17,261 trials
Brain and Central Nervous System Tumors · Extragonadal Germ Cell Tumor · Ovarian Cancer
Primary: Rate of Complete Response — 38; 7; 11 Participants
Pompe Disease
Primary: Number of Participants With a Change in Creatine Kinase (CK) Reflecting Worsening of Muscle Involvement — 1; 0 participants
Diabetes Mellitus, Type 1
Primary: Number of Hypoglycemic Events ≥50 mg/dl 15 Minutes AND ≥ 70 mg/dl 30 Minutes After Initial Treatment — 58; 53 Hypoglycemic Events — p=0.99
Obesity
Primary: Number of Participants With Any Adverse Event (AE), Serious Adverse Event (SAE) or Death During Part A — 3; 10; 0; 0 Participants
Untreated Mantle Cell Lymphoma
Primary: Number of Participants With Toxicity — 8 participants
Recurrent Bladder Carcinoma · Stage II Bladder Cancer · Stage III Bladder Cancer
Primary: Patients With Dose Limiting Toxicity — 0 Participants
Vascular Disease, Peripheral
Primary: Number of Participants With Adverse Events (AE) and Serious Adverse Events (SAE) — 14; 6; 9; 3 Participants
Smith-Lemli-Opitz Syndrome
Primary: Number of Responders — 18 Participants
Transplantation, Homologous · Transplantation, Autologous · Multiple Myeloma
Primary: Incidence of Graft Versus Host Disease (GvHD) — 1 Participants
Iron Deficiency · Iron Deficiency Anemia
Primary: Change in Transferrin Saturation (TSAT) From Baseline to End of Treatment — 21.6; 21.0; 31.7; 20.0 % saturation
Brain Injuries, Traumatic
Primary: CAPS (Cluster D Symptoms) - PRIMARY BEHAVIORAL OUTCOME MEASURE — 13.88; 12.89; 11.23; 10.89 Score
Severe Thalassemia
Primary: Primary Objective: Event-free Survival at 1 Year. — 17 Participants
Lymphoma · Primary Central Nervous System Lymphoma · Recurrent/Refractory Secondary Central Nervous System Lymphoma
Primary: Progression Free Survival — 39 days
Severe Aplastic Anemia · Very Severe Aplastic Anemia · Moderate Aplastic Anemia
Primary: Proportion of Participants With Platelet Response — 0.23 proportion of participants
Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities · Adult Acute Myeloid Leukemia With Del(5q) · Adult Acute Myeloid Leukemia With Inv(16)(p13;q22)
Primary: Rates of Complete Remission and Complete Remission With Incomplete Recovery of Counts — 38; 33; 41; 23 Participants