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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT02164253
Other study ID # 2012_69
Secondary ID 2013-001228-21
Status Completed
Phase Phase 2
First received May 20, 2014
Last updated March 8, 2017
Start date September 2013
Est. completion date December 2016

Study information

Verified date October 2016
Source University Hospital, Lille
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

The FAIR-ALS study is to investigate the safety and efficacy of a scavenger treatment of iron deferiprone, which would reduce the brain iron to limit the development of amyotrophic lateral sclerosis.

It has been shown an excess of iron in the central nervous system carrying a sporadic ALS patients. Iron overload associated with a loss of motor neurons may explain the signs of the disease (atrophy).

The investigators discuss the hypothesis that reducing excess iron, the investigators can reduce the loss of neurons and thus the progression of signs of the disease.


Description:

At the end of the study, it will propose to continue the usual quarterly patient follow up, as recommended by the French ALS centers.

Deferiprone can be administered as part of a compassionate use, for patients who want it and who do not have hypoxemia.

We therefore plan a treatment period compassionate relatively short and less than 3 years.


Recruitment information / eligibility

Status Completed
Enrollment 23
Est. completion date December 2016
Est. primary completion date December 2016
Accepts healthy volunteers No
Gender All
Age group 18 Years to 85 Years
Eligibility Inclusion Criteria:

- Amyotrophic lateral sclerosis defined in accordance to the El Escorial criteria (possible, probable or defined)

- 18 to 85 years old patient, male or female

- Patient with social security cover

Exclusion Criteria:

- Achieved respiratory defined by a FVC <70%

- Evolution of more than 24 months

- Demented subject

- Severe malnutrition

- Patients with treatment potentially at risk of agranulocytosis and neutropenia

- Patients with a history of agranulocytosis or iatrogenic under haematological disease

- Incapable of giving consent

- Indication against MRI

- Indication against lumbar puncture

- Patient refused lumbar puncture

- Hypersensitivity to iron chelators

- Concomitant treatment with antacids containing aluminum

- Presence of another serious illness to life-threatening or disabling cons to the use of the treatment mixture of oxygen and nitrous oxide equally

Study Design


Related Conditions & MeSH terms


Intervention

Drug:
Deferiprone
30 mg/kg per day, oral use

Locations

Country Name City State
France Hôpital Roger Salengro, CHRU de Lille Lille

Sponsors (2)

Lead Sponsor Collaborator
University Hospital, Lille ApoPharma

Country where clinical trial is conducted

France, 

References & Publications (21)

Bensimon G, Lacomblez L, Meininger V. A controlled trial of riluzole in amyotrophic lateral sclerosis. ALS/Riluzole Study Group. N Engl J Med. 1994 Mar 3;330(9):585-91. — View Citation

Boddaert N, Le Quan Sang KH, Rötig A, Leroy-Willig A, Gallet S, Brunelle F, Sidi D, Thalabard JC, Munnich A, Cabantchik ZI. Selective iron chelation in Friedreich ataxia: biologic and clinical implications. Blood. 2007 Jul 1;110(1):401-8. — View Citation

Danzeisen R, Achsel T, Bederke U, Cozzolino M, Crosio C, Ferri A, Frenzel M, Gralla EB, Huber L, Ludolph A, Nencini M, Rotilio G, Valentine JS, Carrì MT. Superoxide dismutase 1 modulates expression of transferrin receptor. J Biol Inorg Chem. 2006 Jun;11(4):489-98. — View Citation

Felice KJ. A longitudinal study comparing thenar motor unit number estimates to other quantitative tests in patients with amyotrophic lateral sclerosis. Muscle Nerve. 1997 Feb;20(2):179-85. — View Citation

Goodall EF, Greenway MJ, van Marion I, Carroll CB, Hardiman O, Morrison KE. Association of the H63D polymorphism in the hemochromatosis gene with sporadic ALS. Neurology. 2005 Sep 27;65(6):934-7. — View Citation

Kong J, Xu Z. Massive mitochondrial degeneration in motor neurons triggers the onset of amyotrophic lateral sclerosis in mice expressing a mutant SOD1. J Neurosci. 1998 May 1;18(9):3241-50. — View Citation

Kwiatkowski A, Ryckewaert G, Jissendi Tchofo P, Moreau C, Vuillaume I, Chinnery PF, Destée A, Defebvre L, Devos D. Long-term improvement under deferiprone in a case of neurodegeneration with brain iron accumulation. Parkinsonism Relat Disord. 2012 Jan;18(1):110-2. doi: 10.1016/j.parkreldis.2011.06.024. — View Citation

Langkammer C, Enzinger C, Quasthoff S, Grafenauer P, Soellinger M, Fazekas F, Ropele S. Mapping of iron deposition in conjunction with assessment of nerve fiber tract integrity in amyotrophic lateral sclerosis. J Magn Reson Imaging. 2010 Jun;31(6):1339-45. doi: 10.1002/jmri.22185. — View Citation

Lin J, Diamanduros A, Chowdhury SA, Scelsa S, Latov N, Sadiq SA. Specific electron transport chain abnormalities in amyotrophic lateral sclerosis. J Neurol. 2009 May;256(5):774-82. doi: 10.1007/s00415-009-5015-8. — View Citation

Miller R, Bradley W, Cudkowicz M, Hubble J, Meininger V, Mitsumoto H, Moore D, Pohlmann H, Sauer D, Silani V, Strong M, Swash M, Vernotica E; TCH346 Study Group.. Phase II/III randomized trial of TCH346 in patients with ALS. Neurology. 2007 Aug 21;69(8):776-84. — View Citation

Moreau C, Devos D, Brunaud-Danel V, Defebvre L, Perez T, Destée A, Tonnel AB, Lassalle P, Just N. Elevated IL-6 and TNF-alpha levels in patients with ALS: inflammation or hypoxia? Neurology. 2005 Dec 27;65(12):1958-60. — View Citation

Moreau C, Devos D, Brunaud-Danel V, Defebvre L, Perez T, Destée A, Tonnel AB, Lassalle P, Just N. Paradoxical response of VEGF expression to hypoxia in CSF of patients with ALS. J Neurol Neurosurg Psychiatry. 2006 Feb;77(2):255-7. — View Citation

Moreau C, Devos D, Gosset P, Brunaud-Danel V, Tonnel AB, Lassalle P, Defebvre L, Destée A. [Mechanisms of deregulated response to hypoxia in sporadic amyotrophic lateral sclerosis: a clinical study]. Rev Neurol (Paris). 2010 Mar;166(3):279-83. doi: 10.1016/j.neurol.2009.05.018. Review. French. — View Citation

Moreau C, Gosset P, Brunaud-Danel V, Lassalle P, Degonne B, Destee A, Defebvre L, Devos D. CSF profiles of angiogenic and inflammatory factors depend on the respiratory status of ALS patients. Amyotroph Lateral Scler. 2009 Jun;10(3):175-81. doi: 10.1080/17482960802651725. — View Citation

Moreau C, Gosset P, Kluza J, Brunaud-Danel V, Lassalle P, Marchetti P, Defebvre L, Destée A, Devos D. Deregulation of the hypoxia inducible factor-1a pathway in monocytes from sporadic amyotrophic lateral sclerosis patients. Neuroscience. 2011 Jan 13;172:110-7. doi: 10.1016/j.neuroscience.2010.10.040. — View Citation

Noto Y, Misawa S, Kanai K, Shibuya K, Isose S, Nasu S, Sekiguchi Y, Fujimaki Y, Nakagawa M, Kuwabara S. Awaji ALS criteria increase the diagnostic sensitivity in patients with bulbar onset. Clin Neurophysiol. 2012 Feb;123(2):382-5. doi: 10.1016/j.clinph.2011.05.030. — View Citation

Schrooten M, Smetcoren C, Robberecht W, Van Damme P. Benefit of the Awaji diagnostic algorithm for amyotrophic lateral sclerosis: a prospective study. Ann Neurol. 2011 Jul;70(1):79-83. doi: 10.1002/ana.22380. — View Citation

Shefner JM, Cudkowicz ME, Zhang H, Schoenfeld D, Jillapalli D; Northeast ALS Consortium.. Revised statistical motor unit number estimation in the Celecoxib/ALS trial. Muscle Nerve. 2007 Feb;35(2):228-34. — View Citation

Shefner JM, Watson ML, Simionescu L, Caress JB, Burns TM, Maragakis NJ, Benatar M, David WS, Sharma KR, Rutkove SB. Multipoint incremental motor unit number estimation as an outcome measure in ALS. Neurology. 2011 Jul 19;77(3):235-41. doi: 10.1212/WNL.0b013e318225aabf. — View Citation

Yuen EC, Olney RK. Longitudinal study of fiber density and motor unit number estimate in patients with amyotrophic lateral sclerosis. Neurology. 1997 Aug;49(2):573-8. — View Citation

Zigmond AS, Snaith RP. The hospital anxiety and depression scale. Acta Psychiatr Scand. 1983 Jun;67(6):361-70. — View Citation

* Note: There are 21 references in allClick here to view all references

Outcome

Type Measure Description Time frame Safety issue
Primary Evolution of Amyotrophic Lateral Sclerosis Functional Rating Scale V3, V6, V9, V12, V15
Secondary Comparison of the progression of ALSFRS-R (Amyotrophic Lateral Sclerosis Functional Rating Scale ) score for 3 months without treatment (V0 to V3) in the period of the first three months under treatment (V3 to V6). SCREENING, V0, V3, V6
Secondary The proportion of patients who become non-self-sufficient after 12 months with the appearance of a sub scores ALSFRS-R less than or equal to 2 on swallowing, cut food using utensils or walk. SCREENING, V0, V3, V6, V9, V12, V15
Secondary Number of patients with anemia at 12 months defined by a hemoglobin / dL Hb <12 g V3, V6, V9, V12, V15
Secondary Number of serious and non-serious adverse events SCREENING, V0, V3, V6, V9, V12, V15
Secondary Progression of respiratory vital capacity tests SCREENING, V0, V3, V6, V9, V12, V15
Secondary Values of R2 * in MRI V3, V6, V19
Secondary Oxidative stress markers analyzed blindly in blood and cerebrospinal fluid V3, V9
Secondary No alteration of energy metabolism in aerobic and anaerobic blood and cerebrospinal fluid V3, V9
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