Shwachman-Diamond Syndrome clinical trials
The registry lists 21 studies for Shwachman-Diamond Syndrome across more than 10 countries. 4 are recruiting or about to start. 4 of them are mainly about Shwachman-Diamond Syndrome (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-12.
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Breast cancer · Recruiting: 2 came in, 3 changed
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A study of drug A after surgery for early breast cancer
Came in · now Recruiting
Drug B with hormone therapy in advanced breast cancer
Changed
· Sites: 4 added, including Seoul and Busan
· Enrollment: 300 → 450 participants
Exercise during chemotherapy
Now Active, not recruiting
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Who runs them
Masonic Cancer Center, University of Minnesota (3) · Diane George (2) · Emory University (2) · Roswell Park Cancer Institute (2) · Fred Hutchinson Cancer Center (2) · Washington University School of Medicine (1) · Bellicum Pharmaceuticals (1) · University Hospital Freiburg (1)
What is being tested here
Drugs under test in Shwachman-Diamond Syndrome 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 |
|---|---|---|---|
| Mavorixafor | 1 | 1 | 1 |
| Fludarabine | 0 | 2 | 5 |
| Busulfan | 0 | 2 | 3 |
| Fludarabine phosphate | 0 | 1 | 3 |
| Melphalan | 0 | 0 | 2 |
| Cyclophosphamide | 0 | 1 | 2 |
| Treosulfan | 0 | 0 | 1 |
| Alemtuzumab | 0 | 1 | 1 |
| Abatacept | 0 | 0 | 1 |
| Antilymphocyte Serum | 0 | 1 | 1 |
Drug classes tested here Alkylating agents 6 · Antimetabolites 5
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.
The study
Who these studies take
Of the 4 Shwachman-Diamond Syndrome studies recruiting now, 3 (75%) set no upper age limit and 0 (0%) stop at 75 or younger. 4 (100%) take children under 18. 0 (0%) are for women only and 0 (0%) for men only; 1 (25%) take healthy volunteers.
| Age | Women | Men |
|---|---|---|
| 10 | 3 (75%) | 3 (75%) |
| 18 | 4 (100%) | 4 (100%) |
| 30 | 4 (100%) | 4 (100%) |
| 40 | 4 (100%) | 4 (100%) |
| 50 | 4 (100%) | 4 (100%) |
| 65 | 4 (100%) | 4 (100%) |
| 75 | 4 (100%) | 4 (100%) |
| 85 | 4 (100%) | 4 (100%) |
Across all 21 studies, recruiting or not: 71% stop at 75 or younger and 62% at 65; 95% take children; 0% are for women only and 0% for men only; 19% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.
Studies
Recruiting first, then most recently updated. Showing 21 of 21. search them.
- Cancer in Inherited Bone Marrow Failure SyndromesRecruitingNCT000272741 country
- A Study of Mavorixafor in Participants With Congenital and Acquired Primary Autoimmune and Idiopathic Chronic Neutropenic Disorders Who Are Experiencing Recurrent and/or Serious InfectionsRecruitingNCT06056297Phase 325 countries
- Shwachman Diamond Syndrome Registry and StudyRecruitingNCT060569081 country
- Shwachman-Diamond Syndrome Global Patient Survey and Partnering PlatformRecruitingNCT069999541 country
- Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904)CompletedNCT04965597Phase 21 country
- CD34+ (Non-Malignant) Stem Cell Selection for Patients Receiving Allogeneic Stem Cell TransplantationActive, not recruitingNCT01966367Phase 1/21 country
- Baby Detect : Genomic Newborn ScreeningCompletedNCT056874741 country
- Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood CancerTerminatedNCT03333486Phase 21 country
- Hematopoietic Stem Cell Transplant for High Risk HemoglobinopathiesTerminatedNCT02179359Not applicable1 country
- Diabetes/ Endocrine Surveillance in SDSTerminatedNCT042754791 country
- CD34+ (Malignant) Stem Cell Selection for Patients Receiving Allogenic Stem Cell TransplantActive, not recruitingNCT02061800Phase 1/21 country
- Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant DiseasesTerminatedNCT02231710Phase 11 country
- Treosulfan and Fludarabine Phosphate Before Donor Stem Cell Transplant in Treating Patients With Nonmalignant Inherited DisordersCompletedNCT00919503Phase 21 country
- Stem Cell Transplant for HemoglobinopathyCompletedNCT00176852Phase 2/31 country
- Bone Marrow Transplant With Abatacept for Non-Malignant DiseasesCompletedNCT01917708Phase 11 country
- Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic MalignanciesCompletedNCT01529827Phase 21 country
- Stem Cell Transplant for Bone Marrow Failure SyndromesCompletedNCT00176878Phase 2/31 country
- Determination of the Postprandial Responses to Two Cereal Foods Differing by Their SDS Content Consumed Alone.CompletedNCT02538653Not applicable1 country
- Alefacept and Allogeneic Hematopoietic Stem Cell TransplantationTerminatedNCT01319851Not applicable1 country
- Assessing Immune Function in Young Patients With Cytopenia That Did Not Respond to TreatmentCompletedNCT0049907010 countries
- Phase II Pilot Study of Granulocyte Colony-Stimulating Factor for Inherited Bone Marrow Failure SyndromesCompletedNCT00004787Phase 2