Clinical Trials on Wolfram Syndrome

Total 34048 results

University of Colorado, Denver

Approved for marketing

Use of 3,4-Diaminopyridine in the Treatment of Lambert-Eaton Syndrome (3 4-DAP)

Lambert Eaton Myasthenic Syndrome

United States
Jacobus Pharmaceutical

Completed

Effectiveness of 3,4-Diaminopyridine in Lambert-Eaton Myasthenic Syndrome (DAPPER)

Lambert-Eaton Myasthenic Syndrome | Eaton-Lambert Myasthenic Syndrome

United States
Minovia Therapeutics Ltd.

Recruiting

Collection of Samples of Bone Marrow Aspiration From Patients With Myelodysplastic Syndrome

Myelodysplastic Syndromes

Israel
M.D. Anderson Cancer Center

Recruiting

A Pilot, Open-Label Study of Luspatercept for Patients With Lower Risk Myelodysplastic Syndromes (MDS)

Myelodysplastic Syndromes

United States
Anhui Provincial Hospital

Recruiting

Safety and Efficacy of Unrelated Umbilical Cord Blood Microtransplantation in Patients With Higher-risk MDS

Myelodysplastic Syndromes

China
GWT-TUD GmbH
Celgene

Recruiting

Assessment of Effectiveness and Safety of Luspatercept in Patients Suffering From Lower-risk Myelodysplastic Syndrome. (LUSPLUS)

Myelodysplastic Syndromes

Austria, Germany, Spain, Switzerland
Akeso

Recruiting

A Trial of AK117 (Anti-CD47) in Patients With Myelodysplastic Syndrome

Myelodysplastic Syndrome

China
University Hospital, Toulouse

Recruiting

Interplay Between Immune and Metabolic Programs in Myelodysplastic Syndromes (IMET-MDS)

Myelodysplastic Syndromes

France
Peking University People's Hospital

Recruiting

RIC Regimen for Low- and Intermediate-risk MDS Receiving Haplo-HSCT

Myelodysplastic Syndromes

China
Institut Paoli-Calmettes

Recruiting

Institut Paoli Calmettes Myelodysplastic Syndromes Database

Myelodysplastic Syndromes

France
Yonsei University

Recruiting

Cohort Study of New Prognostic Factors With Peripheral Blood and Bone Marrow Evaluation at the Time of Diagnosis and Relapse in Myelodysplastic Syndrome

Myelodysplastic Syndrome

Korea, Republic of
Memorial Sloan Kettering Cancer Center
National Cancer Institute (NCI)

Completed

Decitabine and Tretinoin in Treating Patients With Myelodysplastic Syndromes

Myelodysplastic Syndromes

United States
Geron Corporation

Active, not recruiting

Study to Evaluate Imetelstat (GRN163L) in Subjects With International Prognostic Scoring System (IPSS) Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS)

Myelodysplastic Syndromes

United States, Canada, Belgium, Spain, Czechia, Korea, Republic of, United Kingdom, Netherlands, Israel, Germany, Russian Federation, Switzerland, Italy, France, Turkey, Poland, Ukraine
Astex Pharmaceuticals, Inc.

Active, not recruiting

Phase 1-2 Study of Low Dose ASTX727 (ASTX727 LD) in Lower Risk MDS

Myelodysplastic Syndromes

Spain, United States, Belgium, Canada, Germany, Italy
Astex Pharmaceuticals, Inc.

Terminated

A Study of Oral Decitabine/Cedazuridine in Combination With Magrolimab in Participants With Intermediate- to Very High-Risk Myelodysplastic Syndromes (MDS)

Myelodysplastic Syndromes

United States
Novartis Pharmaceuticals

Active, not recruiting

A Study of MBG453 in Combination With Hypomethylating Agents in Subjects With IPSS-R Intermediate, High or Very High Risk Myelodysplastic Syndrome (MDS). (STIMULUS-MDS1)

Myelodysplastic Syndromes

Czechia, Hungary, Japan, Taiwan, United States, Belgium, Italy, United Kingdom, Korea, Republic of, Hong Kong, Austria, Germany, Turkey, Spain, Greece, France, Canada
Shanghai Antengene Corporation Limited

Terminated

A Study to Evaluating the Pharmacokinetics, Safety, and Efficacy of ATG 016 Monotherapy in IPSS-R Intermediate Risk and Above Myelodysplastic Syndrome (MDS) (HATCH)

Myelodysplastic Syndrome

China
Washington University School of Medicine
Jazz Pharmaceuticals

Active, not recruiting

CPX-351 (Vyxeos™) for Transplant Eligible, Higher Risk Patients With Myelodysplastic Syndrome

Myelodysplastic Syndromes

United States
National Cancer Institute (NCI)

Active, not recruiting

Testing the Safety and Efficacy of the Combination of the Antibody Pembrolizumab and Entinostat in Patients With Myelodysplastic Syndrome Who Are Not Responding to Hypomethylating Agents

Myelodysplastic Syndrome

United States
Eisai Inc.

Completed

Escalation Study to Determine Bioavailability of a Single Oral Dose of Decitabine in Patients With Myelodysplastic Syndrome (MDS)

Myelodysplastic Syndrome

United States
Fundación General de la Universidad de Salamanca
Celgene Corporation

Completed

Study of REVLIMID (Lenalidomide) Versus Placebo in Patients With Low Risk Myelodysplastic Syndrome (SINTRA-REV)

Myelodysplastic Syndrome

Germany, Spain, France
Sumitomo Pharma Co., Ltd.

Completed

Study of DSP-7888 in Patients With Myelodysplastic Syndrome (MDS)

Myelodysplastic Syndrome

Japan
Memorial Sloan Kettering Cancer Center
M.D. Anderson Cancer Center; Columbia University; Karyopharm Therapeutics Inc

Completed

Selective Inhibitor of Nuclear Export (SINE) Selinexor (KPT-330) in Patients With Myelodysplastic Syndromes

Myelodysplastic Syndromes

United States
Columbia University
Celgene Corporation

Active, not recruiting

Revlimid in Transfusion Dependent Patients

Myelodysplastic Syndrome

United States
Montefiore Medical Center
Albert Einstein College of Medicine

Withdrawn

Pyrimethamine for Intermediate/High-risk Myelodysplastic Syndromes (MDS)

Myelodysplastic Syndromes

United States
Onconova Therapeutics, Inc.

Completed

Oral Rigosertib in Low Risk MDS Patients Refractory to ESAs

Myelodysplastic Syndromes

United States, France, Germany
Boehringer Ingelheim

Terminated

Phase I/II Trial to Investigate BI 836858 in Myelodysplastic Syndromes

Myelodysplastic Syndromes

United States, Germany
Array Biopharma, now a wholly owned subsidiary...

Completed

A Study of ARRY-614 in Patients With Low or Intermediate-1 Risk Myelodysplastic Syndrome

Myelodysplastic Syndromes

United States
Onconova Therapeutics, Inc.

Withdrawn

Three Dosing Schedules of Oral Rigosertib in MDS Patients

Myelodysplastic Syndrome

United States
PersImmune, Inc
University of California, San Diego

Unknown

Personalized Adoptive Cellular Therapy Targeting MDS Stem Cell Neoantigens (PACTN)

Myelodysplastic Syndromes

United States
Celgene

Completed

Alternative Dosing Regimens of Subcutaneous Azacitidine for Myelodysplastic Syndromes

Myelodysplastic Syndromes

United States
GlaxoSmithKline
Parexel

Terminated

Safety, Clinical Activity, Pharmacokinetics (PK) and Pharmacodynamics Study of GSK2879552, Alone or With Azacitidine, in Subjects With High Risk Myelodysplastic Syndromes (MDS)

Myelodysplastic Syndromes | Myelodysplastic Syndrome

United States, Spain
MedImmune LLC

Completed

Phase 1 Study to Evaluate MEDI4736 in Subjects With Myelodysplastic Syndrome

Myelodysplastic Syndrome

United States, United Kingdom, France, Germany
Case Comprehensive Cancer Center
National Cancer Institute (NCI)

Terminated

RAD001(Everolimus) in Treating Patients With Myelodysplastic Syndromes

Myelodysplastic Syndromes

United States
Opsona Therapeutics Ltd.
M.D. Anderson Cancer Center; Montefiore Medical Center; New York Presbyterian... and other collaborators

Completed

A Phase I/II Study of OPN-305 in Second-line Lower Risk Myelodysplastic Syndrome

Myelodysplastic Syndrome

United States
Opsona Therapeutics Ltd.
M.D. Anderson Cancer Center; Montefiore Medical Center; New York Presbyterian... and other collaborators

Completed

Follow up Protocol to the Phase I/II Study of OPN-305 in Second-line or Third-line Lower Lower Risk Myelodysplastic Syndrome

Myelodysplastic Syndrome

United States
M.D. Anderson Cancer Center
Genzyme, a Sanofi Company

Completed

Decitabine and Clofarabine in Higher Risk Myelodysplastic Syndromes (MDS)

Myelodysplastic Syndrome

United States
M.D. Anderson Cancer Center
National Cancer Institute (NCI)

Completed

Amifostine in Treating Patients With Myelodysplastic Syndrome

Myelodysplastic Syndromes

United States
Sunnybrook Health Sciences Centre
Novartis

Terminated

Azacitidine Plus Deferasirox (ICL670) in Higher Risk Myelodysplastic Syndromes (MDS)

Myelodysplastic Syndromes

Canada
Assistance Publique - Hôpitaux de Paris
Institut National de la Santé Et de la Recherche Médicale, France; Gustave... and other collaborators

Completed

Prospective Study of Molecular Predictors of Survival in Myelodysplastic Syndromes (MDS04)

Myelodysplastic Syndromes

France
Institut Paoli-Calmettes

Unknown

Evaluation of a Question Prompt List for Patients With Myelodysplastic Syndromes (QPL-MDS) (QPL-MDS)

Myelodysplastic Syndromes

France
Celgene

Completed

Study to Determine the Safety and Preliminary Efficacy of CC-1088 in the Treatment of Myelodysplastic Syndromes

Myelodysplastic Syndrome

United States
SymBio Pharmaceuticals

Completed

Long Term Safety Study of SyB L-1101 in Patients With Recurrent/Relapsed or Refractory Myelodysplastic Syndrome (MDS) - Extension Study

Myelodysplastic Syndrome

Japan
SymBio Pharmaceuticals

Completed

Long Term Safety Study of SyB C-1101 in Patients With Recurrent/Relapsed or Refractory Myelodysplastic Syndrome (MDS) - Extension Study

Myelodysplastic Syndrome

Japan
Hoffmann-La Roche

Completed

A Study of NeoRecormon (Epoetin Beta), CellCept (Mycophenolate Mofetil) and Prednisone in Patients With Low or Intermediate Myelodysplastic Syndromes.

Myelodysplastic Syndromes

Spain
Roswell Park Cancer Institute
Genzyme, a Sanofi Company

Terminated

Safety and Tolerability of Oral Clofarabine in Intermediate to High Risk Myelodysplastic Patients

Myelodysplastic Syndrome

United States
University of Rochester

Completed

Bortezomib in Treating Patients With Myelodysplastic Syndromes

Myelodysplastic Syndromes

United States
TetraLogic Pharmaceuticals

Completed

Birinapant With 5-azacitidine in MDS Subjects Who Are Naïve, Have Relapsed or Are Refractory to 5-azacitidine Therapy

Myelodysplastic Syndrome

United States
Washington University School of Medicine

Completed

Lenalidomide for Myelodysplastic Syndrome Refractory to Hypomethylating Agents

Myelodysplastic Syndromes

United States
Seoul St. Mary's Hospital
Celgene Corporation

Unknown

5 Day Versus 7 Day Azacitidine in Lower Risk Myelodysplastic Syndrome

Myelodysplastic Syndrome

Korea, Republic of

Clinical Trials on Wolfram Syndrome

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Medical Conditions

Drug Interventions

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CROs in Cambodia

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Rare Diseases

Drug Interventions

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