alpha-Mannosidosis clinical trials
The registry lists 24 studies for alpha-Mannosidosis across more than 10 countries. 6 are recruiting or about to start. 15 of them are mainly about alpha-Mannosidosis (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-03.
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Who runs them
Zymenex A/S (6) · Chiesi Farmaceutici S.p.A. (6) · Masonic Cancer Center, University of Minnesota (3) · Paul Szabolcs (2) · Joanne Kurtzberg, MD (1) · University of Pittsburgh (1) · Talaris Therapeutics Inc. (1) · Greenwood Genetic Center (1)
What is being tested here
Drugs under test in alpha-Mannosidosis studies, those with the most open studies first. Medicines only given beside them are not counted. How drugs are counted
| Drug | Open | Phase 3 | All |
|---|---|---|---|
| Alemtuzumab | 1 | 0 | 3 |
| Hydroxyurea | 1 | 0 | 1 |
| Fludarabine | 1 | 0 | 1 |
| Melphalan | 1 | 0 | 1 |
| Thiotepa | 1 | 0 | 1 |
| Velmanase alfa | 0 | 5 | 9 |
| Cyclophosphamide | 0 | 0 | 2 |
| Busulfan | 0 | 0 | 2 |
| Cyclosporine | 0 | 0 | 1 |
| Mycophenolic Acid | 0 | 0 | 1 |
Drug classes tested here Alkylating agents 1 open
Coming up
Studies recruiting or running, from their records. “Main data collection” ends at the primary completion date, the sponsor’s own estimate, which often moves.
All 2
- Nov 2026Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT · Paul Szabolcs
- Oct 2027UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells · Joanne Kurtzberg, MD
Who these studies take
Of the 5 alpha-Mannosidosis studies recruiting now, 1 (20%) set no upper age limit and 4 (80%) stop at 75 or younger. 5 (100%) take children under 18. 0 (0%) are for women only and 0 (0%) for men only; 0 (0%) take healthy volunteers.
| Age | Women | Men |
|---|---|---|
| 10 | 4 (80%) | 4 (80%) |
| 18 | 4 (80%) | 4 (80%) |
| 30 | 3 (60%) | 3 (60%) |
| 40 | 3 (60%) | 3 (60%) |
| 50 | 3 (60%) | 3 (60%) |
| 65 | 1 (20%) | 1 (20%) |
| 75 | 1 (20%) | 1 (20%) |
| 85 | 1 (20%) | 1 (20%) |
Across all 24 studies, recruiting or not: 58% stop at 75 or younger and 58% at 65; 100% take children; 0% are for women only and 0% for men only; 0% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.
Studies
Recruiting first, then most recently updated. Showing 24 of 24. search them.
- UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like CellsRecruitingNCT02254863Phase 11 country
- Longitudinal Study of Neurodegenerative DisordersRecruitingNCT033332001 country
- Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RICRecruitingNCT045283551 country
- Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCTRecruitingNCT01962415Phase 21 country
- Analysis of Velmanase Alfa (Lamzede®)'s Effects in the Body of Children With Alpha-Mannosidosis Under the Age 3RecruitingNCT061845031 country
- Expanded Access to Velmanase AlfaAvailableNCT04959240
- MT2013-31: Allo HCT for Metabolic Disorders and Severe OsteopetrosisActive, not recruitingNCT02171104Phase 21 country
- Longitudinal Studies of the GlycoproteinosesCompletedNCT018914221 country
- Lamazym Aftercare Study FR Designed to Provide Treatment for French PatientsCompletedNCT01908712Phase 31 country
- Lamazym Aftercare StudyCompletedNCT01908725Phase 31 country
- Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic DisordersTerminatedNCT01372228Phase 1/21 country
- Biomarker for Mannosidosis Disease (BioMannosidosis)WithdrawnNCT032640403 countries
- Trial on Safety and Efficacy of Velmanase Alfa Treatment in Pediatric Patients With Alpha-MannosidosisCompletedNCT02998879Phase 25 countries
- Interventional Study to Assess Efficacy and Safety of Velmanase Alfa in Patients With Alpha MannosidosisWithdrawnNCT04031066Phase 3
- Evaluation of Long-term Efficacy of Treatment With LamazymCompletedNCT02478840Phase 31 country
- The Natural History of Alpha-MannosidosisCompletedNCT004984204 countries
- Safety Study of Recombinant Human Alpha-mannosidase for the Treatment of Patients With Alpha-mannosidosisCompletedNCT01268358Phase 1
- A Placebo-Controlled Phase 3 Trial of Repeated Lamazym Treatment of Subjects With Alpha-MannosidosisCompletedNCT01681953Phase 35 countries
- Clinical Biomarkers in Alpha-mannosidosisCompletedNCT021415031 country
- Allogeneic Bone Marrow Transplant for Inherited Metabolic DisordersCompletedNCT01043640Phase 21 country
- Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of MetabolismTerminatedNCT00668564Phase 21 country
- Long-term Efficacy and Safety of Lamazym for the Treatment of Patients With Alpha-MannosidosisCompletedNCT01681940Phase 24 countries
- ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic DiseasesTerminatedNCT00654433Phase 31 country
- Dose Finding Study of Recombinant Human Alpha-mannosidase for the Treatment of Patients With Alpha-mannosidosisStatus unknownNCT01285700Phase 21 country