Phase 3 N=19 Plerixafor Versus G-CSF in the Treatment of People With WHIM Syndrome
Myelokathexis · Infections · Neutropenia
Primary: Severity of Infection — 12.5; 9 score on a scale (with no maximum)
Clinical Trial Search
Completed trials with posted results — search by concept, filter by the parameters that matter at the bedside.
Phase 2 N=8 A Dose Determination and Safety Study of X4P-001 (Mavorixafor) in Participants With Warts, Hypogammaglobulinemia, Infections, and Myelokathexis (WHIM) Syndrome
WHIM Syndrome
Primary: Mean Value of the Area Under the Plasma Concentration-time Curve for Absolute Neutrophil Count (AUCANC) — -12574.63; -6206.75; -6949.58; 24175.00 cells*hour/μL
Phase 1 N=6 AMD 3100 for Treatment of Myelokathexis
Neutropenia
Primary: Blood Neutrophil Counts. — 4.48 10^9 per Liter — p=<0.05
Phase 3 N=47 Rilonacept for Treatment of Cryopyrin-Associated Periodic Syndromes (CAPS)
Familial Cold Autoinflammatory Syndrome (FCAS) · Familial Cold Urticaria · Muckle-Wells Syndrome (MWS)
Primary: Change From Baseline to Week-6 (Part A) Endpoint in Mean Key Symptom Score (KSS) — 2.4; 3.1; 2.1; 0.5 Units of a Scale — p=<.0001
Phase 2 N=8 Gene Therapy for Wiskott-Aldrich Syndrome
Wiskott-Aldrich Syndrome (WAS)
Primary: Safety of Reduced Conditioning Regimen — 8 Participants
Phase 2 N=21 Efficacy of Minoxidil in Children With Williams-Beuren Syndrome
Williams Beuren Syndrome
Primary: Variation of Carotid Intima-media Thickness (IMT) Assessed by Vascular Echography — 0.028; 0.012 mm
Phase 2 N=47 Reduced Intensity Conditioning for Hemophagocytic Syndromes or Selected Primary Immune Deficiencies (BMT CTN 1204)
Hemophagocytic Lymphohistiocytosis · Chronic Active Epstein-Barr Virus Infection · Chronic Granulomatous Disease
Primary: Percentage of Participants With Overall Survival (OS) — 80.4; 66.7 percentage of participants
Phase 2 N=31 Study of Ibrutinib in Patients With Symptomatic, Previously Untreated Waldenstrom's Macroglobulinemia, and Impact on Tumor Genomic Evolution Using Whole Genome Sequencing
Waldenstrom's Macroglobulinemia
Primary: Major Response Rate — 26 Participants
Phase 2 N=37 Perifosine in Patients With Relapsed/Refractory Waldenstrom's Macroglobulinemia
Waldenstrom's Macroglobulinemia
Primary: Overall Response (OR) Rate — 38 percentage of patients
Phase 2 N=6 Growth Hormone Treatment in Children With Phelan McDermid Syndrome
Phelan McDermid Syndrome
Primary: ABC - Social Withdrawal Subscale — 10.31; 4.6; 14.8; 6.2 score on a scale
N/A N=19 Mast Cell Activation Test in Allergic Disease
Asthma · Mastocytosis · Mast Cell Activation Syndrome
Primary: Mast Cell Activation Test Results for Validation Group — 6; 2; 0; 4 Participants
N/A N=125 Therapy of HES, PV, Atypical Chronic Myelocytic Leukemia (CML) or Chronic Myelomonocytic Leukemia (CMML), and Mastocytosis With Imatinib Mesylate
Chronic Myelomonocytic Leukemia · Chronic Myeloid Leukemia · Polycythemia Vera
Primary: Number of Participants With a Complete Response (CR) — 0; 0; 2; 0 participants
Phase 2 N=11 Interleukin-1 Trap to Treat Autoinflammatory Diseases
Inflammation · Familial Mediterranean Fever · Still's Disease, Adult-Onset
Primary: Mean Change in Daily Scores — -3.09 units on a scale
Phase 2 N=16 Safety and Efficacy of Subcutaneous Sarilumab in Improving the Quality of Life in People With Indolent Systemic Mastocytosis
Indolent Systemic Mastocytosis
Primary: Percent Change in Quality of Life (QoL) Using Mastocytosis Quality of Life Questionnaire (MC-QoL) — 38.98; 33.02 percentage of change in quality of life — p=0.7740
Phase 2 N=33 Study of ABT-199 (GDC-199) In Patients With Relapsed Or Refractory Waldenström Macroglobulinemia
Waldenstrom Macroglobulinemia
Primary: Overall Response Rate — 26 Participants
Phase 2 N=37 A Phase II Trial of Ofatumumab in Subjects With Waldenstrom's Macroglobulinemia
Waldenstrom Macroglobulinaemia
Primary: Number of Participants With Overall Response (OR) for Cycle 1 (Including the Redosing Cycle), as Assessed by the Investigator — 7; 15 participants
Phase 2 N=25 Thalidomide and Rituximab in Waldenstrom's Macroglobulinemia
Waldenstrom's Macroglobulinemia · Lymphoplasmacytic Lymphoma
Primary: Objective Response Rate — 1; 15; 2; 7 participants
Phase 4 N=18 Role of Immunoglobulin E (IgE) Bearing Cells in Chronic Idiopathic Urticaria (CIU)
Urticaria Chronic
Primary: Time to 50% Decline in Clinical Symptoms Measured by the Urticaria Activity Score Seven Day(UAS7) — 23; 12 days
Phase 3 N=32 Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks in Patients With Hereditary Angioedema
Hereditary Angioedema · Angioneurotic Edema · Genetic Disorders
Primary: Time to Beginning of Relief of Symptoms — 62; 508; 61 minutes — p=0.003
Phase 2 N=5 Magnesium Supplementation in People With XMEN Syndrome
XMEN
Primary: Participants With a ≥0.5 Log Reduction in the Number of EBV-infected B Cells After Magnesium Supplementation as Compared to Placebo - Phase 1 — 0; 0 Participants
Phase 2 N=63 Ibrutinib (PCI-32765) in Waldenstrom's Macroglobulinemia
Waldenstrom's Macroglobulinemia
Primary: Overall Response Rate — 57 Participants
Phase 1 N=17 Evaluation of a New Anti-cancer Immunotherapy in Adult Acute Myeloid Leukemia Patients With a Suboptimal Clinical Response to Induction Chemotherapy
Leukaemia, Myelocytic, Acute
Primary: Number of Patients With Severe Toxicities — 1; 0 Participants
Phase 2 N=20 Abatacept in Treating Adults With Mild Relapsing Wegener's Granulomatosis
Wegener's Granulomatosis
Primary: Safety of Abatacept - Number of Participants With Adverse Events — 7; 16; 14; 1 participants
Phase 2 N=7 IL-11 in Women With Von Willebrand Disease and Refractory Menorrhagia
Von Willebrand Disease
Primary: >50% Reduction in PBAC (Pictorial Blood Assessment Chart) at 6 Months — 71 percentage of participants
Phase 2 N=12 Efficacy of Eltrombopag to Improve Thrombocytopenia of MYH9-related Disease
Blood Platelet Disorders
Primary: Response to Drug Based on Platelet Count at the End of Therapy — 91.6 percentage of participants
Phase 2 N=6 Inhibition of Anaphylaxis by Ibrutinib
Food Allergy · Anaphylaxis Food
Primary: Number of Doses of Ibrutinib for Maximal Suppression of Skin Prick Test Size to Foods — 2 doses — p=<0.0001
Phase 3 N=181 Ibrutinib With Rituximab in Adults With Waldenström's Macroglobulinemia
Waldenström's Macroglobulinemia
Primary: Progression Free Survival (PFS) Based on Independent Review Committee (IRC) Assessment - Kaplan Meier Landmark Estimates at Month 54 — 68.0; 25.3; 39.7 percentage of…
Phase 3 N=38 Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks in Patients With Hereditary Angioedema
Hereditary Angioedema · Angioneurotic Edema
Primary: Time to Beginning of Relief of Symptoms — 68; 122; 258; 62.5 minutes — p=0.001
N/A N=12 PMS Retrospective Study of WIRION Performance in Patients Undergoing Saphenous Vein Graft (SVG) Intervention
Stenosis
Primary: Number of Participants With Adverse Events During the Procedure and up to 30 Days Following Procedure — 5 Participants
N/A N=80 SMYLS: A Self-management Program for Youth Living With Sickle Cell Disease
Sickle Cell Disease
Primary: Rate of Recruitment: Dyads — 2 dyads recruited and enrolled/week