Adults 2 to 110, any sex, with Severe Aplastic Anemia, Refractory or Severe Aplastic Anemia, Relapse. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Number of Participants With Hematological Response at 6 MonthsPrimary· 6 months
Hematological response is defined as no longer satisfying blood count criteria for Severe Aplastic Anemia. Patients were classified as responders if they met two of the following three criteria: ANC greater than 500/ mm'; platelet count greater than 20,000/mm3; and reticulocyte count greater than 40,000/mm3 (60,000/mm3 after January 1993).
Group
Value
95% CI
Refractory
0
Relapsed
18
Adverse events — posted to ClinicalTrials.gov
Time frame: 6 months.
Reporting threshold: 0%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
Refractory
Serious: 2/5 (40%)
Deaths: 5/5
Relapsed
Serious: 22/42 (52%)
Deaths: 13/42
Serious adverse events (15 terms)
Reaction
System
Refractory
Relapsed
Febrile neutropenia
Blood and lymphatic system disorders
—
—
Anaemia
Blood and lymphatic system disorders
—
—
Cardiac failure
Cardiac disorders
—
—
Presyncope
Cardiac disorders
—
—
Gastritis
Gastrointestinal disorders
—
—
Gastrointestinal haemorrhage
Gastrointestinal disorders
—
—
Allergic transfusion reaction
Immune system disorders
—
—
Fungal infection
Infections and infestations
—
—
Pneumonia
Infections and infestations
—
—
Haemorrhagic ovarian cyst
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
—
—
Central nervous system inflammation
Nervous system disorders
—
—
Cerebral ischaemia
Nervous system disorders
—
—
Vaginal ulceration
Reproductive system and breast disorders
—
—
Epistaxis
Respiratory, thoracic and mediastinal disorders
—
—
Rash
Skin and subcutaneous tissue disorders
—
—
Other adverse events (137 terms — click to expand)
This study will evaluate the safety and usefulness of a new immunosuppressive drug, alemtuzumab (Campath ), in patients with severe aplastic anemia (SAA). SAA is a rare and serious blood disorder in which the bone marrow stops making red blood cells, white blood cells and platelets. Alemtuzumab is a monoclonal antibody that attaches to and kills white blood cells called lymphocytes. In certain types of aplastic anemia, lymphocytes are responsible for the destruction of stem cells in the bone marrow, leading to a decrease in blood counts. Because alemtuzumab destroys lymphocytes, it may be effective in treating aplastic anemia. Alemtuzumab is currently approved to treat chronic lymphocytic leukemia and is also helpful in other conditions that require immunosuppression, such as rheumatoid arthritis and immune cytopenias.
Patients 2 years of age and older with severe aplastic anemia whose disease does not respond to immunosuppressive therapy or has recurred following immunosuppressive therapy may be eligible for this study. Participants undergo the following tests and procedures:
* Pretreatment evaluation: Patients have a medical history, physical examination, blood tests, electrocardiogram (EKG), echocardiogram, 24-hour Holter monitor (continuous 24-hour monitoring of electrical activity of the heart), bone marrow biopsy (withdrawal through a needle of a small sample of bone marrow for analysis).
* Placement of a central line, if needed: An intravenous line (tube) is placed into a major vein in the patient's chest. It can stay in the body for the entire treatment period and be used to give chemotherapy or other medications, including antibiotics and blood transfusions, if needed, and to withdraw blood samples.
* Alemtuzumab therapy: Patients are admitted to the NIH Clinical Center for the first few injections for close monitoring of side effects. After receiving an initial small test dose, patients begin the first of ten daily injections under the skin, each lasting about 2 hours. Once patients tolerate the infusions with minimal or no side effects, they may be given the remaining infusions on an outpatient basis. Patients who relapse after their initial response to alemtuzumab are given cyclosporine to see if this drug will boost their immune response.
* Patients receive transfusions, growth factors, and antibiotic therapy, as needed.
* Infection therapy: Patients are given aerosolized pentamidine to protect against lung infections and valacyclovir to protect against herpes infections.
* A blood test is done and vital signs are measured every day while patients receive alemtuzumab.
* Patients have an echocardiogram and 24-hour Holter monitor after the last dose of alemtuzumab.
* Blood tests are done weekly for the first 3 months after alemtuzumab administration, then every other week until 6 months.
Patients return to the NIH for follow-up visits 1 month, 3 months, 6 months, and yearly for 5 years after the last dose of alemtuzumab for the following tests and evaluations:
* Blood test
* Repeat echocardiogram at 3-month visit
* Repeat bone marrow biopsy 6 months and 12 months after alemtuzumab, then as clinically indicated for 5 years.
Publications & conference data
8 peer-reviewed publications reference this trial (live from Europe PMC):
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NCT06948097 — Syk Inhibition in MItigating Lung Allograft Rejection (SIMILAR): A Trial to Evaluate the Safety and Tolerability of Fost
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Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by National Heart, Lung, and Blood Institute (NHLBI)
Last refreshed: 7 July 2020
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT00195624.