Aplastic AnemiaPhase 1NCT00001398
Stem Cell Factor Medication for Aplastic Anemia
A Phase 1 study testing Recombinant Methionyl Human Stem Cell Factor (r-metHuSCF) in people with Aplastic Anemia and Pancytopenia, run by National Heart, Lung, and Blood Institute (NHLBI) at 1 site in United States.
Trial summary
This trial, sponsored by Amgen, Inc., which produces the recombinant methionyl human stem cell factor (r-metHuSCF), also involves two other institutions. The primary objective is determination of the safety of administering multiple doses of r-metHuSCF in the setting of acquired aplastic anemia and evaluation of the… Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Aplastic Anemia, Pancytopenia
- What is being tested?
- Recombinant Methionyl Human Stem Cell Factor (r-metHuSCF)
- How many people
- 40 participants
- Where
- 1 siteUnited States
- Study dates
- Oct 1993to Jun 2002
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Acquired moderate or severe aplastic anemia.
Intolerant to, or failure to durably respond to, ATG/ALG therapy with or without cyclosporin.
Patients may not have received ATG/ALG, therapy for 12 weeks prior to enrollment or cyclosporin for 4 weeks prior to enrollment.
Two years of age or older.
Karnofsky Performance Status greater than or equal to 60 percent.
Adequate organ function as defined by serum creatine less than 2.0 mg/dl and a bilirubin less than 2.0 mg/dl.
Patients (or their parent[s]/responsible guardian[s]) must be able to comprehend and be willing to sign an informed consent prior to starting r-metHuSCF therapy.
No current diagnosis or past history of myelodysplastic syndromes.
No diagnosis of Fanconi's anemia, dyskeratosis congenita, or other congenital forms of aplastic anemia.
No current diagnosis of clinically active paroxysmal nocturnal hemoglobinuria (PNH) defined as patients with clinically significant thrombosis or hemolysis.
No diagnosis of eosinophilic fasciitis.
No treatment with ATG, ALG, or other immunosuppresive agents within 12 weeks of enrollment or treatment with cyclosporine A or IL-3 within 4 weeks of enrollment.
No treatment with hematopoietic growth factors within 2 weeks of enrollment.
No evidence of active uncontrolled infection.
No known allergy to Ecoli-derived products.
No current or recent symptoms of asthma occurring within the past 10 years (including allergic asthma, or asthma induced by cold temperature, infection, or exercise).
No history of anaphylactic/anaphylactoid-type event manifested by disseminated urticaria, laryngeal edema, and/or bronchospasm (or for example: food, insect bites, etc). Patients with drug allergies, manifested solely by rash and/or urticaria, are not excluded. An isolated episode of urticaria occuring more than 3 years earlier is not a contraindication.
No significant nonmalignant disease including previously documented HIV infection, uncontrolled hypertension (diastolic blood pressure greater than 115 mmHg), unstable angina, congestive heart failure (greater than NY Class II), poorly controlled diabetes, coronary angioplasty within 6 months, or uncontrolled atrial or ventricular cardiac arrhythmias.
No pregnancy or breast feeding. Those of childbearing potential must observe adequate birth control measures.
No treatment with an investigational agent (other than hematopoietic growth factors) within 4 weeks of study entry.
No concurrent use of beta adrenergic blocking agents.
No concurrent use, or use within the past 2 weeks, of Monoamine Oxidase Inhibitors (MAO Inhibitors).
No psychiatric, addictive, or any disorder which compromises ability to give truly informed consent for participation in this study.
Trial locations and contacts
1 site in 1 country.
United States1 site
National Heart, Lung and Blood Institute (NHLBI)
Bethesda, Maryland, 20892
About this study
Official title: A Phase I/II Trial of Recombinant Methionyl Human Stem Cell Factor (r-metHuSCF) in Patients Diagnosed With Acquired Aplastic Anemia
This trial, sponsored by Amgen, Inc., which produces the recombinant methionyl human stem cell factor (r-metHuSCF), also involves two other institutions. The primary objective is determination of the safety of administering multiple doses of r-metHuSCF in the setting of acquired aplastic anemia and evaluation of the effect of r-metHuSCF on peripheral blood counts. Potential effects of r-metHuSCF on frequency of need for red cell or platelet transfusions and on bone marrow morphology/cellularity will also be evaluated.
Read the detailed description
This trial, sponsored by Amgen, Inc., which produces the recombinant methionyl human stem cell factor (r-metHuSCF), also involves two other institutions. The primary objective is determination of the safety of administering multiple doses of r-metHuSCF in the setting of acquired aplastic anemia and evaluation of the effect of r-metHuSCF on peripheral blood counts. Potential effects of r-metHuSCF on frequency of need for red cell or platelet transfusions and on bone marrow morphology/cellularity will also be evaluated.
- Conditions
- Aplastic Anemia, Pancytopenia
- Being tested
- Recombinant Methionyl Human Stem Cell Factor (r-metHuSCF)
- Keywords
- Hematopoiesis, Stem Cell Cycling, Bone Marrow Failure, Acquired Aplastic Anemia
- Medical subject headings
- Anemia, Aplastic, Pancytopenia, Bone Marrow Failure Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Recombinant Methionyl Human Stem Cell Factor (r-metHuSCF)Drug
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Nov 1999 (estimated)
- Study startOct 1993
- Study completionJun 2002
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Aplastic AnemiaStudies of the same condition, open and under way.
- More trials run by National Heart, Lung, and Blood Institute (NHLBI)The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Enrolment
- 40 participants
Sponsor and investigators
- Sponsor type
- US National Institutes of Health
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00001398
- Sponsor's protocol ID
- 940016
- Other IDs
- 94-H-0016
- Status last verified
- Jun 2002
- First submitted
- 3 Nov 1999
- Last updated
- 4 Mar 2008
- Results posted
- No
Publications
Camitta BM, Thomas ED, Nathan DG, Gale RP, Kopecky KJ, Rappeport JM, Santos G, Gordon-Smith EC, Storb R. A prospective study of androgens and bone marrow transplantation for treatment of severe aplastic anemia. Blood. 1979 Mar;53(3):504-14. No abstract available.
PubMed 32941Camitta BM, Storb R, Thomas ED. Aplastic anemia (first of two parts): pathogenesis, diagnosis, treatment, and prognosis. N Engl J Med. 1982 Mar 18;306(11):645-52. doi: 10.1056/NEJM198203183061105. No abstract available.
PubMed 7035946Szklo M, Sensenbrenner L, Markowitz J, Weida S, Warm S, Linet M. Incidence of aplastic anemia in metropolitan Baltimore: a population-based study. Blood. 1985 Jul;66(1):115-9.
PubMed 4005425
Study at a glance
From the registry record, last updated by the sponsor on 4 Mar 2008. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I/II Trial of Recombinant Methionyl Human Stem Cell Factor (r-metHuSCF) in Patients Diagnosed With Acquired Aplastic Anemia
- ClinicalTrials.gov ID
- NCT00001398
- Acronym / sponsor ID
- 940016
- Conditions
- Aplastic Anemia, Pancytopenia
- Keywords
- Hematopoiesis, Stem Cell Cycling, Bone Marrow Failure, Acquired Aplastic Anemia
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 40 participants
- Lead sponsor
- National Heart, Lung, and Blood Institute (NHLBI)US National Institutes of Health
- Study start
- Oct 1993
- Study completion
- Jun 2002
- First posted
- 4 Nov 1999 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This trial, sponsored by Amgen, Inc., which produces the recombinant methionyl human stem cell factor (r-metHuSCF), also involves two other institutions. The primary objective is determination of the safety of administering multiple doses of r-metHuSCF in the setting of acquired aplastic anemia and evaluation of the effect of r-metHuSCF on peripheral blood counts. Potential effects of r-metHuSCF on frequency of need for red cell or platelet transfusions and on bone marrow morphology/cellularity will also be evaluated.
Detailed description
This trial, sponsored by Amgen, Inc., which produces the recombinant methionyl human stem cell factor (r-metHuSCF), also involves two other institutions. The primary objective is determination of the safety of administering multiple doses of r-metHuSCF in the setting of acquired aplastic anemia and evaluation of the effect of r-metHuSCF on peripheral blood counts. Potential effects of r-metHuSCF on frequency of need for red cell or platelet transfusions and on bone marrow morphology/cellularity will also be evaluated.
Study design
- Primary purpose
- Treatment
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Recombinant Methionyl Human Stem Cell Factor (r-metHuSCF) | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Mar 2008.
- Background: PubMed 32941Camitta BM, Thomas ED, Nathan DG, Gale RP, Kopecky KJ, Rappeport JM, Santos G, Gordon-Smith EC, Storb R. A prospective study of androgens and bone marrow transplantation for treatment of severe aplastic anemia. Blood. 1979 Mar;53(3):504-14. No abstract available.
- Background: PubMed 7035946Camitta BM, Storb R, Thomas ED. Aplastic anemia (first of two parts): pathogenesis, diagnosis, treatment, and prognosis. N Engl J Med. 1982 Mar 18;306(11):645-52. doi: 10.1056/NEJM198203183061105. No abstract available.
- Background: PubMed 4005425Szklo M, Sensenbrenner L, Markowitz J, Weida S, Warm S, Linet M. Incidence of aplastic anemia in metropolitan Baltimore: a population-based study. Blood. 1985 Jul;66(1):115-9.
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Aplastic Anaemia
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Recombinant Methionyl Human Stem Cell Factor (r-metHuSCF) in people with Aplastic Anemia and Pancytopenia, run by National Heart, Lung, and Blood Institute (NHLBI) at 1 site in United States.
The full registered description is in the About this study section and the official record.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 4 Mar 2008. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00001398, which also lists 3 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Mar 2008; AzTrials last refreshed its copy on 28 Oct 2026.