T-Regulatory Cells in Amyotrophic Lateral Sclerosis

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT03241784
• Date of registration: 27/07/2017
• Prospective Registration: No
• Primary sponsor: Stanley H. Appel, MD
• Public title: T-Regulatory Cells in Amyotrophic Lateral Sclerosis
• Scientific title: Expansion and Infusion of T-Regulatory Cells in Amyotrophic Lateral Sclerosis
• Date of first enrolment: May 16, 2016
• Target sample size: 4
• Recruitment status: Unknown status
• URL: https://clinicaltrials.gov/show/NCT03241784
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1

Countries of recruitment

United States

Contacts

• Name: Stanley H Appel, MD
• Affiliation: Houston Methodist Neurological Institute

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Age 18 years or older.

2. Sporadic or familial ALS diagnosed as possible, laboratory-supported probable,
probable, or definite as defined by revised El Escorial criteria (Appendix 1).

3. 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).

4. Capable of providing informed consent and following trial procedures.

5. Geographically accessible to the site.

6. 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.

7. Subjects must agree not to take live attenuated vaccines (including seasonal flu
vaccine) 30 days before blood collection.

8. Available autologous Tregs product with greater than or equal to 50% expression of
CD4, CD25 and FoxP3 determined by flow-cytometry.

9. Subjects must have been previously evaluated and followed clinically by a
neuromuscular specialist at Houston Methodist Neurological Institute

10. Normal Alanine aminotransferase level (ALT)

11. Normal Serum creatinine level

Exclusion Criteria:

1. Prior use of cells therapies

2. Concurrent use of other experimental ALS therapies

3. Pregnant or breastfeeding or planning to become pregnant or planning a partner's
pregnancy.

4. Other unstable medical or psychiatric illness

5. Known immune deficiency or history of lymphoma or leukemia

6. History of lymphopenia.

7. History of acquired or inherited immune deficiency syndrome, including leukopenia.

8. History of severe untreated chronic obstructive sleep apnea.

9. FVC less than 50% predicted at screening.

10. 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.

11. 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.

12. Clinically significant history of cardiac, oncologic, hepatic, or renal dysfunction,
or other medically significant illness.

13. The presence of any immunologic or autoimmune disease

14. Severe cardiac dysfunction defined clinically, or as a left ventricular ejection
fraction less than 40% of predicted or abnormal EKG findings.



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

ALS (Amyotrophic Lateral Sclerosis)

Intervention(s)

Biological: Interleukin-2

Biological: Autologous T-regulatory lymphocytes

Primary Outcome(s)

Number of participants with treatment-related adverse events as assessed by CTCAE v4.0 [Time Frame: Baseline to two years]

Secondary Outcome(s)

ALSFRS-R (ALS Functional Rating Scale-Revised) [Time Frame: Baseline to week 15]

TH17 and Th1 lymphocytes [Time Frame: Baseline to 3 months post-treatment for a total of two years from baseline]

Treg Suppression [Time Frame: Baseline to 3 months post treatment for a total of two years from baseline]

AALS (Appel ALS) Scale [Time Frame: Baseline to week 15]

T-Regulatory Cells [Time Frame: Baseline to 3 months post treatment for a total of two years from baseline]

Secondary ID(s)

Pro00013616

Source(s) of Monetary Support

Please refer to primary and secondary sponsors