FDA Office of Orphan Products Development: Clinical Trials

73 trials sponsored by FDA Office of Orphan Products Development, newest first

ClinicalTrials Daily  ·  Areas  ·  Conditions  ·  Drugs  ·  Sponsors  ·  

58 completed, 6 terminated, 4 recruiting, 2 active not recruiting. 32 Phase 2, 19 Phase 1, 16 Phase 3, 15 Not applicable.

By phase and status

PhaseTrialsStatus
Phase 232Completed 23, Terminated 5, Recruiting 2, Suspended 1, Withdrawn 1
Phase 119Completed 15, Recruiting 2, Terminated 2
Phase 316Completed 13, Active not recruiting 1, Unknown 1, Withdrawn 1
Not applicable15Completed 14, Active not recruiting 1

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Trials newest first · largest first

Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.

TrialPhaseStatusSponsorEnrollmentStartUpdated
Safety of MT-401-OTS in Patients With Relapsed AML or MDS
MT-401-OTS - Acute Myeloid Leukemia, in Relapse, MDS
Phase 1RecruitingMarker Therapeutics, Inc.402025-06-162025-12-16
Multicenter Trial of ECMO in Children With Severe Cardiac Failure Using the Cardiohelp System
Cardiohelp device (VA-ECMO), Heparin, Bivalirudin - Heart Failure, Cardiogenic Shock
Phase 2RecruitingStanford University502025-04-152026-08-10
Interferon-γ (IFN-γ) With Donor Leukocyte Infusion to Treat Relapsed Acute Myeloid Leukemia and Myelodysplastic Syndromes Post Allogeneic Hematopoietic Stem Cell Transplantation
Interferon gamma-1b, Donor Leukocyte Infusion (DLI) - Acute Myeloid Leukemia, Myelodysplastic Syndromes
Phase 2RecruitingSawa Ito, MD572024-09-232026-08-11
SELVA: A Phase 3 Study Evaluating QTORIN 3.9% Rapamycin Anhydrous Gel in the Treatment of Microcystic Lymphatic Malformations
QTORIN 3.9% Rapamycin Anhydrous Gel - Microcystic Lymphatic Malformation
Phase 3Active not recruitingPalvella Therapeutics, Inc.512024-07-312026-03-27
Danvatirsen Monotherapy Followed by Combination With Venetoclax in Relapsed/Refractory MDS & AML
Danvatirsen, Danvatirsen + Venetoclax - AML/MDS, Acute Myeloid Leukemia
Phase 1RecruitingMontefiore Medical Center382024-05-082026-10-15
Daily Vitamin D for Sickle-cell Respiratory Complications
Daily oral vitamin D3, 3,333 IU, Monthly oral vitamin D3, 100,000 IU, Placebo oral tablet - Sickle Cell Disease, Anemia, Sickle Cell
Phase 2CompletedColumbia University582020-09-152025-09-24
KPT-330 Plus RICE for Relapsed/Refractory Aggressive B-Cell Lymphoma
KPT-330, Rituximab, Etoposide - Diffuse Large B-Cell Lymphoma
Phase 1CompletedWeill Medical College of Cornell University222015-12-112022-04-12
Abatacept as GVHD Prophylaxis Phase 2
Abatacept, placebo - Graft vs Host Disease, Malignancy
Phase 2CompletedBoston Children's Hospital1862013-02-012025-12-11
Efficacy and Safety Study of Nexagon for Persistent Corneal Epithelial Defects
Nexagon, Vehicle only - Persistent Corneal Epithelial Defects
Phase 2TerminatedUniversity of California, San Francisco22011-11-012015-05-07
ABC294640 in Treating Patients With Advanced Solid Tumors
sphingosine kinase-2 inhibitor ABC294640 - Pancreatic Cancer, Unspecified Adult Solid Tumor, Protocol Specific
Phase 1CompletedRedHill Biopharma Limited222011-08-012020-01-07
Efficacy Study of NH001 in Vegetative State & Minimally Conscious State Following a Traumatic Brain Injury
Apomorphine, Placebo - Brain Injury
Phase 2SuspendedNeuroHealing Pharmaceuticals Inc.762010-07-012017-03-31
Alendronate in Juvenile Osteoporosis
Alendronate (Fosamax) - Juvenile Osteoporosis, Low Bone Density
CompletedMedical University of South Carolina112009-07-012018-07-11
Study of Repeat Intranodal Injection of Memgen's Cancer Vaccine, Ad-ISF35, in Subjects With Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma (CLL/SLL)
ISF35 - Chronic Lymphocytic Leukemia, Small Lymphocytic Lymphoma
Phase 2TerminatedJanuario Castro, M.D.62009-01-012015-12-30
Radioimmunoimaging of AL Amyloidosis
124I-labeled monoclonal antibody Mu 11-1F4 - Primary Amyloidosis
Phase 1CompletedUniversity of Tennessee402008-12-012013-09-18
MND-ADA Transduction of CD34+ Cells From Children With ADA-SCID
ADA gene transfer - Severe Combined Immunodeficiency
Phase 2CompletedDonald B. Kohn, M.D.102008-11-012021-04-23
8-Chloro-Adenosine in Chronic Lymphocytic Leukemia
8-Chloro-Adenosine - Leukemia, Chronic Lymphocytic Leukemia
Phase 1CompletedM.D. Anderson Cancer Center112008-06-012016-10-13
A Safety and Efficacy Study of AEGR-733 to Treat Homozygous Familial Hypercholesterolemia (FH)
AEGR-733 - Homozygous Familial Hypercholesterolemia
Phase 3CompletedAegerion Pharmaceuticals, Inc.292007-12-012018-03-20
Clinical Trial of Vincristine vs. Prednisolone for Treatment of Complicated Hemangiomas
Vincristine, Prednisone - Hemangioma
Phase 2TerminatedMedical College of Wisconsin82007-11-012013-06-11
Penicillamine Chelation for Children With Lead Poisoning
d-penicillamine, placebo - Lead Poisoning, Vitamin D Deficiency
Phase 2 / Phase 3WithdrawnFDA Office of Orphan Products Development2007-09-012015-03-26
Safety and PK of Nikkomycin Z for Coccidioides Pneumonia Treatment
nikkomycin Z, Placebo - Coccidioidomycosis
Phase 1 / Phase 2TerminatedUniversity of Arizona62007-09-012025-04-16

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Conditions studied

Rare Diseases (21)  ·  Respiratory Tract Diseases (9)  ·  Genetic Diseases, Inborn (6)  ·  Cystic Fibrosis (5)  ·  Anemia, Sickle Cell (4)  ·  Communicable Diseases (3)  ·  Immune System Diseases (3)  ·  Leukemia, Myeloid, Acute (3)  ·  Urogenital Diseases (3)  ·  Amyloidosis (2)  ·  Bacterial Infections (2)  ·  Fractures, Bone (2)  ·  Gastrointestinal Diseases (2)  ·  Graft vs Host Disease (2)  ·  Heart Defects, Congenital (2)

Interventions

ADI PEG20 (2)  ·  Alendronate (2)  ·  Cholecalciferol (2)  ·  Dexamethasone (2)  ·  Etanercept (2)  ·  Minocycline (2)  ·  Oxandrolone (2)  ·  Prednisone (2)  ·  Solutions (2)  ·  Thalidomide (2)  ·  3-(4-chlorophenyl)-adamantane-1-carboxylic acid (pyridin-4-ylmethyl)amide (1)  ·  4-Aminopyridine (1)  ·  8-chloroadenosine (1)  ·  Abatacept (1)  ·  Administration, Oral (1)

Countries

United States (51)  ·  Canada (8)  ·  Finland (2)  ·  France (2)  ·  Italy (2)  ·  United Kingdom (2)  ·  Belgium (1)  ·  Germany (1)  ·  Israel (1)  ·  Japan (1)  ·  Lithuania (1)  ·  Netherlands (1)  ·  Poland (1)  ·  Russia (1)  ·  South Africa (1)

Top investigators

Deborah A Bowlby, M.D. (2)  ·  Abhimanyu Garg, M.D. (1)  ·  Aditi Shastri, MBBS (1)  ·  Alan Bruce Montgomery (1)  ·  Alan Solomon, MD (1)  ·  Annette S. Feigenbaum, MD (1)  ·  Bennie H Jeng, MD (1)  ·  Beth Drolet, MD (1)  ·  Caroline Ozment, MD (1)  ·  Carolyn Britten, MD (1)  ·  Carolyn C Hoppe, M.D. (1)  ·  Daniel F Hanley, MD (1)  ·  David E Nix, Pharm D (1)  ·  Debra Weiner, MD, PhD (1)

Results publications

PMID 12795371, PMID 11588741, PMID 12905485, PMID 15751091, PMID 15838068, PMID 15986348, PMID 16378799, PMID 16646004, PMID 17554116, PMID 18406958, PMID 20522711, PMID 21180274, PMID 21410393, PMID 21477202, PMID 21940973, PMID 22127969, PMID 22382155, PMID 22474059, PMID 23370203, PMID 23447519, PMID 23463422, PMID 23622922, PMID 24159565, PMID 25405392, PMID 25897792, PMID 25979023, PMID 26228884, PMID 26723464, PMID 26799509, PMID 26825712, PMID 28346229, PMID 29948025, PMID 30846465, PMID 33331662, PMID 33974038, PMID 35914227, PMID 36520008, PMID 36927115, PMID 37319437, PMID 37811711, PMID 37983082, PMID 38583802, PMID 38775255, PMID 9628377

NIH funding

R01FD001847 (1), U01HL068270 (1), U54RR019498 (1)