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Results for “Aplasia Cutis Congenita”

33 trials

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Showing 20 of 33 results

Testing effectiveness (Phase 2)Active Not RecruitingNCT01659606
What this trial is testing

Radiation- and Alkylator-free Bone Marrow Transplantation Regimen for Patients With Dyskeratosis Congenita

Who this might be right for
Dyskeratosis CongenitaHoyeraal Hreidarsson SyndromeRevesz Syndrome+1 more
Boston Children's Hospital 40
Not applicableStudy completedNCT00499070
What this trial is testing

Assessing Immune Function in Young Patients With Cytopenia That Did Not Respond to Treatment

Who this might be right for
Dyskeratosis CongenitaFanconi AnemiaMyelodysplastic Syndromes+2 more
University Hospital Freiburg 119
Not applicableEnrolling By InvitationNCT03655223
What this trial is testing

Early Check: Expanded Screening in Newborns

Who this might be right for
Spinal Muscular AtrophyFragile X SyndromeFragile X - Premutation+182 more
RTI International 30,000
Not applicableLooking for participantsNCT03050268
What this trial is testing

Familial Investigations of Childhood Cancer Predisposition

Who this might be right for
Acute LeukemiaAdenomatous PolyposisAdrenocortical Carcinoma+43 more
St. Jude Children's Research Hospital 1,500
Not applicableLooking for participantsNCT06065852
What this trial is testing

National Registry of Rare Kidney Diseases

Who this might be right for
Adenine Phosphoribosyltransferase DeficiencyAH AmyloidosisAHL Amyloidosis+81 more
UK Kidney Association 35,000
Early research (Phase 1)Study completedNCT00692926
What this trial is testing

Unrelated Umbilical Cord Blood Transplantation Augmented With ALDHbr Umbilical Cord Blood Cells

Who this might be right for
MDSAnemia, AplasticInborn Errors of Metabolism+2 more
Joanne Kurtzberg, MD 37
Testing effectiveness (Phase 2)Ended earlyNCT01001598
What this trial is testing

Safety and Efficacy Trial of Danazol in Patients With Fanconi Anemia or Dyskeratosis Congenita

Who this might be right for
Fanconi AnemiaDyskeratosis Congenita
Boston Children's Hospital 5
Early research (Phase 1)Study completedNCT01586455
What this trial is testing

Human Placental-Derived Stem Cell Transplantation

Who this might be right for
Mucopolysaccharidosis IMucopolysaccharidosis VIAdrenoleukodystrophy+13 more
New York Medical College 43
Testing effectiveness (Phase 2)Study completedNCT00730314
What this trial is testing

Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells

Who this might be right for
Sickle Cell DiseaseThalassemiaAnemia+9 more
Children's Hospital Los Angeles 25
Not applicableLooking for participantsNCT00027274
What this trial is testing

Cancer in Inherited Bone Marrow Failure Syndromes

Who this might be right for
Diamond Blackfan AnemiaDyskeratosis CongenitaFanconi Anemia+2 more
National Cancer Institute (NCI) 4,000
Testing effectiveness (Phase 2)Looking for participantsNCT01962415
What this trial is testing

Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT

Who this might be right for
Primary Immunodeficiency (PID)Congenital Bone Marrow Failure SyndromesInherited Metabolic Disorders (IMD)+4 more
Paul Szabolcs 100
Not applicableStudy completedNCT02162420
What this trial is testing

Hematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic Anemia

Who this might be right for
Dyskeratosis CongenitaAplastic Anemia
Masonic Cancer Center, University of Minnesota 61
Large-scale testing (Phase 3)Study completedNCT00455312
What this trial is testing

Stem Cell Transplant (SCT) for Dyskeratosis Congenita or SAA

Who this might be right for
Dyskeratosis CongenitaAplastic Anemia
Masonic Cancer Center, University of Minnesota 36
Testing effectiveness (Phase 2)Study completedNCT00004787
What this trial is testing

Phase II Pilot Study of Granulocyte Colony-Stimulating Factor for Inherited Bone Marrow Failure Syndromes

Who this might be right for
Shwachman SyndromeFanconi's AnemiaDyskeratosis Congenita+1 more
National Center for Research Resources (NCRR) 20
Not applicableEnded earlyNCT01319851
What this trial is testing

Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation

Who this might be right for
ThalassemiaSickle Cell DiseaseGlanzmann Thrombasthenia+10 more
Emory University 3
Not applicableActive Not RecruitingNCT00542373
What this trial is testing

Widefield Fluorescence and Reflectance Imaging Systems and Oral Tissue Samples in Monitoring Participants at Risk for Developing Oral Cancer

Who this might be right for
ErythroplakiaFanconi AnemiaLichen Planus+5 more
M.D. Anderson Cancer Center 338
Not applicableLooking for participantsNCT04528355
What this trial is testing

Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC

Who this might be right for
Primary Immunodeficiency (PID)Congenital Bone Marrow Failure SyndromesInherited Metabolic Disorders (IMD)+2 more
Paul Szabolcs 50
Early research (Phase 1)Ended earlyNCT02231710
What this trial is testing

Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases

Who this might be right for
Primary Immune Deficiency DisordersHemophagocytic LymphohistiocytosisInherited Bone Marrow Failure Syndrome+2 more
Bellicum Pharmaceuticals 1
Testing effectiveness (Phase 2)Looking for participantsNCT03579875
What this trial is testing

Alpha/Beta TCD HCT in Patients With Inherited BMF Disorders

Who this might be right for
Fanconi AnemiaSevere Aplastic AnemiaMyelodysplastic Syndromes+5 more
Masonic Cancer Center, University of Minnesota 48
Early research (Phase 1)Study completedNCT01917708
What this trial is testing

Bone Marrow Transplant With Abatacept for Non-Malignant Diseases

Who this might be right for
Hurler SyndromeFanconi AnemiaGlanzmann Thrombasthenia+12 more
Emory University 10
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