Acquired Aplastic AnaemiaPhase 2NCT01530555
Prospective Phase II Study of Rabbit Antithymocyte Globulin (ATG, Thymoglobuline®, Genzyme) With Ciclosporin for Patients With Acquired Aplastic Anaemia
A Phase 2 study testing Rabbit ATG, Thymoglobuline (Genzyme) in people with Acquired Aplastic Anaemia, run by King Faisal Specialist Hospital & Research Center.
Trial summary
To assess the tolerability and efficacy of rabbit antithymocyte globulin (ATG, Thymoglobuline®) with ciclosporin (CSA) in the first line treatment of patients with acquired severe aplastic anaemia (SAA), and patients with non-severe aplastic anaemia (NSAA) and who are transfusion dependent. Read more about this study
- Who is it for?
- Ages 16 to 80 years
- Conditions the study is for
- Acquired Aplastic Anaemia
- What is being tested?
- Rabbit ATG, Thymoglobuline (Genzyme)
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 35 participants
- Where
- No sites listed yet
- Study dates
- Apr 2008to Jul 2013
- Phase
- Phase 2
- 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 16 to 80 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- To define aplastic anaemia there must be at least two of the following: (1) haemoglobin < 10g/dl; (2) platelet count < 50 x 109/l; (3) neutrophil count < 1.5 x 109/l, and a hypocellular bone marrow on bone marrow biopsy
- Time from diagnosis to study registration ≤ 6mths
- No prior treatment except for haemopoietic growth factors given for no more than 4 weeks, and androgens.
- Age ≥ 16yrs (≥ 18yrs in Germany in accordance with German law), with no upper age limit.
Who cannot join
- Eligibility for an HLA-matched sibling donor transplant for SAA patients
- Prior therapy with ATG or CSA
- Haematopoeitic growth factors more than 4 weeks before study enrollment
- Diagnosis of Fanconi anaemia, dyskeratosis congenita or congenital bone marrow failure syndrome
- Evidence of myelodysplastic disease
- Paroxysmal nocturnal haemoglobinuria with evidence of significant haemolysis, history of PNH associated thrombosis or a PNH clone > 50% by flow cytometry
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- Diagnosis or previous history of carcinoma (except local cervical, basal cell, squamous cells, or melanoma)
- Subject is pregnant (e.g. positive HCG test) or is breast feeding
- Severe uncontrolled infection or unexplained fever > 38oC
- Subjects who have hepatic, renal cardiac, metabolic or other concurrent diseases of such severity that life expectancy is less than 3 months
About this study
To assess the tolerability and efficacy of rabbit antithymocyte globulin (ATG, Thymoglobuline®) with ciclosporin (CSA) in the first line treatment of patients with acquired severe aplastic anaemia (SAA), and patients with non-severe aplastic anaemia (NSAA) and who are transfusion dependent. To compare the response rate of the combination of rabbit ATG (Thymoglobuline® and CSA from this pilot study with the response rate observed in a series of matched AA patients; treated after 1994 with the combination of horse ATG (Lymphoglobuline®) and CSA; obtained from the EBMT database (comparative study).
- Conditions
- Acquired Aplastic Anaemia
- Being tested
- Rabbit ATG, Thymoglobuline (Genzyme)
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Rabbit ATG, Thymoglobuline (Genzyme)Drug
- Rabbit ATG, Thymoglobuline® (Genzyme) 1.5 vials/10kg (3.75mg/kg) daily for 5 days given as an intravenous infusion over 12-18 hours.
- Ciclosporin (CSA) 5mg/kg/day orally from day +1 for a minimum of 6 months
| Study group | Role | What they receive |
|---|---|---|
| Treatment arm | Experimental | 1. Rabbit ATG, Thymoglobuline (Genzyme) 1.5 vials/10kg (3.75mg/kg) daily for 5 days given as an intravenous infusion over 12-18 hours.
2. Ciclosporin (CSA) 5mg/kg/day orally from day +1 for a minimum of 6 months, with later tailing according to individual patient response. Aim to maintain trough whole blood CSA levels between 150 and 250 ng/ml. Receives: Drug: Rabbit ATG, Thymoglobuline (Genzyme) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Response
Primary outcome is response at 6 months post ATG treatment
Measured: 2 years
Secondary outcomes (1)
Over all survival
Secondary outcome variables include overall survival and failure free survival at 2 years post ATG treatment
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Feb 2012 (estimated)
- Study startApr 2008
- Primary completionJul 2013
- Study completionJul 2013
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acquired Aplastic AnaemiaStudies of the same condition, open and under way.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 35 participants (actual)
Sponsor and investigators
- Lead sponsor
- King Faisal Specialist Hospital & Research Center
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Ahmed Al Zahrani, MD, Principal investigator, King Faisal Specialist Hospital & Research Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01530555
- Sponsor's protocol ID
- 2081-005
- Status last verified
- Feb 2012
- First submitted
- 24 Jan 2012
- Last updated
- 25 Feb 2016
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Feb 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Prospective Phase II Study of Rabbit Antithymocyte Globulin (ATG, Thymoglobuline®, Genzyme) With Ciclosporin for Patients With Acquired Aplastic Anaemia
- ClinicalTrials.gov ID
- NCT01530555
- Acronym / sponsor ID
- 2081-005
- Conditions
- Acquired Aplastic Anaemia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 35 participants (actual)
- Lead sponsor
- King Faisal Specialist Hospital & Research CenterUniversity, hospital or other
- Study start
- Apr 2008
- Primary completion
- Jul 2013 (actual)
- Study completion
- Jul 2013 (actual)
- First posted
- 10 Feb 2012 (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.
To assess the tolerability and efficacy of rabbit antithymocyte globulin (ATG, Thymoglobuline®) with ciclosporin (CSA) in the first line treatment of patients with acquired severe aplastic anaemia (SAA), and patients with non-severe aplastic anaemia (NSAA) and who are transfusion dependent. To compare the response rate of the combination of rabbit ATG (Thymoglobuline® and CSA from this pilot study with the response rate observed in a series of matched AA patients; treated after 1994 with the combination of horse ATG (Lymphoglobuline®) and CSA; obtained from the EBMT database (comparative study).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment arm | Experimental | Drug: Rabbit ATG, Thymoglobuline (Genzyme)1. Rabbit ATG, Thymoglobuline (Genzyme) 1.5 vials/10kg (3.75mg/kg) daily for 5 days given as an intravenous infusion over 12-18 hours. 2. Ciclosporin (CSA) 5mg/kg/day orally from day +1 for a minimum of 6 months, with later tailing according to individual patient response. Aim to maintain trough whole blood CSA levels between 150 and 250 ng/ml. |
Who may qualify
- Ages
- 16 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- To define aplastic anaemia there must be at least two of the following: (1) haemoglobin < 10g/dl; (2) platelet count < 50 x 109/l; (3) neutrophil count < 1.5 x 109/l, and a hypocellular bone marrow on bone marrow biopsy
- Time from diagnosis to study registration ≤ 6mths
- No prior treatment except for haemopoietic growth factors given for no more than 4 weeks, and androgens.
- Age ≥ 16yrs (≥ 18yrs in Germany in accordance with German law), with no upper age limit.
Key exclusion criteria (10 listed)
- Eligibility for an HLA-matched sibling donor transplant for SAA patients
- Prior therapy with ATG or CSA
- Haematopoeitic growth factors more than 4 weeks before study enrollment
- Diagnosis of Fanconi anaemia, dyskeratosis congenita or congenital bone marrow failure syndrome
- Evidence of myelodysplastic disease
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 |
|---|---|---|
| Rabbit ATG, Thymoglobuline (Genzyme)1. Rabbit ATG, Thymoglobuline® (Genzyme) 1.5 vials/10kg (3.75mg/kg) daily for 5 days given as an intravenous infusion over 12-18 hours. 2. Ciclosporin (CSA) 5mg/kg/day orally from day +1 for a minimum of 6 months | Drug | Treatment arm |
Primary outcome
Response
Time frame: 2 years
Primary outcome is response at 6 months post ATG treatment
1 secondary outcome
Over all survival
Time frame: 2 years
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 25 Feb 2016.
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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.
Ahmed Al Zahrani, MD principal investigator
King Faisal Specialist Hospital & Research Center
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Rabbit ATG, Thymoglobuline (Genzyme) in people with Acquired Aplastic Anaemia, run by King Faisal Specialist Hospital & Research Center.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 25 Feb 2016. Status can change between updates, so confirm with the study contact before making plans.
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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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.
What does the primary outcome measure?
Response (time frame: 2 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 25 Feb 2016.