Status:
UNKNOWN
T-Regulatory Cells in Amyotrophic Lateral Sclerosis
Lead Sponsor:
Stanley H. Appel, MD
Conditions:
ALS (Amyotrophic Lateral Sclerosis)
Eligibility:
All Genders
18+ years
Phase:
PHASE1
Brief Summary
This is an open-label pilot study to determine the safety and tolerability of infusions of autologous CD4+ CD25+ regulatory T cells with concomitant subcutaneous IL-2 injections in 4 subjects with ALS...
Detailed Description
This Pilot Study will consist of 4 ALS subjects who will undergo 4 infusions of autologous expanded Tregs with concomitant subcutaneous injections of IL-2 (2 x 105 IU/m2) 3 times weekly for 52 weeks o...
Eligibility Criteria
Inclusion
- Age 18 years or older.
- Sporadic or familial ALS diagnosed as possible, laboratory-supported probable, probable, or definite as defined by revised El Escorial criteria (Appendix 1).
- Subjects must not have taken riluzole for at least 30 days, or be on a stable dose of riluzole for at least 30 days (riluzole-naïve subjects are permitted in the study).
- Capable of providing informed consent and following trial procedures.
- Geographically accessible to the site.
- Women must not be able to become pregnant (e.g. post-menopausal, surgically sterile, or using adequate birth control methods) for the duration of the study and three months after study completion. Adequate contraception includes: abstinence, hormonal contraception (oral contraception, implanted contraception, injected contraception or other hormonal (patch or contraceptive ring, for example) contraception), intrauterine device (IUD) in place for ≥ 3 months, barrier method in conjunction with spermicide, or another adequate method.
- Subjects must agree not to take live attenuated vaccines (including seasonal flu vaccine) 30 days before blood collection.
- Available autologous Tregs product with greater than or equal to 50% expression of CD4, CD25 and FoxP3 determined by flow-cytometry.
- Subjects must have been previously evaluated and followed clinically by a neuromuscular specialist at Houston Methodist Neurological Institute
- Normal Alanine aminotransferase level (ALT)
- Normal Serum creatinine level
Exclusion
- Prior use of cells therapies
- Concurrent use of other experimental ALS therapies
- Pregnant or breastfeeding or planning to become pregnant or planning a partner's pregnancy.
- Other unstable medical or psychiatric illness
- Known immune deficiency or history of lymphoma or leukemia
- History of lymphopenia.
- History of acquired or inherited immune deficiency syndrome, including leukopenia.
- History of severe untreated chronic obstructive sleep apnea.
- FVC less than 50% predicted at screening.
- Exposure to any other agent currently under investigation for the treatment of subjects with ALS (off-label use or investigational) within 30 days of the Baseline Visit.
- The presence of unstable psychiatric disease, cognitive impairment, or dementia that would impair ability of the subject to provide informed consent, according to the PI's judgment, or a history of active substance abuse within the prior year.
- Clinically significant history of cardiac, oncologic, hepatic, or renal dysfunction, or other medically significant illness.
- The presence of any immunologic or autoimmune disease
- Severe cardiac dysfunction defined clinically, or as a left ventricular ejection fraction less than 40% of predicted or abnormal EKG findings.
Key Trial Info
Start Date :
May 16 2016
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
March 1 2018
Estimated Enrollment :
4 Patients enrolled
Trial Details
Trial ID
NCT03241784
Start Date
May 16 2016
End Date
March 1 2018
Last Update
March 26 2018
Active Locations (1)
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1
Methodist Neurological Institute
Houston, Texas, United States, 77030