Evidence map›Paper›PMID 33963971›Full record

Trial reportAdvances in therapy2021

A Randomized Phase 1 Safety, Pharmacokinetic and Pharmacodynamic Study of the Novel Myostatin Inhibitor Apitegromab (SRK-015): A Potential Treatment for Spinal Muscular Atrophy.

Doreen Barrett, Sanela Bilic, Yung Chyung, Shaun M Cote, Ryan Iarrobino, Katherine Kacena, Ashish Kalra, Kimberly Long, George Nomikos, Amy Place and 2 more

Open access · hybridAbstract readClinical Trial, Phase IRandomized Controlled Trial
PubMed Full text at PMC Publisher
In one paragraph

Trial report in Advances in therapy, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 30 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
30citing papers in PubMed, 1 pooled it
3.8field-weighted citation impact, top 6% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

30 citing papers in PubMed, 1 synthesis or guideline pooled it, 38 citations in OpenAlex.

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4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

12 authors at 3 institutions in 1 country.

Doreen BarrettScholar Rock, Inc., 301 Binney Street, 3rd Floor, Cambridge, MA, 02142, USA.
Sanela BilicVanadro, LLC, Urbandale, IA, USA.
Yung ChyungScholar Rock, Inc., 301 Binney Street, 3rd Floor, Cambridge, MA, 02142, USA.
Shaun M CoteScholar Rock, Inc., 301 Binney Street, 3rd Floor, Cambridge, MA, 02142, USA.
Ryan IarrobinoScholar Rock, Inc., 301 Binney Street, 3rd Floor, Cambridge, MA, 02142, USA.
Katherine KacenaKatherine Kacena Consulting, Natick, MA, USA.
Ashish KalraScholar Rock, Inc., 301 Binney Street, 3rd Floor, Cambridge, MA, 02142, USA.
Kimberly LongScholar Rock, Inc., 301 Binney Street, 3rd Floor, Cambridge, MA, 02142, USA.
George NomikosScholar Rock, Inc., 301 Binney Street, 3rd Floor, Cambridge, MA, 02142, USA. gnomikos@scholarrock.com.ORCID http://orcid.org/0000-0003-2846-1426
Amy PlaceScholar Rock, Inc., 301 Binney Street, 3rd Floor, Cambridge, MA, 02142, USA.
James Gordon StillClinical Development Solutions, LLC, Raleigh, NC, USA.
Leela VrishabhendraMedpace Clinical Pharmacology, Cincinnati, OH, USA.
Scholar Rock (United States) · USClinical Solutions · USMedpace (United States) · US

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

introductionApitegromab (SRK-015) is an anti-promyostatin monoclonal antibody under development to improve motor function in patients with spinal muscular atrophy, a rare neuromuscular disease. This phase 1 double-blind, placebo-controlled study assessed safety, pharmacokinetic parameters, pharmacodynamics (serum latent myostatin), and immunogenicity of single and multiple ascending doses of apitegromab in healthy adult subjects.

methodsSubjects were administered single intravenous ascending doses of apitegromab of 1, 3, 10, 20, 30 mg/kg or placebo, and multiple intravenous ascending doses of apitegromab of 10, 20, 30 mg/kg or placebo.

resultsFollowing single ascending doses, the pharmacokinetic parameters of apitegromab appeared to be similar across all dose groups, following a biphasic pattern of decline in the concentration-time curve. The mean apparent terminal t

conclusionThese results support continued investigation of apitegromab for the treatment of patients with milder forms (type 2 and 3) of spinal muscular atrophy.

Indexed as

Muscular Atrophy, SpinalMyostatinAdultArea Under CurveDose-Response Relationship, DrugDouble-Blind MethodDrug Administration ScheduleHumansMyostatinApitegromabHuman monoclonal antibodyMyostatinPharmacodynamicsPharmacokineticsPhase 1 studySafetySpinal muscular atrophySRK-015Tolerability

Identifiers

PMID33963971
PMCPMC8189951
OpenAlexW3159808529

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC
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Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.