A placebo-controlled trial of recombinant human ciliary neurotrophic (rhCNTF) factor in amyotrophic lateral sclerosis

Robert G. Miller, Jack H. Petajan, Wilson W. Bryan, Carmel Armon, Richard J. Barohn, Jessie C. Goodpasture, Rebecca J. Hoagland, Gareth J. Parry, Mark A Ross, Scott C. Stromatt

Research output: Contribution to journalArticle

304 Citations (Scopus)

Abstract

Preclinical investigations indicated that recombinant human ciliary neurotrophic factor (rhCNTF) may have potential as therapy for amyotrophic lateral sclerosis (ALS). We evaluated the safety and efficacy of rhCNTF in a prospective, double-blind, placebo-controlled trial in 570 patients with ALS. Patients were randomized to receive 0.5, 2, or 5 μg/kg/day rhCNTF, or placebo, for 6 months. The primary efficacy end point was the change from baseline to the last on-treatment value of a combination megascore for limb strength (maximum voluntary isometric contraction) and pulmonary function. Secondary end points included individual arm and leg megascores, pulmonary function tests, an activities-of-daily-living outcome measure, and survival. The four treatment groups were similar at baseline with respect to age, sex, disease duration, and muscle strength values. At all doses tested, rhCNTF had no beneficial effect on the primary or secondary end points. Certain adverse events, as follows, appeared to be dose related: injection site reactions, cough, asthenia, nausea, anorexia, weight loss, and increased salivation. There was an increased number of deaths at the highest dose level. rhCNTF had no benefical effect on any measure of ALS progression. There were increased adverse events in the 5 μg/kg group and increased deaths.

Original languageEnglish (US)
Pages (from-to)256-260
Number of pages5
JournalAnnals of Neurology
Volume39
Issue number2
DOIs
StatePublished - 1996
Externally publishedYes

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Amyotrophic Lateral Sclerosis
Placebos
Salivation
Asthenia
Isometric Contraction
Respiratory Function Tests
Muscle Strength
Anorexia
Activities of Daily Living
Cough
Nausea
Weight Loss
Leg
Arm
Therapeutics
Extremities
Outcome Assessment (Health Care)
axokine
Safety
Lung

ASJC Scopus subject areas

  • Neuroscience(all)

Cite this

Miller, R. G., Petajan, J. H., Bryan, W. W., Armon, C., Barohn, R. J., Goodpasture, J. C., ... Stromatt, S. C. (1996). A placebo-controlled trial of recombinant human ciliary neurotrophic (rhCNTF) factor in amyotrophic lateral sclerosis. Annals of Neurology, 39(2), 256-260. https://doi.org/10.1002/ana.410390215

A placebo-controlled trial of recombinant human ciliary neurotrophic (rhCNTF) factor in amyotrophic lateral sclerosis. / Miller, Robert G.; Petajan, Jack H.; Bryan, Wilson W.; Armon, Carmel; Barohn, Richard J.; Goodpasture, Jessie C.; Hoagland, Rebecca J.; Parry, Gareth J.; Ross, Mark A; Stromatt, Scott C.

In: Annals of Neurology, Vol. 39, No. 2, 1996, p. 256-260.

Research output: Contribution to journalArticle

Miller, RG, Petajan, JH, Bryan, WW, Armon, C, Barohn, RJ, Goodpasture, JC, Hoagland, RJ, Parry, GJ, Ross, MA & Stromatt, SC 1996, 'A placebo-controlled trial of recombinant human ciliary neurotrophic (rhCNTF) factor in amyotrophic lateral sclerosis', Annals of Neurology, vol. 39, no. 2, pp. 256-260. https://doi.org/10.1002/ana.410390215
Miller, Robert G. ; Petajan, Jack H. ; Bryan, Wilson W. ; Armon, Carmel ; Barohn, Richard J. ; Goodpasture, Jessie C. ; Hoagland, Rebecca J. ; Parry, Gareth J. ; Ross, Mark A ; Stromatt, Scott C. / A placebo-controlled trial of recombinant human ciliary neurotrophic (rhCNTF) factor in amyotrophic lateral sclerosis. In: Annals of Neurology. 1996 ; Vol. 39, No. 2. pp. 256-260.
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