Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Alemtuzumab · 1 trial · 1 indication
Primary endpoint: In each group, the Number of participants with Graft Failure at the 2 years endpoint will be estimated using the Kaplan Meier product limit estimator.
| Arm | Type | Description |
|---|---|---|
| Related donor | EXPERIMENTAL | Matched sibling donors (9-10/10 marrow/PBSC or 5-6/6 UCB (single) with a total TNC dose of greater than 5 x 107/kg recipient weight), age 2-30 years after conditioning regimen Alemtuzumab , Fludarabine, and Melphalan. 1\) Patients will receive a conditioning regimen composed of Alemtuzumab, Fludarabine, and Melphalan as detailed in the table below. Day Treatment * -22 Alemtuzumab 3mg IV (test dose) * -21 Alemtuzumab 10mg IV * -20 Alemtuzumab 15mg IV * -19 Alemtuzumab 20mg IV * -8 Fludarabine 30mg/m2 IV * -7 Fludarabine 30mg/m2 IV * -6 Fludarabine 30mg/m2 IV * -5 Fludarabine 30mg/m2 IV * -4 Fludarabine 30mg/m2 IV * -3 Melphalan 140mg/m2 IV * -2 Rest Day * -1 Rest Day * 0 Stem Cell Infusion |
| Name | Type | Description |
|---|---|---|
| Alemtuzumab | DRUG | Adjusted Ideal Body Weight Formula: AIBW = IBW + \[(0.4) x (ABW - IBW)\] b) Medications i.) Alemtuzumab I. Hb S% must be \< or = 45% within 7 days prior to initiation of Alemtuzumab II. Iron chelation and hydroxyurea must be discontinued \>48 hours before initiating therapy III. Alemtuzumab will be diluted in 100mL of 0.9% NS and infused at a rate as below |
| Fludarabine | DRUG | I. Fludarabine should be diluted in 100 ml 0.9%NS and given over 30 minutes. II. A daily dose of an antiemetic should be given 30 minutes prior to administration of the Fludarabine |
| Melphalan | DRUG | I. Melphalan should be diluted in 0.9%NS to a concentration of 0.1 -0.45 mg/mL and given over 45 minutes. \*Entire dose must be infused within 60 minutes of reconstitution in Pharmacy. II. A daily dose of an antiemetic should be given 30 minutes prior to administration of the Melphalan III. Patients should be encouraged to suck on a popsicle or something similar during the Melphalan infusion. |
| Stem Cells | PROCEDURE | Infusion of Hematopoietic Stem Cells |
Inclusion Criteria: * Patient Eligibility 1\) Matched sibling donors (9-10/10 marrow/PBSC or 5-6/6 UCB (single or double) with a total TNC dose of greater than 5 x 107/kg recipient weight) 1. Age 2-30 2. Hb SS, S-thal0, S-thal+, SC 3. Evidence of ongoing hemolysis: Hb\<10, retic \>5%, LDH ...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Novo Nordisk A/S Sponsored ADR Class B | NVO | 5 | PHASE3 | Etavopivat Low dose |
| Novartis AG Sponsored ADR | NVS | 4 | PHASE3 | Crizanlizumab |
| Sanofi SA Sponsored ADR | SNY | 2 | PHASE3 | PCV21, 20vPCV |
| Vertex Pharmaceuticals Incorporated | VRTX | 3 | PHASE3 | CTX001 |
| Agios Pharmaceuticals, Inc. | AGIO | 2 | PHASE2 | Mitapivat |
| Pfizer Inc. | PFE | 1 | PHASE2 | Osivelotor |
| Bristol-Myers Squibb Company | BMY | 1 | PHASE1 | BMS-986470, Famotidine, Pantoprazole |
| Fulcrum Therapeutics, Inc. | FULC | 1 | PHASE2 | Pociredir |
| Beam Therapeutics, Inc. | BEAM | 2 | PHASE1 | BEAM-101 |
| Editas Medicine, Inc. | EDIT | 2 | PHASE1 | EDIT-301 |
| Disc Medicine, Inc. | IRON | 1 | PHASE1 | DISC-3405 |
| Illumina, Inc. | ILMN | 1 | - | Undisclosed |
Alemtuzumab is being studied for use in Sickle Cell Disease as part of a stem cell transplantation regimen. It is an investigational small molecule in Phase 3 clinical development for this hematology indication. The drug is not approved and remains under clinical investigation.
Alemtuzumab targets CD52, a protein found on the surface of mature lymphocytes. By binding to CD52, it leads to the depletion of these immune cells, which is useful in conditioning patients before stem cell transplantation. This mechanism is being evaluated in the context of Sickle Cell Disease treatment.
Alemtuzumab is being developed by Meridian Bioscience Inc, which trades under the ticker VIVO. The company is conducting clinical research on this drug for Sickle Cell Disease, with the asset currently in Phase 3 development.
Alemtuzumab is in Phase 3 clinical development for Sickle Cell Disease. It is an investigational drug, meaning it has not been approved by regulatory authorities. The Phase 3 trial has been completed, with results not yet reported in this context.
Alemtuzumab is associated with one completed Phase 3 trial, NCT01877837, titled 'Stem Cell Transplantation for Sickle Cell Anemia'. This trial enrolled 30 participants in the United States, with a minimum age of 2 years, and was uncontrolled, meaning there was no comparator arm.
Alemtuzumab is also known as Campath, a brand name for the same drug. In the context of Sickle Cell Disease, it is being investigated under the name Alemtuzumab, but it is the same compound. This alternative name is important for literature searches and understanding its clinical history.