Safety, pharmacokinetics, and preliminary assessment of efficacy of mecasermin (recombinant human IGF-1) for the treatment of Rett syndrome.

Khwaja OS, Ho E, Barnes KV, O'Leary HM, Pereira LM, Finkelstein Y, Nelson CA 3rd, Vogel-Farley V, DeGregorio G, Holm IA, Khatwa U, Kapur K and 10 more

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Alexander ME, Finnegan DM, Cantwell NG, Walco AC, Rappaport L, Gregas M, Fichorova RN, Shannon MW, Sur M, Kaufmann WE

Proceedings of the National Academy of Sciences of the United States of America12 Mar 2014Cited by 163 (Europe PMC)

PMID24623853PMCIDPMC3970488DOI10.1073/pnas.1311141111PepBankPUB-24623853

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Summary

The text of this summary is not shown here, because the article license does not allow reuse. Read it at the source (opens in a new tab)

Text rights: Publisher copyright. Results still quote short attributed sentences.

Results from this study8

8 results from studies that gave the peptide. 8 results from people.

01HumanNon-randomized trialSafety outcome
In girls with MECP2 mutations, mecasermin treatment produced no evidence of hypoglycemia.
In plain wordsIn girls with MECP2 mutations, no evidence of low blood sugar appeared during mecasermin treatment.
Peptide
IGF-1 (mecasermin)
Outcome
Not applicableHypoglycemia
Study
n = 12
Outcome group
Adverse events · Metabolic and weight
Studied dose
40-120 μg/kg twice daily, then maximum dose · 4-wk MAD and 20-wk OLEAs given in this study (human).
Source sentences
Summary
Twelve subjects completed the MAD and 10 the entire study, without evidence of hypoglycemia or serious adverse events.
2 context sentences(design, population, supporting numbers)
Summary · context
We evaluated the safety, tolerability, and pharmacokinetic profiles of IGF-1 in 12 girls with MECP2 mutations (9 with RTT).
Summary · context
This phase 1 trial included a 4-wk multiple ascending dose (MAD) (40-120 μg/kg twice daily) period and a 20-wk open-label extension (OLE) at the maximum dose.
Report a problemFND-2462385301
02HumanNon-randomized trialSafety outcome
In girls with MECP2 mutations, mecasermin treatment produced no evidence of serious adverse events.
In plain wordsIn girls with MECP2 mutations, no evidence of serious adverse events appeared during mecasermin treatment.
Peptide
IGF-1 (mecasermin)
Outcome
Not applicableSerious adverse events
Study
n = 12
Outcome group
Adverse events · Whole body
Studied dose
40-120 μg/kg twice daily, then maximum dose · 4-wk MAD and 20-wk OLEAs given in this study (human).
Source sentences
Summary
Twelve subjects completed the MAD and 10 the entire study, without evidence of hypoglycemia or serious adverse events.
2 context sentences(design, population, supporting numbers)
Summary · context
We evaluated the safety, tolerability, and pharmacokinetic profiles of IGF-1 in 12 girls with MECP2 mutations (9 with RTT).
Summary · context
This phase 1 trial included a 4-wk multiple ascending dose (MAD) (40-120 μg/kg twice daily) period and a 20-wk open-label extension (OLE) at the maximum dose.
Report a problemFND-2462385302
03HumanNon-randomized trialPharmacokinetics
In girls with MECP2 mutations, mecasermin increased cerebrospinal fluid IGF‑1 by the end of the ascending-dose period, indicating CNS exposure.
In plain wordsIn girls with MECP2 mutations, mecasermin increased IGF‑1 in spinal fluid by the end of the ascending-dose period.
Peptide
IGF-1 (mecasermin)
Outcome
IncreaseCSF IGF-1 levels
Study
n = 12
Outcome group
Pharmacokinetics · Pharmacology
Studied dose
40-120 μg/kg twice daily · 4-wkAs given in this study (human).
Source sentences
Summary
Mecasermin reached the CNS compartment as evidenced by the increase in cerebrospinal fluid IGF-1 levels at the end of the MAD.
2 context sentences(design, population, supporting numbers)
Summary · context
We evaluated the safety, tolerability, and pharmacokinetic profiles of IGF-1 in 12 girls with MECP2 mutations (9 with RTT).
Summary · context
This phase 1 trial included a 4-wk multiple ascending dose (MAD) (40-120 μg/kg twice daily) period and a 20-wk open-label extension (OLE) at the maximum dose.
Report a problemFND-2462385304
04HumanNon-randomized trialPharmacokinetics
In girls with MECP2 mutations, mecasermin followed nonlinear kinetics, with greater distribution in the peripheral compartment.
In plain wordsIn girls with MECP2 mutations, mecasermin followed nonlinear drug kinetics, with greater distribution in the peripheral compartment.
Peptide
IGF-1 (mecasermin)
Outcome
Not applicablePharmacokinetic profile (nonlinear kinetics, peripheral distribution)
Study
n = 12
Outcome group
Pharmacokinetics · Pharmacology
Studied dose
40-120 μg/kg twice daily, then maximum dose · 4-wk MAD and 20-wk OLEAs given in this study (human).
Source sentences
Summary
The drug followed nonlinear kinetics, with greater distribution in the peripheral compartment.
2 context sentences(design, population, supporting numbers)
Summary · context
We evaluated the safety, tolerability, and pharmacokinetic profiles of IGF-1 in 12 girls with MECP2 mutations (9 with RTT).
Summary · context
This phase 1 trial included a 4-wk multiple ascending dose (MAD) (40-120 μg/kg twice daily) period and a 20-wk open-label extension (OLE) at the maximum dose.
Report a problemFND-2462385305
05HumanNon-randomized trialEfficacy outcome
In girls studied for Rett syndrome, apnea improved during the open-label mecasermin extension.
In plain wordsIn girls studied for Rett syndrome, breathing pauses improved during the open-label mecasermin extension.
Peptide
IGF-1 (mecasermin)
Outcome
DecreaseApnea
Study
n: not reported in source
Outcome group
Respiratory function · Respiratory
Studied dose
maximum dose (MAD range 40-120 μg/kg twice daily) · 20-wk OLEAs given in this study (human).
Source sentences
Summary
Cardiorespiratory measures showed that apnea improved during the OLE.
2 context sentences(design, population, supporting numbers)
Summary · context
We evaluated the safety, tolerability, and pharmacokinetic profiles of IGF-1 in 12 girls with MECP2 mutations (9 with RTT).
Summary · context
This phase 1 trial included a 4-wk multiple ascending dose (MAD) (40-120 μg/kg twice daily) period and a 20-wk open-label extension (OLE) at the maximum dose.
Report a problemFND-2462385307
06HumanNon-randomized trialEfficacy outcome
In girls studied for Rett syndrome, anxiety measures improved during the open-label mecasermin extension.
In plain wordsIn girls studied for Rett syndrome, anxiety measures improved during the open-label mecasermin extension.
Peptide
IGF-1 (mecasermin)
Outcome
DecreaseAnxiety
Study
n: not reported in source
Outcome group
Anxiety and mood · Mental health and mood
Studied dose
maximum dose (MAD range 40-120 μg/kg twice daily) · 20-wk OLEAs given in this study (human).
Source sentences
Summary
Some neurobehavioral parameters, specifically measures of anxiety and mood also improved during the OLE.
2 context sentences(design, population, supporting numbers)
Summary · context
We evaluated the safety, tolerability, and pharmacokinetic profiles of IGF-1 in 12 girls with MECP2 mutations (9 with RTT).
Summary · context
This phase 1 trial included a 4-wk multiple ascending dose (MAD) (40-120 μg/kg twice daily) period and a 20-wk open-label extension (OLE) at the maximum dose.
Report a problemFND-2462385308
07HumanNon-randomized trialEfficacy outcome
In girls studied for Rett syndrome, mood measures improved during the open-label mecasermin extension.
In plain wordsIn girls studied for Rett syndrome, mood measures improved during the open-label mecasermin extension.
Peptide
IGF-1 (mecasermin)
Outcome
Direction not reportedMood
Study
n: not reported in source
Outcome group
Anxiety and mood · Mental health and mood
Studied dose
maximum dose (MAD range 40-120 μg/kg twice daily) · 20-wk OLEAs given in this study (human).
Source sentences
Summary
Some neurobehavioral parameters, specifically measures of anxiety and mood also improved during the OLE.
2 context sentences(design, population, supporting numbers)
Summary · context
We evaluated the safety, tolerability, and pharmacokinetic profiles of IGF-1 in 12 girls with MECP2 mutations (9 with RTT).
Summary · context
This phase 1 trial included a 4-wk multiple ascending dose (MAD) (40-120 μg/kg twice daily) period and a 20-wk open-label extension (OLE) at the maximum dose.
Report a problemFND-2462385309
08HumanNon-randomized trialMechanism
In girls studied for Rett syndrome, mecasermin-associated mood and anxiety improvements were supported by reversal of right frontal alpha band asymmetry.
In plain wordsIn girls studied for Rett syndrome, reversal of a brain-wave asymmetry supported the mood and anxiety improvements during mecasermin treatment.
Peptide
IGF-1 (mecasermin)
Outcome
Direction not reportedRight frontal alpha band asymmetry
Study
n: not reported in source
Outcome group
Anxiety and mood · Nervous system and cognition
Studied dose
Dose not stated
Source sentences
Summary
These improvements in mood and anxiety scores were supported by reversal of right frontal alpha band asymmetry on EEG, an index of anxiety and depression.
2 context sentences(design, population, supporting numbers)
Summary · context
We evaluated the safety, tolerability, and pharmacokinetic profiles of IGF-1 in 12 girls with MECP2 mutations (9 with RTT).
Summary · context
This phase 1 trial included a 4-wk multiple ascending dose (MAD) (40-120 μg/kg twice daily) period and a 20-wk open-label extension (OLE) at the maximum dose.
Report a problemFND-2462385310

AI wrote and checked these results. AI can make mistakes, so check important results against the source.

Registered trials1

Trials this study reports on, or that list this study as a reference. Registry data from ClinicalTrials.gov.

1 trial · 1 completed · 1 with posted results · source ClinicalTrials.gov (opens in a new tab)

CompletedPhase 2Results postedNCT01777542

Treatment of Rett Syndrome With Recombinant Human IGF-1

Boston Children's Hospitaln = 30Jan 2013 to Nov 2016

Rett Syndrome

Trial page ClinicalTrials.govUpdated 26 Mar 2018

Version history

PepBank keeps every version of the data on this study. Status changes from Retraction Watch, Europe PMC and Crossref are listed with their source.

Version 2Current
14 Oct 2026, 05:02 UTC
Study results updated
Version 1
14 Oct 2026, 01:46 UTC
First added · source update from Europe PMC

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