 18 participants. Completed in 1 March 2024.
1 March 2024
Quick facts
| Lead sponsor | Denali Therapeutics Inc. |
|---|---|
| Status | Completed |
| Study type | OBSERVATIONAL |
| Enrollment | 18 |
| Start date | 23 October 2019 |
| Primary completion | 1 March 2024 |
| Estimated completion | 1 March 2024 |
| Sites | 6 locations across United Kingdom, Netherlands, United States |
Drugs / interventions tested
- No Intervention
Conditions studied
- Mucopolysaccharidosis II — all drugs for Mucopolysaccharidosis II →
Sponsor
Denali Therapeutics Inc. — full company profile →
Who can join
Under 30, any sex, with Mucopolysaccharidosis II. Patients with the condition only — healthy volunteers not accepted.
Sponsor's own description
This is a six-part prospective, multicenter, multiregional observational study of patients with mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome, to assess biomarkers potentially related to disease severity and/or treatment response and prospectively assess the progression of disease in participants with MPS II who are aged ≤30 years at the time of enrollment.
Publications & conference data
No peer-reviewed publications indexed yet for this trial. Completed trials usually publish results within 12-18 months.
Verify or expand the search:
- PubMed search for NCT04007536
- Europe PMC full search
- ASCO Meeting Library
- ESMO Meeting Library
- bioRxiv preprints
- medRxiv preprints
- Google Scholar
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Other recruiting trials for Mucopolysaccharidosis II
Currently open trials in the same condition.
- NCT05619900 — Registry of Patients Diagnosed With Lysosomal Storage Diseases · recruiting
- NCT04573023 — A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II (STARLIGHT) · Phase 3 · active not recruiting
- NCT04628871 — Long Term Follow-up (LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIX · active not recruiting
- NCT04348136 — An Extension Study of JR-141 in Patients With Mucopolysaccharidosis Type II · Phase 2, PHASE3 · active not recruiting
- NCT03708965 — An Extension Study of JR-141-BR21 in Patients With Mucopolysaccharidosis II · Phase 2 · active not recruiting
Other Denali Therapeutics Inc. trials
Trials by the same sponsor.
- NCT06602193 — Safety and Pharmacodynamic Effects of BIIB122 in Participants With LRRK2-Associated Parkinson's Disease (LRRK2-PD) · Phase 2 · recruiting
- NCT06281158 — A Phase 1, Open-label Study of the Absorption, Metabolism, and Excretion of [14C]-DNL343 Following a Single Oral Dose in · Phase 1 · completed
- NCT05450549 — A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of DNL919 in Healthy Participants · Phase 1 · completed
- NCT05006352 — A Study to Determine the Safety, Pharmacokinetics, and Pharmacodynamics of DNL343 in Participants With Amyotrophic Later · Phase 1 · completed
- NCT04581772 — A Study to Evaluate the Bioavailability and Safety of DNL343 in Healthy Volunteers · Phase 1 · completed
Verify against primary sources
- ClinicalTrials.gov — authoritative US registry record
- WHO ICTRP — international registry index
- EU Clinical Trials Register
- Sponsor press releases (Google)
- Trial protocol + status: ClinicalTrials.gov NCT04007536 (US National Library of Medicine, public domain)
- Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
- Sponsor: as reported to ClinicalTrials.gov by Denali Therapeutics Inc.
- Last refreshed: 10 June 2024
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT04007536.
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing